A Phase 2 interventional study of Rilonacept and Placebo in Familial Mediterranean Fever, sponsored by The Cleveland Clinic. Completed at 5 sites in United States. Open to participants aged 4 Years and older. Per ClinicalTrials.gov, last updated 2013-02-11.
Sponsored by The Cleveland Clinic · Phase 2, Interventional, and Treatment
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Familial Mediterranean fever (FMF) is a genetic disease resulting in recurrent attacks of fever, abdominal pain, chest pain, arthritis and rash. There are 5-15% of patients who continue to have FMF attacks despite treatment with colchicine or who cannot tolerate colchicine. Currently there are no alternatives to colchicine. Pyrin, the protein that has a defect in FMF has an important role in the regulation of a molecule called interleukin (IL)-1 beta production and activity. This molecule is very important in the process of inflammation in FMF.
Therefore we propose to use IL-1 Trap (Rilonacept), a medication that binds and neutralizes IL-1.
We will enroll in this study 17 subjects from the age of 4 years, including adults with active FMF despite colchicine therapy. Subjects will receive in random order two 3-month courses of Rilonacept at 2.2 mg/kg (maximum 160 mg) by weekly subcutaneous injection and two 3-month courses of placebo injection. If patients have at least two FMF attacks during a treatment course they will be able to get if they choose the other treatment until the end of that treatment course. Our hypothesis is that Rilonacept will decrease the number of acute FMF attacks and will be safe to use. This study may confirm the importance of IL-1 in the cause of FMF.
Funding source - FDA Office of Orphan Products Development
Familial Mediterranean fever (FMF) is an autosomal recessive autoinflammatory genetic disorder resulting in recurrent attacks of fever, serositis, arthritis and rash. Late complications of untreated FMF include the development of renal amyloidosis. FMF is a rare orphan disease in the United States. Treatment with colchicine is effective in reducing the frequency of episodes in most patients and the development of amyloidosis in nearly all patients. However, there are still 5-15% of patients who continue to have acute FMF attacks despite colchicine therapy or are intolerant of colchicine, usually from gastrointestinal adverse effects. Currently there are no effective alternatives to colchicine. Pyrin, the mutated protein in FMF has an important role in the regulation of IL-1 beta production and activity. Mutations in pyrin result in increased IL-1 beta levels in mice and humans. IL-1 beta is an important pro-inflammatory cytokine. Thus, we hypothesize that inhibition of IL-1 will decrease acute attacks in patients with FMF. We propose to use IL-1 Trap (Rilonacept), a fusion protein consisting of human IL-1 cytokine receptor extracellular domains and the FC portion of human IgG1 that binds and neutralizes IL-1.
We will enroll 17 subjects from the age of 4 years, including adults, from multiple centers in the United States with active FMF (at least 1 attack per month) despite receiving at least 1.2-1.5 mg/d of colchicine (dose dependent on age) or are intolerant of colchicine. Subjects will be diagnosed by clinical criteria with at least one heterozygote mutation of the MEFV (pyrin) gene. After screening subjects will be monitored for a month to observe for acute FMF attacks or if they did not develop an attack in that month until they develop two attacks. We will then use a single-subject alternating treatments design with subjects receiving in random order two 3-month courses of Rilonacept at 2.2 mg/kg (max 160 mg) by weekly SC injection and two 3-month courses of comparable volume placebo. Subjects will continue the usual colchicine dose they were on when they started the study. Subjects with 2 acute FMF attacks during a treatment course will be able to crossover to the other treatment arm until the end of that treatment course. There will be 10 study visits: 1. Screening. 2. Treatment baseline after one month or after subjects have developed FMF attacks as described above. After 1 month of each treatment course and at the end of each treatment course (overall 8 visits). At each visit subjects will return completed diary forms, used and unused drug, queried on adverse effects, undergo a physical examination and laboratory tests obtained for inflammation, safety and in some visits for translational studies. Subjects will also fill out quality of life questionnaires and give an overall estimation of the disease activity. Results will be analyzed by traditional frequency statistics (using an intent to treat analysis) and by Bayesian hierarchical modeling. Our primary aim is to assess the efficacy of Rilonacept in decreasing the number of acute FMF attacks while monitoring drug safety.
The significance of the study includes short and long-term benefits. Fewer FMF attacks will result in less functional impairment and a higher quality of life in colchicine resistant or intolerant patients. Once weekly injections have the potential to improve treatment compliance. Fewer acute attacks of arthritis may prevent the development of chronic joint damage. In the long-term, better FMF control may prevent amyloidosis. This study may confirm the importance of IL-1 in the pathogenesis of FMF and provide support for an FDA filing for use of Rilonacept in FMF. The study design may serve as a template for trials of new biologic drugs for rare diseases.
61 studies on the registry are indexed under Familial Mediterranean Fever; 27 are open to participants now.
This study's enrollment of 14 is below the median of 40 across 27 interventional studies indexed under Familial Mediterranean Fever.
Browse Familial Mediterranean Fever studies →The Cleveland Clinic is the lead sponsor of 832 studies on the registry; 120 are open to participants now.
Of its 90 completed or terminated interventional studies of FDA-regulated products, 73 (81%) have results posted.
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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.
A planned gap after a treatment, so that its effects can lessen before the study treatment or a study assessment. Ask the study team and the doctor who prescribed it before changing any medicine.
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.
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.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
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.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
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.
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.
Vaccines made with a weakened form of the germ, such as measles–mumps–rubella (MMR), chickenpox and yellow fever. They can be risky when the immune system is weakened. Flu injections are not live; the nasal flu spray is.
Washout period
A planned gap after a treatment, so that its effects can lessen before the study treatment or a study assessment. Ask the study team and the doctor who prescribed it before changing any medicine.
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.
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.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
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.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
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.
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.
Live vaccines
Vaccines made with a weakened form of the germ, such as measles–mumps–rubella (MMR), chickenpox and yellow fever. They can be risky when the immune system is weakened. Flu injections are not live; the nasal flu spray is.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Treatment Arm A: Rilonacept (IL-1 Trap) at a dose of 2.2 mg/kg/wk (max 160 mg)given by subcutaneous injection for 3 months plus colchicine at a stable dose for those subjects already taking colchicine, or without colchicine for those intolerant or non-compliant with colchicine. Since the colchicine dose is stable throughout the study for each subject, at the prestudy dose, colchicine was not considered an intervention
Drug: Rilonacept
Treatment Arm B: Placebo given by subcutaneous injection weekly with or without colchicine for 3 months. Since the colchicine dose is stable throughout the study for each subject, at the prestudy dose, colchicine was not considered an intervention.
Drug: Placebo
2.2 mg/kg/wk by subcutaneous injection, for 3 months
Also known as: Arcalyst, IL-1 Trap
placebo by subcutaneous injection weekly for 3 months
To Assess the Efficacy of Rilonacept in Decreasing the Number of Acute FMF Attacks.
Difference in number of attacks per treatment month between rilonacept and placebo
Time frame: attacks were assessed at the end of each 3 month treatment course (overall up to 6 month of rilonacept and 6 months of placebo, each)
To Determine if There is a Medically Important Difference Between the Safety Profiles of Rilonacept vs. Placebo.
Differences in adverse events (AEs) between rilonacept and placebo per patient-month of treatment. We separately analyzed injection site reactions and infectious adverse events. Other adverse events were too small in number to analyze. The upper table (and first statistical analysis) regards injection site reactions and lower table (and second statistical analysis) regards infections.
Time frame: 12 months of entire study length
To Determine the Difference in the Length of Attacks During Treatment With Rilonacept vs. Placebo.
This outcome was the difference in days in the length of attacks between rilonacept and placebo.
Time frame: 12 months
Percentage of Treatment Courses Without FMF Attacks in Rilonacept Courses as Compared to Placebo Courses.
The percentage of rilonacept and placebo treatment courses without FMF attacks.
Time frame: Each treatment course of up to 3 months
To Determine the Proportion of Courses in Which Subjects Attained at Least a 50% Decrease in Acute FMF Attacks During Rilonacept Courses as Compared to Placebo Courses.
Differences between rilonacept and placebo in the percentage of courses that attained at least a 50% decrease in FMF attacks when compared to attacks in the screening period.
Time frame: Up to 3 months for each treatment course
To Determine Differences in the Time to the Development of Attacks Between the Treatment Arms (Rilonacept vs. Placebo).
In a survival analysis we measured the difference (in days) until the development of the first and second attack within a treatment course of up to 3 months and examined differences in this parameter between rilonacept and placebo. Data regarding the development of the second attack are reported below. In regards to the first attack there were no significant differences between rilonacept and placebo (20 days (7.5,\>90)for rilonacept; 15 (8,32) for placebo, P=0.066).
Time frame: 3 months
To Determine the Differences in the Erythrocyte Sedimentation Rate Between the Treatment Arms (Rilonacept vs. Placebo).
Erythrocyte sedimentation rate - ESR (mm/h)
Time frame: 3 months (each treatment course, overall 12 months)
To Determine the Differences in C-Reactive Protein Between the Treatment Arms (Rilonacept vs. Placebo)
Differences between the treatment courses in the C-Reactive Protein levels mg/L
Time frame: 3 months (each treatment course, overall 12 months)
To Determine the Differences in the Platelet Count Between the Treatment Arms (Rilonacept vs. Placebo)
The difference between the treatment arms in the platelet count X 10 to the power of 9
Time frame: 3 months (each treatment course, overall 12 months)
To Determine the Differences in the Fibrinogen Levels Between the Treatment Arms (Rilonacept vs. Placebo)
The differences between treatment arms in the fibrinogen level (micromol/L)
Time frame: 3 months (each treatment course, overall 12 months)
To Determine the Differences in Serum Amyloid A Levels Between the Treatment Arms (Rilonacept vs. Placebo)
The difference between the treatment arms in serum amyloid A levels (mg/L)
Time frame: 3 months (each treatment course, overall 12 months)
To Determine the Differences in the Quality of Life Between the Treatment Arms (Rilonacept vs. Placebo).
Differences in the health-related quality of life (HRQOL) during treatment with rilonacept vs. placebo. HRQOl was measured by the Childhood Health Questionnaire which was adopted also for adults. There are 2 summary scores: 1. Physical summary score. 2. Psychosocial summary score. The data reported below in the upper table is the physical summary composite score and in the lower table the psychosocial summary composite score. Scores were from 0-100 (higher is better) with a score of 50 representing the mean of the normal population.
Time frame: 12 months
To Determine the Differences in the FMF Severity Score of the Subjects Between the Treatment Arms (Rilonacept vs. Placebo).
Differences in the Armenian Evaluation Score between rilonacept and placebo courses. The Armenian Evaluation Score is a composite score of disease severity based on the frequency, duration and character of attacks (degree of fever and severity of serositis). It was adapted to calculate a score for a 3-month treatment course. The lowest (best) score is 0 and higher values are worse. In theory there is no upper limit to the scale. The total score is reported (there are no subscales).
Time frame: overall 12 months
To Determine the Differences in the Proportion of Time Subjects Received Rilonacept vs Placebo
The proportion of time within the trial that participants received rilonacept as opposed to placebo. The reason for this outcome is that participants who had at least 2 attacks within an individual treatment course were able to "escape" in a blinded manner to the other treatment arm until the end of that treatment course and then resume the original randomization sequence. Thus participants may have been treated for a longer time with one treatment arm or the other.
Time frame: 12 months
Participants were recruited from October 2008 until February 2010. The study was conducted in 6 clinics in the United States in FMF clinics or clinics in regions where a higher prevalence of FMF is expected based on local population ethnicity.
| Milestone | Rilonacept-Placebo-Rilonacept-Placebo | Rilonacept-Placebo-Placebo-Rilonacept | Placebo-Rilonacept-Placebo-Rilonacept | Placebo-Rilonacept-Rilonacept-Placebo |
|---|---|---|---|---|
| Started | 4 | 3 | 3 | 4 |
| Completed | 3 | 3 | 3 | 3 |
| Not completed | 1 | 0 | 0 | 1 |
| Withdrew: Withdrawal by subject | 1 | 0 | 0 | 0 |
| Withdrew: Lost to follow-up | 0 | 0 | 0 | 1 |
| Milestone | Rilonacept-Placebo-Rilonacept-Placebo | Rilonacept-Placebo-Placebo-Rilonacept | Placebo-Rilonacept-Placebo-Rilonacept | Placebo-Rilonacept-Rilonacept-Placebo |
|---|---|---|---|---|
| Started | 3 | 3 | 3 | 3 |
| Completed | 2 | 3 | 3 | 3 |
| Not completed | 1 | 0 | 0 | 0 |
| Withdrew: Lack of efficacy | 1 | 0 | 0 | 0 |
| Milestone | Rilonacept-Placebo-Rilonacept-Placebo | Rilonacept-Placebo-Placebo-Rilonacept | Placebo-Rilonacept-Placebo-Rilonacept | Placebo-Rilonacept-Rilonacept-Placebo |
|---|---|---|---|---|
| Started | 2 | 3 | 3 | 3 |
| Completed | 2 | 3 | 3 | 3 |
| Not completed | 0 | 0 | 0 | 0 |
| Milestone | Rilonacept-Placebo-Rilonacept-Placebo | Rilonacept-Placebo-Placebo-Rilonacept | Placebo-Rilonacept-Placebo-Rilonacept | Placebo-Rilonacept-Rilonacept-Placebo |
|---|---|---|---|---|
| Started | 2 | 3 | 3 | 3 |
| Completed | 2 | 3 | 3 | 3 |
| Not completed | 0 | 0 | 0 | 0 |
Difference in number of attacks per treatment month between rilonacept and placebo
| number of attacks per month | Rilonacept | Placebo |
|---|---|---|
| To Assess the Efficacy of Rilonacept in Decreasing the Number of Acute FMF Attacks. | 0.77 (0.18 to 1.20) | 2.00 (0.90 to 2.40) |
Differences in adverse events (AEs) between rilonacept and placebo per patient-month of treatment. We separately analyzed injection site reactions and infectious adverse events. Other adverse events were too small in number to analyze. The upper table (and first statistical analysis) regards injection site reactions and lower table (and second statistical analysis) regards infections.
| AEs per patient-month of treatment | Rilonacept | Placebo |
|---|---|---|
| Injection site reactions | 1 (0 to 1.04) | 0 (0 to 0.36) |
| Infections | 0 (0 to 0.12) | 0.18 (0 to 0.29) |
This outcome was the difference in days in the length of attacks between rilonacept and placebo.
| Number of days | Rilonacept | Placebo |
|---|---|---|
| To Determine the Difference in the Length of Attacks During Treatment With Rilonacept vs. Placebo. | 2.8 (1 to 3.3) | 3.2 (2.7 to 4) |
The percentage of rilonacept and placebo treatment courses without FMF attacks.
| Percentage of courses | Rilonacept | Placebo |
|---|---|---|
| Percentage of Treatment Courses Without FMF Attacks in Rilonacept Courses as Compared to Placebo Courses. | 29 | 0 |
Differences between rilonacept and placebo in the percentage of courses that attained at least a 50% decrease in FMF attacks when compared to attacks in the screening period.
| Percentage of courses | Rilonacept | Placebo |
|---|---|---|
| To Determine the Proportion of Courses in Which Subjects Attained at Least a 50% Decrease in Acute FMF Attacks During Rilonacept Courses as Compared to Placebo Courses. | 75 | 35 |
In a survival analysis we measured the difference (in days) until the development of the first and second attack within a treatment course of up to 3 months and examined differences in this parameter between rilonacept and placebo. Data regarding the development of the second attack are reported below. In regards to the first attack there were no significant differences between rilonacept and placebo (20 days (7.5,\>90)for rilonacept; 15 (8,32) for placebo, P=0.066).
| days until second attack | Rilonacept | Placebo |
|---|---|---|
| To Determine Differences in the Time to the Development of Attacks Between the Treatment Arms (Rilonacept vs. Placebo). | 90 (25 to 90) | 36 (15 to 68) |
Erythrocyte sedimentation rate - ESR (mm/h)
| mm/h | Rilonacept | Placebo |
|---|---|---|
| To Determine the Differences in the Erythrocyte Sedimentation Rate Between the Treatment Arms (Rilonacept vs. Placebo). | 5.8 (2 to 16) | 14 (7 to 19) |
Differences between the treatment courses in the C-Reactive Protein levels mg/L
| mg/L | Rilonacept | Placebo |
|---|---|---|
| To Determine the Differences in C-Reactive Protein Between the Treatment Arms (Rilonacept vs. Placebo) | 2 (2 to 4) | 4 (2 to 9) |
The difference between the treatment arms in the platelet count X 10 to the power of 9
| cell count X10 to the power of 9 | Rilonacept | Placebo |
|---|---|---|
| To Determine the Differences in the Platelet Count Between the Treatment Arms (Rilonacept vs. Placebo) | 262 (246 to 318) | 334 (305 to 352) |
The differences between treatment arms in the fibrinogen level (micromol/L)
| micromol/L | Rilonacept | Placebo |
|---|---|---|
| To Determine the Differences in the Fibrinogen Levels Between the Treatment Arms (Rilonacept vs. Placebo) | 6.56 (6.41 to 9.70) | 9.56 (9.26 to 10.17) |
The difference between the treatment arms in serum amyloid A levels (mg/L)
| mg/L | Rilonacept | Placebo |
|---|---|---|
| To Determine the Differences in Serum Amyloid A Levels Between the Treatment Arms (Rilonacept vs. Placebo) | 13 (0 to 40) | 15 (0 to 192) |
Differences in the health-related quality of life (HRQOL) during treatment with rilonacept vs. placebo. HRQOl was measured by the Childhood Health Questionnaire which was adopted also for adults. There are 2 summary scores: 1. Physical summary score. 2. Psychosocial summary score. The data reported below in the upper table is the physical summary composite score and in the lower table the psychosocial summary composite score. Scores were from 0-100 (higher is better) with a score of 50 representing the mean of the normal population.
| Composite HRQOL summary score | Rilonacept | Placebo |
|---|---|---|
| Physical HRQOL summary composite score | 33 (25.8 to 38.1) | 20.3 (10.5 to 32.7) |
| Psychosocial HRQOL summary composite score | 53.3 (48.6 to 57.2) | 49.8 (42.5 to 59) |
Differences in the Armenian Evaluation Score between rilonacept and placebo courses. The Armenian Evaluation Score is a composite score of disease severity based on the frequency, duration and character of attacks (degree of fever and severity of serositis). It was adapted to calculate a score for a 3-month treatment course. The lowest (best) score is 0 and higher values are worse. In theory there is no upper limit to the scale. The total score is reported (there are no subscales).
| units on a scale | Rilonacept | Placebo |
|---|---|---|
| To Determine the Differences in the FMF Severity Score of the Subjects Between the Treatment Arms (Rilonacept vs. Placebo). | 5.5 (3 to 13) | 11 (7.8 to 12.5) |
The proportion of time within the trial that participants received rilonacept as opposed to placebo. The reason for this outcome is that participants who had at least 2 attacks within an individual treatment course were able to "escape" in a blinded manner to the other treatment arm until the end of that treatment course and then resume the original randomization sequence. Thus participants may have been treated for a longer time with one treatment arm or the other.
| Percentage of time treated | Rilonacept | Placebo |
|---|---|---|
| To Determine the Differences in the Proportion of Time Subjects Received Rilonacept vs Placebo | 52.5 (50.5 to 70.5) | 47.5 (29.5 to 49.5) |
Collected over 1 year. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Placebo | — | 3/14 (21.4%) | 12/14 (85.7%) |
| Rilonacept | — | 3/13 (23.1%) | 11/13 (84.6%) |
| Event | Placebo | Rilonacept |
|---|---|---|
| Hospitalization for FMF attackImmune system disorders | 2/14 | 2/13 |
| PneumoniaInfections and infestations | 0/14 | 1/13 |
| Respiratory InfectionInfections and infestations | 1/14 | 0/13 |
| Event | Placebo | Rilonacept |
|---|---|---|
| Injection site reactionInjury, poisoning and procedural complications | 5/14 | 7/13 |
| Upper respiratory infection/OtitisInfections and infestations | 3/14 | 0/13 |
| Arthralgia/bone painMusculoskeletal and connective tissue disorders | 1/14 | 2/13 |
| SinusitisInfections and infestations | 1/14 | 1/13 |
| Epstein Barr virusInfections and infestations | 0/14 | 1/13 |
| HypertensionGeneral disorders | 0/14 | 1/13 |
| HeadachesNervous system disorders | 1/14 | 1/13 |
| OtitisInfections and infestations | 1/14 | 1/13 |
| InfluenzaInfections and infestations | 0/14 | 1/13 |
| Loss of appetiteGastrointestinal disorders | 0/14 | 1/13 |
| Age, Categorical(Participants) | All Patients Received Both Rilonacept and Placebo |
|---|---|
| <=18 years | 4 |
| Between 18 and 65 years | 10 |
| >=65 years | 0 |
| Age Continuous(years) | All Patients Received Both Rilonacept and Placebo |
|---|---|
| Mean | 24.4 ± 11.8 |
| Sex: Female, Male(Participants) | All Patients Received Both Rilonacept and Placebo |
|---|---|
| Female | 6 |
| Male | 8 |
| Region of Enrollment(participants) | All Patients Received Both Rilonacept and Placebo |
|---|---|
| United States | 14 |
The record gives a start of Aug 2008, a primary completion of Sep 2011 and a completion of Sep 2011. It does not say whether these dates are actual or estimated.
Results were first posted in Oct 2012, 1 year 1 month after primary completion.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Feb 2013. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Rilonacept
Familial Mediterranean Fever →