NCT05448391

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.

Trial Health

15
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Timeline
Completed

Started Sep 2022

Status
withdrawn

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

July 1, 2022

Completed
6 days until next milestone

First Posted

Study publicly available on registry

July 7, 2022

Completed
2 months until next milestone

Study Start

First participant enrolled

September 13, 2022

Completed
1.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 30, 2023

Completed
1.1 years until next milestone

Study Completion

Last participant's last visit for all outcomes

December 30, 2024

Completed
Last Updated

February 13, 2023

Status Verified

February 1, 2023

Enrollment Period

1.2 years

First QC Date

July 1, 2022

Last Update Submit

February 10, 2023

Conditions

Familial Mediterranean Fever

Outcome Measures

Primary Outcomes (1)

  • Incidence of treatment-emergent adverse events (TEAEs) and serious TEAEs

    Baseline to Week 12

Secondary Outcomes (1)

  • 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

Study Arms (1)

RIST4721 400 mg

EXPERIMENTAL

RIST4721 400 mg: 4 active (100 mg) tablets once daily for 12 weeks

Drug: RIST4721

Interventions

RIST4721DRUG

RIST4721 tablets, 100 mg are blue, oval, biconvex film-coated tablets

RIST4721 400 mg

Eligibility Criteria

Age18 Years - 74 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • Breastfeeding or pregnant
  • Known immunodeficiency or subject is immunocompromised
  • Active/latent infection with HBV, HCV, HIV, SARS-CoV-2 or TB

Contact the study team to confirm eligibility.

Sponsors & Collaborators

MeSH Terms

Conditions

Familial Mediterranean Fever

Condition Hierarchy (Ancestors)

Hereditary Autoinflammatory DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities
0

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 1, 2022

First Posted

July 7, 2022

Study Start

September 13, 2022

Primary Completion

November 30, 2023

Study Completion

December 30, 2024

Last Updated

February 13, 2023

Record last verified: 2023-02

Data Sharing

IPD Sharing
Will not share