Back to resultsA Study to Evaluate RIST4721 in Familial Mediterranean Fever (FMF)
Primary Purpose
Familial Mediterranean Fever
Status
Withdrawn
Phase
Phase 2
Locations
Study Type
Interventional
Intervention
RIST4721
Sponsored by
About this trial
This is an interventional treatment trial for Familial Mediterranean Fever
Eligibility Criteria
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
Sites / Locations
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
RIST4721 400 mg
Arm Description
RIST4721 400 mg: 4 active (100 mg) tablets once daily for 12 weeks
Outcomes
Primary Outcome Measures
Incidence of treatment-emergent adverse events (TEAEs) and serious TEAEs
Secondary Outcome Measures
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
Full Information
NCT ID
NCT05448391
First Posted
July 1, 2022
Last Updated
February 10, 2023
Sponsor
Aristea Therapeutics, Inc.
1. Study Identification
Unique Protocol Identification Number
NCT05448391
Brief Title
A Study to Evaluate RIST4721 in Familial Mediterranean Fever (FMF)
Official Title
An Open-label, Single-arm, Phase 2 Study to Evaluate the Safety and Efficacy of RIST4721 in Subjects With Familial Mediterranean Fever
Study Type
Interventional
2. Study Status
Record Verification Date
February 2023
Overall Recruitment Status
Withdrawn
Why Stopped
Due to safety findings in other ongoing Phase 2 trials.
Study Start Date
September 13, 2022 (Actual)
Primary Completion Date
November 30, 2023 (Anticipated)
Study Completion Date
December 30, 2024 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Aristea Therapeutics, Inc.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Familial Mediterranean Fever
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
0 (Actual)
8. Arms, Groups, and Interventions
Arm Title
RIST4721 400 mg
Arm Type
Experimental
Arm Description
RIST4721 400 mg: 4 active (100 mg) tablets once daily for 12 weeks
Intervention Type
Drug
Intervention Name(s)
RIST4721
Intervention Description
RIST4721 tablets, 100 mg are blue, oval, biconvex film-coated tablets
Primary Outcome Measure Information:
Title
Incidence of treatment-emergent adverse events (TEAEs) and serious TEAEs
Time Frame
Baseline to Week 12
Secondary Outcome Measure Information:
Title
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
Time Frame
Baseline to Week 12
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
74 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
A Study to Evaluate RIST4721 in Familial Mediterranean Fever (FMF)
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