ClinicaliQ Trial Snapshot
- KHENERFIN Study: A Trial to Evaluate the Efficacy and Safety of Sonlicromanol in Primary Mitochondrial Diseases — Recruiting • Phase III • Cardiology / Cardiovascular • NCT06451757.
- Treatment being tested: Sonlicromanol, a novel investigational medicine designed to reduce fatigue symptoms and improve physical function (including balance control) in patients with primary mitochondrial diseases.
- Patient eligibility overview: Adults with genetically confirmed primary mitochondrial disease who experience clinically significant fatigue; specific inclusion/exclusion criteria apply and should be reviewed with the trial site.
Use This Page For
- Quick orientation before opening the registry record.
- Checking recruitment status, phase and sponsor at a glance.
- Connecting this trial to nearby guidelines, Drug Science and education.
What This Trial Is Studying
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.…
Eligibility Snapshot
- 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):
- 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.
Use the source registry for the full inclusion and exclusion criteria before discussing referral or enrolment.