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KHENERFIN Study: A Trial to Evaluate the Efficacy and Safety of Sonlicromanol in Primary Mitochondrial Diseases
NCT06451757 · View on ClinicalTrials.gov ↗
Study 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.
Conditions Studied
Interventions
- DRUG Placebo
- DRUG Sonlicromanol
Study Locations (10)
Massachusetts
- Massachusetts General Hospital — Boston
Ohio
- Cleveland Clinic Neurological Institute Mellen Center — Cleveland
Texas
- The University of Texas Health Science Center at Houston — Houston
Region Sjælland
- Rigshospitalet, University of Copenhagen — Kopenhagen
Gironde
- CHU de Bordeaux - Hôpital Pellegrin Service Gynecologie Obstetrique — Bordeaux
Paris
- Groupe Hospitalier Pitie-Salpetriere - Charles-Foix Clinical Investigation Center Paris-Est — Paris
Other
- Klinikum der Universität München Friedrich-Baur-Institut — München
Milano
- Fondazione IRCCS Istituto Neurologico Carlo Besta — Milan
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 220 participants |
| Start Date | 2026-02 |
| Est. Completion | 2028-09 |
| Phase | Phase 3 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT06451757
The ClinicalTrials.gov registry entry for NCT06451757 describes a study currently listed as recruiting. It is categorized as Phase 3, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 220 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Khondrion, which has 1 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 4 conditions, with Mitochondrial Diseases appearing as the primary indexed condition, and to 2 interventions — of which Placebo is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT06451757 reports 10 study locations spanning 10 distinct geographic areas — top geographies include Massachusetts, Ohio, Texas. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT06451757 about?
NCT06451757 is a clinical study titled "KHENERFIN Study: A Trial to Evaluate the Efficacy and Safety of Sonlicromanol in Primary Mitochondrial Diseases". 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 pe...
What is the current status of trial NCT06451757?
This trial is currently recruiting. It is a Phase 3 study. The enrollment target is 220 participants. The study started on 2026-02. Estimated completion is 2028-09.
What conditions does trial NCT06451757 study?
This clinical trial studies the following conditions: Mitochondrial Diseases, Maternally Inherited Diabetes and Deafness (MIDD), Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like Episodes (MELAS), Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT06451757?
The interventions under investigation include: Placebo (DRUG), Sonlicromanol (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT06451757?
This trial is sponsored by Khondrion, which has 1 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT06451757 being conducted?
This trial has 10 study locations across Massachusetts, Ohio, Texas, Region Sjælland, Gironde. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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