Clinical Trial: A Dose-escalating Clinical Trial With KH176

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: A Phase I, Randomized, Double Blind, Placebo-controlled, Dose-escalating Clinical Trial With KH176

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 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

Detailed Summary:
Sponsor: Khondrion BV

Current Primary Outcome:

  • Safety and tolerability (assessment of adverse events, routine clinical laboratory, vital signs, ECG, physical exam) [ Time Frame: 4 months ]
    assessment of adverse events, routine clinical laboratory, vital signs, ECG, physical exam
  • Pharmacokinetics of KH176 [ Time Frame: 7 days ]
    assessment of time to reach peak plasma concentration
  • Pharmacokinetics of KH176 [ Time Frame: 7 days ]
    assessment of peak plasma concentration
  • Pharmacokinetics of KH176 [ Time Frame: 7 days ]
    assessment of the area under the plasma concentration versus time curve (AUC)
  • Pharmacokinetics of KH176 [ Time Frame: 7 days ]
    assessment of half life


Original Primary Outcome: Same as current

Current Secondary Outcome: Pharmacodynamics of KH176 [ Time Frame: 7 days ]

change from baseline in biochemistry related to OXPHOS (glutathione, lactate)


Original Secondary Outcome: Same as current

Information By: Khondrion BV

Dates:
Date Received: April 19, 2015
Date Started: May 2015
Date Completion:
Last Updated: February 5, 2016
Last Verified: February 2016