KHENERFIN Study: A Trial to Evaluate the Efficacy and Safety of Sonlicromanol in Primary Mitochondrial Diseases
Condition(s) studied
Investigational drug(s) / intervention(s)
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
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)
| Facility | City | Region | Status |
|---|---|---|---|
| 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 |
Other trials for the same condition
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