KHENERFIN Phase III sonlicromanol trial in adults with mitochondrial DNA m.3243A>G primary mitochondrial disease

Phase 3, randomised, double-blind trial in Mitochondriopathy and Mitochondrial disease due to m.3243A>G mitochondrial DNA variant (m.3243A>G) comparing Sonlicromanol with Matching placebo (2 arms). Primary endpoints: Change from baseline at Week 52 in Neuro-QoL Fatigue SF v1 score and Change from baseline at Week 52 in 5 Times Sit-to-Stand Test duration. Planned start Mar 2026; status: authorised.