A Phase 2/3 interventional study of RPH-104 and Placebo in Pericarditis, sponsored by R-Pharm International, LLC. Completed at 2 sites in Russian Federation. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2022-10-31.
Sponsored by R-Pharm International, LLC (part of R-Pharm) · Phase 2/3, Interventional, and Treatment
The goal of this study was to evaluate the Efficacy and Safety of RPH-104 Treatment in patients in comparison to placebo with Idiopathic Recurrent Pericarditis
The study included the following periods:
Run-in treatment period (12 weeks, Days 0-84 - for the patients receiving non-steroidal anti-inflammatory drugs (NSAIDs) and/or colchicine as the therapy for recurrent pericarditis (underlying disease), or 24 weeks, Days 0-168 - for the patients receiving glucocorticosteroids (GCSs) as monotherapy or in combination with NSAIDs and/or colchicine; Depending on the Protocol version, during the period, the patients received the product according to a schedule of 80 mg every two weeks, starting from Day 0 (Protocol version 2.0/3.0). The Protocol amendment (Version 4.0) was accompanied by the adjustment of the treatment regimen and, accordingly, the patients enrolled in the study according to Version 4.0 received the product starting with a loading dose of 160 mg (the first injection on Day 0) and then 80 mg on Day 7, Day 14, and then once every 2 weeks (1 time/2 weeks).
The treatment response during the run-in treatment period was defined as the resolution of the relapse observed at enrollment in the study, and the absence of new relapses of pericarditis in patients enrolled in the study with signs of pericarditis recurrence; absence of new relapses of pericarditis in patients enrolled without signs of relapse. In the absence of treatment response assessed on Day 14 of the study and then during the preparatory open-label period, the use of RPH-104 in patients was discontinued, and they were prescribed treatment with other drugs at the Investigator's choice.
After two weeks of open-label therapy (starting from Day 14 of the run-in treatment period), patients with a response to the study drug treatment began to discontinue the previous treatment of the underlying disease. Monotherapy with NSAIDs, colchicine, or their combination was discontinued simultaneously. The GCS dose was gradually tapered over 12 weeks with subsequent complete discontinuation (thus, these patients received RPH-104 in combination with GCS for 14 weeks and RPH-104 as monotherapy for the next 10 weeks).
The patients who demonstrated response to therapy with RPH-104 during the run-in treatment period, were transferred to the randomized withdrawal period.
Randomized withdrawal period (24 weeks: Day 0 since the randomization - Day 168 since the randomization) included treatment with RPH-104 or placebo depending on the randomization group (1:1 ratio) once in 2 weeks and the efficacy and safety monitoring procedures; Patients have been receiving therapy for a period of 24 weeks: the study drug group (to receive RPH-104 80 mg SC) or placebo group (to receive an equivalent volume of placebo subcutaneous (SC)).
In case of recurrence of pericarditis during the period of randomized withdrawal (regarded as a lack of response), the treatment group was unblinded. Patients in the placebo group were prescribed open-label therapy with RPH-104 as a single dose of 160 mg subcutaneously (first injection), followed by administration of 80 mg every 7 days, 14 days after the first injection. Also, at the Investigator's discretion, the use of NSAIDs and/or colchicine was allowed for relapse treatment in these patients. The response to therapy in such patients was assessed 3 days and 7 days after the first open-label injection of the study drug, according to the results of which, if the relapse was not resolved, the same treatment regimen was continued (including NSAIDs and/or colchicine). Evaluation of the drug efficacy in these patients was carried out 14 days after switching to active study drug treatment. In case of resolution of the developed relapse, the use of NSAIDs/colchicine was to be discontinued simultaneously, the use of the study drug was to be continued at a dose of 80 mg once every other week with an efficacy assessment every 4 weeks until the end of the randomized withdrawal period of the study.
Patients from the RPH-104 group (as well as patients from the placebo group who were switched to the study drug therapy due to a disease recurrence, those in whom the recurrence did not resolve within 14 days, or patients who developed a new recurrence after the resolution of the previous one) were switched to treatment with other drugs at the discretion of the Investigator; these patients had to come to a follow-up safety visit in 2 and 8 weeks after the administration of the last dose of the study drug.
Thus, after the end of randomized withdrawal period, the patients who responded to therapy with RPH-104 were asked to transfer to the open-label study to evaluate the long-term safety and efficacy of RPH-104. Non-responders, as well as patients who did not agree to participate in the open-label study of safety and efficacy, had to come to follow-up safety visits.
Thus, the maximum duration of treatment in this study was 36 weeks (for patients who were receiving NSAIDs and/or colchicine at the study enrollment) and 48 weeks (as a monotherapy or in combination with NSAIDs and/or colchicine).
The total maximal duration of the study was planned to be approximately 60 weeks.
A total of 20 patients with idiopathic recurrent pericarditis were planned to be randomized into the study. Taking into account potential dropouts during the screening and the run-in treatment period, the number of screened patients (signing the Informed Consent Form) could be as large as 35.
54 studies on the registry are indexed under Pericarditis; 23 are open to participants now.
This study's enrollment of 20 is below the median of 60 across 29 interventional studies indexed under Pericarditis.
Browse Pericarditis studies →R-Pharm International, LLC is the lead sponsor of 17 studies on the registry; 4 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Confirmed diagnosis of idiopathic recurrent pericarditis, established on the basis of the European Society of Cardiology (ESC) diagnostic criteria (2015):
OR Consent of sexually active male subjects to use highly effective contraceptive methods throughout the study, starting from the beginning of the screening and for at least 8 weeks after discontinuation of the study drug.
Highly effective methods of contraception include the following:
use of a combination of any two of the following (a+b or a+c or b+c):
Exclusion Criteria:
Any conditions or signs in the patient that, according to the investigator's judgement, indicate a disorder (suppression) of the patient's immune response and/or significantly increase the risk of immunomodulatory therapy, including, but not limited to, the following:
A history of active tuberculosis or the presence of risk factors or signs indicating the presence of active or latent infection caused by M. Tuberculosis, including but not limited to the following:
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.
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.
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 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.
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.
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.
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.
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
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 number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
A blood test of average blood sugar over the past two to three months, given as a percentage or in mmol/mol. 6.5% (48 mmol/mol) or more is the usual threshold for diabetes, normally confirmed by a second test.
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.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
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.
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.
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.
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.
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.
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.
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.
Autoimmune disease
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
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.
Creatinine clearance (CrCl)
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
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.
Absolute neutrophil count (ANC)
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HbA1c
A blood test of average blood sugar over the past two to three months, given as a percentage or in mmol/mol. 6.5% (48 mmol/mol) or more is the usual threshold for diabetes, normally confirmed by a second test.
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.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
subjects will receive RPH-104 at a dose of 80 mg subcutaneously once every 2 weeks
Biological: RPH-104
subjects will receive placebo subcutaneously once every 2 weeks
Drug: Placebo
solution for subcutaneous administration 40 mg/mL, 2 mL in the 4-mL transparent glass vial
Normal Saline (0.9% Sodium Chloride solution for Injection), 5 mL in a polypropylene ampoule, 10 ampoules per labeled package
Time (days) to the recurrence within 24 weeks after randomization
Time (days) to the recurrence within 24 weeks after randomization in patients with idiopathic recurrent pericarditis treated with RPH-104 compared to placebo.
Time frame: up to 24 weeks after randomization
Proportion of patients demonstrating a response to RPH-104 treatment
Proportion of patients demonstrating a response to RPH-104 treatment on Day 3, Day 7, Day 14 of the run-in treatment period
Time frame: Day 3, Day 7, Day 14 of the run-in treatment period
The proportion of patients switched to active treatment with RPH-104
The proportion of patients switched to active treatment with RPH-104 due to the development of a relapse during the period of randomized withdrawal, with a response to treatment in 3 days, 7 and 14 days after the study drug administration.
Time frame: Day 3, Day 7, Day 14 of the run-in treatment period
Proportion of patients with pericarditis recurrence throughout the run-in treatment period
Proportion of patients with pericarditis recurrence throughout the run-in treatment period.
Time frame: up to 24 weeks
Proportion of patients, treated with RPH-104 with pericarditis recurrence recurrence within 24 weeks after the randomization, compared to placebo.
Proportion of patients, treated with RPH-104, with pericarditis recurrence within 24 weeks after the randomization, compared to placebo.
Time frame: up to 24 weeks
Proportion of patients who completely discontinued corticosteroids within 12 weeks following the response to therapy throughout the run-in treatment period.
Proportion of patients who completely discontinued corticosteroids within 12 weeks following the response to therapy throughout the run-in treatment period
Time frame: up to 24 weeks
Change in chest pain from baseline assessed by the patients during the run-in treatment period
Change in chest pain from baseline (Day 0 of the run-in treatment period) assessed by the patients using a numeric rating scale (NRS) during the run-in treatment period. NRS contains 11 values from 0 to 10 points, where 0 is no pain, 10 is unbearable pain.
Time frame: from Day 0 up to 24 weeks
Change in chest pain assessed by the patients during the randomized withdrawal period
Change in chest pain from the assessment on the Day 0 of the randomized withdrawal period assessed by the patients using NRS during the randomized withdrawal period in patients treated with RPH-104 compared to placebo. NRS contains 11 values from 0 to 10 points, where 0 is no pain, 10 is unbearable pain.
Time frame: from Day 0 of the randomized withdrawal period up to 24 weeks
Change in C-reactive protein (CRP) level during the run-in treatment period
Change in C-reactive protein (CRP) level from baseline (Day 0 of the run-in treatment period) during the run-in treatment period.
Time frame: from Day 0 of up to 24 weeks
Change in C-reactive protein (CRP) level during the randomized withdrawal period
Change of the CRP level from Day 0 of the randomized withdrawal period during the randomized withdrawal period for RPH-104 administration versus placebo.
Time frame: from Day 0 of the randomized withdrawal period up to 24 weeks
Change of the size of pericardial effusion from baseline based on echocardiography (Echo-CG) data during the run-in treatment period
Change of the size of pericardial effusion from baseline (Day 0 of the run-in treatment period) based on Echo-CG data during the run-in treatment period.
Time frame: from Day 0 up to 24 weeks
Change of the size of pericardial effusion from baseline based on Echo-CG data during the randomized withdrawal period
Change of the size of pericardial effusion according to the echocardiography data from Day 0 of the randomized withdrawal period during the randomized withdrawal period for RPH-104 administration versus placebo.
Time frame: from Day 0 of the randomized withdrawal period up to 24 weeks
Change in physician global assessment of disease activity from baseline assessed using a numeric rating scale during the run-in treatment period
Change in physician global assessment of disease activity from baseline (Day 0 of the run-in treatment period) assessed using a numeric rating scale during the run-in treatment period. Global disease activity is assessed by the physician using a NRS containing 11 values from 0 to 10 points, where 0 is the absence of active disease, 10 is the maximum activity of the disease.
Time frame: from Day 0 up to 24 weeks
Change in physician global assessment of disease activity assessed using a numeric rating scale during the randomized withdrawal period
Change in physician global assessment of disease activity from the assessment on the Day 0 of the randomized withdrawal period assessed using a numeric rating scale during the randomized withdrawal period in patients treated with RPH-104 compared to placebo. Global disease activity is assessed by the physician using a NRS containing 11 values from 0 to 10 points, where 0 is the absence of active disease, 10 is the maximum activity of the disease.
Time frame: from Day 0 of the randomized withdrawal period up to 24 weeks
Change in patient's overall health self-assessment during the run-in treatment period
Change from baseline (Day 0 of the preparatory therapy period) of the patient's overall health self-assessment on the numerical rating scale during the preparatory therapy period. The patient's overall health is assessed using a NRS containing 11 values from 0 to 10 points, where 0 is very good, 10 is very poor.
Time frame: from Day 0 up to 24 weeks
Change in patient's overall health self-assessment during the randomized withdrawal period
Change of the patient's overall health self-assessment on the numerical rating scale from Day 0 of the randomized withdrawal period during the randomized withdrawal period of RPH-104 administration versus placebo.
Time frame: from Day 0 of the randomized withdrawal period up to 24 weeks
Change in quality of life from baseline based on data of the SF-36 questionnaire during the run-in treatment period
Change in quality of life from baseline (Day 0 of the run-in treatment period) based on data of the SF-36® questionnaire during the run-in treatment period
Time frame: from Day 0 up to 24 weeks
Change in quality of life based on data of the SF-36 questionnaire during the randomized withdrawal period
Change in quality of life from the assessment on the Day 0 of the randomized withdrawal period based on data of the SF-36 questionnaire during the randomized withdrawal period in patients treated with RPH-104 compared to placebo.
Time frame: from Day 0 of the randomized withdrawal period up to 24 weeks
Plan to share: No
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Ran from Feb 2020 to primary completion in Jan 2022, 23 months.
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Studies of the drug tested here: Goflikicept