KHENERFIN Study: A trial evaluating the efficacy and safety of sonlicromanol in primary mitochondrial diseases (KH176-301)

The main criteria for participation

This international Phase 3 trial is recruiting, notably in France, at least 220 adult patients with a primary mitochondrial disease caused by the m.3243A>G mutation in the MT-TL1 gene, presenting a chronic fatigue for at least 3 months confirmed by the Neuro-QoL questionnaire, and a myopathy evaluated via the Five-Times-Sit-to-Stand (FTSTS) test.

The objective

The trial aims to evaluate whether sonlicromanol, compared to a placebo, reduces symptoms of fatigue and their impact on daily life, and whether it improves physical capabilities (balance control and lower limb muscle strength). It will also assess the safety and tolerability of the treatment over the long term.

In practice

The trial lasts approximately 60 weeks.

Following an evaluation period of approximately 4 weeks, each eligible patient is placed by lottery into the group receiving sonlicromanol or the group receiving the placebo. In both cases, the medication is a tablet to be taken twice daily. Neither the patient nor the medical staff knows who is receiving which treatment.

Treatment or placebo administration lasts for one year (52 weeks). During this period, and up to two weeks after the final dose, various assessments for fatigue, mobility, etc., along with blood tests, will be conducted regularly.