Can a new drug slow friedreich ataxia? Long-Term trial underway

NCT ID NCT05515536

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

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 2 times

Summary

This study is testing the long-term safety of a drug called vatiquinone in 130 people with Friedreich ataxia, a rare genetic disease that affects movement and coordination. All participants have taken vatiquinone before in earlier studies. Researchers will track side effects and measure changes in disease severity over three years.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: vatiquinone
What this could lead to: If successful, this could show that vatiquinone is safe and may help slow the progression of Friedreich ataxia over years.
What could go wrong: This is an open-label study with no placebo group, so results may be less reliable. It only includes people who already tolerated the drug, which may not reflect the broader population.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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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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