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Clinical trialNCT07681713Not, yet, recruitingPhase 3Interventional

Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA)

SponsorPTC TherapeuticsEnrollment120Friedreich's Ataxia
View the registry record on ClinicalTrials.gov

Summary

The lay summary the sponsor registered on ClinicalTrials.gov.

The purpose of this study is to confirm the treatment effects of vatiquinone on the key measures of FA disease progression.

Investigators

No overall officials are registered on this study record. Site-level investigators, where published, appear with their sites below. Why? →

A profile link is shown only when the registered name resolves to a person record with a confident, namesake-checked match; ambiguous names stay unlinked.

Sites

9 registered facilities, with the per-site recruitment status where the registry publishes one. ClinicalTrials.gov does not publish per-site enrollment counts.

Belgium
  • Hôpital Érasme (hôpital Universitaire de Bruxelles)

    Brussels, Belgium

    Gilles Naeije
Brazil
  • University of Campinas

    Campinas, São Paulo, Brazil

    Marcondes França
Canada
  • Centre Hospitalier de L'Universite de Montreal Chum

    Montreal, Quebec, Canada

    Antoine Duquette
France
  • Institut Du Cerveau Et de la Moelle Epinière

    Paris, France

    Alexandra Durr
Spain
  • Hospital Sant Joan de Deu

    Barcelona, Spain

    Alejandra Darling
United States
  • University of California Los Angeles

    Los Angeles, California, United States

    Susan Perlman
  • University of South Florida

    Tampa, Florida, United States

  • University of Iowa

    Iowa City, Iowa, United States

    Katherine Mathews
  • Children'S Hospital of Philadelphia

    Philadelphia, Pennsylvania, United States

    David Lynch

Site investigator names are published by ClinicalTrials.gov only while a study recruits, so completed studies list sites without them.

Drugs studied

The drug and biological interventions registered on this study, normalized to their RxNorm ingredient where possible.

Vatiquinone
Linked drugs open the directory filtered to KOLs working with them.clinicaltrials.gov ↗

Registration & key dates

Registry attributes as recorded on the ClinicalTrials.gov study record.

Start
15 Aug 2026
Primary completion
15 Mar 2029
Completion
15 Mar 2029
Last update posted
14 Aug 2026
Registered enrollment
120
Registry id
NCT07681713

Cross-registrations

EU-CTR / CTIS (EudraCT)2026-526947-37-00

Source: ClinicalTrials.gov, via the CTTI AACT database, data as of 24 Aug 2026. Registry facts are shown as registered by the study sponsor; findmyKOL does not interpret them.