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Sponsored by: |
Regeneron Pharmaceuticals |
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Information provided by: | Regeneron Pharmaceuticals |
ClinicalTrials.gov Identifier: | NCT00288704 |
Inflammatory symptoms of CAPS are due to mutations in a gene called "cryopyrin" (CIAS1; also known as NALP3 or PYPAF1). These mutations result in the body's overproduction of interleukin-1 (IL-1), a protein that stimulates the inflammatory process. IL-1 Trap is an experimental drug candidate that is designed to bind to the interleukin-1 cytokine and prevent it from binding to its receptors in the body.
Condition | Intervention | Phase |
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Familial Cold Autoinflammatory Syndrome Familial Cold Urticaria Muckle-Wells Syndrome Genetic Diseases, Inborn |
Biological: IL-1 Trap |
Phase II Phase III |
Study Type: | Interventional |
Study Design: | Treatment, Randomized, Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Placebo Control, Parallel Assignment, Safety/Efficacy Study |
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 |
Enrollment: | 104 |
Study Start Date: | December 2005 |
Study Completion Date: | August 2008 |
Primary Completion Date: | June 2008 (Final data collection date for primary outcome measure) |
Arms | Assigned Interventions |
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Part A: Placebo Comparator
Double blind, placebo controlled
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Biological: IL-1 Trap
Rilonacept was supplied by Regeneron as a lyophilized powder in sealed, sterile, single-use, 20-mL vials at a concentration of 80 mg/mL when reconstituted with 2.3-mL of sterile water for injection. The lyophilized formulation contained histidine, sodium citrate, PEG 3350, polysorbate 20, glycine, arginine, and sucrose (pH 6.5).
Biological: IL-1 Trap
Rilonacept was supplied by Regeneron as a lyophilized powder in sealed, sterile, single-use, 20-mL vials at a concentration of 80 mg/mL when reconstituted with 2.3-mL of sterile water for injection. The lyophilized formulation contained histidine, sodium citrate, PEG 3350, Rilonacept was supplied by Regeneron as a lyophilized powder in sealed, sterile, single-use, 20-mL vials at a concentration of 80 mg/mL when reconstituted with 2.3-mL of sterile water for injection. The lyophilized formulation contained histidine, sodium citrate, PEG 3350, polysorbate 20, glycine, arginine, and sucrose (pH 6.5). polysorbate 20, glycine, arginine, and sucrose (pH 6.5).
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Part B: Placebo Comparator
Double Blind, placebo controlled
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Biological: IL-1 Trap
Rilonacept was supplied by Regeneron as a lyophilized powder in sealed, sterile, single-use, 20-mL vials at a concentration of 80 mg/mL when reconstituted with 2.3-mL of sterile water for injection. The lyophilized formulation contained histidine, sodium citrate, PEG 3350, polysorbate 20, glycine, arginine, and sucrose (pH 6.5).
Biological: IL-1 Trap
Rilonacept was supplied by Regeneron as a lyophilized powder in sealed, sterile, single-use, 20-mL vials at a concentration of 80 mg/mL when reconstituted with 2.3-mL of sterile water for injection. The lyophilized formulation contained histidine, sodium citrate, PEG 3350, Rilonacept was supplied by Regeneron as a lyophilized powder in sealed, sterile, single-use, 20-mL vials at a concentration of 80 mg/mL when reconstituted with 2.3-mL of sterile water for injection. The lyophilized formulation contained histidine, sodium citrate, PEG 3350, polysorbate 20, glycine, arginine, and sucrose (pH 6.5). polysorbate 20, glycine, arginine, and sucrose (pH 6.5).
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24 Week Open Label Extension: Active Comparator
24 week open label extension phase
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Biological: IL-1 Trap
Rilonacept was supplied by Regeneron as a lyophilized powder in sealed, sterile, single-use, 20-mL vials at a concentration of 80 mg/mL when reconstituted with 2.3-mL of sterile water for injection. The lyophilized formulation contained histidine, sodium citrate, PEG 3350, polysorbate 20, glycine, arginine, and sucrose (pH 6.5).
Biological: IL-1 Trap
Rilonacept was supplied by Regeneron as a lyophilized powder in sealed, sterile, single-use, 20-mL vials at a concentration of 80 mg/mL when reconstituted with 2.3-mL of sterile water for injection. The lyophilized formulation contained histidine, sodium citrate, PEG 3350, Rilonacept was supplied by Regeneron as a lyophilized powder in sealed, sterile, single-use, 20-mL vials at a concentration of 80 mg/mL when reconstituted with 2.3-mL of sterile water for injection. The lyophilized formulation contained histidine, sodium citrate, PEG 3350, polysorbate 20, glycine, arginine, and sucrose (pH 6.5). polysorbate 20, glycine, arginine, and sucrose (pH 6.5).
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64 Week Open Label extension: Active Comparator
64 week open label extension phase
|
Biological: IL-1 Trap
Rilonacept was supplied by Regeneron as a lyophilized powder in sealed, sterile, single-use, 20-mL vials at a concentration of 80 mg/mL when reconstituted with 2.3-mL of sterile water for injection. The lyophilized formulation contained histidine, sodium citrate, PEG 3350, polysorbate 20, glycine, arginine, and sucrose (pH 6.5).
Biological: IL-1 Trap
Rilonacept was supplied by Regeneron as a lyophilized powder in sealed, sterile, single-use, 20-mL vials at a concentration of 80 mg/mL when reconstituted with 2.3-mL of sterile water for injection. The lyophilized formulation contained histidine, sodium citrate, PEG 3350, Rilonacept was supplied by Regeneron as a lyophilized powder in sealed, sterile, single-use, 20-mL vials at a concentration of 80 mg/mL when reconstituted with 2.3-mL of sterile water for injection. The lyophilized formulation contained histidine, sodium citrate, PEG 3350, polysorbate 20, glycine, arginine, and sucrose (pH 6.5). polysorbate 20, glycine, arginine, and sucrose (pH 6.5).
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Primary Objective:
The primary objective of this study are to assess the effect of rilonacept on the clinical signs and symptoms of CAPS when used for chronic therapy
Secondary Objective(s):
The secondary objectives are as follows:
This is a multi-center, initially double-blind, placebo-controlled study (Parts A and B) followed by extended open-label phases designed to assess the efficacy, safety, and tolerability of weekly subcutaneous (SC) doses of 160 mg of rilonacept in adult subjects with active CAPS. 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 the 24-week long Parts A and B phase of the study. Subjects then continued treatment in a 24-week open-label extension phase (OLE) and a further 64-week long-term open-label extension (LTOLE), and a 6-week post-treatment follow-up period. Amendments 4 and 6 allowed eligible adult and pediatric subjects to enroll directly into the open-label phases of the trial.
Ages Eligible for Study: | 18 Years and older |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Study Director: | Scott Mellis, MD, PhD | Regeneron Pharmaceuticals |
Study Director: | Steven P Weinstein, MD, PhD | Regeneron Pharmaceuticals |
Responsible Party: | Regeneron Pharmaceuticals, Inc. ( Robert Evans, PharmD ) |
Study ID Numbers: | IL1T-AI-0505 |
Study First Received: | February 6, 2006 |
Last Updated: | March 2, 2009 |
ClinicalTrials.gov Identifier: | NCT00288704 History of Changes |
Health Authority: | United States: Food and Drug Administration |
Familial Cold Autoinflammatory Syndrome (FCAS) Muckle-Wells Syndrome (MWS) CIAS1 NALP3 |
PYPAF1 Cryopyrin CAPS Interleukin-1 |
Metabolic Diseases Skin Diseases Glycine Citric Acid Urticaria Wells Syndrome Familial Cold Autoinflammatory Syndrome |
Amyloidosis Hypersensitivity Genetic Diseases, Inborn Hypersensitivity, Immediate Muckle-Wells Syndrome Metabolic Disorder |
Hypersensitivity Skin Diseases, Vascular Amyloidosis Metabolic Diseases Pathologic Processes Disease |
Immune System Diseases Skin Diseases Genetic Diseases, Inborn Syndrome Hypersensitivity, Immediate Urticaria |