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), MIDD
(Maternally Inherited Diabetes and Deafness), Leigh's Disease and LHON (Leber's Hereditary
Optic Neuropathy). The current Proof of Concept study aims to explore the effects of
treatment with KH176 for 4 weeks on clinical signs and symptoms and biomarkers of
mitochondrial disease and to evaluate the safety and pharmacokinetics of KH176 in patients
with m.3242A>G related mitochondrial disease.
Phase:
Phase 2
Details
Lead Sponsor:
Khondrion BV
Collaborators:
Radboud Center for Mitochondrial Medicine (RCMM) Radboud University