Hope for mitochondrial disease: new drug targets debilitating fatigue

NCT ID NCT06451757

First seen Jun 27, 2026 · Last updated Jun 30, 2026 · Updated 2 times

Summary

This study tests whether the drug sonlicromanol can reduce fatigue and improve physical abilities like balance and leg strength in adults with a specific genetic form of mitochondrial disease. About 220 participants will take either the drug or a placebo twice daily for 52 weeks. The main outcomes are changes in fatigue scores and a sit-to-stand test.

What this could mean

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

Active substance

sonlicromanol

What this could lead to

If it works, this could provide a treatment to ease fatigue and improve daily physical abilities for people with mitochondrial disease.

What could go wrong

This is a Phase 3 trial, but it's still experimental. The drug may not prove better than placebo, and side effects are possible. Results may not apply to all mitochondrial disease types.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

inborn mitochondrial metabolism disorder maternally-inherited diabetes and deafness MELAS syndrome mitochondrial disease

As listed by the trial registrant

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

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • CHU de Bordeaux - Hôpital Pellegrin Service Gynecologie Obstetrique

    NOT_YET_RECRUITING

    Bordeaux, Gironde, 33000, France

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Cleveland Clinic Neurological Institute Mellen Center

    NOT_YET_RECRUITING

    Cleveland, Ohio, 44195, United States

    Contact Email: •••••@•••••

    Contact

  • Fondazione IRCCS Istituto Neurologico Carlo Besta

    RECRUITING

    Milan, 20133, Italy

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Groupe Hospitalier Pitie-Salpetriere - Charles-Foix Clinical Investigation Center Paris-Est

    NOT_YET_RECRUITING

    Paris, Paris, 75013, France

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Klinikum der Universität München Friedrich-Baur-Institut

    RECRUITING

    München, 80336, Germany

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • Massachusetts General Hospital

    NOT_YET_RECRUITING

    Boston, Massachusetts, 02114, United States

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Radboud University Medical Center

    RECRUITING

    Nijmegen, Gelderland, 6525, Netherlands

    Contact Email: •••••@•••••

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

  • Rigshospitalet, University of Copenhagen

    NOT_YET_RECRUITING

    Kopenhagen, Region Sjælland, DK2100, Denmark

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • The University of Texas Health Science Center at Houston

    RECRUITING

    Houston, Texas, 77030, United States

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

    Contact

  • University College London Hospitals NHS Foundation Trust National Hospital for Neurology and Neurosurgery

    RECRUITING

    London, Greater London, WC1N 3BG, United Kingdom

    Contact Email: •••••@•••••

    Contact