Long-Term Open-Label Study to Assess the Safety and Efficacy of Vatiquinone in Patients With Friedreich Ataxia

Phase 3

Recruiting

• Conditions: Friedreich ataxia

• Registration Number: 2024-516505-23-00
• Lead Sponsor: PTC Therapeutics Inc.

Brief Summary

To assess the long-term safety of vatiquinone in subjects with FA previously exposed to vatiquinone

Detailed Description

Not available

Study Timeline

• Study First Posted: 2024-10-24
• Last Update Posted: 2024-11-25

Recruitment & Eligibility

• Status: Ongoing, recruiting
• Sex: Not specified
• Target Recruitment: 34

Inclusion Criteria

Subjects with FA who completed and directly rolled over from a previous vatiquinone clinical study.

Males and females of childbearing potential must be willing to use an effective method of contraception (eg, implants, injectables, transdermal patch, combined oral contraceptives, barrier methods, and intrauterine devices) from the time consent is signed until 30 days after last dose of study drug or Early Termination Visit. Male subjects must agree not to donate sperm during the study and for at least 30 days after the last dose of study drug or Early Termination Visit. Note: Double-barrier method (ie, condom with spermicide) is required if no other methods of contraception are in use.

Subject provides consent to participate.

Exclusion Criteria

Current participation in any other interventional study

Pregnancy or breastfeeding

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Adverse events (AEs)/serious adverse events (SAEs), laboratory abnormalities, vital signs, and electrocardiograms (ECGs)		Adverse events (AEs)/serious adverse events (SAEs), laboratory abnormalities, vital signs, and electrocardiograms (ECGs)

Secondary Outcome Measures

Name	Time	Method
Change from baseline in the mFARS and its 4 subscales (Upright Stability, Upper Limb, Lower Limb, Bulbar)		Change from baseline in the mFARS and its 4 subscales (Upright Stability, Upper Limb, Lower Limb, Bulbar)

Trial Locations

Locations (4)

Assistance Publique Hopitaux De Paris; 🇫🇷 Paris, France

Universitaetsklinikum Tuebingen AöR; 🇩🇪 Tuebingen, Germany

Ospedale Pediatrico Bambino Gesu; 🇮🇹 Rome, Italy

Hospital Sant Joan De Deu Barcelona; 🇪🇸 Esplugues De Llobregat, Spain

Assistance Publique Hopitaux De Paris

🇫🇷Paris, France

Alexandra Durr

Site contact

+33142161347

elodie.petit@icm-institute.org