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Trial record 2 of 2 for:    KH176 | Mitochondrial Diseases

A Dose-escalating Clinical Trial With KH176

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ClinicalTrials.gov Identifier: NCT02544217
Recruitment Status : Completed
First Posted : September 9, 2015
Last Update Posted : February 8, 2016
Sponsor:
Collaborator:
Drug Research Unit Ghent, Belgium
Information provided by (Responsible Party):
Khondrion BV

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

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

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 30 participants
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
Study Start Date : May 2015
Actual Primary Completion Date : September 2015
Actual Study Completion Date : October 2015


Arm Intervention/treatment
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



Primary Outcome Measures :
  1. 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

  2. Pharmacokinetics of KH176 [ Time Frame: 7 days ]
    assessment of time to reach peak plasma concentration

  3. Pharmacokinetics of KH176 [ Time Frame: 7 days ]
    assessment of peak plasma concentration

  4. Pharmacokinetics of KH176 [ Time Frame: 7 days ]
    assessment of the area under the plasma concentration versus time curve (AUC)

  5. Pharmacokinetics of KH176 [ Time Frame: 7 days ]
    assessment of half life


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



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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

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

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Khondrion BV
ClinicalTrials.gov Identifier: NCT02544217     History of Changes
Other Study ID Numbers: KH176-101
First Posted: September 9, 2015    Key Record Dates
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