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

NCT ID NCT05515536

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

130 people

The number who actually took part.

Started

Dec 2022

Expected to finish

Dec 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

Children (under 18), adults (18 to 64) and older adults (65 and over)

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Participants with FA who completed and directly rolled over from a previous vatiquinone clinical study. * Males and females of childbearing potential must be willing to use an effective method of contraception (for example, implants, injectables, transdermal patches, combined oral contraceptives, barrier methods, and intrauterine devices) from the time consent is signed until 30 days after the last dose of study drug or Early Termination Visit. Exclusion Criteria: * Current participation in any other interventional study * Pregnancy or breastfeeding

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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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