Ignite Creation Date:
2024-05-06 @ 3:38 PM
Last Modification Date:
2024-10-26 @ 1:53 PM
Study NCT ID:
NCT04692766
Status:
COMPLETED
Last Update Posted:
2022-10-31
First Post:
2020-12-29
Brief Title:
Study to Evaluate the Efficacy and Safety of RPH-104 Treatment in Patients With Idiopathic Recurrent Pericarditis
Sponsor:
R-Pharm International LLC
Organization:
R-Pharm
Study Overview
Official Title:
A Double-blind Randomized Placebo-controlled Study to Evaluate the Efficacy and Safety of RPH-104 Treatment in Patients With Idiopathic Recurrent Pericarditis
Status:
COMPLETED
Status Verified Date:
2022-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
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
Detailed Description:
The study included the following periods
1 Screening period up to 4 weeks During the screening period the patients eligibility for the study was evaluated based on the inclusionnon-inclusion criteria Those patients who were considered eligible for participation in the study were enrolled in the run-in treatment period
2 Run-in treatment period 12 weeks Days 0-84 - for the patients receiving non-steroidal anti-inflammatory drugs NSAIDs andor 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 andor 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 2030 The Protocol amendment Version 40 was accompanied by the adjustment of the treatment regimen and accordingly the patients enrolled in the study according to Version 40 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 time2 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 Investigators 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
3 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 11 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 Investigators discretion the use of NSAIDs andor 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 andor 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 NSAIDscolchicine 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
4 Safety follow-up period included monitoring of safety for 8 weeks after the last dosing of the study drug
Thus the maximum duration of treatment in this study was 36 weeks for patients who were receiving NSAIDs andor colchicine at the study enrollment and 48 weeks as a monotherapy or in combination with NSAIDs andor 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
1 Screening period up to 4 weeks During the screening period the patients eligibility for the study was evaluated based on the inclusionnon-inclusion criteria Those patients who were considered eligible for participation in the study were enrolled in the run-in treatment period
2 Run-in treatment period 12 weeks Days 0-84 - for the patients receiving non-steroidal anti-inflammatory drugs NSAIDs andor 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 andor 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 2030 The Protocol amendment Version 40 was accompanied by the adjustment of the treatment regimen and accordingly the patients enrolled in the study according to Version 40 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 time2 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 Investigators 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
3 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 11 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 Investigators discretion the use of NSAIDs andor 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 andor 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 NSAIDscolchicine 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
4 Safety follow-up period included monitoring of safety for 8 weeks after the last dosing of the study drug
Thus the maximum duration of treatment in this study was 36 weeks for patients who were receiving NSAIDs andor colchicine at the study enrollment and 48 weeks as a monotherapy or in combination with NSAIDs andor 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
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None