A Pilot Open-Label Study to Investigate the Safety and Anti-inflammatory Effects of VX-765, an Orally Administered Interleukin-1ß Converting Enzyme Inhibitor, in Patients with Muckle-Wells Syndrome

Phase 1

• Conditions: Muckle-Wells syndrome

• Registration Number: EUCTR2004-000283-27-GB
• Lead Sponsor: Vertex Pharmaceuticals Incorporated

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2005-02-14
• Study Completion: 2005-06-13
• Last Update Posted: 13 October 2020

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 6

Inclusion Criteria

Confirmed diagnosis of Muckle-Wells syndrome, as defined by mutations of the NALP3/CIAS1 gene, associated with laboratory evidence of acute phase response; symptoms of rash, conjunctivitis, fever, fatigue, or arthralgia; SAA greater than 10 mg/L on 2 consecutive occasions, patient agreement to discontinue anti-inflammatory medications.
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Pregnancy/lactation, any chronic inflammatory disorder other than Muckle-Wells Syndrome, medication that may alter drug absorption, recent use of other investigational drugs, recent blood/plasma donation, drug allergy/hypersensitivity

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified