Ignite Creation Date:
2024-05-05 @ 4:38 PM
Last Modification Date:
2024-10-26 @ 9:22 AM
Study NCT ID:
NCT00288704
Status:
COMPLETED
Last Update Posted:
2011-12-06
First Post:
2006-02-06
Brief Title:
Rilonacept for Treatment of Cryopyrin-Associated Periodic Syndromes CAPS
Sponsor:
Regeneron Pharmaceuticals
Organization:
Regeneron Pharmaceuticals
Study Overview
Official Title:
IL1T-AI-0505 A Multi-center Double-Blind Placebo-Controlled Study of the Safety Tolerability Efficacy of Rilonacept in Subjects With Cryopyrin-Associated Periodic Syndromes CAPS Using Parallel Group Randomized Withdrawal Designs
Status:
COMPLETED
Status Verified Date:
2011-12
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:
Inflammatory symptoms of Cryopyrin-Associated Periodic Syndrome CAPS are due to mutations in a the NLRP-3 gene previously known as Cold Induced Autoinflammatory Syndrome-1 or CIAS1 These mutations result in the bodys overproduction of interleukin-1 IL-1 a protein that stimulates the inflammatory process IL-1 Trap rilonacept was designed to bind to the interleukin-1 cytokine and prevent it from binding to its receptors in the body
Detailed Description:
Primary Objective
The primary objective of this study was to assess the effect of rilonacept on the clinical signs and symptoms of Cryopyrin-Associated Periodic Syndrome CAPS when used for chronic therapy as evaluated by the subjects themselves over time using a validated patient-reported outcomes tool
Secondary Objectives
The secondary objectives were as follows
To determine the safety and tolerability of rilonacept in subjects with CAPS
To assess the effect of rilonacept on laboratory measures of inflammation such as acute phase reactants
This was a multi-center two-part double-blind placebo-controlled study Parts A and B designed to assess the efficacy safety and tolerability of weekly subcutaneous SC doses of 160 mg of rilonacept in adult subjects with active CAPS These phases were followed by extended open-label phases After written informed consent was obtained subjects who met the protocol eligibility criteria were enrolled at one of 27 study sites in the United States The study consisted of a 3-week screening period preceding Part A a 6-week long double-blind randomized phase of the study All subjects were then treated with single-blind rilonacept for 9-weeks followed by a subsequent 9-week double-blind withdrawal phase during which subjects were re-randomized to either rilonacept or placebo Subjects then continued treatment in a 24-week open-label extension phase OLE and a further 112-week long-term open-label extension LTOLE during which all subjects received rilonacept and a 6-week post-treatment follow-up period Amendments 4 and 6 allowed eligible adult and pediatric subjects aged 7 and above to enroll directly into the open-label phases of the trial
For reporting purposes the 24-week OLE and the 112-week LTOLE was considered one Open Label Extension OLE phase This occurred after the 24-week double blind Parts A and B phase In other words OLE Week 1 corresponded to the week 25 in the study
OLE Week 72 was the final timepoint where efficacy was measured Safety continued after that timepoint until the end of the study
The primary objective of this study was to assess the effect of rilonacept on the clinical signs and symptoms of Cryopyrin-Associated Periodic Syndrome CAPS when used for chronic therapy as evaluated by the subjects themselves over time using a validated patient-reported outcomes tool
Secondary Objectives
The secondary objectives were as follows
To determine the safety and tolerability of rilonacept in subjects with CAPS
To assess the effect of rilonacept on laboratory measures of inflammation such as acute phase reactants
This was a multi-center two-part double-blind placebo-controlled study Parts A and B designed to assess the efficacy safety and tolerability of weekly subcutaneous SC doses of 160 mg of rilonacept in adult subjects with active CAPS These phases were followed by extended open-label phases After written informed consent was obtained subjects who met the protocol eligibility criteria were enrolled at one of 27 study sites in the United States The study consisted of a 3-week screening period preceding Part A a 6-week long double-blind randomized phase of the study All subjects were then treated with single-blind rilonacept for 9-weeks followed by a subsequent 9-week double-blind withdrawal phase during which subjects were re-randomized to either rilonacept or placebo Subjects then continued treatment in a 24-week open-label extension phase OLE and a further 112-week long-term open-label extension LTOLE during which all subjects received rilonacept and a 6-week post-treatment follow-up period Amendments 4 and 6 allowed eligible adult and pediatric subjects aged 7 and above to enroll directly into the open-label phases of the trial
For reporting purposes the 24-week OLE and the 112-week LTOLE was considered one Open Label Extension OLE phase This occurred after the 24-week double blind Parts A and B phase In other words OLE Week 1 corresponded to the week 25 in the study
OLE Week 72 was the final timepoint where efficacy was measured Safety continued after that timepoint until the end of the study
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None