NCT00288704

Rilonacept for Treatment of Cryopyrin-Associated Periodic Syndromes (CAPS)

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
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 104 (actual)
• Sponsor: Regeneron Pharmaceuticals · Industry
• Sex: All
• Age: 7 Years
• Healthy volunteers: Not accepted

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 body's 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 Objective(s): 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.

Conditions

• Familial Cold Autoinflammatory Syndrome (FCAS)
• Familial Cold Urticaria
• Muckle-Wells Syndrome (MWS)
• Genetic Diseases, Inborn

Interventions

Type	Name	Description
DRUG	rilonacept 160 mg	Rilonacept was given by subcutaneous injection. It was administered weekly at the dose of 160mg. On Day 1, subjects received two injections of rilonacept (for a total of 320 mg).
DRUG	Placebo	Subcutaneous injection of Placebo occurred during first 6 weeks of the study or during randomized withdrawal (weeks 15-24). On Day 1, subjects received two placebo injections.
DRUG	rilonacept 160 mg	Rilonacept was given by subcutaneous injection. It was administered weekly at the dose of 160mg. No loading dose was given for subjects who entered directly into the open-label.

Timeline

• Start date: 2005-12-01
• Primary completion: 2008-06-01
• Completion: 2008-08-01
• First posted: 2006-02-08
• Last updated: 2011-12-06
• Results posted: 2009-11-13

Locations

24 sites across 1 country: United States