Familial Mediterranean Fever
Conditions
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.
Interventions
DRUGRIST4721
RIST4721 tablets, 100 mg are blue, oval, biconvex film-coated tablets
Sponsors
Aristea Therapeutics, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 74 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence of treatment-emergent adverse events (TEAEs) and serious TEAEs | Baseline to Week 12 |
Secondary
| Measure | Time frame |
|---|---|
| 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 |
Outcome results
None listed