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Long-Term safety of mitochondrial drug confirmed in 101 patients

NCT ID NCT05218655

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
Running, but no longer taking on new participants.
Completed This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Jun 27, 2026

Summary

This study looked at the safety of vatiquinone in 101 people with inherited mitochondrial disease who had already taken the drug in a previous study or treatment plan. The goal was to track any side effects until the drug became commercially available or the program ended. Participants had conditions like Leigh syndrome, MELAS, or other mitochondrial diseases.

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

101 people

The number who actually took part.

Started

Jun 2022

Finished

Apr 2025

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 inherited mitochondrial disease including Leigh syndrome, Alpers syndrome, mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes (MELAS), myoclonic epilepsy with ragged-red fibers (MERRF), pontocerebellar hypoplasia type 6 (PCH6), or other mitochondrial disease who participated in a previous vatiquinone clinical study or treatment plan. * Women of childbearing potential must have a negative pregnancy test at screening/baseline and agree to abstinence or the use of at least 1 of the highly effective forms of contraception as specified in the protocol (with a failure rate of \<1% per year when used consistently and correctly). Highly effective contraception or abstinence must be continued for the duration of the study, and for up to 30 days after the last dose of study drug. * Fertile men who are sexually active with women of childbearing potential and who have not had a vasectomy, must agree to use a barrier method of birth control during the study and for up to 30 days after the last dose of study drug. Exclusion Criteria: * Current participation in any other interventional study. * Pregnancy or breast feeding.

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Conditions

The condition(s) this trial relates to.

Diffuse Cerebral Sclerosis of Schilder Drug Resistant Epilepsy Leigh syndrome MELAS syndrome MERRF syndrome mitochondrial disease mitochondrial DNA depletion syndrome 4a mitochondrial encephalomyopathy pontocerebellar hypoplasia type 6

As listed by the trial registrant

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

Inherited mitochondrial disease

Contacts and locations

Locations

  • Akron Children's Hospital

    Akron, Ohio, 44308, United States

  • Baylor College of Medicine

    Houston, Texas, 77030, United States

  • CHU Montpellier - Hopital Saint-Eloi

    Montpellier, 34295, France

  • Child Neurology Center of Northwest Florida

    Gulf Breeze, Florida, 32561, United States

  • Children's Hospital of Philadelphia - CHOP

    Philadelphia, Pennsylvania, 19104, United States

  • Children's National

    Washington D.C., District of Columbia, 20010, United States

  • Chu Angers

    Angers, 49933, France

  • Cleveland Clinic

    Cleveland, Ohio, 44195, United States

  • Columbia University Medical Center - CUMC

    New York, New York, 10032, United States

  • Great Ormond Street Hospital for Children NHS Foundation Trust

    London, WC1N 3JH, United Kingdom

  • Hopital Necker-Enfants Malades

    Paris, 75015, France

  • Hospital Ruber Internacional

    Madrid, 28034, Spain

  • Hospital Sant Joan de Déu

    Barcelona, 08950, Spain

  • Hospital Universitario 12 de Octubre

    Madrid, 28003, Spain

  • Hôpital de Hautepierre - Hôpitaux Universitaires de Strasbourg

    Strasbourg, 67000, France

  • Instytut Pomnik - Centrum Zdrowia Dziecka

    Warsaw, 04-730, Poland

  • Medical University of South Carolina - MUSC

    Charleston, South Carolina, 29425, United States

  • Ospedale Pediatrico Bambino Gesù

    Roma, 00165, Italy

  • PTC Clinical Site

    Japanese City, Japan

  • Seattle Children Hospital

    Seattle, Washington, 98105, United States

  • Stanford University

    Stanford, California, 94305, United States

  • The Johns Hopkins University

    Baltimore, Maryland, 21205, United States

  • The Newcastle Upon Tyne Hospitals NHS Foundation Trust

    Newcastle upon Tyne, NE1 4LP, United Kingdom

  • UT Health The University of Texas

    Houston, Texas, 77030, United States

  • University of California, San Diego Altman Clinical and Translational Research Institute

    La Jolla, California, 92037, United States

  • Yale Medicine

    New Haven, Connecticut, 06511, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.