Hope for friedreich ataxia: experimental drug vatiquinone put to the test

NCT ID NCT04577352

Completed Disease control Sponsor: PTC Therapeutics Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tested an experimental drug called vatiquinone in 146 people with Friedreich ataxia, a rare genetic disease that affects movement and coordination. Participants took either the drug or a placebo for 72 weeks to see if it slowed worsening of symptoms, measured by a standard rating scale. The goal was to find a treatment that helps control the disease, not cure it.

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Conditions

The condition(s) this trial relates to.

Friedreich ataxia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

FRIEDREICH ATAXIA

Contacts and locations

Locations

CBR Neurogenetic Research Clinic, University of Auckland

Auckland, 1023, New Zealand
CHU Sainte-Justine

Montreal, Quebec, H3T1C5, Canada
Centre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM)

Montreal, Quebec, H2X 0A9, Canada
Department of Neurology and Hertie-Institute for Clinical Brain Research German Center of Neurodegenerative Diseases (DZNE)

Tübingen, 72076, Germany
Hospital Sant Joan de Déu Barcelona Unidad de Enfermedades Neuromusculares

Barcelona, 08950, Spain
Hôpital Pitié-Salpêtrière, Institut du Cerveau (Paris Brain Institute)

Paris, 75646, France
Murdoch Children's Research Institute

Parkville, Victoria, 3052, Australia
Ospedale Pediatrico Bambino Gesu' IRCCS

Roma, 00165, Italy
The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States
UCLA

Los Angeles, California, 90095, United States
University of Campinas (UNICAMP) - School of Medical Sciences, Dept of Neurology

São Paulo, 13083-887, Brazil
University of Florida

Gainesville, Florida, 32608, United States
University of Iowa

Iowa City, Iowa, 52242, United States
University of South Florida

Tampa, Florida, 33612, United States

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