A Phase 3 interventional study of Rilonacept and Placebo in Recurrent Pericarditis, sponsored by Kiniksa Pharmaceuticals (UK), Ltd.. Completed at 21 sites in 4 countries. Open to participants aged 12 Years and older. Per ClinicalTrials.gov, last updated 2023-09-06.
Sponsored by Kiniksa Pharmaceuticals (UK), Ltd. · Phase 3, Interventional, and Treatment
The primary objective of this study was to assess the efficacy of rilonacept treatment in participants with recurrent pericarditis.
In the single-blind run-in (RI) period, rilonacept 320 mg (or 4.4 mg/kg in pediatric participants ≥12 and \<18 years old) subcutaneous (SC), followed by 160 mg (or 2.2 mg/kg in pediatric participants ≥12 and \<18 years old) injections once weekly.
During the Randomized-Withdrawal (RW) period, eligible participants are randomized 1:1 to double-blinded administration of study drug:
Participants with pericarditis recurrence who meet the protocol criteria for bailout rilonacept (report at least 1 day with pericarditis pain ≥4 on the 11-point numerical rating scale (NRS) and have 1 C-reactive protein (CRP) value ≥ 1 mg/dL [either on the same day or separated by no more than 7 days]) receive bailout rilonacept (2 open-label injections of 160 mg rilonacept [or 4.4 mg/kg for pediatric subjects]) irrespective of randomized treatment assignment and as soon as at least 5 days have passed since the last study drug injection.
Upon completion of the RW period (i.e., when the prespecified number of primary efficacy endpoints [clinical events committee-confirmed pericarditis recurrence] events have occurred), all participants who did not discontinue study drug have an option to continue treatment with open-label rilonacept in the Long-Term Extension (LTE) or to withdraw from the study. Participants still in the RI period at the time that the RW period has ended and the LTE is opened will have the option to enter the LTE directly when they have completed the RI period and have met the definition of clinical response or to withdraw from the study.
54 studies on the registry are indexed under Pericarditis; 23 are open to participants now.
This study's enrollment of 86 is above the median of 60 across 29 interventional studies indexed under Pericarditis.
Browse Pericarditis studies →Kiniksa Pharmaceuticals (UK), Ltd. is the lead sponsor of 2 studies on the registry; none are open to participants now.
Counted across the registry records on this site, refreshed daily.
Key Inclusion Criteria:
Key Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Periods have stopped for good: usually no period for 12 months in a row without another medical reason, sometimes confirmed by a blood test (FSH).
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Postmenopausal
Periods have stopped for good: usually no period for 12 months in a row without another medical reason, sometimes confirmed by a blood test (FSH).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Metastases
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Excipients
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
RI period: single-blind rilonacept 320 mg (or 4.4 mg/kg in pediatric participants) SC, followed by 160 mg (or 2.2 mg/kg in pediatric participants) injections once weekly. RW period: eligible participants randomized to double-blinded administration of rilonacept 160 mg (or 2.2 mg/kg in pediatric participants) SC injections once weekly. Participants with pericarditis recurrence who meet the protocol criteria for bailout rilonacept (report at least 1 day with pericarditis pain ≥4 on the 11-point NRS and have 1 CRP value ≥ 1 mg/dL \[either on the same day or separated by no more than 7 days\]) receive bailout rilonacept (2 open-label injections of 160 mg rilonacept \[or 4.4 mg/kg for pediatric participants\]) irrespective of randomized treatment assignment and as soon as at least 5 days have passed since the last study drug injection. LTE period: open-label administration of rilonacept 160 mg (or 2.2 mg/kg in pediatric participants) SC injections once weekly.
Drug: Rilonacept
RI period: single-blind rilonacept 320 mg (or 4.4 mg/kg in pediatric participants) SC, followed by 160 mg (or 2.2 mg/kg in pediatric participants) injections once weekly. RW period: eligible participants randomized to placebo SC injections once weekly. Participants with pericarditis recurrence who meet the protocol criteria for bailout rilonacept (report at least 1 day with pericarditis pain ≥4 on the 11-point NRS and have 1 CRP value ≥ 1 mg/dL \[either on the same day or separated by no more than 7 days\]) receive bailout rilonacept (2 open-label injections of 160 mg rilonacept \[or 4.4 mg/kg for pediatric participants\]) irrespective of randomized treatment assignment and as soon as at least 5 days have passed since the last study drug injection. LTE period: open-label administration of rilonacept 160 mg (or 2.2 mg/kg in pediatric participants) SC injections once weekly.
Drug: Rilonacept · Drug: Placebo
Rilonacept 320 mg (or 4.4 mg/kg in pediatric participants ≥12 and \<18 years old) SC , followed by 160 mg (or 2.2 mg/kg in pediatric participants ≥12 and \<18 years old) injections once weekly
Also known as: KPL-914, Arcalyst® for Cryopyrin-Associated Periodic Syndromes (CAPS)
Placebo SC injections once weekly
Time to Pericarditis Recurrence in the RW Period
Time to pericarditis recurrence (from randomization to 1st recurrence). Kaplan-Meier. Clinical Events Committee (CEC)-confirmed recurrences used for primary analysis. Recurrence defined as recurrence typical pericarditis pain with supportive objective evidence. CEC-adjudicated recurrences defined as:1) Re-appearance/worsening pericarditis pain (1 NRS ≥ 4) AND elevated CRP (≥1.0 mg/dL) on same day/separated by ≤ 7 days OR 2) Re-appearance/worsening pericarditis pain (1 NRS ≥ 4) AND abnormal CRP (\> 0.5 mg/dL) on same day/separated by ≤ 7 days AND 1 supportive evidence OR 3) Re-appearance/worsening pericarditis pain (no NRS ≥ 4) AND elevated CRP (≥ 1.0 mg/dL) not attributable to other causes AND 1 supportive evidence. Supportive evidence: White blood cell count \> upper limit normal, fever \> 38C, pericardial rub, electrocardiogram changes consistent with pericarditis, new/worsening pericardial effusion (echocardiogram), new/worsening pericardial inflammation (magnetic resonance imaging).
Time frame: RW Period (mean 24.8 weeks)
Major Secondary Efficacy Endpoint: Percentage of Participants Who Maintained Clinical Response at Week 16 of the RW Period
Participants were asked to select the score that best describes their average level of pericarditis pain over the previous 24 hours using an 11-point numerical rating scale (NRS), where zero (0) indicates 'no pain' and ten (10) indicates 'pain as bad as it could be'. Clinical response was defined as a weekly average of daily pericarditis pain on the NRS ≤ 2.0 and C-reactive protein (CRP) level ≤ 0.5 mg/dL, and on monotherapy of randomized study drug at Week 16.
Time frame: RW Period Week 16
Major Secondary Efficacy Endpoint: Percentage of Days With No or Minimal Pericarditis Pain at Week 16 of the RW Period
Participants were asked to select the score that best describes their average level of pericarditis pain over the previous 24 hours using an 11-point numerical rating scale (NRS), where zero (0) indicates 'no pain' and ten (10) indicates 'pain as bad as it could be'. No or minimal pain was defined as non-missing NRS ≤ 2. The percentage of days with no or minimal pericarditis pain in the first 16 weeks was calculated for each participant using 16×7 as the denominator. Missing values in pain diary were counted as 0 day with no or minimal pain. Days of using oral rescue therapy or corticosteroid count as 0 day with no or minimal pain. If bailout rilonacept was used, each administration (loading dose or not) was counted as 7 days without qualifying no or minimal pain.
Time frame: RW Period Week 16
Major Secondary Efficacy Endpoint: Percentage of Participants With Absent or Minimal Pericarditis Symptoms Based on the Patient Global Impression of Pericarditis Severity (PGIPS) at Week 16 of the RW Period
Percentage of participants with no or minimal pericarditis symptoms at Week 16, based on the PGIPS, a single-item measure of the participant's impression of the overall severity of pericarditis symptoms at the time the questionnaire is administered using a 7-point rating scale ranging from absent (0=no recurrent pericarditis symptoms) to very severe (6=recurrent pericarditis symptoms that cannot be ignored and markedly limits daily activities). The exact 95% CI is calculated with randomization strata pooled. Participants who had received bailout rilonacept or rescue medication before the time point were considered nonresponders.
Time frame: RW Period Week 16
Percentage of Participants Who Maintained Clinical Response at Week 24 of the RW Period
Participants were asked to select the score that best describes their average level of pericarditis pain over the previous 24 hours using an 11-point numerical rating scale (NRS), where zero (0) indicates 'no pain' and ten (10) indicates 'pain as bad as it could be'. Clinical response was defined as a weekly average of daily pericarditis pain on the NRS ≤ 2.0 and C-reactive protein (CRP) level ≤ 0.5 mg/dL, and on monotherapy of randomized study drug at Week 24.
Time frame: RW Period Week 24
Percentage of Participants Who Maintained Clinical Response at Week 8 of the RW Period
Participants were asked to select the score that best describes their average level of pericarditis pain over the previous 24 hours using an 11-point numerical rating scale (NRS), where zero (0) indicates 'no pain' and ten (10) indicates 'pain as bad as it could be'. Clinical response was defined as a weekly average of daily pericarditis pain on the NRS ≤ 2.0 and C-reactive protein (CRP) level ≤ 0.5 mg/dL, and on monotherapy of randomized study drug at Week 8.
Time frame: RW Period Week 8
Percentage of Days With No or Minimal Pericarditis Pain at Week 24 of the RW Period
Participants were asked to select the score that best describes their average level of pericarditis pain over the previous 24 hours using an 11-point numerical rating scale (NRS), where zero (0) indicates 'no pain' and ten (10) indicates 'pain as bad as it could be'. No or minimal pain was defined as non-missing NRS ≤ 2. The percentage of days with no or minimal pericarditis pain in the first 24 weeks was calculated for each participant using 24×7 as the denominator. Missing values in pain diary were counted as 0 day with no or minimal pain. Days of using oral rescue therapy or corticosteroid count as 0 day with no or minimal pain. If bailout rilonacept was used, each administration (loading dose or not) was counted as 7 days without qualifying no or minimal pain.
Time frame: RW Period Week 24
Percentage of Days With No or Minimal Pericarditis Pain at Week 8 of the RW Period
Participants were asked to select the score that best describes their average level of pericarditis pain over the previous 24 hours using an 11-point numerical rating scale (NRS), where zero (0) indicates 'no pain' and ten (10) indicates 'pain as bad as it could be'. No or minimal pain was defined as non-missing NRS ≤ 2. The percentage of days with no or minimal pericarditis pain in the first 24 weeks was calculated for each participant using 8×7 as the denominator. Missing values in pain diary were counted as 0 day with no or minimal pain. Days of using oral rescue therapy or corticosteroid count as 0 day with no or minimal pain. If bailout rilonacept was used, each administration (loading dose or not) was counted as 7 days without qualifying no or minimal pain.
Time frame: RW Period Week 8
Percentage of Participants With Absent or Minimal Pericarditis Symptoms at Week 24 of the RW Period Based on the PGIPS
Percentage of participants with no or minimal pericarditis symptoms at Week 24, based on the PGIPS, a single-item measure of the participant's impression of the overall severity of pericarditis symptoms at the time the questionnaire is administered using a 7-point rating scale ranging from absent (0=no recurrent pericarditis symptoms) to very severe (6=recurrent pericarditis symptoms cannot be ignored and markedly limits daily activities). The exact 95% CI is calculated with randomization strata pooled. Participants who had received bailout rilonacept or rescue medication before the time point were considered nonresponders.
Time frame: RW Period Week 24
Percentage of Participants With Absent or Minimal Pericarditis Symptoms at Week 8 of the RW Period Based on the PGIPS
Percentage of participants with no or minimal pericarditis symptoms at Week 16, based on the Patient Global Impression of Pericarditis Severity (PGI-PS). The PGI-PS is a single-item measure of the participant's impression of the overall severity of pericarditis symptoms at the time the questionnaire is administered, using a 7-point rating scale ranging from absent (no recurrent pericarditis symptoms) to very severe (recurrent pericarditis symptoms cannot be ignored). The exact 95% CI is calculated with randomization strata pooled. Participants who had received bailout rilonacept or rescue medication before the time point were considered nonresponders.
Time frame: RW Period Week 8
Percentage of Participants Without Pericarditis Recurrence in the First 24 Weeks of the RW Period
Pericarditis recurrence is defined as the recurrence of typical pericarditis pain associated with supportive objective evidence of pericarditis. At any time during the RW period, participants who experienced a suspected recurrence of pericarditis symptoms reported to the study site/clinic for a scheduled or unscheduled visit, during which clinical assessments were performed to gather all the necessary diagnostic data to confirm or rule out the presence of pericarditis recurrence. A pericarditis recurrence event adjudication package was then prepared for adjudication by CEC. Kaplan-Meier estimate.
Time frame: up to 24 weeks in the RW Period
Time to Pericarditis Pain ≥ 4 on the NRS in the RW Period
Participants were asked to select the score that best described their average level of pericarditis pain over the previous 24 hours using an 11-point numerical rating scale (NRS), where zero (0) indicates 'no pain' and ten (10) indicates 'pain as bad as it could be'.
Time frame: RW Period (mean 24.8 weeks)
Time to CRP Level ≥ 1 mg/dL in the RW Period
Kaplan-Meier estimate.
Time frame: RW Period (mean 24.8 weeks)
Time to Pericardial Rub in the RW Period
Kaplan-Meier estimate. A pericardial rub (also called a pericardial friction rub) is an audible medical sign used in the diagnosis of pericarditis.
Time frame: RW Period (mean 24.8 weeks)
Time to Widespread ST-segment Elevation or PR-segment Depression on Electrocardiogram (ECG) in the RW Period
Kaplan-Meier estimate. ST-segment elevation and PR-segment depression are ECG changes in the evolution of acute pericarditis.
Time frame: RW Period (mean 24.8 weeks)
Time to New or Worsening Pericardial Effusion on Echocardiography (ECHO) in the RW Period
Pericardial effusion based on ECHO was evaluated by the central laboratory during the RW period. Kaplan-Meier estimate.
Time frame: RW Period (mean 24.8 weeks)
Number of Participants in ECHO Pericardial Effusion Size Categories at RW Period Baseline, RW Week 24 and Worst Post-baseline in the RW Period
Pericardial effusion based on ECHO was evaluated by the central laboratory during the RW period. "None or trivial/physiologic" is considered to be normal. The "RW Worst Post-baseline" category denotes the largest size of pericardial effusion category in which a participant was reported at any time during the RW period (post-baseline).
Time frame: RW Period Baseline, RW Period Week 24, RW Period (mean 24.8 weeks)
Change From RW Period Baseline Over Time in CRP Levels in RW Period
Estimated from ANCOVA models including treatment arm as fix effect, baseline value, baseline value by treatment interaction, randomization strata as covariates.
Time frame: RW Period Baseline, RW Period Weeks 4, 8, 12, 16, 20, 24, 32, 40, 48, 56
Change From RW Period Baseline Over Time in Weekly Average of Pericarditis Pain in the RW Period
Participants were asked to select the score that best described their average level of pericarditis pain over the previous 24 hours using an 11-point numerical rating scale (NRS), where zero (0) indicates 'no pain' and ten (10) indicates 'pain as bad as it could be'. Estimated from ANCOVA models including treatment arm as fix effect, baseline value, baseline value by treatment interaction, randomization strata as covariates.
Time frame: RW Period Baseline, RW Period Weeks 1-50, 54
Percentage of Participants With Absent or Minimal Pericarditis Symptoms Over Time After RW Period Week 24 Based on the PGIPS
The PGIPS, a single-item measure of the participant's impression of the overall severity of pericarditis symptoms at the time the questionnaire is administered using a 7-point rating scale ranging from absent (0=no recurrent pericarditis symptoms) to very severe (6=recurrent pericarditis symptoms cannot be ignored and markedly limits daily activities). The exact 95% CI is calculated with randomization strata pooled. Participants who had received bailout rilonacept or rescue medication before the time point were considered nonresponders.
Time frame: RW Period Weeks 32, 40, 48, 56
Percentage of Participants With Absent or Minimal Pericarditis Activity Over Time in the RW Period Based on the Physician Global Assessment of Pericarditis Activity (PGA-PA)
The PGA-PA is a single-item, clinician-reported outcome measure that Investigators use to rate their impression of the patient's overall pericarditis disease activity at the time the assessment is completed, using a rating scale ranging from absent to very severe. The Investigator selected the box that best described a participant's pericarditis activity at the time of occurrence of the assessment: Absent, Minimal, Mild, Moderate, Moderately Severe, Severe, Very Severe.
Time frame: RW Period Baseline, RW Period Weeks 8, 16, 24, 32, 40, 48, 56
Change From RW Period Baseline in Short Form-36 (SF-36) Physical and Mental Component Scores at RW Period Week 24
The SF-36 determines participants' overall quality of life by assessing 1) limitations in physical functioning due to health problems; 2) limitations in usual role because of physical health problems; 3) bodily pain; 4) general health perceptions; 5) vitality; 6) limitations in social functioning because of physical or emotional problems; 7) limitations in usual role due to emotional problems; and 8) general mental health. Items 1-4 primarily contribute to the physical component summary (PCS) score of the SF-36. Items 5-8 primarily contribute to the mental component summary (MCS) score of the SF-36. Scores on each item are summed and averaged (range: 0=worst to 100=best) with higher scores indicating better health. Increases from baseline indicate improvement.
Time frame: RW Period Baseline, RW Period Week 24
Change From RW Baseline in SF-36 Individual Scores at RW Period Week 24
The SF-36 determines participants' overall quality of life by assessing 1) limitations in physical functioning due to health problems; 2) limitations in usual role because of physical health problems; 3) bodily pain; 4) general health perceptions; 5) vitality; 6) limitations in social functioning because of physical or emotional problems; 7) limitations in usual role due to emotional problems; and 8) general mental health. Scores on each item are summed and averaged (range: 0=worst to 100=best) with higher scores indicating better health. Increases from baseline indicate improvement.
Time frame: RW Period Baseline, RW Period Week 24
Change From RW Period Baseline in the Short Form Health Survey-6 Domains (SF-6D) Utility Index Score at RW Period Week 24
The SF-6D is calculated based on responses to 11 items on the SF-36, that correspond to 6 domains: physical functioning, role participation (combined role-physical and role-emotional), social functioning, bodily pain, mental health, and vitality. Individual respondents can be classified on any of 4 to 6 levels of functioning or limitations for each of 6 domains, thus allowing a respondent to be classified into any of 18,000 possible unique health states. Using a standard gamble technique, each of these health states were mapped onto the SF-6D index score, which ranges from 0.00 (worst possible health state/death) to 1.00 (best possible health state/perfect health).
Time frame: RW Period Baseline, RW Period Week 24
Change From RW Period Baseline in 5-level EuroQoL-5 Dimensions (EQ-5D-5L) Individual Scores and Index Value to RW Period Week 24
The EQ-5D-5L is a self-reported health status questionnaire that consists of 6 questions used to calculate a health utility score for use in health economic analysis. There are 2 components to the EQ-5D-5L: a 5-item health state profile that assesses mobility, self-care, usual activities, pain/discomfort, and anxiety/depression used to obtain an Index Utility Score, as well as a visual analogue scale (VAS) that measures health state. Individual and index scores range from 0 to 1, with low scores representing a higher level of dysfunction. The VAS is designed to rate the participant's current health state on a scale from 0 to 100, where 0 represents the worst imaginable health state and 100 represents the best imaginable health state.
Time frame: RW Period Baseline, RW Period Week 24
Change From RW Period Baseline in Insomnia Severity Index (ISI) Total Score at RW Period Week 24
Participant's sleep quality was assessed with the ISI survey questionnaire. The ISI is a 7-item survey, with each question having 5 possible answers (none, mild, moderate, severe, or very severe), scored as 0, 1, 2, 3, or 4, respectively. Scores are summed for a total score, which ranges from 0 to 28. Lower scores are considered good, better, or healthy, and increasingly higher scores are considered to indicate greater insomnia. Clinical interpretation of the total score is as follows: 0 to 7 = no clinically significant insomnia; 8 to 14 = subthreshold insomnia; 15 to 21 = clinical insomnia (moderate severity); 22 to 28 = clinical insomnia (severe).
Time frame: RW Period Baseline, RW Period Week 24
Change in ISI Categories From RW Period Baseline to RW Period Week 24
Participant's sleep quality was assessed with the ISI survey questionnaire. The ISI is a 7-item survey, with each question having 5 possible answers (none, mild, moderate, severe, or very severe), scored as 0, 1, 2, 3, or 4, respectively. Scores are summed for a total score, which ranges from 0 to 28. Lower scores are considered good, better, or healthy, and increasingly higher scores are considered to indicate greater insomnia. Clinical interpretation of the total score is as follows: 0 to 7 = no clinically significant insomnia; 8 to 14 = subthreshold insomnia; 15 to 21 = clinical insomnia (moderate severity); 22 to 28 = clinical insomnia (severe).
Time frame: RW Period Baseline (BL), RW Period Week (Wk) 24
Percentage of Participants Using Oral Rescue Therapy (ORT), Corticosteroid, or Bailout Rilonacept for Pericarditis Every 4 Weeks Cumulatively in the RW Period
ORT included analgesics, NSAIDs, and/or colchicine. ORT use while waiting for at least 5 days since previous administration of study drug before receiving bailout, or within 5 days before the assessment of pericarditis recurrence, was excluded.
Time frame: RW Period (mean 24.8 weeks)
Percentage of Participants Using ORT for Pericarditis in the First 24 Weeks of RW Period
ORT included analgesics, NSAIDs, and/or colchicine.
Time frame: RW Period (up to Week 24)
Percentage of Participants With Pericardial Delayed Hyperenhancement, Myocardial Delayed Hyperenhancement or Pericardial Effusion on Magnetic Resonance Imaging (MRI) at RW Week 24
MRI assessments were performed in a subgroup of participants (MRI substudy) to assess the percentage of participants with: * Pericardial delayed hyperenhancement * Myocardial delayed hyperenhancement * Pericardial effusion
Time frame: RW Period Week 24
Time From First Dose to Pain Response in the RI Period
Participants were asked to select the score that best described their average level of pericarditis pain over the previous 24 hours using an 11-point numerical rating scale (NRS), where zero (0) indicates 'no pain' and ten (10) indicates 'pain as bad as it could be'. Time to pain response was defined as number of days from first dose to the first day a participant's daily pain NRS was ≤ 2 of the 3 days over which the rolling average daily pain NRS was ≤ 2.
Time frame: RI Period (up to 12 weeks)
Time From First Dose to CRP Normalization in the RI Period
Time to CRP normalization, defined as CRP ≤ 0.5 mg/dL, was censored at treatment discontinuation, taking prohibited medication, or Week 12, whichever occurred first.
Time frame: RI Period (up to 12 weeks)
Time From First Dose to Rilonacept Monotherapy in RI Period
Time to rilonacept monotherapy was defined as the number of weeks from first dose to the first day of achieving monotherapy.
Time frame: RI Period (up to 12 weeks)
Time From First Dose to Treatment Response in RI Period
Time to treatment response is defined as time from first dose to the first day of pain response, and CRP ≤ 0.5 mg/dL within 7 days before or after pain response. Treatment response day will be the first day that the above criterion is met. If pain response occurs before CRP ≤ 0.5 mg/dL, each 3-day rolling average of NRS should be ≤ 2.0 from the day of pain response to the day of CRP ≤ 0.5 mg/dL. The response day will be the day of pain response. If CRP ≤0.5 mg/dL occurs before pain response, the response day will also be the day of pain response.
Time frame: RI Period (up to 12 weeks)
Percentage of Participants Achieving Clinical Response at RI Period Week 12
Participants were asked to select the score that best described their average level of pericarditis pain over the previous 24 hours using an 11-point numerical rating scale (NRS), where zero (0) indicates 'no pain' and ten (10) indicates 'pain as bad as it could be'. Clinical Response was defined as a weekly average of daily pericarditis pain of ≤ 2.0 on the 11-point NRS and CRP level ≤ 0.5 mg/dL and participants must have been able to stop background SOC pericarditis therapy by Week 10.
Time frame: RI Period Week 12
Percentage of Participants With CRP Normalization at RI Period Week 12
CRP normalization was defined as CRP ≤ 0.5 mg/dL.
Time frame: RI Period Week 12
Change From Baseline Over Time in Weekly Average of Pericarditis Pain NRS Score in RI Period
Participants were asked to select the score that best described their average level of pericarditis pain over the previous 24 hours using an 11-point numerical rating scale (NRS), where zero (0) indicates 'no pain' and ten (10) indicates 'pain as bad as it could be'.
Time frame: RI Period Baseline, RI Period Weeks 1-12
Change From Baseline Over Time in CRP Levels in RI Period
Time frame: RI Period Baseline, RI Period Day 4, Weeks 1, 2, 4, 6, 12
Percentage of Participants With Resolution of Pericarditis-Related ECHO and ECG Abnormalities at Week 12 of the RI Period
Time frame: RI Period Baseline, RI Period Week 12
Percentage of Days With No or Minimal Pain in the RI Period While on Treatment
No or minimal pain is defined as non-missing daily NRS ≤ 2, where participants were asked to select the score that best describes their average level of pericarditis pain over the previous 24 hours using an 11-point NRS, where zero (0) indicates 'no pain' and ten (10) indicates 'pain as bad as it could be'.
Time frame: RI Period (up to Week 12)
Percentage of Participants With No or Minimal Pericarditis Symptoms Over Time in the RI Period, Based on the PGIPS
The PGIPS, a single-item measure of the participant's impression of the overall severity of pericarditis symptoms at the time the questionnaire is administered using a 7-point rating scale ranging from absent (0=no recurrent pericarditis symptoms) to very severe (6=recurrent pericarditis symptoms cannot be ignored and markedly limits daily activities). The exact 95% CI is calculated with randomization strata pooled. Participants who had received bailout rilonacept or rescue medication before the time point were considered nonresponders.
Time frame: RI Period Baseline, RI Period Weeks 6 and 12
Percentage of Participants With No or Minimal Pericarditis Activity Over Time in the RI Period, Based on the PGA-PA
The PGA-PA is a single-item, clinician-reported outcome measure that Investigators use to rate their impression of the patient's overall pericarditis disease activity at the time the assessment is completed, using a rating scale ranging from absent to very severe. The Investigator selected the box that best described a participant's pericarditis activity at the time of occurrence of the assessment: Absent, Minimal, Mild, Moderate, Moderately Severe, Severe, Very Severe. The exact 95% CI is calculated with randomization strata pooled. Participants who had received bailout rilonacept or rescue medication before the time point were considered nonresponders.
Time frame: RI Period Baseline, RI Period Weeks 6 and 12
Change From RI Period Baseline in the SF-36 Domain Scores and Physical and Mental Scores to RI Period Week 12
The SF-36 determines participants' overall quality of life by assessing 1) limitations in physical functioning due to health problems; 2) limitations in usual role because of physical health problems; 3) bodily pain; 4) general health perceptions; 5) vitality; 6) limitations in social functioning because of physical or emotional problems; 7) limitations in usual role due to emotional problems; and 8) general mental health. Scores on each item are summed and averaged (range: 0=worst to 100=best) with higher scores indicating better health. Increases from baseline indicate improvement.
Time frame: RI Period Baseline, RI Period Week 12
Change From RI Period Baseline in SF-6D Scores at RI Period Week 12
The SF-6D is calculated based on responses to 11 items on the SF-36, that correspond to 6 domains: physical functioning, role participation (combined role-physical and role-emotional), social functioning, bodily pain, mental health, and vitality. Individual respondents can be classified on any of 4 to 6 levels of functioning or limitations for each of 6 domains, thus allowing a respondent to be classified into any of 18,000 possible unique health states. Using a standard gamble technique, each of these health states were mapped onto the SF-6D index score, which ranges from 0.00 (worst possible health state/death) to 1.00 (best possible health state/perfect health).
Time frame: RI Period Baseline, RI Period Week 12
Change From RI Period Baseline in 5-level EuroQoL-5 Dimensions (EQ-5D-5L) Individual Scores and Index Value at RI Period Week 12
The EQ-5D-5L is a self-reported health status questionnaire that consists of 6 questions used to calculate a health utility score for use in health economic analysis. There are 2 components to the EQ-5D-5L: a 5-item health state profile that assesses mobility, self-care, usual activities, pain/discomfort, and anxiety/depression used to obtain an Index Utility Score, as well as a visual analogue scale (VAS) that measures health state. Individual and index scores range from 0 to 1, with low scores representing a higher level of dysfunction. The VAS is designed to rate the participant's current health state on a scale from 0 to 100, where 0 represents the worst imaginable health state and 100 represents the best imaginable health state.
Time frame: RI Period Baseline, RI Period Week 12
Change From RI Period Baseline in ISI Total Score at RI Period Week 12
Participant's sleep quality was assessed with the ISI survey questionnaire. The ISI is a 7-item survey, with each question having 5 possible answers (none, mild, moderate, severe, or very severe), scored as 0, 1, 2, 3, or 4, respectively. Scores are summed for a total score, which ranges from 0 to 28. Lower scores are considered good, better, or healthy, and increasingly higher scores are considered to indicate greater insomnia. Clinical interpretation of the total score is as follows: 0 to 7 = no clinically significant insomnia; 8 to 14 = subthreshold insomnia; 15 to 21 = clinical insomnia (moderate severity); 22 to 28 = clinical insomnia (severe).
Time frame: RI Period Baseline, RI Period Week 12
Change in ISI Categories From RI Period Baseline to RI Period Week 12
Participant's sleep quality was assessed with the ISI survey questionnaire. The ISI is a 7-item survey, with each question having 5 possible answers (none, mild, moderate, severe, or very severe), scored as 0, 1, 2, 3, or 4, respectively. Scores are summed for a total score, which ranges from 0 to 28. Lower scores are considered good, better, or healthy, and increasingly higher scores are considered to indicate greater insomnia. Clinical interpretation of the total score is as follows: 0 to 7 = no clinically significant insomnia; 8 to 14 = subthreshold insomnia; 15 to 21 = clinical insomnia (moderate severity); 22 to 28 = clinical insomnia (severe).
Time frame: RI Period Baseline (BL), RI Period Week (Wk) 12
Number of Participants Who Were Off Background Pericarditis Medication on or Before RI Period Weeks 4, 8, 10, and 12
Time frame: RI Period Baseline, RI Period Weeks 4, 8, 10, 12
Annualized Rate of Pericarditis Recurrence in the Long-Term Extension (LTE) Period Based on Investigator's Assessment (Based on Investigators' Judgement)
Annualized Recurrence Rate is defined as the number of recurrences in LTE periods for all participants/Sum of participant years in LTE periods for all participants. Participant years in LTE period is defined as the time from 1st dose date of LTE period to the date of End of Study, or data cutoff date, whichever is earlier. The 95% CI was calculated using an exact method with Poisson distribution. Pericarditis recurrence is defined as the recurrence of typical pericarditis pain associated with supportive objective evidence of pericarditis.
Time frame: LTE Period, through LTE Follow up (up to Week 48)
Change From LTE Baseline Over Time in CRP Levels
Time frame: LTE Baseline, LTE Week 12, LTE Week 24
Percentage of Participants With Absent or Minimal Pericarditis Activity In the LTE Period Based on the PGIPS
Percentage of participants with no or minimal pericarditis symptoms in the LTE Period, based on the PGIPS, a single-item measure of the participant's impression of the overall severity of pericarditis symptoms at the time the questionnaire is administered using a 7-point rating scale ranging from absent (0=no recurrent pericarditis symptoms) to very severe (6=recurrent pericarditis symptoms that cannot be ignored and markedly limits daily activities).
Time frame: LTE Baseline, LTE Month 12, LTE Month 24
Percentage of Participants With Absent or Minimal Pericarditis Activity Over Time in the LTE Period Based on the PGA-PA
The PGA-PA is a single-item, clinician-reported outcome measure that Investigators use to rate their impression of the patient's overall pericarditis disease activity at the time the assessment is completed, using a rating scale ranging from absent to very severe. The Investigator selected the box that best described a participant's pericarditis activity at the time of occurrence of the assessment: Absent, Minimal, Mild, Moderate, Moderately Severe, Severe, Very Severe.
Time frame: LTE Baseline, LTE Week 12, LTE Week 24
Change From LTE Baseline in the SF-36 Domain Scores and Physical and Mental Scores
The SF-36 determines participants' overall quality of life by assessing 1) limitations in physical functioning due to health problems; 2) limitations in usual role because of physical health problems; 3) bodily pain; 4) general health perceptions; 5) vitality; 6) limitations in social functioning because of physical or emotional problems; 7) limitations in usual role due to emotional problems; and 8) general mental health. Scores on each item are summed and averaged (range: 0=worst to 100=best) with higher scores indicating better health. Increases from baseline indicate improvement.
Time frame: LTE Baseline, LTE Week 24
Change From LTE Baseline in SF-6D Health Utility Index Score
The SF-6D is calculated based on responses to 11 items on the SF-36, that correspond to 6 domains: physical functioning, role participation (combined role-physical and role-emotional), social functioning, bodily pain, mental health, and vitality. Individual respondents can be classified on any of 4 to 6 levels of functioning or limitations for each of six domains, thus allowing a respondent to be classified into any of 18,000 possible unique health states. Using a standard gamble technique, each of these health states were mapped onto the SF-6D index score, which ranges from 0.00 (worst possible health state/death) to 1.00 (best possible health state/perfect health).
Time frame: LTE Baseline, LTE Week 24
Change From LTE Baseline in EQ-5D-5L Individual Scores and Index Value
The EQ-5D-5L is a self-reported health status questionnaire that consists of 6 questions used to calculate a health utility score for use in health economic analysis. There are 2 components to the EQ-5D-5L: a 5-item health state profile that assesses mobility, self-care, usual activities, pain/discomfort, and anxiety/depression used to obtain an Index Utility Score, as well as a visual analogue scale (VAS) that measures health state. Individual and index scores range from 0 to 1, with low scores representing a higher level of dysfunction. The VAS is designed to rate the participant's current health state on a scale from 0 to 100, where 0 represents the worst imaginable health state and 100 represents the best imaginable health state.
Time frame: LTE Baseline, LTE Week 24
Change From LTE Baseline in ISI Total Score
Participant's sleep quality was assessed with the ISI survey questionnaire. The ISI is a 7-item survey, with each question having 5 possible answers (none, mild, moderate, severe, or very severe), scored as 0, 1, 2, 3, or 4, respectively. Scores are summed for a total score, which ranges from 0 to 28. Lower scores are considered good, better, or healthy, and increasingly higher scores are considered to indicate greater insomnia. Clinical interpretation of the total score is as follows: 0 to 7 = no clinically significant insomnia; 8 to 14 = subthreshold insomnia; 15 to 21 = clinical insomnia (moderate severity); 22 to 28 = clinical insomnia (severe).
Time frame: LTE Baseline, LTE Week 24
Change From LTE Baseline in ISI Categories
Participant's sleep quality was assessed with the ISI survey questionnaire. The ISI is a 7-item survey, with each question having 5 possible answers (none, mild, moderate, severe, or very severe), scored as 0, 1, 2, 3, or 4, respectively. Scores are summed for a total score, which ranges from 0 to 28. Lower scores are considered good, better, or healthy, and increasingly higher scores are considered to indicate greater insomnia. Clinical interpretation of the total score is as follows: 0 to 7 = no clinically significant insomnia; 8 to 14 = subthreshold insomnia; 15 to 21 = clinical insomnia (moderate severity); 22 to 28 = clinical insomnia (severe).
Time frame: LTE Baseline (BL), LTE Week 24
Percentage of Participants Requiring Addition of Standard of Care (SOC) Pericarditis Therapy Every 4 Weeks Cumulatively in the LTE Period
Time frame: LTE Period, through LTE Follow up (up to Week 24)
Change From LTE Baseline in Pericardial Signs in ECHO
Time frame: LTE Baseline, LTE Week 24
Change From LTE Baseline in Pericardial Signs in ECG
Time frame: LTE Baseline, LTE Week 24
Change From LTE Baseline in Pericardial Signs in MRI
Time frame: LTE Baseline, LTE Week 24
Number of Participants With Pericardial Delayed Hyperenhancement, Myocardial Delayed Hyperenhancement or Pericardial Effusion at LTE Period Week 24
Time frame: LTE Week 24
Annualized Rate of Pericarditis Recurrence in LTE Periods Based on Investigator's Assessment
Annualized Recurrence Rate is defined as number of recurrences in LTE periods for all participants/Sum of participant years in LTE period for all participants. Participant years in LTE period is defined as the time from 1st dose date of LTE period to the date of End of Study, or data cutoff date, whichever is earlier. The 95% CI was calculated using an exact method with Poisson distribution. Pericarditis recurrence was defined as the recurrence of typical pericarditis pain associated with supportive objective evidence of pericarditis. At any time during the RW period, participants who experienced a suspected recurrence of pericarditis symptoms reported to the study site/clinic for a scheduled or unscheduled visit, during which clinical assessments were performed to gather all the necessary diagnostic data to confirm or rule out the presence of pericarditis recurrence.
Time frame: LTE Period, through LTE Follow up (up to Week 48)
Annualized Rate of Pericarditis Recurrence in RW Period Based on CEC Adjudication
Annualized Recurrence Rate is defined as the number of recurrences in RW period for all participants/Sum of participant years in RW period for all participants. Participant years in RW period is defined as the time from randomization date to the date of EORW or last dose date + 6 weeks, whichever is earlier. The 95% CI was calculated using an exact method with Poisson distribution. Pericarditis recurrence was defined as the recurrence of typical pericarditis pain associated with supportive objective evidence of pericarditis. At any time during the RW period, participants who experienced a suspected recurrence of pericarditis symptoms reported to the study site/clinic for a scheduled or unscheduled visit, during which clinical assessments were performed to gather all the necessary diagnostic data to confirm or rule out the presence of pericarditis recurrence.
Time frame: RW Period (mean 24.8 weeks)
| Milestone | Run-in Period | Randomized Withdrawal Period: Rilonacept | Randomized Withdrawal Period: Placebo | Long Term Extension: Non-Randomized | Long Term Extension: Randomized, Recurrence (RW) | Long Term Extension: Randomized, No Recurrence (RW) |
|---|---|---|---|---|---|---|
| Started | 86 | 0 | 0 | 0 | 0 | 0 |
| Continue to rw period | 61 | 0 | 0 | 0 | 0 | 0 |
| Continue directly to lte period | 15 | 0 | 0 | 0 | 0 | 0 |
| Completed | 76 | 0 | 0 | 0 | 0 | 0 |
| Not completed | 10 | 0 | 0 | 0 | 0 | 0 |
| Withdrew: Lack of efficacy | 3 | 0 | 0 | 0 | 0 | 0 |
| Withdrew: Did not meet clinical response criteria | 3 | 0 | 0 | 0 | 0 | 0 |
| Withdrew: Adverse event | 4 | 0 | 0 | 0 | 0 | 0 |
| Milestone | Run-in Period | Randomized Withdrawal Period: Rilonacept | Randomized Withdrawal Period: Placebo | Long Term Extension: Non-Randomized | Long Term Extension: Randomized, Recurrence (RW) | Long Term Extension: Randomized, No Recurrence (RW) |
|---|---|---|---|---|---|---|
| Started | 0 | 30 | 31 | 0 | 0 | 0 |
| Completed | 0 | 29 | 30 | 0 | 0 | 0 |
| Not completed | 0 | 1 | 1 | 0 | 0 | 0 |
| Withdrew: Completed, not continuing into lte | 0 | 1 | 0 | 0 | 0 | 0 |
| Withdrew: Withdrawal by subject | 0 | 0 | 1 | 0 | 0 | 0 |
| Milestone | Run-in Period | Randomized Withdrawal Period: Rilonacept | Randomized Withdrawal Period: Placebo | Long Term Extension: Non-Randomized | Long Term Extension: Randomized, Recurrence (RW) | Long Term Extension: Randomized, No Recurrence (RW) |
|---|---|---|---|---|---|---|
| Started | 0 | 0 | 0 | 15 | 25 | 34 |
| Completed | 0 | 0 | 0 | 13 | 24 | 32 |
| Not completed | 0 | 0 | 0 | 2 | 1 | 2 |
| Withdrew: Lost to follow-up | 0 | 0 | 0 | 1 | 0 | 0 |
| Withdrew: Withdrawal by subject | 0 | 0 | 0 | 1 | 0 | 2 |
| Withdrew: Investigator decision due to serious adverse event | 0 | 0 | 0 | 0 | 1 | 0 |
Time to pericarditis recurrence (from randomization to 1st recurrence). Kaplan-Meier. Clinical Events Committee (CEC)-confirmed recurrences used for primary analysis. Recurrence defined as recurrence typical pericarditis pain with supportive objective evidence. CEC-adjudicated recurrences defined as:1) Re-appearance/worsening pericarditis pain (1 NRS ≥ 4) AND elevated CRP (≥1.0 mg/dL) on same day/separated by ≤ 7 days OR 2) Re-appearance/worsening pericarditis pain (1 NRS ≥ 4) AND abnormal CRP (\> 0.5 mg/dL) on same day/separated by ≤ 7 days AND 1 supportive evidence OR 3) Re-appearance/worsening pericarditis pain (no NRS ≥ 4) AND elevated CRP (≥ 1.0 mg/dL) not attributable to other causes AND 1 supportive evidence. Supportive evidence: White blood cell count \> upper limit normal, fever \> 38C, pericardial rub, electrocardiogram changes consistent with pericarditis, new/worsening pericardial effusion (echocardiogram), new/worsening pericardial inflammation (magnetic resonance imaging).
| weeks | Randomized Withdrawal Period: Rilonacept | Randomized Withdrawal Period: Placebo |
|---|---|---|
| Time to Pericarditis Recurrence in the RW Period | NA (NA to NA) | 8.6 (4.0 to 11.7) |
Participants were asked to select the score that best describes their average level of pericarditis pain over the previous 24 hours using an 11-point numerical rating scale (NRS), where zero (0) indicates 'no pain' and ten (10) indicates 'pain as bad as it could be'. Clinical response was defined as a weekly average of daily pericarditis pain on the NRS ≤ 2.0 and C-reactive protein (CRP) level ≤ 0.5 mg/dL, and on monotherapy of randomized study drug at Week 16.
| percentage of participants | Randomized Withdrawal Period: Rilonacept | Randomized Withdrawal Period: Placebo |
|---|---|---|
| Major Secondary Efficacy Endpoint: Percentage of Participants Who Maintained Clinical Response at Week 16 of the RW Period | 81.0 (58.1 to 94.6) | 20.0 (5.7 to 43.7) |
Participants were asked to select the score that best describes their average level of pericarditis pain over the previous 24 hours using an 11-point numerical rating scale (NRS), where zero (0) indicates 'no pain' and ten (10) indicates 'pain as bad as it could be'. No or minimal pain was defined as non-missing NRS ≤ 2. The percentage of days with no or minimal pericarditis pain in the first 16 weeks was calculated for each participant using 16×7 as the denominator. Missing values in pain diary were counted as 0 day with no or minimal pain. Days of using oral rescue therapy or corticosteroid count as 0 day with no or minimal pain. If bailout rilonacept was used, each administration (loading dose or not) was counted as 7 days without qualifying no or minimal pain.
| percentage of days | Randomized Withdrawal Period: Rilonacept | Randomized Withdrawal Period: Placebo |
|---|---|---|
| Major Secondary Efficacy Endpoint: Percentage of Days With No or Minimal Pericarditis Pain at Week 16 of the RW Period | 98.6 ± 7.55 | 47.4 ± 7.26 |
Percentage of participants with no or minimal pericarditis symptoms at Week 16, based on the PGIPS, a single-item measure of the participant's impression of the overall severity of pericarditis symptoms at the time the questionnaire is administered using a 7-point rating scale ranging from absent (0=no recurrent pericarditis symptoms) to very severe (6=recurrent pericarditis symptoms that cannot be ignored and markedly limits daily activities). The exact 95% CI is calculated with randomization strata pooled. Participants who had received bailout rilonacept or rescue medication before the time point were considered nonresponders.
| percentage of participants | Randomized Withdrawal Period: Rilonacept | Randomized Withdrawal Period: Placebo |
|---|---|---|
| Major Secondary Efficacy Endpoint: Percentage of Participants With Absent or Minimal Pericarditis Symptoms Based on the Patient Global Impression of Pericarditis Severity (PGIPS) at Week 16 of the RW Period | 81.0 (58.1 to 94.6) | 25.0 (8.7 to 49.1) |
Participants were asked to select the score that best describes their average level of pericarditis pain over the previous 24 hours using an 11-point numerical rating scale (NRS), where zero (0) indicates 'no pain' and ten (10) indicates 'pain as bad as it could be'. Clinical response was defined as a weekly average of daily pericarditis pain on the NRS ≤ 2.0 and C-reactive protein (CRP) level ≤ 0.5 mg/dL, and on monotherapy of randomized study drug at Week 24.
| percentage of participants | Randomized Withdrawal Period: Rilonacept | Randomized Withdrawal Period: Placebo |
|---|---|---|
| Percentage of Participants Who Maintained Clinical Response at Week 24 of the RW Period | 76.5 (50.1 to 93.2) | 20.0 (4.3 to 48.1) |
Participants were asked to select the score that best describes their average level of pericarditis pain over the previous 24 hours using an 11-point numerical rating scale (NRS), where zero (0) indicates 'no pain' and ten (10) indicates 'pain as bad as it could be'. Clinical response was defined as a weekly average of daily pericarditis pain on the NRS ≤ 2.0 and C-reactive protein (CRP) level ≤ 0.5 mg/dL, and on monotherapy of randomized study drug at Week 8.
| percentage of participants | Randomized Withdrawal Period: Rilonacept | Randomized Withdrawal Period: Placebo |
|---|---|---|
| Percentage of Participants Who Maintained Clinical Response at Week 8 of the RW Period | 77.8 (57.7 to 91.4) | 25.8 (11.9 to 44.6) |
Participants were asked to select the score that best describes their average level of pericarditis pain over the previous 24 hours using an 11-point numerical rating scale (NRS), where zero (0) indicates 'no pain' and ten (10) indicates 'pain as bad as it could be'. No or minimal pain was defined as non-missing NRS ≤ 2. The percentage of days with no or minimal pericarditis pain in the first 24 weeks was calculated for each participant using 24×7 as the denominator. Missing values in pain diary were counted as 0 day with no or minimal pain. Days of using oral rescue therapy or corticosteroid count as 0 day with no or minimal pain. If bailout rilonacept was used, each administration (loading dose or not) was counted as 7 days without qualifying no or minimal pain.
| percentage of days | Randomized Withdrawal Period: Rilonacept | Randomized Withdrawal Period: Placebo |
|---|---|---|
| Percentage of Days With No or Minimal Pericarditis Pain at Week 24 of the RW Period | 100.6 ± 8.02 | 48.7 ± 7.64 |
Participants were asked to select the score that best describes their average level of pericarditis pain over the previous 24 hours using an 11-point numerical rating scale (NRS), where zero (0) indicates 'no pain' and ten (10) indicates 'pain as bad as it could be'. No or minimal pain was defined as non-missing NRS ≤ 2. The percentage of days with no or minimal pericarditis pain in the first 24 weeks was calculated for each participant using 8×7 as the denominator. Missing values in pain diary were counted as 0 day with no or minimal pain. Days of using oral rescue therapy or corticosteroid count as 0 day with no or minimal pain. If bailout rilonacept was used, each administration (loading dose or not) was counted as 7 days without qualifying no or minimal pain.
| percentage of days | Randomized Withdrawal Period: Rilonacept | Randomized Withdrawal Period: Placebo |
|---|---|---|
| Percentage of Days With No or Minimal Pericarditis Pain at Week 8 of the RW Period | 97.9 ± 7.64 | 57.3 ± 7.19 |
Percentage of participants with no or minimal pericarditis symptoms at Week 24, based on the PGIPS, a single-item measure of the participant's impression of the overall severity of pericarditis symptoms at the time the questionnaire is administered using a 7-point rating scale ranging from absent (0=no recurrent pericarditis symptoms) to very severe (6=recurrent pericarditis symptoms cannot be ignored and markedly limits daily activities). The exact 95% CI is calculated with randomization strata pooled. Participants who had received bailout rilonacept or rescue medication before the time point were considered nonresponders.
| percentage of participants | Randomized Withdrawal Period: Rilonacept | Randomized Withdrawal Period: Placebo |
|---|---|---|
| Percentage of Participants With Absent or Minimal Pericarditis Symptoms at Week 24 of the RW Period Based on the PGIPS | 88.2 (63.6 to 98.5) | 20.0 (4.3 to 48.1) |
Percentage of participants with no or minimal pericarditis symptoms at Week 16, based on the Patient Global Impression of Pericarditis Severity (PGI-PS). The PGI-PS is a single-item measure of the participant's impression of the overall severity of pericarditis symptoms at the time the questionnaire is administered, using a 7-point rating scale ranging from absent (no recurrent pericarditis symptoms) to very severe (recurrent pericarditis symptoms cannot be ignored). The exact 95% CI is calculated with randomization strata pooled. Participants who had received bailout rilonacept or rescue medication before the time point were considered nonresponders.
| percentage of participants | Randomized Withdrawal Period: Rilonacept | Randomized Withdrawal Period: Placebo |
|---|---|---|
| Percentage of Participants With Absent or Minimal Pericarditis Symptoms at Week 8 of the RW Period Based on the PGIPS | 85.2 (66.3 to 95.8) | 32.3 (16.7 to 51.4) |
Pericarditis recurrence is defined as the recurrence of typical pericarditis pain associated with supportive objective evidence of pericarditis. At any time during the RW period, participants who experienced a suspected recurrence of pericarditis symptoms reported to the study site/clinic for a scheduled or unscheduled visit, during which clinical assessments were performed to gather all the necessary diagnostic data to confirm or rule out the presence of pericarditis recurrence. A pericarditis recurrence event adjudication package was then prepared for adjudication by CEC. Kaplan-Meier estimate.
| percentage of participants | Randomized Withdrawal Period: Rilonacept | Randomized Withdrawal Period: Placebo |
|---|---|---|
| Percentage of Participants Without Pericarditis Recurrence in the First 24 Weeks of the RW Period | 95.7 (72.9 to 99.4) | 19.9 (6.5 to 38.6) |
Participants were asked to select the score that best described their average level of pericarditis pain over the previous 24 hours using an 11-point numerical rating scale (NRS), where zero (0) indicates 'no pain' and ten (10) indicates 'pain as bad as it could be'.
| weeks | Randomized Withdrawal Period: Rilonacept | Randomized Withdrawal Period: Placebo |
|---|---|---|
| Time to Pericarditis Pain ≥ 4 on the NRS in the RW Period | NA (33.1 to NA) | 4.1 (2.9 to 6.6) |
Kaplan-Meier estimate.
| weeks | Randomized Withdrawal Period: Rilonacept | Randomized Withdrawal Period: Placebo |
|---|---|---|
| Time to CRP Level ≥ 1 mg/dL in the RW Period | NA (NA to NA) | 6.9 (4.0 to 10.1) |
Kaplan-Meier estimate. A pericardial rub (also called a pericardial friction rub) is an audible medical sign used in the diagnosis of pericarditis.
| weeks | Randomized Withdrawal Period: Rilonacept | Randomized Withdrawal Period: Placebo |
|---|---|---|
| Time to Pericardial Rub in the RW Period | NA (NA to NA) | NA (NA to NA) |
Kaplan-Meier estimate. ST-segment elevation and PR-segment depression are ECG changes in the evolution of acute pericarditis.
| weeks | Randomized Withdrawal Period: Rilonacept | Randomized Withdrawal Period: Placebo |
|---|---|---|
| Time to Widespread ST-segment Elevation or PR-segment Depression on Electrocardiogram (ECG) in the RW Period | NA (NA to NA) | NA (NA to NA) |
Pericardial effusion based on ECHO was evaluated by the central laboratory during the RW period. Kaplan-Meier estimate.
| weeks | Randomized Withdrawal Period: Rilonacept | Randomized Withdrawal Period: Placebo |
|---|---|---|
| Time to New or Worsening Pericardial Effusion on Echocardiography (ECHO) in the RW Period | NA (NA to NA) | NA (NA to NA) |
Pericardial effusion based on ECHO was evaluated by the central laboratory during the RW period. "None or trivial/physiologic" is considered to be normal. The "RW Worst Post-baseline" category denotes the largest size of pericardial effusion category in which a participant was reported at any time during the RW period (post-baseline).
| Participants | Randomized Withdrawal Period: Rilonacept, Before Bailout Rilonacept | Randomized Withdrawal Period: Rilonacept, Including Bailout Rilonacept | Randomized Withdrawal Period: Placebo, Before Bailout Rilonacept | Randomized Withdrawal Period: Placebo, Including Bailout Rilonacept |
|---|---|---|---|---|
| RW Baseline — None or Trivial/Physiologic | 28 | 28 | 30 | 30 |
| RW Baseline — Small | 1 | 1 | 1 | 1 |
| RW Baseline — Moderate | 1 | 1 | 0 | 0 |
| RW Baseline — Large | 0 | 0 | 0 | 0 |
| RW Baseline — Very Large | 0 | 0 | 0 | 0 |
| RW Baseline — Missing/Not Done | 0 | 0 | 0 | 0 |
| RW Week 24 — None or Trivial/Physiologic | 12 | 12 | 3 | 13 |
| RW Week 24 — Small | 0 | 0 | 0 | 0 |
| RW Week 24 — Moderate | 0 | 0 | 0 | 0 |
| RW Week 24 — Large | 0 | 0 | 0 | 0 |
| RW Week 24 — Very Large | 0 | 0 | 0 | 0 |
| RW Week 24 — Missing/Not Done | 0 | 0 | 0 | 0 |
| RW Worst Post-baseline — None or Trivial/Physiologic | 28 | 28 | 14 | 20 |
| RW Worst Post-baseline — Small | 0 | 0 | 4 | 7 |
| RW Worst Post-baseline — Moderate | 0 | 0 | 1 | 1 |
| RW Worst Post-baseline — Large | 0 | 0 | 0 | 0 |
| RW Worst Post-baseline — Very Large | 0 | 0 | 0 | 0 |
| RW Worst Post-baseline — Missing/Not Done | 0 | 0 | 0 | 0 |
Estimated from ANCOVA models including treatment arm as fix effect, baseline value, baseline value by treatment interaction, randomization strata as covariates.
| mg/dL | Randomized Withdrawal Period: Rilonacept, Before Bailout Rilonacept | Randomized Withdrawal Period: Rilonacept, Including Bailout Rilonacept | Randomized Withdrawal Period: Placebo, Before Bailout Rilonacept | Randomized Withdrawal Period: Placebo, Including Bailout Rilonacept |
|---|---|---|---|---|
| Change at RW Week 4 | -0.01 ± 0.058 | -0.05 ± 1.112 | 0.15 ± 0.061 | 1.46 ± 0.861 |
| Change at RW Week 8 | 0.12 ± 0.308 | 0.11 ± 0.300 | 0.45 ± 0.331 | 0.25 ± 0.246 |
| Change at RW Week 12 | 0.04 ± 0.034 | -0.04 ± 0.085 | 0.03 ± 0.043 | 0.08 ± 0.077 |
| Change at RW Week 16 | 0.40 ± 0.148 | 0.27 ± 0.119 | 0.04 ± 0.227 | 0.18 ± 0.109 |
| Change at RW Week 20 | -0.04 ± 0.048 | -0.10 ± 0.181 | 0.10 ± 0.083 | 0.16 ± 0.174 |
| Change at RW Week 24 | -0.04 ± 0.032 | -0.05 ± 0.047 | 0.06 ± 0.056 | 0.02 ± 0.045 |
| Change at RW Week 32 | -0.05 ± 0.016 | -0.02 ± 0.059 | NA ± NA | 0.07 ± 0.055 |
| Change at RW Week 40 | 0.04 ± 0.010 | 0.08 ± 0.298 | NA ± NA | 0.74 ± 0.290 |
| Change at RW Week 48 | 0.02 ± 0.000 | -0.03 ± 0.008 | — | 0.06 ± 0.017 |
| Change at RW Week 56 | — | — | — | 0.00 ± NA |
Participants were asked to select the score that best described their average level of pericarditis pain over the previous 24 hours using an 11-point numerical rating scale (NRS), where zero (0) indicates 'no pain' and ten (10) indicates 'pain as bad as it could be'. Estimated from ANCOVA models including treatment arm as fix effect, baseline value, baseline value by treatment interaction, randomization strata as covariates.
| units on a scale | Randomized Withdrawal Period: Rilonacept, Before Bailout Rilonacept | Randomized Withdrawal Period: Rilonacept, Including Bailout Rilonacept | Randomized Withdrawal Period: Placebo, Before Bailout Rilonacept | Randomized Withdrawal Period: Placebo, Including Bailout Rilonacept |
|---|---|---|---|---|
| Change at RW Week 1 | -0.08 ± 0.103 | -0.08 ± 0.103 | 0.17 ± 0.099 | 0.17 ± 0.099 |
| Change at RW Week 2 | -0.21 ± 0.237 | -0.25 ± 0.273 | 0.57 ± 0.231 | 0.68 ± 0.263 |
| Change at RW Week 3 | -0.08 ± 0.296 | -0.15 ± 0.330 | 1.12 ± 0.278 | 1.29 ± 0.302 |
| Change at RW Week 4 | -0.16 ± 0.322 | -0.11 ± 0.468 | 1.05 ± 0.333 | 1.84 ± 0.428 |
| Change at RW Week 5 | -0.16 ± 0.314 | -0.21 ± 0.351 | 1.27 ± 0.332 | 1.34 ± 0.320 |
| Change at RW Week 6 | 0.22 ± 0.296 | 0.02 ± 0.310 | 0.96 ± 0.290 | 0.84 ± 0.265 |
| Change at RW Week 7 | 0.06 ± 0.273 | 0.12 ± 0.248 | 0.87 ± 0.304 | 0.86 ± 0.224 |
| Change at RW Week 8 | 0.10 ± 0.226 | 0.11 ± 0.205 | 0.77 ± 0.253 | 0.58 ± 0.189 |
| Change at RW Week 9 | -0.24 ± 0.310 | 0.05 ± 0.320 | 1.45 ± 0.402 | 1.10 ± 0.301 |
| Change at RW Week 10 | -0.17 ± 0.270 | -0.21 ± 0.262 | 1.11 ± 0.343 | 0.62 ± 0.240 |
| Change at RW Week 11 | -0.20 ± 0.200 | -0.16 ± 0.272 | 0.43 ± 0.267 | 0.46 ± 0.250 |
| Change at RW Week 12 | 0.07 ± 0.174 | -0.03 ± 0.248 | 0.36 ± 0.235 | 0.32 ± 0.225 |
| Change at RW Week 13 | -0.02 ± 0.331 | 0.06 ± 0.256 | 0.25 ± 0.386 | 0.07 ± 0.220 |
| Change at RW Week 14 | 0.14 ± 0.186 | 0.08 ± 0.185 | 0.24 ± 0.262 | 0.06 ± 0.170 |
| Change at RW Week 15 | 0.32 ± 0.109 | 0.19 ± 0.149 | -0.08 ± 0.235 | 0.15 ± 0.136 |
| Change at RW Week 16 | 0.12 ± 0.110 | -0.07 ± 0.167 | NA ± NA | 0.09 ± 0.153 |
| Change at RW Week 17 | 0.19 ± 0.129 | 0.01 ± 0.183 | NA ± NA | 0.05 ± 0.165 |
| Change at RW Week 18 | 0.13 ± 0.149 | 0.05 ± 0.157 | NA ± NA | -0.05 ± 0.131 |
| Change at RW Week 19 | 0.04 ± 0.149 | -0.05 ± 0.167 | NA ± NA | -0.04 ± 0.139 |
| Change at RW Week 20 | -0.08 ± 0.111 | -0.22 ± 0.228 | NA ± NA | 0.30 ± 0.226 |
| Change at RW Week 21 | 0.04 ± 0.156 | 0.02 ± 0.162 | NA ± NA | 0.23 ± 0.160 |
| Change at RW Week 22 | -0.08 ± 0.097 | -0.12 ± 0.124 | NA ± NA | 0.01 ± 0.120 |
| Change at RW Week 23 | -0.01 ± 0.103 | -0.03 ± 0.145 | NA ± NA | -0.01 ± 0.140 |
| Change at RW Week 24 | 0.02 ± 0.122 | -0.04 ± 0.138 | NA ± NA | 0.02 ± 0.132 |
| Change at RW Week 25 | 0.12 ± 0.122 | 0.09 ± 0.109 | NA ± NA | 0.13 ± 0.110 |
| Change at RW Week 26 | 0.19 ± 0.145 | 0.16 ± 0.127 | NA ± NA | 0.29 ± 0.131 |
| Change at RW Week 27 | 0.23 ± 0.193 | 0.16 ± 0.154 | NA ± NA | 0.15 ± 0.157 |
| Change at RW Week 28 | 0.38 ± 0.449 | 0.33 ± 0.339 | NA ± NA | 0.18 ± 0.340 |
| Change at RW Week 29 | 0.26 ± 0.160 | 0.29 ± 0.233 | NA ± NA | 0.12 ± 0.227 |
| Change at RW Week 30 | 0.25 ± 0.296 | 0.39 ± 0.259 | NA ± NA | 0.64 ± 0.250 |
| Change at RW Week 31 | 0.06 ± 0.110 | 0.02 ± 0.109 | NA ± NA | 0.33 ± 0.105 |
| Change at RW Week 32 | 0.47 ± 0.081 | 0.20 ± 0.141 | NA ± NA | 0.23 ± 0.118 |
| Change at RW Week 33 | 0.62 ± 0.016 | 0.31 ± 0.167 | NA ± NA | 0.20 ± 0.151 |
| Change at RW Week 34 | 1.64 ± 0.086 | 0.95 ± 0.387 | NA ± NA | 0.27 ± 0.329 |
| Change at RW Week 35 | 0.75 ± 0.055 | 0.62 ± 0.347 | NA ± NA | 0.11 ± 0.295 |
| Change at RW Week 36 | 0.25 ± 0.093 | 0.15 ± 0.348 | NA ± NA | 0.20 ± 0.346 |
| Change at RW Week 37 | -0.02 ± 0.243 | -0.13 ± 0.404 | NA ± NA | 0.16 ± 0.367 |
| Change at RW Week 38 | 0.56 ± 0.022 | 0.40 ± 0.177 | NA ± NA | 0.04 ± 0.162 |
| Change at RW Week 39 | 0.96 ± 0.260 | 0.55 ± 0.578 | NA ± NA | 0.51 ± 0.592 |
| Change at RW Week 40 | NA ± NA | 0.17 ± 1.253 | NA ± NA | 0.44 ± 1.002 |
| Change at RW Week 41 | 0.92 ± 0.293 | 0.72 ± 0.723 | — | 0.91 ± 0.939 |
| Change at RW Week 42 | 0.77 ± 0.488 | 0.79 ± 0.262 | — | 0.60 ± 0.342 |
| Change at RW Week 43 | 1.03 ± 0.619 | 0.87 ± 0.449 | — | 0.88 ± 0.587 |
| Change at RW Week 44 | 0.84 ± 0.553 | 0.64 ± 0.590 | — | 0.75 ± 0.766 |
| Change at RW Week 45 | 0.77 ± 0.567 | 0.85 ± 0.384 | — | 3.77 ± 1.564 |
| Change at RW Week 46 | 0.18 ± 0.680 | 0.39 ± 0.471 | — | NA ± NA |
| Change at RW Week 47 | 0.07 ± 0.000 | 0.16 ± 0.261 | — | 3.10 ± 1.070 |
| Change at RW Week 48 | 0.03 ± 0.000 | 0.32 ± 0.273 | — | 6.18 ± 1.490 |
| Change at RW Week 49 | -0.50 ± 0.000 | -0.13 ± 0.115 | — | 5.77 ± 0.630 |
| Change at RW Week 50 | 0.00 ± NA | 0.00 ± NA | — | — |
| Change at RW Week 54 | — | — | — | 0.58 ± 0.000 |
The PGIPS, a single-item measure of the participant's impression of the overall severity of pericarditis symptoms at the time the questionnaire is administered using a 7-point rating scale ranging from absent (0=no recurrent pericarditis symptoms) to very severe (6=recurrent pericarditis symptoms cannot be ignored and markedly limits daily activities). The exact 95% CI is calculated with randomization strata pooled. Participants who had received bailout rilonacept or rescue medication before the time point were considered nonresponders.
| percentage of participants | Randomized Withdrawal Period: Rilonacept, Before Bailout Rilonacept | Randomized Withdrawal Period: Rilonacept, Including Bailout Rilonacept | Randomized Withdrawal Period: Placebo, Before Bailout Rilonacept | Randomized Withdrawal Period: Placebo, Including Bailout Rilonacept |
|---|---|---|---|---|
| Week 32 | 90.9 (58.72 to 99.77) | 83.3 (51.59 to 97.91) | 50.0 (1.26 to 98.74) | 70.0 (34.75 to 93.33) |
| Week 40 | 60.0 (14.66 to 94.73) | 66.7 (22.28 to 95.67) | 100 (2.50 to 100.00) | 83.3 (35.88 to 99.58) |
| Week 48 | 100 (15.81 to 100.00) | 100 (15.81 to 100.00) | — | 50.0 (1.26 to 98.74) |
| Week 56 | — | — | — | 100 (2.50 to 100.00) |
The PGA-PA is a single-item, clinician-reported outcome measure that Investigators use to rate their impression of the patient's overall pericarditis disease activity at the time the assessment is completed, using a rating scale ranging from absent to very severe. The Investigator selected the box that best described a participant's pericarditis activity at the time of occurrence of the assessment: Absent, Minimal, Mild, Moderate, Moderately Severe, Severe, Very Severe.
| percentage of participants | Randomized Withdrawal Period: Rilonacept, Before Bailout Rilonacept | Randomized Withdrawal Period: Rilonacept, Including Bailout Rilonacept | Randomized Withdrawal Period: Placebo, Before Bailout Rilonacept | Randomized Withdrawal Period: Placebo, Including Bailout Rilonacept |
|---|---|---|---|---|
| Baseline | 96.7 (82.78 to 99.92) | 96.7 (82.78 to 99.92) | 100 (88.78 to 100.00) | 100 (88.78 to 100.00) |
| Week 8 | 100 (85.18 to 100.00) | 100 (85.18 to 100.00) | 71.4 (41.90 to 91.61) | 85.2 (66.27 to 95.81) |
| Week 16 | 95.0 (75.13 to 99.87) | 95.0 (75.13 to 99.87) | 100 (47.82 to 100.00) | 95.0 (75.13 to 99.87) |
| Week 24 | 100 (78.20 to 100.00) | 100 (78.20 to 100.00) | 100 (29.24 to 100.00) | 100 (76.84 to 100.00) |
| Week 32 | 90.9 (58.72 to 99.77) | 83.3 (51.59 to 97.91) | 50.0 (1.26 to 98.74) | 90.9 (58.72 to 99.77) |
| Week 40 | 66.7 (9.43 to 99.16) | 75.0 (19.41 to 99.37) | 100 (2.50 to 100.00) | 100 (54.07 to 100.00) |
| Week 48 | 50.0 (1.26 to 98.74) | 50.0 (1.26 to 98.74) | — | 50.0 (1.26 to 98.74) |
| Week 56 | — | — | — | 100 (2.50 to 100.00) |
The SF-36 determines participants' overall quality of life by assessing 1) limitations in physical functioning due to health problems; 2) limitations in usual role because of physical health problems; 3) bodily pain; 4) general health perceptions; 5) vitality; 6) limitations in social functioning because of physical or emotional problems; 7) limitations in usual role due to emotional problems; and 8) general mental health. Items 1-4 primarily contribute to the physical component summary (PCS) score of the SF-36. Items 5-8 primarily contribute to the mental component summary (MCS) score of the SF-36. Scores on each item are summed and averaged (range: 0=worst to 100=best) with higher scores indicating better health. Increases from baseline indicate improvement.
| score on a scale | Randomized Withdrawal Period: Rilonacept, Before Bailout Rilonacept | Randomized Withdrawal Period: Rilonacept, Including Bailout Rilonacept | Randomized Withdrawal Period: Placebo, Before Bailout Rilonacept | Randomized Withdrawal Period: Placebo, Including Bailout Rilonacept |
|---|---|---|---|---|
| Change at RW Week 24 in PCS | 0.427 ± 5.8566 | 0.427 ± 5.8566 | 0.363 ± 2.3866 | 0.354 ± 2.7169 |
| Change at RW Week 24 in MCS | 3.081 ± 11.3204 | 3.081 ± 11.3204 | -0.050 ± 2.7318 | -0.575 ± 5.5922 |
The SF-36 determines participants' overall quality of life by assessing 1) limitations in physical functioning due to health problems; 2) limitations in usual role because of physical health problems; 3) bodily pain; 4) general health perceptions; 5) vitality; 6) limitations in social functioning because of physical or emotional problems; 7) limitations in usual role due to emotional problems; and 8) general mental health. Scores on each item are summed and averaged (range: 0=worst to 100=best) with higher scores indicating better health. Increases from baseline indicate improvement.
| score on a scale | Randomized Withdrawal Period: Rilonacept, Before Bailout Rilonacept | Randomized Withdrawal Period: Rilonacept, Including Bailout Rilonacept | Randomized Withdrawal Period: Placebo, Before Bailout Rilonacept | Randomized Withdrawal Period: Placebo, Including Bailout Rilonacept |
|---|---|---|---|---|
| Change at RW Week 24 in Physical Functioning | 2.427 ± 5.3887 | 2.427 ± 5.3887 | -0.637 ± 1.1027 | 0.273 ± 4.0332 |
| Change at RW Week 24 in Role Physical | 2.995 ± 5.7997 | 2.995 ± 5.7997 | 1.500 ± 2.5981 | 1.123 ± 3.4967 |
| Change at RW Week 24 in Bodily Pain | -0.485 ± 9.7799 | -0.485 ± 9.7799 | 0.807 ± 5.2864 | 0.921 ± 6.2063 |
| Change at RW Week 24 in General Health | -0.919 ± 4.9051 | -0.919 ± 4.9051 | -1.587 ± 5.9895 | -1.901 ± 6.8070 |
| Change at RW Week 24 in Vitality | 3.168 ± 9.8845 | 3.168 ± 9.8845 | 1.980 ± 6.8589 | 0.636 ± 6.1850 |
| Change at RW Week 24 in Social Functioning | 0.669 ± 8.6544 | 0.669 ± 8.6544 | 0.000 ± 0.0000 | -1.791 ± 4.2209 |
| Change at RW Week 24 in Role Emotional | 3.251 ± 10.7297 | 3.251 ± 10.7297 | 0.000 ± 0.0000 | 0.249 ± 7.0264 |
| Change at RW Week 24 in Mental Health | 3.314 ± 8.5900 | 3.314 ± 8.5900 | -0.873 ± 3.9923 | -0.187 ± 6.7646 |
The SF-6D is calculated based on responses to 11 items on the SF-36, that correspond to 6 domains: physical functioning, role participation (combined role-physical and role-emotional), social functioning, bodily pain, mental health, and vitality. Individual respondents can be classified on any of 4 to 6 levels of functioning or limitations for each of 6 domains, thus allowing a respondent to be classified into any of 18,000 possible unique health states. Using a standard gamble technique, each of these health states were mapped onto the SF-6D index score, which ranges from 0.00 (worst possible health state/death) to 1.00 (best possible health state/perfect health).
| score on a scale | Randomized Withdrawal Period: Rilonacept, Before Bailout Rilonacept | Randomized Withdrawal Period: Rilonacept, Including Bailout Rilonacept | Randomized Withdrawal Period: Placebo, Before Bailout Rilonacept | Randomized Withdrawal Period: Placebo, Including Bailout Rilonacept |
|---|---|---|---|---|
| Change From RW Period Baseline in the Short Form Health Survey-6 Domains (SF-6D) Utility Index Score at RW Period Week 24 | 0.0385 ± 0.10879 | 0.0385 ± 0.10879 | -0.0027 ± 0.05705 | -0.0005 ± 0.09871 |
The EQ-5D-5L is a self-reported health status questionnaire that consists of 6 questions used to calculate a health utility score for use in health economic analysis. There are 2 components to the EQ-5D-5L: a 5-item health state profile that assesses mobility, self-care, usual activities, pain/discomfort, and anxiety/depression used to obtain an Index Utility Score, as well as a visual analogue scale (VAS) that measures health state. Individual and index scores range from 0 to 1, with low scores representing a higher level of dysfunction. The VAS is designed to rate the participant's current health state on a scale from 0 to 100, where 0 represents the worst imaginable health state and 100 represents the best imaginable health state.
| score on a scale | Randomized Withdrawal Period: Rilonacept, Before Bailout Rilonacept | Randomized Withdrawal Period: Rilonacept, Including Bailout Rilonacept | Randomized Withdrawal Period: Placebo, Before Bailout Rilonacept | Randomized Withdrawal Period: Placebo, Including Bailout Rilonacept |
|---|---|---|---|---|
| Change at RW Week 24 in Mobility | -0.1 ± 0.52 | -0.1 ± 0.52 | 0.0 ± 0.00 | -0.1 ± 0.53 |
| Change at RW Week 24 in Self-Care | -0.1 ± 0.26 | -0.1 ± 0.26 | 0.0 ± 0.00 | 0.1 ± 0.27 |
| Change at RW Week 24 in Usual Activities | -0.1 ± 0.70 | -0.1 ± 0.70 | 0.0 ± 0.00 | 0.1 ± 0.36 |
| Change at RW Week 24 in Pain/Discomfort | 0.1 ± 0.92 | 0.1 ± 0.92 | 0.0 ± 1.00 | 0.1 ± 0.47 |
| Change at RW Week 24 in Anxiety/Depression | -0.1 ± 0.83 | -0.1 ± 0.83 | -0.3 ± 0.58 | 0.0 ± 0.55 |
| Change at RW Week 24 in Index Score | 0.0033 ± 0.09812 | 0.0033 ± 0.09812 | 0.0413 ± 0.15635 | 0.0029 ± 0.08592 |
| Change at RW Week 24 in VAS Score | 7.5 ± 17.32 | 7.5 ± 17.32 | 10.0 ± 10.15 | -8.3 ± 29.07 |
Participant's sleep quality was assessed with the ISI survey questionnaire. The ISI is a 7-item survey, with each question having 5 possible answers (none, mild, moderate, severe, or very severe), scored as 0, 1, 2, 3, or 4, respectively. Scores are summed for a total score, which ranges from 0 to 28. Lower scores are considered good, better, or healthy, and increasingly higher scores are considered to indicate greater insomnia. Clinical interpretation of the total score is as follows: 0 to 7 = no clinically significant insomnia; 8 to 14 = subthreshold insomnia; 15 to 21 = clinical insomnia (moderate severity); 22 to 28 = clinical insomnia (severe).
| score on a scale | Randomized Withdrawal Period: Rilonacept, Before Bailout Rilonacept | Randomized Withdrawal Period: Rilonacept, Including Bailout Rilonacept | Randomized Withdrawal Period: Placebo, Before Bailout Rilonacept | Randomized Withdrawal Period: Placebo, Including Bailout Rilonacept |
|---|---|---|---|---|
| Change From RW Period Baseline in Insomnia Severity Index (ISI) Total Score at RW Period Week 24 | -0.7 ± 4.75 | -0.7 ± 4.75 | -0.3 ± 3.79 | -0.2 ± 2.99 |
Participant's sleep quality was assessed with the ISI survey questionnaire. The ISI is a 7-item survey, with each question having 5 possible answers (none, mild, moderate, severe, or very severe), scored as 0, 1, 2, 3, or 4, respectively. Scores are summed for a total score, which ranges from 0 to 28. Lower scores are considered good, better, or healthy, and increasingly higher scores are considered to indicate greater insomnia. Clinical interpretation of the total score is as follows: 0 to 7 = no clinically significant insomnia; 8 to 14 = subthreshold insomnia; 15 to 21 = clinical insomnia (moderate severity); 22 to 28 = clinical insomnia (severe).
| Participants | Randomized Withdrawal Period: Rilonacept, Before Bailout Rilonacept | Randomized Withdrawal Period: Rilonacept, Including Bailout Rilonacept | Randomized Withdrawal Period: Placebo, Before Bailout Rilonacept | Randomized Withdrawal Period: Placebo, Including Bailout Rilonacept |
|---|---|---|---|---|
| BL No Clinically Significant Insomnia — Wk 24 No Clinically Significant Insomnia | 7 | 7 | 2 | 9 |
| BL No Clinically Significant Insomnia — Wk 24 Subthreshold Insomnia | 3 | 3 | 1 | 1 |
| BL No Clinically Significant Insomnia — Wk 24 Clinical Insomnia, Moderate Severity | 0 | 0 | 0 | 0 |
| BL No Clinically Significant Insomnia — Wk 24 Clinical Insomnia, Severe | 0 | 0 | 0 | 0 |
| BL No Clinically Significant Insomnia — Wk 24 Missing | 2 | 2 | 8 | 1 |
| BL Subthreshold Insomnia — Wk 24 No Clinically Significant Insomnia | 2 | 2 | 0 | 2 |
| BL Subthreshold Insomnia — Wk 24 Subthreshold Insomnia | 1 | 1 | 0 | 2 |
| BL Subthreshold Insomnia — Wk 24 Clinical Insomnia, Moderate Severity | 0 | 0 | 0 | 0 |
| BL Subthreshold Insomnia — Wk 24 Clinical Insomnia, Severe | 0 | 0 | 0 | 0 |
| BL Subthreshold Insomnia — Wk 24 Missing | 0 | 0 | 4 | 0 |
| BL Clinical Insomnia, Moderate Severity — Wk 24 No Clinically Significant Insomnia | 0 | 0 | — | — |
| BL Clinical Insomnia, Moderate Severity — Wk 24 Subthreshold Insomnia | 2 | 2 | — | — |
| BL Clinical Insomnia, Moderate Severity — Wk 24 Clinical Insomnia, Moderate Severity | 0 | 0 | — | — |
| BL Clinical Insomnia, Moderate Severity — Wk 24 Clinical Insomnia, Severe | 0 | 0 | — | — |
| BL Clinical Insomnia, Moderate Severity — Wk 24 Missing | 0 | 0 | — | — |
| BL Clinical Insomnia, Severe — Wk 24 No Clinically Significant Insomnia | — | 0 | — | — |
| BL Clinical Insomnia, Severe — Wk 24 Subthreshold Insomnia | — | — | — | — |
| BL Clinical Insomnia, Severe — Wk 24 Clinical Insomnia, Moderate Severity | — | — | — | — |
| BL Clinical Insomnia, Severe — Wk 24 Clinical Insomnia, Severe | — | — | — | — |
| BL Clinical Insomnia, Severe — Wk 24 Missing | — | — | — | — |
| BL Missing — Wk 24 No Clinically Significant Insomnia | — | — | — | — |
| BL Missing — Wk 24 Subthreshold Insomnia | — | — | — | — |
| BL Missing — Wk 24 Clinical Insomnia, Moderate Severity | — | — | — | — |
| BL Missing — Wk 24 Clinical Insomnia, Severe | — | — | — | — |
| BL Missing — Wk 24 Missing | — | — | — | — |
ORT included analgesics, NSAIDs, and/or colchicine. ORT use while waiting for at least 5 days since previous administration of study drug before receiving bailout, or within 5 days before the assessment of pericarditis recurrence, was excluded.
| percentage of participants | Randomized Withdrawal Period: Rilonacept | Randomized Withdrawal Period: Placebo |
|---|---|---|
| ORT : First Use ≤ 4 Weeks | 0 | 3.2 |
| ORT : First Use ≤ 8 Weeks | 0 | 6.5 |
| ORT : First Use ≤ 12 Weeks | 0 | 6.5 |
| ORT : First Use ≤ 16 Weeks | 3.3 | 6.5 |
| ORT : First Use ≤ 20 Weeks | 3.3 | 6.5 |
| ORT : First Use ≤ 24 Weeks | 3.3 | 6.5 |
| ORT : All Weeks (Including > 24 Weeks in RW Period) | 3.3 | 6.5 |
| Corticosteroid : First Use ≤ 4 Weeks | 0 | 0 |
| Corticosteroid : First Use ≤ 8 Weeks | 0 | 0 |
| Corticosteroid : First Use ≤ 12 Weeks | 0 | 0 |
| Corticosteroid : First Use ≤ 16 Weeks | 0 | 0 |
| Corticosteroid : First Use ≤ 20 Weeks | 0 | 0 |
| Corticosteroid : First Use ≤ 24 Weeks | 0 | 0 |
| Corticosteroid : All Weeks (Including > 24 Weeks in RW Period) | 0 | 0 |
| Bailout : First Use ≤ 4 Weeks | 0 | 29.0 |
| Bailout : First Use ≤ 8 Weeks | 0 | 48.4 |
| Bailout : First Use ≤ 12 Weeks | 0 | 61.3 |
| Bailout : First Use ≤ 16 Weeks | 0 | 67.7 |
| Bailout : First Use ≤ 20 Weeks | 0 | 71.0 |
| Bailout : First Use ≤ 24 Weeks | 0 | 71.0 |
| Bailout : All Weeks (Including > 24 Weeks in RW Period) | 3.3 | 74.2 |
| Total (any of the above) : First Use ≤ 4 Weeks | 0 | 32.3 |
| Total (any of the above) : First Use ≤ 8 Weeks | 0 | 51.6 |
| Total (any of the above) : First Use ≤ 12 Weeks | 0 | 64.5 |
| Total (any of the above) : First Use ≤ 16 Weeks | 3.3 | 71.0 |
| Total (any of the above) : First Use ≤ 20 Weeks | 3.3 | 74.2 |
| Total (any of the above) : First Use ≤ 24 Weeks | 3.3 | 74.2 |
| Total (any of the above) : All Weeks (Including > 24 Weeks in RW Period) | 6.7 | 77.4 |
ORT included analgesics, NSAIDs, and/or colchicine.
| percentage of participants | Randomized Withdrawal Period: Rilonacept | Randomized Withdrawal Period: Placebo |
|---|---|---|
| Percentage of Participants Using ORT for Pericarditis in the First 24 Weeks of RW Period | 3.3 | 6.5 |
MRI assessments were performed in a subgroup of participants (MRI substudy) to assess the percentage of participants with: * Pericardial delayed hyperenhancement * Myocardial delayed hyperenhancement * Pericardial effusion
| percentage of participants | Randomized Withdrawal Period: Rilonacept, Before Bailout Rilonacept | Randomized Withdrawal Period: Rilonacept, Including Bailout Rilonacept | Randomized Withdrawal Period: Placebo | Randomized Withdrawal Period: Placebo, Including Bailout Rilonacept |
|---|---|---|---|---|
| Pericardial Delayed Hyperenhancement | 66.7 | 66.7 | 28.6 | 57.1 |
| Myocardial Delayed Hyperenhancement | 0 | 0 | 0 | 0 |
| Pericardial Effusion | 0 | 0 | 0 | 0 |
Participants were asked to select the score that best described their average level of pericarditis pain over the previous 24 hours using an 11-point numerical rating scale (NRS), where zero (0) indicates 'no pain' and ten (10) indicates 'pain as bad as it could be'. Time to pain response was defined as number of days from first dose to the first day a participant's daily pain NRS was ≤ 2 of the 3 days over which the rolling average daily pain NRS was ≤ 2.
| days | Run-in Period |
|---|---|
| Time From First Dose to Pain Response in the RI Period | 5.0 (4.0 to 6.0) |
Time to CRP normalization, defined as CRP ≤ 0.5 mg/dL, was censored at treatment discontinuation, taking prohibited medication, or Week 12, whichever occurred first.
| days | Run-in Period |
|---|---|
| Time From First Dose to CRP Normalization in the RI Period | 7.0 (5.0 to 8.0) |
Time to rilonacept monotherapy was defined as the number of weeks from first dose to the first day of achieving monotherapy.
| weeks | Run-in Period |
|---|---|
| Time From First Dose to Rilonacept Monotherapy in RI Period | 7.3 (7.0 to 8.1) |
Time to treatment response is defined as time from first dose to the first day of pain response, and CRP ≤ 0.5 mg/dL within 7 days before or after pain response. Treatment response day will be the first day that the above criterion is met. If pain response occurs before CRP ≤ 0.5 mg/dL, each 3-day rolling average of NRS should be ≤ 2.0 from the day of pain response to the day of CRP ≤ 0.5 mg/dL. The response day will be the day of pain response. If CRP ≤0.5 mg/dL occurs before pain response, the response day will also be the day of pain response.
| days | Run-in Period |
|---|---|
| Time From First Dose to Treatment Response in RI Period | 5.0 (4.0 to 7.0) |
Participants were asked to select the score that best described their average level of pericarditis pain over the previous 24 hours using an 11-point numerical rating scale (NRS), where zero (0) indicates 'no pain' and ten (10) indicates 'pain as bad as it could be'. Clinical Response was defined as a weekly average of daily pericarditis pain of ≤ 2.0 on the 11-point NRS and CRP level ≤ 0.5 mg/dL and participants must have been able to stop background SOC pericarditis therapy by Week 10.
| percentage of participants | Run-in Period |
|---|---|
| Achieved Clinical Response | 84.9 |
| Did Not Achieve Clinical Response | 15.1 |
CRP normalization was defined as CRP ≤ 0.5 mg/dL.
| percentage of participants | Run-in Period |
|---|---|
| Percentage of Participants With CRP Normalization at RI Period Week 12 | 98.6 |
Participants were asked to select the score that best described their average level of pericarditis pain over the previous 24 hours using an 11-point numerical rating scale (NRS), where zero (0) indicates 'no pain' and ten (10) indicates 'pain as bad as it could be'.
| score on a scale | Run-in Period |
|---|---|
| RI Baseline | 4.48 ± 2.510 |
| Change at RI Week 1 | -2.31 ± 2.187 |
| Change at RI Week 2 | -3.18 ± 2.605 |
| Change at RI Week 3 | -3.28 ± 2.547 |
| Change at RI Week 4 | -3.51 ± 2.614 |
| Change at RI Week 5 | -3.55 ± 2.661 |
| Change at RI Week 6 | -3.70 ± 2.615 |
| Change at RI Week 7 | -3.81 ± 2.663 |
| Change at RI Week 8 | -3.92 ± 2.569 |
| Change at RI Week 9 | -3.81 ± 2.727 |
| Change at RI Week 10 | -3.83 ± 2.649 |
| Change at RI Week 11 | -4.05 ± 2.596 |
| Change at RI Week 12 | -4.00 ± 2.658 |
| mg/dL | Run-in Period |
|---|---|
| RI Baseline | 3.72 ± 5.719 |
| Change at RI Day 4 | -2.80 ± 4.582 |
| Change at RI Week 1 | -3.48 ± 5.539 |
| Change at RI Week 2 | -3.55 ± 5.717 |
| Change at RI Week 4 | -3.46 ± 5.809 |
| Change at RI Week 6 | -3.55 ± 5.899 |
| Change at RI Week 12 | -3.60 ± 5.916 |
| percentage of participants | Run-in Period: ECHO Abnormality at Baseline | Run-in Period: ECG Abnormality (ST-Elevation) at Baseline | Run-in Period: ECG Abnormality (PR Depression) at Baseline |
|---|---|---|---|
| Percentage of Participants With Resolution of Pericarditis-Related ECHO and ECG Abnormalities at Week 12 of the RI Period | 63.6 | 100 | 100 |
No or minimal pain is defined as non-missing daily NRS ≤ 2, where participants were asked to select the score that best describes their average level of pericarditis pain over the previous 24 hours using an 11-point NRS, where zero (0) indicates 'no pain' and ten (10) indicates 'pain as bad as it could be'.
| percentage of days | Run-in Period |
|---|---|
| Percentage of Days With No or Minimal Pain in the RI Period While on Treatment | 80.66 ± 21.388 |
The PGIPS, a single-item measure of the participant's impression of the overall severity of pericarditis symptoms at the time the questionnaire is administered using a 7-point rating scale ranging from absent (0=no recurrent pericarditis symptoms) to very severe (6=recurrent pericarditis symptoms cannot be ignored and markedly limits daily activities). The exact 95% CI is calculated with randomization strata pooled. Participants who had received bailout rilonacept or rescue medication before the time point were considered nonresponders.
| percentage of participants | Run-in Period |
|---|---|
| Baseline | 18.1 (10.48 to 28.05) |
| Week 6 | 89.5 (80.31 to 95.34) |
| Week 12 | 92.6 (84.57 to 97.23) |
The PGA-PA is a single-item, clinician-reported outcome measure that Investigators use to rate their impression of the patient's overall pericarditis disease activity at the time the assessment is completed, using a rating scale ranging from absent to very severe. The Investigator selected the box that best described a participant's pericarditis activity at the time of occurrence of the assessment: Absent, Minimal, Mild, Moderate, Moderately Severe, Severe, Very Severe. The exact 95% CI is calculated with randomization strata pooled. Participants who had received bailout rilonacept or rescue medication before the time point were considered nonresponders.
| percentage of participants | Run-in Period |
|---|---|
| RI Baseline | 10.6 (4.96 to 19.15) |
| RI Week 6 | 94.8 (87.23 to 98.57) |
| RI Week 12 | 98.7 (93.06 to 99.97) |
The SF-36 determines participants' overall quality of life by assessing 1) limitations in physical functioning due to health problems; 2) limitations in usual role because of physical health problems; 3) bodily pain; 4) general health perceptions; 5) vitality; 6) limitations in social functioning because of physical or emotional problems; 7) limitations in usual role due to emotional problems; and 8) general mental health. Scores on each item are summed and averaged (range: 0=worst to 100=best) with higher scores indicating better health. Increases from baseline indicate improvement.
| score on a scale | Run-in Period |
|---|---|
| Physical Component Score | 13.8 ± 8.7 |
| Mental Component Score | 9.2 ± 9.3 |
| Physical Functioning | 10.1 ± 9.2 |
| Role-Physical | 13.8 ± 9.4 |
| Bodily Pain | 19.1 ± 10.3 |
| General Health | 7.7 ± 8.2 |
| Vitality | 13.4 ± 9.9 |
| Social Functioning | 15.1 ± 10.6 |
| Role-Emotional | 7.7 ± 10.0 |
| Mental Health | 9.6 ± 9.0 |
The SF-6D is calculated based on responses to 11 items on the SF-36, that correspond to 6 domains: physical functioning, role participation (combined role-physical and role-emotional), social functioning, bodily pain, mental health, and vitality. Individual respondents can be classified on any of 4 to 6 levels of functioning or limitations for each of 6 domains, thus allowing a respondent to be classified into any of 18,000 possible unique health states. Using a standard gamble technique, each of these health states were mapped onto the SF-6D index score, which ranges from 0.00 (worst possible health state/death) to 1.00 (best possible health state/perfect health).
| score on a scale | Run-in Period |
|---|---|
| Change From RI Period Baseline in SF-6D Scores at RI Period Week 12 | 0.1828 ± 0.12658 |
The EQ-5D-5L is a self-reported health status questionnaire that consists of 6 questions used to calculate a health utility score for use in health economic analysis. There are 2 components to the EQ-5D-5L: a 5-item health state profile that assesses mobility, self-care, usual activities, pain/discomfort, and anxiety/depression used to obtain an Index Utility Score, as well as a visual analogue scale (VAS) that measures health state. Individual and index scores range from 0 to 1, with low scores representing a higher level of dysfunction. The VAS is designed to rate the participant's current health state on a scale from 0 to 100, where 0 represents the worst imaginable health state and 100 represents the best imaginable health state.
| score on a scale | Run-in Period |
|---|---|
| Mobility | -0.2 ± 0.84 |
| Self-Care | -0.1 ± 0.57 |
| Usual Activities | -1.1 ± 1.16 |
| Pain/Discomfort | -1.5 ± 1.18 |
| Anxiety/Depression | -0.5 ± 0.90 |
| Index Score | 0.1626 ± 0.16814 |
| VAS Score | 23.0 ± 23.41 |
Participant's sleep quality was assessed with the ISI survey questionnaire. The ISI is a 7-item survey, with each question having 5 possible answers (none, mild, moderate, severe, or very severe), scored as 0, 1, 2, 3, or 4, respectively. Scores are summed for a total score, which ranges from 0 to 28. Lower scores are considered good, better, or healthy, and increasingly higher scores are considered to indicate greater insomnia. Clinical interpretation of the total score is as follows: 0 to 7 = no clinically significant insomnia; 8 to 14 = subthreshold insomnia; 15 to 21 = clinical insomnia (moderate severity); 22 to 28 = clinical insomnia (severe).
| score on a scale | Run-in Period |
|---|---|
| Change From RI Period Baseline in ISI Total Score at RI Period Week 12 | -4.9 ± 6.31 |
Participant's sleep quality was assessed with the ISI survey questionnaire. The ISI is a 7-item survey, with each question having 5 possible answers (none, mild, moderate, severe, or very severe), scored as 0, 1, 2, 3, or 4, respectively. Scores are summed for a total score, which ranges from 0 to 28. Lower scores are considered good, better, or healthy, and increasingly higher scores are considered to indicate greater insomnia. Clinical interpretation of the total score is as follows: 0 to 7 = no clinically significant insomnia; 8 to 14 = subthreshold insomnia; 15 to 21 = clinical insomnia (moderate severity); 22 to 28 = clinical insomnia (severe).
| Participants | Run-in Period |
|---|---|
| BL No Clinically Significant Insomnia — Wk 12 No Clinically Significant Insomnia | 23 |
| BL No Clinically Significant Insomnia — Wk 12 Subthreshold Insomnia | 1 |
| BL No Clinically Significant Insomnia — Wk 12 Clinical Insomnia, Moderate Severity | 1 |
| BL No Clinically Significant Insomnia — Wk 12 Clinical Insomnia, Severe | 0 |
| BL No Clinically Significant Insomnia — Wk 12 Missing | 1 |
| BL Subthreshold Insomnia — Wk 12 No Clinically Significant Insomnia | 18 |
| BL Subthreshold Insomnia — Wk 12 Subthreshold Insomnia | 7 |
| BL Subthreshold Insomnia — Wk 12 Clinical Insomnia, Moderate Severity | 0 |
| BL Subthreshold Insomnia — Wk 12 Clinical Insomnia, Severe | 0 |
| BL Subthreshold Insomnia — Wk 12 Missing | 2 |
| BL Clinical Insomnia, Moderate Severity — Wk 12 No Clinically Significant Insomnia | 7 |
| BL Clinical Insomnia, Moderate Severity — Wk 12 Subthreshold Insomnia | 10 |
| BL Clinical Insomnia, Moderate Severity — Wk 12 Clinical Insomnia, Moderate Severity | 2 |
| BL Clinical Insomnia, Moderate Severity — Wk 12 Clinical Insomnia, Severe | 0 |
| BL Clinical Insomnia, Moderate Severity — Wk 12 Missing | 0 |
| BL Clinical Insomnia, Severe — Wk 12 No Clinically Significant Insomnia | 2 |
| BL Clinical Insomnia, Severe — Wk 12 Subthreshold Insomnia | 0 |
| BL Clinical Insomnia, Severe — Wk 12 Clinical Insomnia, Moderate Severity | 1 |
| BL Clinical Insomnia, Severe — Wk 12 Clinical Insomnia, Severe | 0 |
| BL Clinical Insomnia, Severe — Wk 12 Missing | 1 |
| BL Missing — Wk 12 No Clinically Significant Insomnia | 3 |
| BL Missing — Wk 12 Subthreshold Insomnia | 0 |
| BL Missing — Wk 12 Clinical Insomnia, Moderate Severity | 0 |
| BL Missing — Wk 12 Clinical Insomnia, Severe | 0 |
| BL Missing — Wk 12 Missing | 0 |
| Participants | Run-in Period |
|---|---|
| RI Baseline — Not Receiving Background Pericarditis Medication | 7 |
| RI Baseline — Receiving Background Pericarditis Medication | 79 |
| RI Week 4 — Not Receiving Background Pericarditis Medication | 15 |
| RI Week 4 — Receiving Background Pericarditis Medication | 69 |
| RI Week 8 — Not Receiving Background Pericarditis Medication | 49 |
| RI Week 8 — Receiving Background Pericarditis Medication | 32 |
| RI Week 10 — Not Receiving Background Pericarditis Medication | 71 |
| RI Week 10 — Receiving Background Pericarditis Medication | 9 |
| RI Week 12 — Not Receiving Background Pericarditis Medication | 76 |
| RI Week 12 — Receiving Background Pericarditis Medication | 3 |
Annualized Recurrence Rate is defined as the number of recurrences in LTE periods for all participants/Sum of participant years in LTE periods for all participants. Participant years in LTE period is defined as the time from 1st dose date of LTE period to the date of End of Study, or data cutoff date, whichever is earlier. The 95% CI was calculated using an exact method with Poisson distribution. Pericarditis recurrence is defined as the recurrence of typical pericarditis pain associated with supportive objective evidence of pericarditis.
| recurrences per participant per year | Long Term Extension: Non-Randomized | Long Term Extension: Randomized, Recurrence (RW) | Long Term Extension: Randomized, No Recurrence (RW) | Long Term Extension: Overall |
|---|---|---|---|---|
| Annualized Rate of Pericarditis Recurrence in the Long-Term Extension (LTE) Period Based on Investigator's Assessment (Based on Investigators' Judgement) | 0.088 (0.011 to 0.319) | 0.168 (0.062 to 0.367) | 0.125 (0.046 to 0.273) | 0.132 (0.072 to 0.221) |
| mg/dL | Long Term Extension: Non-Randomized | Long Term Extension: Randomized, Recurrence (RW) | Long Term Extension: Randomized, No Recurrence (RW) | Long Term Extension: Overall |
|---|---|---|---|---|
| LTE Baseline | 0.18 ± 0.294 | 0.21 ± 0.249 | 0.16 ± 0.251 | 0.18 ± 0.257 |
| Change at Week 12 | 0.01 ± 0.249 | 0.00 ± 0.127 | -0.06 ± 0.255 | -0.03 ± 0.219 |
| Change at Week 24 | 0.03 ± 0.182 | 0.17 ± 0.909 | -0.06 ± 0.269 | 0.04 ± 0.579 |
Percentage of participants with no or minimal pericarditis symptoms in the LTE Period, based on the PGIPS, a single-item measure of the participant's impression of the overall severity of pericarditis symptoms at the time the questionnaire is administered using a 7-point rating scale ranging from absent (0=no recurrent pericarditis symptoms) to very severe (6=recurrent pericarditis symptoms that cannot be ignored and markedly limits daily activities).
| percentage of participants | Long Term Extension: Non-Randomized | Long Term Extension: Randomized, Recurrence (RW) | Long Term Extension: Randomized, No Recurrence (RW) | Long Term Extension: Overall |
|---|---|---|---|---|
| LTE Baseline | 100.0 (78.20 to 100.0) | 88.0 (68.78 to 97.45) | 91.2 (76.32 to 98.14) | 91.9 (83.18 to 96.97) |
| LTE Week 12 | 93.3 (68.05 to 99.83) | 95.8 (78.88 to 99.89) | 100.0 (89.11 to 100.00) | 97.2 (90.19 to 99.66) |
| LTE Week 24 | 86.7 (59.54 to 98.34) | 78.3 (56.30 to 92.54) | 93.3 (77.93 to 99.18) | 86.8 (76.36 to 93.77) |
The PGA-PA is a single-item, clinician-reported outcome measure that Investigators use to rate their impression of the patient's overall pericarditis disease activity at the time the assessment is completed, using a rating scale ranging from absent to very severe. The Investigator selected the box that best described a participant's pericarditis activity at the time of occurrence of the assessment: Absent, Minimal, Mild, Moderate, Moderately Severe, Severe, Very Severe.
| percentage of participants | Long Term Extension: Non-Randomized | Long Term Extension: Randomized, Recurrence (RW) | Long Term Extension: Randomized, No Recurrence (RW) | Long-Term Extension: Overall |
|---|---|---|---|---|
| LTE Baseline | 100.0 (78.20 to 100.0) | 92.0 (73.97 to 99.02) | 94.1 (80.32 to 99.28) | 94.6 (86.73 to 98.51) |
| LTE Week 12 | 100.0 (78.20 to 100.00) | 91.7 (73.00 to 98.97) | 97.0 (84.24 to 99.92) | 95.8 (88.30 to 99.13) |
| LTE Week 24 | 93.3 (68.05 to 99.83) | 88.0 (68.78 to 97.45) | 96.8 (83.30 to 99.92) | 93.0 (84.33 to 97.67) |
The SF-36 determines participants' overall quality of life by assessing 1) limitations in physical functioning due to health problems; 2) limitations in usual role because of physical health problems; 3) bodily pain; 4) general health perceptions; 5) vitality; 6) limitations in social functioning because of physical or emotional problems; 7) limitations in usual role due to emotional problems; and 8) general mental health. Scores on each item are summed and averaged (range: 0=worst to 100=best) with higher scores indicating better health. Increases from baseline indicate improvement.
| score on a scale | Long Term Extension: Non-Randomized | Long Term Extension: Randomized, Recurrence (RW) | Long Term Extension: Randomized, No Recurrence (RW) | Long-Term Extension: Overall |
|---|---|---|---|---|
| Physical Functioning: Change at LTE Week 24 | 2.596 ± 8.4132 | 0.125 ± 6.4017 | -0.463 ± 5.7067 | 0.391 ± 6.5919 |
| Role-Physical: Change at LTE Week 24 | -1.443 ± 6.0141 | 2.052 ± 9.8327 | -1.627 ± 6.6000 | -0.306 ± 7.8579 |
| Bodily Pain: Change at LTE Week 24 | 3.082 ± 10.2083 | 1.140 ± 9.6306 | -0.820 ± 8.0536 | 0.690 ± 9.0827 |
| General Health: Change at LTE Week 24 | 1.324 ± 10.1092 | 0.932 ± 7.0108 | 0.762 ± 6.1724 | 0.940 ± 7.3166 |
| Vitality: Change at LTE Week 24 | 1.910 ± 8.2069 | 1.679 ± 9.3889 | -0.307 ± 6.6591 | 0.855 ± 7.9681 |
| Social Functioning: Change at LTE Week 24 | -0.716 ± 7.8320 | 0.219 ± 10.0797 | 0.172 ± 6.4927 | 0.000 ± 8.0595 |
| Role-Emotional: Change at LTE Week 24 | 0.496 ± 6.3849 | 0.758 ± 5.6497 | -0.360 ± 8.0771 | 0.211 ± 6.8796 |
| Mental Health: Change at LTE Week 24 | 2.988 ± 9.8321 | 0.455 ± 8.2976 | 1.353 ± 8.2616 | 1.387 ± 8.5381 |
| Physical Score: Change at LTE Week 24 | 1.210 ± 6.5951 | 1.168 ± 8.0082 | -1.079 ± 6.3170 | 0.190 ± 6.9912 |
| Mental Score: Change at LTE Week 24 | 1.186 ± 7.5539 | 0.596 ± 6.6219 | 0.850 ± 8.8099 | 0.833 ± 7.7298 |
The SF-6D is calculated based on responses to 11 items on the SF-36, that correspond to 6 domains: physical functioning, role participation (combined role-physical and role-emotional), social functioning, bodily pain, mental health, and vitality. Individual respondents can be classified on any of 4 to 6 levels of functioning or limitations for each of six domains, thus allowing a respondent to be classified into any of 18,000 possible unique health states. Using a standard gamble technique, each of these health states were mapped onto the SF-6D index score, which ranges from 0.00 (worst possible health state/death) to 1.00 (best possible health state/perfect health).
| score on a scale | Long Term Extension: Non-Randomized | Long Term Extension: Randomized, Recurrence (RW) | Long Term Extension: Randomized, No Recurrence (RW) | Long Term Extension: Overall |
|---|---|---|---|---|
| Change From LTE Baseline in SF-6D Health Utility Index Score | -0.106 ± 0.4449 | 0.163 ± 0.4368 | 0.003 ± 0.0781 | 0.035 ± 0.3425 |
The EQ-5D-5L is a self-reported health status questionnaire that consists of 6 questions used to calculate a health utility score for use in health economic analysis. There are 2 components to the EQ-5D-5L: a 5-item health state profile that assesses mobility, self-care, usual activities, pain/discomfort, and anxiety/depression used to obtain an Index Utility Score, as well as a visual analogue scale (VAS) that measures health state. Individual and index scores range from 0 to 1, with low scores representing a higher level of dysfunction. The VAS is designed to rate the participant's current health state on a scale from 0 to 100, where 0 represents the worst imaginable health state and 100 represents the best imaginable health state.
| score on a scale | Long Term Extension: Non-Randomized | Long Term Extension: Randomized, Recurrence (RW) | Long Term Extension: Randomized, No Recurrence (RW) | Long-Term Extension: Overall |
|---|---|---|---|---|
| Mobility: Change at LTE Week 24 | -0.2 ± 0.70 | 0.0 ± 0.77 | 0.1 ± 0.48 | 0.0 ± 0.64 |
| Self-Care: Change at LTE Week 24 | 0.1 ± 1.00 | -0.1 ± 0.29 | 0.0 ± 0.18 | 0.0 ± 0.49 |
| Usual Activities: Change at LTE Week 24 | -0.1 ± 1.35 | 0.0 ± 0.80 | -0.1 ± 0.45 | -0.1 ± 0.81 |
| Pain/Discomfort: Change at Week 24 | -0.3 ± 1.20 | -0.2 ± 0.85 | 0.0 ± 0.67 | -0.1 ± 0.86 |
| Anxiety/Depression: Change at Week 24 | -0.1 ± 0.73 | 0.1 ± 0.67 | 0.0 ± 0.72 | 0.0 ± 0.70 |
| Index Score: Change at Week 24 | 0.0546 ± 0.19544 | 0.0290 ± 0.10339 | -0.0004 ± 0.10212 | 0.0212 ± 0.12704 |
| VAS Score: Change at Week 24 | 6.2 ± 25.95 | 12.6 ± 27.07 | -0.2 ± 9.48 | 5.5 ± 21.19 |
Participant's sleep quality was assessed with the ISI survey questionnaire. The ISI is a 7-item survey, with each question having 5 possible answers (none, mild, moderate, severe, or very severe), scored as 0, 1, 2, 3, or 4, respectively. Scores are summed for a total score, which ranges from 0 to 28. Lower scores are considered good, better, or healthy, and increasingly higher scores are considered to indicate greater insomnia. Clinical interpretation of the total score is as follows: 0 to 7 = no clinically significant insomnia; 8 to 14 = subthreshold insomnia; 15 to 21 = clinical insomnia (moderate severity); 22 to 28 = clinical insomnia (severe).
| score on a scale | Long Term Extension: Non-Randomized | Long Term Extension: Randomized, Recurrence (RW) | Long Term Extension: Randomized, No Recurrence (RW) | Long-Term Extension: Overall |
|---|---|---|---|---|
| Change From LTE Baseline in ISI Total Score | -0.3 ± 1.87 | -0.2 ± 6.87 | -1.1 ± 3.58 | -0.1 ± 0.89 |
Participant's sleep quality was assessed with the ISI survey questionnaire. The ISI is a 7-item survey, with each question having 5 possible answers (none, mild, moderate, severe, or very severe), scored as 0, 1, 2, 3, or 4, respectively. Scores are summed for a total score, which ranges from 0 to 28. Lower scores are considered good, better, or healthy, and increasingly higher scores are considered to indicate greater insomnia. Clinical interpretation of the total score is as follows: 0 to 7 = no clinically significant insomnia; 8 to 14 = subthreshold insomnia; 15 to 21 = clinical insomnia (moderate severity); 22 to 28 = clinical insomnia (severe).
| Participants | Long Term Extension: Non-Randomized | Long Term Extension: Randomized, Recurrence (RW) | Long Term Extension: Randomized, No Recurrence (RW) | Long-Term Extension: Overall |
|---|---|---|---|---|
| LTE BL No Clinically Significant Insomnia — LTE Week 24 No Clinically Significant Insomnia | 9 | 11 | 18 | 38 |
| LTE BL No Clinically Significant Insomnia — LTE Week 24 Subthreshold Insomnia | 1 | 2 | 4 | 7 |
| LTE BL No Clinically Significant Insomnia — LTE Week 24 Clinical Insomnia (Moderate Severity) | 0 | 0 | 0 | 0 |
| LTE BL No Clinically Significant Insomnia — LTE Week 24 Clinical Insomnia (Severe) | 0 | 1 | 0 | 1 |
| LTE BL No Clinically Significant Insomnia — LTE Week 24 Missing | 0 | 0 | 1 | 1 |
| LTE BL Subthreshold Insomnia — LTE Week 24 No Clinically Significant Insomnia | 0 | 2 | 3 | 5 |
| LTE BL Subthreshold Insomnia — LTE Week 24 Subthreshold Insomnia | 2 | 2 | 2 | 6 |
| LTE BL Subthreshold Insomnia — LTE Week 24 Clinical Insomnia (Moderate Severity) | 0 | 1 | 0 | 1 |
| LTE BL Subthreshold Insomnia — LTE Week 24 Clinical Insomnia (Severe) | 0 | 0 | 0 | 0 |
| LTE BL Subthreshold Insomnia — LTE Week 24 Missing | 0 | 1 | 1 | 2 |
| LTE BL Clinical Insomnia (Moderate Severity) — LTE Week 24 No Clinically Significant Insomnia | 0 | 1 | 1 | 2 |
| LTE BL Clinical Insomnia (Moderate Severity) — LTE Week 24 Subthreshold Insomnia | 0 | 2 | 0 | 2 |
| LTE BL Clinical Insomnia (Moderate Severity) — LTE Week 24 Clinical Insomnia (Moderate Severity) | 0 | 0 | 1 | 1 |
| LTE BL Clinical Insomnia (Moderate Severity) — LTE Week 24 Clinical Insomnia (Severe) | 0 | 0 | 0 | 0 |
| LTE BL Clinical Insomnia (Moderate Severity) — LTE Week 24 Missing | 0 | 0 | 1 | 1 |
| LTE BL Clinical Insomnia (Severe) — LTE Week 24 No Clinically Significant Insomnia | 0 | 0 | 0 | 0 |
| LTE BL Clinical Insomnia (Severe) — LTE Week 24 Subthreshold Insomnia | 0 | 0 | 0 | 0 |
| LTE BL Clinical Insomnia (Severe) — LTE Week 24 Clinical Insomnia (Moderate Severity) | 0 | 0 | 0 | 0 |
| LTE BL Clinical Insomnia (Severe) — LTE Week 24 Clinical Insomnia (Severe) | 0 | 0 | 0 | 0 |
| LTE BL Clinical Insomnia (Severe) — LTE Week 24 Missing | 0 | 1 | 0 | 1 |
| LTE BL Missing — LTE Week 24 No Clinically Significant Insomnia | 0 | 0 | 0 | 0 |
| LTE BL Missing — LTE Week 24 Subthreshold Insomnia | 0 | 0 | 0 | 0 |
| LTE BL Missing — LTE Week 24 Clinical Insomnia (Moderate Severity) | 0 | 0 | 0 | 0 |
| LTE BL Missing — LTE Week 24 Clinical Insomnia (Severe) | 0 | 0 | 0 | 0 |
| LTE BL Missing — LTE Week 24 Missing | 0 | 0 | 0 | 0 |
No measurements were reported for this outcome.
| Participants | Long Term Extension: Non-Randomized | Long Term Extension: Randomized, Recurrence (RW) | Long Term Extension: Randomized, No Recurrence (RW) | Long-Term Extension: Overall |
|---|---|---|---|---|
| No Change | 3 | 4 | 7 | 14 |
| New | 0 | 0 | 1 | 1 |
| Improving | 1 | 0 | 1 | 2 |
| Resolved | 0 | 1 | 1 | 2 |
| Missing | 3 | 2 | 0 | 5 |
| Not Applicable | 8 | 17 | 14 | 39 |
| Participants | Long Term Extension: Non-Randomized | Long Term Extension: Randomized, Recurrence (RW) | Long Term Extension: Randomized, No Recurrence (RW) | Long-Term Extension: Overall |
|---|---|---|---|---|
| LTE Baseline — Normal | 7 | 16 | 21 | 44 |
| LTE Baseline — Abnormal-Consistent With Pericarditis | 0 | 1 | 1 | 2 |
| LTE Baseline — Abnormal-Not Consistent With Pericarditis But Clinically Significant | 0 | 0 | 1 | 1 |
| LTE Baseline — Abnormal-Not Consistent With Pericarditis But Not Clinically Significant | 8 | 8 | 11 | 27 |
| LTE Week 24 — Normal | 8 | 15 | 19 | 42 |
| LTE Week 24 — Abnormal-Consistent With Pericarditis | 0 | 1 | 1 | 2 |
| LTE Week 24 — Abnormal-Not Consistent With Pericarditis But Clinically Significant | 1 | 0 | 2 | 3 |
| LTE Week 24 — Abnormal-Not Consistent With Pericarditis But Not Clinically Significant | 6 | 9 | 9 | 24 |
| participants | Long Term Extension: Non-Randomized | Long Term Extension: Randomized, Recurrence (RW) | Long Term Extension: Randomized, No Recurrence (RW) | Long Term Extension: Overall |
|---|---|---|---|---|
| Pericardial Delayed Hyperenhancement Baseline Yes -> Week 24 Yes | 2 | 5 | 2 | 9 |
| Pericardial Delayed Hyperenhancement Baseline Yes -> Week 24 No | 1 | 1 | 0 | 2 |
| Pericardial Delayed Hyperenhancement Baseline Yes -> Week 24 Not Done | 1 | 3 | 8 | 12 |
| Pericardial Delayed Hyperenhancement Baseline No -> Week 24 Yes | — | 0 | 0 | 0 |
| Pericardial Delayed Hyperenhancement Baseline No -> Week 24 No | — | 1 | 2 | 3 |
| Pericardial Delayed Hyperenhancement Baseline No -> Week 24 Not Done | — | 1 | 2 | 3 |
| Pericardial Delayed Hyperenhancement Baseline Missing -> Week 24 Yes | 0 | 0 | 0 | 0 |
| Pericardial Delayed Hyperenhancement Baseline Missing -> Week 24 No | 0 | 1 | 0 | 1 |
| Pericardial Delayed Hyperenhancement Baseline Missing -> Week 24 Not Done | 4 | 1 | 2 | 7 |
| Myocardial Delayed Hyperenhancement Baseline Yes -> Week 24 Yes | 0 | 0 | 0 | 0 |
| Myocardial Delayed Hyperenhancement Baseline Yes -> Week 24 No | 1 | 0 | 2 | 3 |
| Myocardial Delayed Hyperenhancement Baseline Yes -> Week 24 Not Done | 1 | 2 | 1 | 4 |
| Myocardial Delayed Hyperenhancement Baseline No -> Week 24 Yes | 0 | 0 | 1 | 1 |
| Myocardial Delayed Hyperenhancement Baseline No -> Week 24 No | 2 | 6 | 1 | 9 |
| Myocardial Delayed Hyperenhancement Baseline No -> Week 24 Not Done | 0 | 3 | 9 | 12 |
| Myocardial Delayed Hyperenhancement Baseline Missing -> Week 24 Yes | 0 | 0 | 0 | 0 |
| Myocardial Delayed Hyperenhancement Baseline Missing -> Week 24 No | 0 | 1 | 0 | 1 |
| Myocardial Delayed Hyperenhancement Baseline Missing -> Week 24 Not Done | 4 | 1 | 2 | 7 |
| Pericardial Effusion Baseline Yes -> Week 24 Yes | 0 | 0 | — | 0 |
| Pericardial Effusion Baseline Yes -> Week 24 No | 1 | 1 | — | 2 |
| Pericardial Effusion Baseline Yes -> Week 24 Not Done | 0 | 0 | — | 0 |
| Pericardial Effusion Baseline No -> Week 24 Yes | 0 | 0 | 0 | 0 |
| Pericardial Effusion Baseline No -> Week 24 No | 3 | 6 | 4 | 13 |
| Pericardial Effusion Baseline No -> Week 24 Not Done | 0 | 4 | 10 | 14 |
| Pericardial Effusion Baseline Missing -> Week 24 Yes | 0 | 0 | 0 | 0 |
| Pericardial Effusion Baseline Missing -> Week 24 No | 0 | 1 | 0 | 1 |
| Pericardial Effusion Baseline Missing -> Week 24 Not Done | 4 | 1 | 2 | 7 |
| Participants | Long Term Extension: Non-Randomized | Long Term Extension: Randomized, Recurrence (RW) | Long Term Extension: Randomized, No Recurrence (RW) | Long Term Extension: Overall |
|---|---|---|---|---|
| LTE BL: Pericardial Delayed Hyperenhancement=Yes — LTE Week 24=Yes | 2 | 5 | 2 | 9 |
| LTE BL: Pericardial Delayed Hyperenhancement=Yes — LTE Week 24=No | 1 | 1 | 0 | 2 |
| LTE BL: Pericardial Delayed Hyperenhancement=Yes — LTE Week 24=Missing | 1 | 3 | 8 | 12 |
| LTE BL: Pericardial Delayed Hyperenhancement=No — LTE Week 24=Yes | — | 0 | 0 | 0 |
| LTE BL: Pericardial Delayed Hyperenhancement=No — LTE Week 24=No | — | 1 | 2 | 3 |
| LTE BL: Pericardial Delayed Hyperenhancement=No — LTE Week 24=Missing | — | 1 | 2 | 3 |
| LTE BL: Pericardial Delayed Hyperenhancement=Missing — LTE Week 24=Yes | 0 | 0 | 0 | 0 |
| LTE BL: Pericardial Delayed Hyperenhancement=Missing — LTE Week 24=No | 0 | 1 | 0 | 1 |
| LTE BL: Pericardial Delayed Hyperenhancement=Missing — LTE Week 24=Missing | 4 | 1 | 2 | 7 |
| LTE BL: Myocardial Delayed Hyperenhancement=Yes — LTE Week 24=Yes | 0 | 0 | 0 | 0 |
| LTE BL: Myocardial Delayed Hyperenhancement=Yes — LTE Week 24=No | 1 | 0 | 2 | 3 |
| LTE BL: Myocardial Delayed Hyperenhancement=Yes — LTE Week 24=Missing | 1 | 2 | 1 | 4 |
| LTE BL: Myocardial Delayed Hyperenhancement=No — LTE Week 24=Yes | 0 | 0 | 1 | 1 |
| LTE BL: Myocardial Delayed Hyperenhancement=No — LTE Week 24=No | 2 | 6 | 1 | 9 |
| LTE BL: Myocardial Delayed Hyperenhancement=No — LTE Week 24=Missing | 0 | 3 | 9 | 12 |
| LTE BL: Myocardial Delayed Hyperenhancement=Missing — LTE Week 24=Yes | 0 | 0 | 0 | 0 |
| LTE BL: Myocardial Delayed Hyperenhancement=Missing — LTE Week 24=No | 0 | 1 | 0 | 1 |
| LTE BL: Myocardial Delayed Hyperenhancement=Missing — LTE Week 24=Missing | 4 | 1 | 2 | 7 |
| LTE BL: Pericardial Effusion=Yes — LTE Week 24=Yes | 0 | 0 | — | 0 |
| LTE BL: Pericardial Effusion=Yes — LTE Week 24=No | 1 | 1 | — | 2 |
| LTE BL: Pericardial Effusion=Yes — LTE Week 24=Missing | 0 | 0 | — | 0 |
| LTE BL: Pericardial Effusion=No — LTE Week 24=Yes | 0 | 0 | 0 | 0 |
| LTE BL: Pericardial Effusion=No — LTE Week 24=No | 3 | 6 | 4 | 13 |
| LTE BL: Pericardial Effusion=No — LTE Week 24=Missing | 0 | 4 | 10 | 14 |
| LTE BL: Pericardial Effusion=Missing — LTE Week 24=Yes | 0 | 0 | 0 | 0 |
| LTE BL: Pericardial Effusion=Missing — LTE Week 24=No | 0 | 1 | 0 | 1 |
| LTE BL: Pericardial Effusion=Missing — LTE Week 24=Missing | 4 | 1 | 2 | 7 |
Annualized Recurrence Rate is defined as number of recurrences in LTE periods for all participants/Sum of participant years in LTE period for all participants. Participant years in LTE period is defined as the time from 1st dose date of LTE period to the date of End of Study, or data cutoff date, whichever is earlier. The 95% CI was calculated using an exact method with Poisson distribution. Pericarditis recurrence was defined as the recurrence of typical pericarditis pain associated with supportive objective evidence of pericarditis. At any time during the RW period, participants who experienced a suspected recurrence of pericarditis symptoms reported to the study site/clinic for a scheduled or unscheduled visit, during which clinical assessments were performed to gather all the necessary diagnostic data to confirm or rule out the presence of pericarditis recurrence.
| recurrences per participant per year | Long Term Extension: Non-Randomized | Long Term Extension: Randomized, Recurrence (RW) | Long Term Extension: Randomized, No Recurrence (RW) | Long-Term Extension: Overall |
|---|---|---|---|---|
| Annualized Rate of Pericarditis Recurrence in LTE Periods Based on Investigator's Assessment | 0.088 (0.011 to 0.319) | 0.168 (0.062 to 0.367) | 0.125 (0.046 to 0.273) | 0.132 (0.072 to 0.221) |
Annualized Recurrence Rate is defined as the number of recurrences in RW period for all participants/Sum of participant years in RW period for all participants. Participant years in RW period is defined as the time from randomization date to the date of EORW or last dose date + 6 weeks, whichever is earlier. The 95% CI was calculated using an exact method with Poisson distribution. Pericarditis recurrence was defined as the recurrence of typical pericarditis pain associated with supportive objective evidence of pericarditis. At any time during the RW period, participants who experienced a suspected recurrence of pericarditis symptoms reported to the study site/clinic for a scheduled or unscheduled visit, during which clinical assessments were performed to gather all the necessary diagnostic data to confirm or rule out the presence of pericarditis recurrence.
| recurrences per participant per year | Randomized Withdrawal Period: Rilonacept |
|---|---|
| Annualized Rate of Pericarditis Recurrence in RW Period Based on CEC Adjudication | 0.137 (0.017 to 0.494) |
Collected over All Cause Mortality: From enrollment through end of study; up to 156 weeks. Adverse events (AEs), RI Period: from first dose of study drug to the RW period randomization visit; mean duration 11.61 weeks. AEs, RW Period Rilonacept Only, Before Bailout: from RW period randomization until bailout rilonacept administration; mean duration 24.40 weeks. AEs, RW Period Rilonacept, Including Bailout Rilonacept: from RW period randomization until LTE rilonacept administration; mean duration 25.07 weeks.. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Run-in Period | 0/86 (0%) | 1/86 (1.2%) | 55/86 (64%) |
| Randomized Withdrawal Period: Rilonacept Only, Before Bailout Rilonacept | 0/30 (0%) | 1/30 (3.3%) | 18/30 (60%) |
| Randomized Withdrawal Period: Rilonacept, Including Bailout Rilonacept | 0/30 (0%) | 1/30 (3.3%) | 18/30 (60%) |
| Randomized Withdrawal Period: Placebo Only, Before Bailout Rilonacept | 0/31 (0%) | 1/31 (3.2%) | 7/31 (22.6%) |
| Randomized Withdrawal Period: Placebo, Including Bailout Rilonacept | 0/31 (0%) | 3/31 (9.7%) | 13/31 (41.9%) |
| Long-Term Extension Period: Overall | 0/74 (0%) | 6/74 (8.1%) | 43/74 (58.1%) |
| Event | Run-in Period | Randomized Withdrawal Period: Rilonacept Only, Before Bailout Rilonacept | Randomized Withdrawal Period: Rilonacept, Including Bailout Rilonacept | Randomized Withdrawal Period: Placebo Only, Before Bailout Rilonacept | Randomized Withdrawal Period: Placebo, Including Bailout Rilonacept | Long-Term Extension Period: Overall |
|---|---|---|---|---|---|---|
| Squamous cell carcinomaNeoplasms benign, malignant and unspecified (incl cysts and polyps) | 0/86 | 1/30 | 1/30 | 0/31 | 0/31 | 0/74 |
| Cardiac flutterCardiac disorders | 0/86 | 0/30 | 0/30 | 1/31 | 1/31 | 0/74 |
| IleusGastrointestinal disorders | 0/86 | 0/30 | 0/30 | 0/31 | 1/31 | 0/74 |
| PyrexiaGeneral disorders | 0/86 | 0/30 | 0/30 | 0/31 | 1/31 | 0/74 |
| Acute myocardial infarctionCardiac disorders | 0/86 | 0/30 | 0/30 | 0/31 | 0/31 | 1/74 |
| Aortic valve diseaseCardiac disorders | 0/86 | 0/30 | 0/30 | 0/31 | 0/31 | 1/74 |
| Aortic valve incompetenceCardiac disorders | 0/86 | 0/30 | 0/30 | 0/31 | 0/31 | 1/74 |
| Left ventricular failureCardiac disorders | 0/86 | 0/30 | 0/30 | 0/31 | 0/31 | 1/74 |
| PericarditisCardiac disorders | 0/86 | 0/30 | 0/30 | 0/31 | 0/31 | 1/74 |
| Bile duct stoneHepatobiliary disorders | 0/86 | 0/30 | 0/30 | 0/31 | 0/31 | 1/74 |
| Event | Run-in Period | Randomized Withdrawal Period: Rilonacept Only, Before Bailout Rilonacept | Randomized Withdrawal Period: Rilonacept, Including Bailout Rilonacept | Randomized Withdrawal Period: Placebo Only, Before Bailout Rilonacept | Randomized Withdrawal Period: Placebo, Including Bailout Rilonacept | Long-Term Extension Period: Overall |
|---|---|---|---|---|---|---|
| Injection site erythemaGeneral disorders | 18/86 | 6/30 | 7/30 | 0/31 | 1/31 | 3/74 |
| Injection site pruritusGeneral disorders | 5/86 | 4/30 | 5/30 | 0/31 | 1/31 | 0/74 |
| Musculoskeletal chest painMusculoskeletal and connective tissue disorders | 3/86 | 1/30 | 1/30 | 4/31 | 4/31 | 11/74 |
| Corona virus infectionInfections and infestations | 0/86 | 0/30 | 0/30 | 0/31 | 0/31 | 10/74 |
| MyalgiaMusculoskeletal and connective tissue disorders | 9/86 | 1/30 | 1/30 | 0/31 | 0/31 | 0/74 |
| Non-cardiac chest painGeneral disorders | 1/86 | 3/30 | 3/30 | 1/31 | 1/31 | 4/74 |
| SinusitisInfections and infestations | 1/86 | 3/30 | 3/30 | 0/31 | 0/31 | 2/74 |
| Urinary tract infectionInfections and infestations | 1/86 | 3/30 | 3/30 | 0/31 | 1/31 | 5/74 |
| High density lipoprotein increasedInvestigations | 0/86 | 3/30 | 3/30 | 0/31 | 0/31 | 1/74 |
| Oropharyngeal painRespiratory, thoracic and mediastinal disorders | 1/86 | 3/30 | 3/30 | 0/31 | 0/31 | 2/74 |
All participants entering the double-blind Randomized Withdrawal Treatment Period.
| Age, Continuous(years) | Randomized Withdrawal Period: Rilonacept | Randomized Withdrawal Period: Placebo | Total |
|---|---|---|---|
| Mean | 48.0 ± 15.70 | 44.8 ± 14.47 | 46.4 ± 15.05 |
| Age, Customized(Participants) | Randomized Withdrawal Period: Rilonacept | Randomized Withdrawal Period: Placebo | Total |
|---|---|---|---|
| 12 to 17 years | 1 | 2 | 3 |
| 18 to 64 years | 24 | 27 | 51 |
| 65 to 78 years | 5 | 2 | 7 |
| Sex: Female, Male(Participants) | Randomized Withdrawal Period: Rilonacept | Randomized Withdrawal Period: Placebo | Total |
|---|---|---|---|
| Female | 16 | 16 | 32 |
| Male | 14 | 15 | 29 |
| Ethnicity (NIH/OMB)(Participants) | Randomized Withdrawal Period: Rilonacept | Randomized Withdrawal Period: Placebo | Total |
|---|---|---|---|
| Hispanic or Latino | 0 | 0 | 0 |
| Not Hispanic or Latino | 30 | 31 | 61 |
| Unknown or Not Reported | 0 | 0 | 0 |
| Race/Ethnicity, Customized(Participants) | Randomized Withdrawal Period: Rilonacept | Randomized Withdrawal Period: Placebo | Total |
|---|---|---|---|
| White | 28 | 28 | 56 |
| Black or African American | 1 | 3 | 4 |
| Other, Not Specified | 1 | 0 | 1 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No
Ran from Jan 2019 to primary completion in May 2020, 16 months.
Results were first posted in Jul 2021, 1 year 1 month after primary completion.
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Studies of the drug tested here: Rilonacept
Kiniksa Pharmaceuticals (UK), Ltd.