Research Research themes Metabolic disorders Mitochondrial drug developed at Radboudumc is safe in healthy male volunteers

A phase I study showed that KH176 could be safely administered to healthy male volunteers. The results of this study helped to determine the dose that was predicted to be safe in patients with mitochondrial disease, as well as the safety measures that should be taken when KH176 is administered to patients. Due to these first results, a phase II study with KH176 in adult mitochondrial disease patients was initiated, which has recently been completed. This study represents the first clinical step in the development of a drug for patients with mitochondrial disease. Orphanet J Rare Dis 2017