{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"submitter":["Janssen MCH"],"funding":["Road4Energy","Foundations Energy4All","Join4Energy","Ride4Kids","ZonMW PMRare","ZonMw","Tim Foundation","Khondrion BV"],"pagination":["101-111"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC6704357"],"repository":["biostudies-literature"],"omics_type":["Unknown"],"volume":["105(1)"],"pubmed_abstract":["KH176 is a potent intracellular reduction-oxidation-modulating compound developed to treat mitochondrial disease. We studied tolerability, safety, pharmacokinetics, pharmacodynamics, and efficacy of twice daily oral 100 mg KH176 for 28 days in a double-blind, randomized, placebo-controlled, two-way crossover phase IIA study in 18 adult m.3243A>G patients without cardiovascular involvement. Efficacy parameters included clinical and functional outcome measures and biomarkers. The trial was registered within ClinicalTrials.gov (NCT02909400), the European Clinical Trials Database (2016-001696-79), and ISRCTN (43372293) (The KHENERGY study). Twice daily oral 100 mg KH176 was well tolerated and appeared safe. No serious treatment-emergent adverse events were reported. No significant improvements"],"journal":["Clinical pharmacology and therapeutics"],"pubmed_title":["The KHENERGY Study: Safety and Efficacy of KH176 in Mitochondrial m.3243A>G Spectrum Disorders."],"pmcid":["PMC6704357"],"funding_grant_id":["113302003"],"pubmed_authors":["Janssen MCH","Beyrath J","Pickkers P","Hemelaar P","Groothuis J","de Laat P","Koene S","Spaans E","Beukema R","Verhaak C","Smeitink J"],"additional_accession":[]},"is_claimable":false,"name":"The KHENERGY Study: Safety and Efficacy of KH176 in Mitochondrial m.3243A>G Spectrum Disorders.","description":"KH176 is a potent intracellular reduction-oxidation-modulating compound developed to treat mitochondrial disease. We studied tolerability, safety, pharmacokinetics, pharmacodynamics, and efficacy of twice daily oral 100 mg KH176 for 28 days in a double-blind, randomized, placebo-controlled, two-way crossover phase IIA study in 18 adult m.3243A>G patients without cardiovascular involvement. Efficacy parameters included clinical and functional outcome measures and biomarkers. The trial was registered within ClinicalTrials.gov (NCT02909400), the European Clinical Trials Database (2016-001696-79), and ISRCTN (43372293) (The KHENERGY study). Twice daily oral 100 mg KH176 was well tolerated and appeared safe. No serious treatment-emergent adverse events were reported. No significant improvements","dates":{"release":"2019-01-01T00:00:00Z","publication":"2019 Jan","modification":"2026-05-01T04:45:40.794Z","creation":"2019-08-31T07:06:55Z"},"accession":"S-EPMC6704357","cross_references":{"pubmed":["30058726"],"doi":["10.1002/cpt.1197"]}}