Phase 3
N=47
Rilonacept for Treatment of Cryopyrin-Associated Periodic Syndromes (CAPS)
Familial Cold Autoinflammatory Syndrome (FCAS) · Familial Cold Urticaria · Muckle-Wells Syndrome (MWS) · Genetic Diseases, Inborn
Bottom Line
View on ClinicalTrials.gov: NCT00288704 ↗Enrolled (actual)
47
Serious AEs
5.5%
Results posted
Nov 2009
Primary outcome: Primary: Change From Baseline to Week-6 (Part A) Endpoint in Mean Key Symptom Score (KSS) — 2.4; 3.1; 2.1; 0.5 Units of a Scale — p=<.0001
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- rilonacept 160 mg (Drug); Placebo (Drug)
- Age
- Pediatric, Adult, Older Adult · 7+ yrs
- Sex
- All
- Sponsor
- Regeneron Pharmaceuticals
- Primary completion
- Jun 2008
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline to Week-6 (Part A) Endpoint in Mean Key Symptom Score (KSS) |
2.4; 3.1; 2.1; 0.5; -0.3; -2.6 | <.0001 sig |
| PRIMARY Mean Change in Key Symptom Score (KSS) From Week 15 to Week 24 (During the Randomized Withdrawal Phase or Part B) |
0.2; 0.3; 1.2; 0.4; 0.9; 0.1 | <0.001 sig |
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.
Eligibility Criteria
Inclusion Criteria
- Double-blind phases: adults age 18 and above; Open-label extension: Adults and children aged 7 years and older.
- Was diagnosed with Familial Cold Auto-inflammatory Syndrome (FCAS) or Muckle-Wells Syndrome (MWS) based upon clinical signs and symptoms
- Had documented mutation in NLRP-3 (Cold Induced Autoinflammatory Syndrome-1 or CIAS1) in subject or relative, and willingness to have a confirmatory genetic (Deoxyribonucleic acid or DNA) test (cheek swab).
- Was able to understand and comply with study procedures and was able to provide informed consent
- If female, was not currently pregnant and was willing to use contraception during the study
Exclusion Criteria
- Had evidence of untreated tuberculosis or other conditions/therapies that made the subject inappropriate for this study.
Data sourced from ClinicalTrials.gov (NCT00288704). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.