KHENERFIN Study: Sonlicromanol for Primary Mitochondrial Diseases
The KHENERFIN study is testing sonlicromanol to see if it can reduce tiredness (fatigue) and improve physical abilities like balance and leg strength in people with primary mitochondrial diseases. This study is for adults aged 18 and older who have a specific genetic change (m.3243A>G mutation) in their mitochondrial DNA. You would receive either sonlicromanol or a placebo (an inactive pill that looks the same) twice a day for 52 weeks. The study aims to see how well sonlicromanol works and if it's safe. The study plans to enroll 220 participants, but its current status is unclear.
- Study design
- This is an interventional study comparing sonlicromanol to a placebo. It is a double-blind study, meaning neither you nor the study team will know if you are receiving sonlicromanol or the placebo. The study plans to enroll 220 participants.
- What's involved
- You would take either sonlicromanol or a placebo twice daily for 52 weeks. You would have visits at screening, baseline (Day 1), and at weeks 13, 26, 39, and 52 for assessments like fatigue questionnaires and a sit-to-stand test.
- Compensation
- Not stated in the trial record.
- Follow-up
- Your progress will be measured through the end of the 52-week treatment period.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
KHENERFIN Study: A Trial to Evaluate the Efficacy and Safety of Sonlicromanol in Primary Mitochondrial Diseases
At a glance
Conditions
Where it's being run
10 sites across 10 statesWho to contact
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What this trial measures
- Neuro-QoL Fatigue Short Form v1Screening, 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.