Date: 2015-05-27
Type of information: Initiation of the trial
phase: 1
Announcement: initiation of the trial
Company: Khondrion (The Netherlands)
Product: KH176 - (S)-6-hydroxy-2,5,7,8-tetramethyl-N-((R)-piperidin-3-yl)chroman-2-carboxamide hydrochloride
Action mechanism:
Disease: mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes (MELAS syndrome), Leber\'s hereditary optic neuropathy (LHON), Leigh syndrome, mitochondrial disease, mitochondrial DNA tRNALeu(UUR) m.3243A
Therapeutic area: Rare diseases - Genetic diseases
Country: Belgium
Trial
details: 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 LHON (Leber\'s Hereditary Optic Neuropathy). 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 (NCT02544217)
Latest
news: * On May 27, 2015, Khondrion, the Dutch biopharmaceutical company focusing on small molecule therapeutics for mitochondrial diseases, announced the initiation of a Phase 1 Clinical Trial with its frontrunner compound KH176. The trial will be a randomized, double blind, placebo-controlled, dose-escalating study.
