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Trial record 2 of 2 for:    KH176 | Mitochondrial Diseases

A Dose-escalating Clinical Trial With KH176

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT02544217
First Posted: September 9, 2015
Last Update Posted: February 8, 2016
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Collaborator:
Drug Research Unit Ghent, Belgium
Information provided by (Responsible Party):
Khondrion BV
  Purpose
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

Condition Intervention Phase
MELAS LHON Leigh Syndrome Mitochondrial Disease Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation Drug: KH176 Drug: placebo Phase 1

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: A Phase I, Randomized, Double Blind, Placebo-controlled, Dose-escalating Clinical Trial With KH176

Resource links provided by NLM:


Further study details as provided by Khondrion BV:

Primary Outcome Measures:
  • 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


Secondary Outcome Measures:
  • Pharmacodynamics of KH176 [ Time Frame: 7 days ]
    change from baseline in biochemistry related to OXPHOS (glutathione, lactate)


Enrollment: 30
Study Start Date: May 2015
Study Completion Date: October 2015
Primary Completion Date: September 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Single Escalating
2 alternating groups receiving escalating single doses of active/placebo
Drug: KH176 Drug: placebo
Experimental: Multiple Escalating
3 multiple escalating groups, receiving active/placebo
Drug: KH176 Drug: placebo

  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 55 Years   (Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   Yes
Criteria

Inclusion Criteria:

  • Healthy as assessed by medical history, physical examination, Vital Signs, Clinical Laboratory, ECG

Exclusion Criteria:

  • Allergies,
  • Concomitant medication,
  • concomitant disease,
  • relevant surgery,
  • recent blood donation
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02544217


Locations
Belgium
Drug Research Unit Ghent
Ghent, Belgium
Sponsors and Collaborators
Khondrion BV
Drug Research Unit Ghent, Belgium
  More Information

Responsible Party: Khondrion BV
ClinicalTrials.gov Identifier: NCT02544217     History of Changes
Other Study ID Numbers: KH176-101
First Submitted: April 19, 2015
First Posted: September 9, 2015
Last Update Posted: February 8, 2016
Last Verified: February 2016

Additional relevant MeSH terms:
Mitochondrial Diseases
Leigh Disease
Metabolic Diseases
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Pyruvate Metabolism, Inborn Errors
Carbohydrate Metabolism, Inborn Errors