A Study to Evaluate RIST4721 in Familial Mediterranean Fever (FMF)
- Registration Number
- NCT05448391
- Lead Sponsor
- Aristea Therapeutics, Inc.
- Brief Summary
A 12-week Open-label, Single-arm, Phase 2 Study to Evaluate the Safety and Efficacy of RIST4721 in Subjects with Familial Mediterranean Fever followed by an additional Open-label Extension Phase.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- WITHDRAWN
- Sex
- All
- Target Recruitment
- Not specified
Inclusion Criteria
- Diagnosis of type 1 FMF disease but no active flare at the time of screening according to Tel Hashomer criteria or EUROFEVER/Paediatric Rheumatology International Trials Organization (PRINTO) criteria for FMF
- Documented to be heterozygous or homozygous for at least one of the known clearly pathogenic MEFV gene exon 10 mutations
- Males and females must be willing to use birth control as indicated
Exclusion Criteria
- Breastfeeding or pregnant
- Known immunodeficiency or subject is immunocompromised
- Active/latent infection with HBV, HCV, HIV, SARS-CoV-2 or TB
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description RIST4721 400 mg RIST4721 RIST4721 400 mg: 4 active (100 mg) tablets once daily for 12 weeks
- Primary Outcome Measures
Name Time Method Incidence of treatment-emergent adverse events (TEAEs) and serious TEAEs Baseline to Week 12
- Secondary Outcome Measures
Name Time Method Proportion of responders who achieve resolution of their index flare after initiating study treatment and do not experience a new flare from the time of resolution of the index flare until Week 12 Baseline to Week 12