Hope for mitochondrial disease: new drug targets debilitating fatigue

NCT ID NCT06451757

Recruiting now Symptom relief Sponsor: Khondrion BV Source: ClinicalTrials.gov ↗

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.

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.

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.

MATERNALLY INHERITED DIABETES AND DEAFNESS (MIDD) MITOCHONDRIAL DISEASES MITOCHONDRIAL DNA TRNALEU(UUR) M.3243A<G MUTATION MITOCHONDRIAL ENCEPHALOMYOPATHY, LACTIC ACIDOSIS AND STROKE-LIKE EPISODES (MELAS)

Contacts and locations

Locations

CHU de Bordeaux - Hôpital Pellegrin Service Gynecologie Obstetrique
NOT_YET_RECRUITING

Bordeaux, Gironde, 33000, France
Cleveland Clinic Neurological Institute Mellen Center
NOT_YET_RECRUITING

Cleveland, Ohio, 44195, United States
Fondazione IRCCS Istituto Neurologico Carlo Besta
RECRUITING

Milan, 20133, Italy
Groupe Hospitalier Pitie-Salpetriere - Charles-Foix Clinical Investigation Center Paris-Est
NOT_YET_RECRUITING

Paris, Paris, 75013, France
Klinikum der Universität München Friedrich-Baur-Institut
RECRUITING

München, 80336, Germany
Massachusetts General Hospital
NOT_YET_RECRUITING

Boston, Massachusetts, 02114, United States
Radboud University Medical Center
RECRUITING

Nijmegen, Gelderland, 6525, Netherlands
Rigshospitalet, University of Copenhagen
NOT_YET_RECRUITING

Kopenhagen, Region Sjælland, DK2100, Denmark
The University of Texas Health Science Center at Houston
RECRUITING

Houston, Texas, 77030, United States
University College London Hospitals NHS Foundation Trust National Hospital for Neurology and Neurosurgery
RECRUITING

London, Greater London, WC1N 3BG, United Kingdom

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