Conditions: MELAS LHON Leigh Syndrome Mitochondrial Disease Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation
Allocation: RANDOMIZED | Intervention model: CROSSOVER
Enrollment: 32
Healthy volunteers: yes

Brief summary:
Mitochondrial Diseases are rare progressive, multi-system, often early fatal disorders affecting both children and adults. KH176 is a novel chemical entity currently under development for the treatment of inherited mitochondrial diseases, including MELAS (Mitochondrial Encephalomyopathy, Lactic acidosis, and Stroke-like episodes), Leigh's Disease and Leber's Hereditary Optic Neuropathy (LHON). KH176 is a potent intracellular redox modulating agent targeting the reactive oxygen species which are important in the pathogenesis of disorders of mitochondrial oxidative phosphorylation. After demonstrating a favourable safety profile in the pre-clinical testing, the safety, tolerability and pharmacokinetic and pharmacodynamic characteristics of the compound will now be evaluated in healthy male subjects in this trial

Detailed description:

Arms:
arm 1: 2 alternating groups receiving escalating single doses of active/placebo arm 2: 3 multiple escalating groups, receiving active/placebo

Interventions: intervention 1: KH176 intervention 2: placebo
Intervention descriptions: intervention 1: None intervention 2: None

Protocol document: