🏛️ 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 Checklist

Full 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.

💡 How to Participate

  1. Check the checklist above to see if you may qualify.
  2. Review the official NIH record below to find the coordinating site.
  3. Ask about the visit schedule, what is covered at no cost, and any travel support.
  4. Confirm with your own doctor before making a decision.
🏛️ View Official NIH Record ↗