The KHENERGY Study: Safety and Efficacy of KH176 in Mitochondrial m.3243A>G Spectrum Disorders.
Journal
Clinical pharmacology and therapeutics
ISSN: 1532-6535
Titre abrégé: Clin Pharmacol Ther
Pays: United States
ID NLM: 0372741
Informations de publication
Date de publication:
01 2019
01 2019
Historique:
pubmed:
31
7
2018
medline:
16
10
2019
entrez:
31
7
2018
Statut:
ppublish
Résumé
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 in gait parameters or other outcome measures were obtained, except for a positive effect on alertness and mood, although a coincidence due to multiplicity cannot be ignored. The results of the study provide first data on safety and efficacy of KH176 in patients with mitochondrial disease and will be instrumental in designing future clinical trials.
Identifiants
pubmed: 30058726
doi: 10.1002/cpt.1197
pmc: PMC6704357
doi:
Substances chimiques
Antioxidants
0
Chromans
0
DNA, Mitochondrial
0
6-hydroxy-2,5,7,8-tetramethylchroman-2-carboxylic acid
S18UL9710X
Banques de données
ClinicalTrials.gov
['NCT02909400']
EudraCT
['2016-001696-79']
ISRCTN
['ISRCTN43372293']
Types de publication
Clinical Trial, Phase II
Journal Article
Randomized Controlled Trial
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
101-111Informations de copyright
© 2018 American Society for Clinical Pharmacology and Therapeutics.
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