RecruitingPhase 3NCT06451757

KHENERFIN Study: A Trial to Evaluate the Efficacy and Safety of Sonlicromanol in Primary Mitochondrial Diseases

A Phase III, Randomised, Double-blind, Placebo-controlled, Parallel-group, Pivotal Trial to Assess the Efficacy and Safety of Sonlicromanol in Adult Subjects With a Genetically Confirmed Mitochondrial DNA tRNALeu(UUR) m.3243A>G Variant


Sponsor

Khondrion BV

Enrollment

220 participants

Start Date

Feb 1, 2026

Study Type

INTERVENTIONAL

Conditions

Summary

The KHENERFIN study aims to determine whether the study medicine, sonlicromanol, is able to reduce symptoms of fatigue and the impact of fatigue on daily life, and whether sonlicromanol is able to improve physical abilities of people like balance control and lower limb skeletal muscle strength in people with mitochondrial disease. In this study, the effects of sonlicromanol are compared against a placebo, a tablet identical in appearance and taste but without the active drug. Participants take either sonlicromanol or placebo twice daily for a treatment duration of 52 weeks. In addition to these primary objectives, the study evaluates the efficacy of sonlicromanol on secondary and exploratory outcomes, as well as its safety and tolerability after one year of treatment.


Eligibility

Min Age: 18 Years

Plain Language Summary

Simplified for easier understanding

This study tests a drug called sonlicromanol in people with a specific inherited mitochondrial disease (a genetic condition where the body's energy-producing cells don't work properly). The trial focuses on patients with the m.3243A>G mutation who experience fatigue and muscle weakness. **You may be eligible if...** - You are 18 or older with a confirmed m.3243A>G mitochondrial DNA mutation - You have been experiencing chronic fatigue for at least 3 months - You have measurable muscle weakness (assessed by a sit-to-stand test) - Your mutation level in the blood or other tissue is 20% or higher **You may NOT be eligible if...** - You have taken an investigational drug within the past 3 months - You have severe organ problems that could interfere with the study - You are pregnant or planning to become pregnant Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGSonlicromanol

Administration of 90 mg sonlicromanol (100 mg sonlicromanol.HCl) twice daily during 52 weeks

DRUGPlacebo

Administration of 100 mg placebo twice daily during 52 weeks


Locations(10)

Massachusetts General Hospital

Boston, Massachusetts, United States

Cleveland Clinic Neurological Institute Mellen Center

Cleveland, Ohio, United States

The University of Texas Health Science Center at Houston

Houston, Texas, United States

Rigshospitalet, University of Copenhagen

Kopenhagen, Region Sjælland, Denmark

CHU de Bordeaux - Hôpital Pellegrin Service Gynecologie Obstetrique

Bordeaux, Gironde, France

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

Paris, Paris, France

Klinikum der Universität München Friedrich-Baur-Institut

München, Germany

Fondazione IRCCS Istituto Neurologico Carlo Besta

Milan, Milano, Italy

Radboud University Medical Center

Nijmegen, Gelderland, Netherlands

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

London, Greater London, United Kingdom

View Full Details on ClinicalTrials.gov

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NCT06451757


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