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
Khondrion BV
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.
Description
The KHENERFIN study is investigating the medicine sonlicromanol. The study aims to see if sonlicromanol can reduce symptoms of fatigue and reduce the impact of fatigue on daily life. The study also investigates if sonlicromanol improves physical abilities like balance control and lower limb skeletal muscle strength in people with mitochondrial disease. In addition to these primary objectives, the study evaluates the efficacy of sonlicromanol on selected secondary and exploratory outcomes. It also assesses the safety and tolerability of sonlicromanol. This study is a placebo controlled, double…
Eligibility
- Age range
- 18+ years
- Sex
- All
- 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…
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 HospitalBoston, Massachusetts
- Cleveland Clinic Neurological Institute Mellen CenterCleveland, Ohio
- The University of Texas Health Science Center at HoustonHouston, Texas
- Rigshospitalet, University of CopenhagenKopenhagen, Region Sjælland
- CHU de Bordeaux - Hôpital Pellegrin Service Gynecologie ObstetriqueBordeaux, Gironde
- Groupe Hospitalier Pitie-Salpetriere - Charles-Foix Clinical Investigation Center Paris-EstParis, Paris