A Study to Evaluate RIST4721 in Familial Mediterranean Fever (FMF)

An Open-label, Single-arm, Phase 2 Study to Evaluate the Safety and Efficacy of RIST4721 in Subjects With Familial Mediterranean Fever

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.

Study Overview

• Status: Withdrawn
• Conditions: Familial Mediterranean Fever
• Intervention / Treatment: Drug: RIST4721

• Study Type: Interventional
• Phase: Phase 2

Contacts and Locations

Study Locations

• Israel
  • Haifa, Israel, 3109601
    • Recruiting
    • Rambam Health Care Campus
• Turkey
  • Ankara, Turkey, 06560
    • Recruiting
    • Gazi University Hospital, Department of Rheumatology
  • Istanbul, Turkey, 34093
    • Recruiting
    • Istanbul Faculty of Medicine
  • Istanbul, Turkey, 34096
    • Recruiting
    • Istanbul University Cerrahpasa Faculty of Medicine
  • İzmir, Turkey, 35150
    • Recruiting
    • Izmir Katip Çelebi University Faculty of Medicine

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 74 years (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: RIST4721 400 mg; RIST4721 400 mg: 4 active (100 mg) tablets once daily for 12 weeks	Drug: RIST4721; RIST4721 tablets, 100 mg are blue, oval, biconvex film-coated tablets

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Incidence of treatment-emergent adverse events (TEAEs) and serious TEAEs	Baseline to Week 12

Secondary Outcome Measures

Outcome 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

Collaborators and Investigators

• Sponsor: Aristea Therapeutics, Inc.

Study record dates

Study Major Dates

• Study Start (ACTUAL): September 13, 2022
• Primary Completion (ANTICIPATED): November 30, 2023
• Study Completion (ANTICIPATED): December 30, 2024

Study Registration Dates

• First Submitted: July 1, 2022
• First Submitted That Met QC Criteria: July 6, 2022
• First Posted (ACTUAL): July 7, 2022

Study Record Updates

• Last Update Posted (ACTUAL): February 13, 2023
• Last Update Submitted That Met QC Criteria: February 10, 2023
• Last Verified: February 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Skin Diseases; Infections; Genetic Diseases, Inborn; Gram-Negative Bacterial Infections; Bacterial Infections; Bacterial Infections and Mycoses; Skin Diseases, Genetic; Body Temperature Changes; Fever; Brucellosis; Familial Mediterranean Fever; Hereditary Autoinflammatory Diseases
• Other Study ID Numbers: RIST4721-212

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• 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