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Recruiting Phase 3

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

NCT06451757 · tracked via the Priya Life Science France tracker
Sponsor
Khondrion BV
Phase
Phase 3
Started
2026-04-14
Last updated
2026-10-14

Condition(s) studied

Mitochondrial DiseasesMaternally Inherited Diabetes and Deafness (MIDD)Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like Episodes (MELAS)Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation

Investigational drug(s) / intervention(s)

Sonlicromanol →Placebo

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

Placebo: Administration of 100 mg placebo twice daily during 52 weeks

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.

Eligibility

Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
Inclusion criteria 1. Signed Informed Consent 2. Males and females aged ≥18 years with a multi-system primary mitochondrial disease. 3. A confirmed mitochondrial DNA tRNALeu(UUR) m.3243A\>G mutation (m.3243A\>G PMD) plus an age adjusted heteroplasmy percentage ≥ 20% in white blood cells \[=blood heteroplasmy/0.977(age+12)\]. Or in urine (urinary epithelial cells), or buccal smear or skeletal muscle (results (obtained per local guidance) ≥ 20% must be available prior to the subject being randomized). 4. Presence of chronic fatigue (not attributable to other etiologies than PMD): 1. Patient self-reported chronic fatigue for at least 3 months prior to the Screening Visit and recorded in the clinical patient files; AND 2. Presence of fatigue (raw total score \>22), assessed by Neuro-QoL SFv1-F at Screening. 5. Presence of mitochondrial myopathy defined as: 5xSST at Screening and Baseline should be ≥ 11 seconds and participant must demonstrate the ability to complete the test at baseline (i.e., complete the test within 30 seconds). 6\. Other Inclusion criteria per protocol. Exclusion criteria 1. Treatment with any IMP within 3 months (or 5 times the half-life of the IMP, whichever is longer) prior to screening or plans to use an IMP (other than the study intervention) during the study. 2. Bone deformities, motor abnormalities or chronic ulcers that in the opinion of the PI may interfere with and/or confound the interpretation of the subject's performance during the 5 times sit to stand test (5XSST). 3. Surgery of gastrointestinal tract that might interfere with drug absorption. Or severe GI dysmotility, chronic vomiting, diarrhea, bouts of pseudo-obstruction which will impair appropriate IMP absorption in the opinion of the investigator. 4. Clinically significant respiratory disease and/or cardiac disease (medical history or current clinical findings) in the opinion of the investigator. 5. Prior interventional cardiac procedure (e.g., cardiac catheterization, angioplasty/percutaneous coronary intervention, balloon valvuloplasty, etc.) within 3 months prior to screening. 6. Structural heart disease based on cardiac MRI or Echocardiography (e.g., clinically significant valve disease; i.e., aortic or mitral valve stenosis or regurgitation) and / or Left ventricular dysfunction (LVEF \<52% in men and \< 54% in women) and / or congestive cardiac failure (more severe than grade II) and / or Maximum left-ventricular septal or posterior wall thickness \> 15 mm and / or Evidence of active myocardial ischaemia, ECG evidence and / or recent history of myocardial infarction (\< 6-months) and / or coronary angioplasty / coronary bypass surgery (\< 12 months). 7. Family history of unexplained/uninvestigated syncope or congenital long and short QT syndrome or sudden death (under the age of 60) and / or Abnormal QTcF interval \[QTcF \> 450 msec (men) or QTcF \> 470 msec (women) = abnormally long QTcF or QTcF \< QT \< 320ms = abnormally short QtcF\] and / or Intermittent or persistent WPW (pre-excitation) syndrome on ECG \[ie: abnormally wide QRS \> 120ms with slurred up-stroke ('delta' wave) and PR-interval \< 120ms\] and / or ECG evidence of atrial fibrillation (permanent, persistent or paroxysmal) and / or High grade AV Block \[2nd-degree AV block Type II (ie: 2:1) or complete AV block (3rd degree) or intermittent conduction system abnormalities with the exception of any of the following: * 1st degree atrioventricular (AV)-block * 2nd degree AV-block Type 1 (Mobitz Type 1 / Wenckebach type) * Right bundle branch block. 8. History of acute heart failure (within the last 3 months). 9. Higher degree of AV-blocks (AVB II° or III°). 10. Other exclusion criteria per protocol

Primary outcome measure(s)

  • Neuro-QoL Fatigue Short Form v1 — Screening, Baseline (Day 1), Weeks 13, 26, 39, 52 (End of Trial Visit)
    Change from baseline compared to placebo at week 52 and at other relevant study visits of the Quality of Life in Neurological Disorders Fatigue Short Form version 1 (Neuro-QoL Fatigue - SF v1): The Neuro-QoL Fatigue SF v1 is an 8-item self-assessment questionnaire evaluating the perception of fatigue and its impact in daily life activities. Each question is scored as following: 1=Never, 2=Rarely, 3=Sometimes, 4=Often, and 5=Always. Total raw scores range from 8-40. T-scores are calculated from the short form scoring table provided in the instruments´ manual. T-score distributions rescale raw scores into standardized scores with a mean of 50 and a standard deviation (SD) of 10. Change from baseline: Negative numbers mean less fatigue, better outcome, positive score means more fatigue, worse outcome.
  • Five Times Sit-To-Stand Test (5XSST) — Screening, Baseline (Day 1), Weeks 13, 26, 39, 52 (End of Trial Visit)
    Change from Baseline compared to placebo at week 52 and at other relevant study visits of the 5xSST in total time (in seconds) to complete the 5xSTS. The 5xSST scoring is based on the amount of time (to the nearest decimal in seconds a subject is able to transfer from a seated to a standing position and back to sitting five times. Inability to complete five repetitions without assistance or use of upper extremity support indicates 'failure to perform of test, any modifications should be documented. Participant is directed to stand up straight as quickly as possible 5 times, without stopping in between, keeping arms folded across the chest. When 5xSST is not reached within 30.0 seconds the test is stopped and the actual number of sit to stands reached within those 30 seconds is recorded. Faster times (in sec) denotes better performance.

Trial sites (10)

FacilityCityRegionStatus
Massachusetts General Hospital Boston Massachusetts Not Yet Recruiting
Cleveland Clinic Neurological Institute Mellen Center Cleveland Ohio Not Yet Recruiting
The University of Texas Health Science Center at Houston Houston Texas Recruiting
Rigshospitalet, University of Copenhagen Kopenhagen Region Sjælland Recruiting
CHU de Bordeaux - Hôpital Pellegrin Service Gynecologie Obstetrique Bordeaux Gironde Not Yet Recruiting
Groupe Hospitalier Pitie-Salpetriere - Charles-Foix Clinical Investigation Center Paris-Est Paris Paris Recruiting
Klinikum der Universität München Friedrich-Baur-Institut München Germany Recruiting
Fondazione IRCCS Istituto Neurologico Carlo Besta Milan Italy Recruiting
Radboud University Medical Center Nijmegen Gelderland Recruiting
University College London Hospitals NHS Foundation Trust National Hospital for Neurology and Neurosurgery London Greater London Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06451757 on ClinicalTrials.gov ↗ ← All trials in France