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.

Condition studied

Primary Mitochondrial Disease, Mitochondrial DNA 3243A>G variant and Associated Phenotypes (MIDD, MELAS, Multi System Phenotypes

Investigational medicinal product(s)

Eligibility

Primary endpoint

Change from baseline at Week 52 in the: 1. NeuroQoL Fatigue SF v1 score, Change from baseline at Week 52 in the: 2. Duration of the 5 Times Sit-to-Stand Test (5xSST)

Endpoint detail

Change from baseline at Week 52 in the: 1. 36-Item Short Form Survey (SF-36): sub-score physical functioning, Change from baseline at Week 52 in the: 2. Fatigue Severity Scale (FSS), Change from baseline at Week 52 in the: 3. Beck Depression Inventory (BDI-2) total score, Change from baseline at Week 52 in the: 4. Patient reported global impression of severity (PGI-S), Change from baseline at Week 52 in the: 5. Clinician reported global impression of severity (CGI-S)