🏛️ Verified NIH Record • Updated 2025-10-29
A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia (NCT05515536)
Long-Term Open-Label Study to Assess the Safety and Efficacy of Vatiquinone in Patients With Friedreich Ataxia
🔵 Active, Not Recruiting Phase3 💊 Investigational Drug / Biologic Sponsor: PTC Therapeutics
🩺 Plain-English Summary (8th-Grade Level)
The primary objective of this study is to assess the long-term safety of vatiquinone in participants with Friedreich ataxia (FA) previously exposed to vatiquinone.
Who can join: Review the eligibility criteria below.
Location: UCLA — Los Angeles, California
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (14 Sites)
- UCLA — Los Angeles, California
- University of Florida — Gainesville, Florida
- University of South Florida — Tampa, Florida
- University of Iowa — Iowa City, Iowa
- The Children's Hospital of Philadelphia — Philadelphia, Pennsylvania
- Murdoch Children's Research Institute — Parkville, Victoria
- University of Campinas (UNICAMP) - School of Medical Sciences, Dept of Neurology — São Paulo,
- Centre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM) — Montreal, Quebec
- CHU Sainte-Justine — Montreal, Quebec
- Hôpital Pitié-Salpêtrière, Institut du Cerveau (Paris Brain Institute) — Paris,
- Department of Neurology and Hertie-Institute for Clinical Brain Research German Center of Neurodegenerative Diseases (DZNE) — Tübingen,
- Ospedale Pediatrico Bambino Gesu' IRCCS — Roma,
- CBR Neurogenetic Research Clinic, University of Auckland — Auckland,
- Hospital Sant Joan de Déu Barcelona Unidad de Enfermedades Neuromusculares — Barcelona,
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Friedreich Ataxia
- A Study to Find Out How BIIB141 (Omaveloxolone) is Processed in the Body and to Learn More About Its Safety in Participants With Friedreich's Ataxia Aged 2 to 15 Years Old — Philadelphia, Pennsylvania
- A Natural History Study to TRACK Brain and Spinal Cord Changes in Individuals with Friedreich Ataxia (TRACK-FA) — Gainesville, Florida
- FA Clinical Outcome Measures — Los Angeles, California
- Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia — Los Angeles, California
- Clinical Course Of Disease In Participants With FA-CM — La Jolla, California
Explore by condition: All Friedreich Ataxia clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.