A Dose-escalating Clinical Trial With KH176

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

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 Leber's Hereditary Optic Neuropathy (LHON). 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

Study Overview

• Status: Completed
• Conditions: Mitochondrial Disease; MELAS; Leigh Syndrome; LHON; Mitochondrial DNA tRNALeu(UUR) m.3243A
• Intervention / Treatment: Drug: placebo; Drug: KH176

• Study Type: Interventional
• Enrollment (Actual): 32
• Phase: Phase 1

Contacts and Locations

Study Locations

• Belgium
  • Ghent, Belgium
    • Drug Research Unit Ghent

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 55 years (Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Male

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Crossover Assignment
• Masking: Double

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Single Escalating; 2 alternating groups receiving escalating single doses of active/placebo	Drug: placebo; Drug: KH176
Experimental: Multiple Escalating; 3 multiple escalating groups, receiving active/placebo	Drug: placebo; Drug: KH176

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
SAD: Change From Baseline in ECG Results by Timepoint: Corrected QT Interval According to Fridericia's Formula (QTcF)	The baseline ECG recording consists of a triplicate recording (3 recordings of a least 3 complexes within a 5 minute window) prior to dosing on Day 1. For the evaluation of the corrected QT intervals the average of the 3 recordings will be taken as baseline. Other ECG recordings will be singlet (one recording of at least 3 complexes) prior to dosing. QTcF is calculated as the quotient between the QT interval in milliseconds and the cube root of the RR interval in seconds, according to Fridericia's formula.	Baseline, 1, 2, 4, 6, 8, 12, 24 hours, 7 day follow up
Pharmacodynamics of KH176	Change from baseline in biochemistry related to Oxidative Phosphorylation (OXPHOS) (glutathione, lactate); MAD group	Day 1, day 7
Relationship to Study Drug and Severity of Treatment-emergent Adverse Events		4 months
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Red Blood Cell Count. SAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), 24h post dose, FU (7 days after last dosing)
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Sedimentation Rate (SAD Group)		Baseline (pre-dose Day1), 24h post dose, FU (7 days after last dosing)
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Sedimentation Rate MAD Group		Baseline (pre-dose Day 1), Day 3, Day 8, FU (7 days after last dosing)
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Hematocrit SAD Group	Hematocrit is a measure of the ratio of red blood cells (RBCs) in the blood by volume. Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), 24 h postdose, FU (7 days after last dosing)
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Mean Corpuscular Haemoglobin. SAD	Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), 24 h post dose, FU (7 days after last dosing)
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Mean Corpuscular Hemoglobin Concentration - SAD	Erythrocyte mean corpuscular hemoglobin concentration (MCHC) is a measure of the average concentration of hemoglobin per red blood cell. Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), 24 h post dose, FU (7 days after last dosing)
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Mean Corpuscular Volume - SAD	Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), 24 h post dose, FU (7 days after last dosing)
MAD: Change From Baseline in ECG Results by Time Point: QTcF	The baseline ECG recording consists of a triplicate recording (3 recordings of a least 3 complexes within a 5 minute window) prior to dosing on Day 1. For the evaluation of the corrected QT intervals the average of the 3 recordings will be taken as baseline. Other ECG recordings will be singlet (one recording of at least 3 complexes) prior to dosing, or around the expected Cmax on Day 1 or 7 for the multiple-dose part. QTcF is calculated as the quotient between the QT interval in milliseconds and the cube root of the RR interval in seconds, according to Fridericia's formula.	Baseline, Day1, Day 2, Day 3, Day 4, Day 5, Day 6, Day 7
Change From Baseline in Hematology Laboratory Test Results by Timepoint: White Blood Cell Count. SAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), 24h post dose, FU (7 days after last dosing)
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Lymphocytes. SAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Neutrophils. SAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), 24h post dose, FU (7 days after last dosing)
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Hematocrit MAD Group	Hematocrit is a measure of the ratio of red blood cells (RBCs) in the blood by volume. Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value	Baseline (pre-dose Day 1), Day 3, Day 8, FU (7 days post dose)
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Eosinophils. SAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), 24h post dose, FU (7 days after last dosing)
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Thrombocytes. SAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), 24h, FU (7 days post-dose)
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Basophils. SAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value ateach timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), 24h post dose, FU (7 days post-dose)
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Monocytes. SAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), 24h post dose, FU (7 days post-dose)
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Mean Corpuscular Volume. MAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), Day 3, Day 8, FU (7 days post dose)
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Hemoglobin. MAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), Day 3, Day 8, FU (7 days post dose)
Change From Baseline in Hematology Laboratory Test Results by Timepoint: White Blood Cell Count. MAD	Blood samples were collected from participants at the indicated time points for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each time point are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), Day 3, Day 8, FU (7 days post dose)
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Neutrophils. MAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), Day 3, Day 8, FU (7 days post dose)
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Mean Corpuscular Haemoglobin Concentration. MAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), Day 3, Day 8, FU (7 days post dose)
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Lymphocytes. MAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), Day 3, Day 8, FU (7 days post dose)
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Mean Corpuscular Hemoglobin. MAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), Day 3, Day 8, FU (7 days post dose)
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Red Blood Cell Count. MAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), Day 3, Day 8, FU (7 days post dose)
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Eosinophils. MAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), Day 3, Day 8, FU (7 days post dose)
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Monocytes. MAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), Day 3, Day 8, FU (7 days post dose)
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Thrombocytes. MAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), Day 3, Day 8, FU (7 days post dose)
Change From Baseline in Hematology Laboratory Test Results by Timepoint: Basophils. MAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), Day 3, Day 8, FU (7 days post dose)
Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Aspartate Aminotransferase. SAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)
Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Total Protein. SAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)
Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Gamma-GT. SAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value	Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)
Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Alkaline Phosphatase. SAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)
Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Creatinine. SAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)
Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Chloride. SAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)
Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Total Bilirubin. SAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)
Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Alanine Aminotransferase. SAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)
Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Creatinine Kinase. SAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)
Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Urea. SAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)
Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Sodium. SAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)
Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Potassium. SAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)
Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Phosphate. SAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)
Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Calcium (Corrected for Albumin). SAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)
Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Uric Acid. SAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)
Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Triiodothyronine (T3). SAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)
Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Lipase. SAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)
Change From Baseline in Chemistry Laboratory Test Results by Timepoint: (Fasting) Glucose. SAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)
Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Human Serum Albumin. SAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)
Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Cholesterol. SAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)
Change From Baseline in Chemistry Laboratory Test Results by Timepoint: High Density Lipoproteins. SAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)
Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Triglycerides. SAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)
Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Low Density Lipoproteins. SAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)
Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Bicarbonate. SAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)
Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Thyroid-stimulating Hormone. SAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)
Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Thyroxine (T4). SAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)
Change From Baseline in Chemistry Laboratory Test Results by Timepoint: Lactate. SAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of clinical chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day 1), 24h post dose, FU (7 days post dose)
Change From Baseline in Chemistry Laboratory Test Results by Time Point: Total Protein. MAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of hematology parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline (pre-dose Day1), Day 3, Day 8, FU (one week after last dosing)
Change From Baseline in Chemistry Laboratory Test Results by Time Point: Alkaline Phosphatase. MAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline, Day 3, Day 8, FU (one week after last dosing)
Change From Baseline in Chemistry Laboratory Test Results by Time Point: Aspartate Aminotransferase. MAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline, Day 3, Day 8, FU (one week after last dosing)
Change From Baseline in Chemistry Laboratory Test Results by Time Point: Creatine Kinase. MAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline, Day 3, Day 8, FU (one week after last dosing)
Change From Baseline in Chemistry Laboratory Test Results by Time Point: Gamma GT. MAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline, Day 3, Day 8, FU (one week after last dosing)
Change From Baseline in Chemistry Laboratory Test Results by Time Point: Total Bilirubin. MAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline, Day 3, Day 8, FU (one week after last dosing)
Change From Baseline in Chemistry Laboratory Test Results by Time Point: Urea. MAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline, Day 3, Day 8, FU (one week after last dosing)
Change From Baseline in Chemistry Laboratory Test Results by Time Point: Creatinine. MAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline, Day 3, Day 8, FU (one week after last dosing)
Change From Baseline in Chemistry Laboratory Test Results by Time Point: Sodium. MAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline, Day 3, Day 8, FU (one week after last dosing)
Change From Baseline in Chemistry Laboratory Test Results by Time Point: Potassium. MAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline, Day 3, Day 8, FU (one week after last dosing)
Change From Baseline in Chemistry Laboratory Test Results by Time Point: Chloride. MAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline, Day 3, Day 8, FU (one week after last dosing)
Change From Baseline in Chemistry Laboratory Test Results by Time Point: Calcium (Corrected for Albumin). MAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline, Day 3, Day 8, FU (one week after last dosing)
Change From Baseline in Chemistry Laboratory Test Results by Time Point: Uric Acid. MAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline, Day 3, Day 8, FU (one week after last dosing)
Change From Baseline in Chemistry Laboratory Test Results by Time Point: Lipase. MAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline, Day 3, Day 8, FU (one week after last dosing)
Change From Baseline in Chemistry Laboratory Test Results by Time Point: Alanine Aminotransferase. MAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline, Day 3, Day 8, FU (one week after last dosing)
Change From Baseline in Chemistry Laboratory Test Results by Time Point: Phosphate. MAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline, Day 3, Day 8, FU (one week after last dosing)
Change From Baseline in Chemistry Laboratory Test Results by Time Point: Human Serum Albumin. MAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline, Day 3, Day 8, FU (one week after last dosing)
Change From Baseline in Chemistry Laboratory Test Results by Time Point: Fasting Glucose. MAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline, Day 3, Day 8, FU (one week after last dosing)
Change From Baseline in Chemistry Laboratory Test Results by Time Point: Cholesterol. MAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline, Day 3, Day 8, FU (one week after last dosing)
Change From Baseline in Chemistry Laboratory Test Results by Time Point: Triglycerides. MAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline, Day 3, Day 8, FU (one week after last dosing)
Change From Baseline in Chemistry Laboratory Test Results by Time Point: Low Density Lipoproteins. MAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline, Day 3, Day 8, FU (one week after last dosing)
Change From Baseline in Chemistry Laboratory Test Results by Time Point: High Density Lipoproteins. MAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline, Day 3, Day 8, FU (one week after last dosing)
Change From Baseline in Chemistry Laboratory Test Results by Time Point: Thyroid-stimulating Hormone. MAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline, Day 3, Day 8, FU (one week after last dosing)
Change From Baseline in Chemistry Laboratory Test Results by Time Point: Trilodothyronine (T3). MAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline, Day 3, Day 8, FU (one week after last dosing)
Change From Baseline in Chemistry Laboratory Test Results by Time Point: Thyroxine (T4). MAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline, Day 3, Day 8, FU (one week after last dosing)
Change From Baseline in Chemistry Laboratory Test Results by Time Point: Bicarbonate. MAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline, Day 3, Day 8, FU (one week after last dosing)
Change From Baseline in Chemistry Laboratory Test Results by Time Point: Lactate. MAD Group	Blood samples were collected from participants at the indicated timepoints for evaluation of chemistry parameters. The baseline value at visit and the change from baseline value at each timepoint are reported. The change from baseline value was calculated by subtracting the post-baseline value from the baseline value.	Baseline, Day 3, Day 8, FU (one week after last dosing)
Phospholipidosis	Change from Day 1 to Day 7 in di-docosahexaenoyl (22:6)-bis(monoacylglycerol) phosphate (di-22:6-BMP) and normalized di-22:6-BMP (urine) - MAD	Day 1, Day 7
MAD: Change From Baseline in ECG Results by Time Point: Corrected QT Interval According to Barret's Formaula (QTcB)	The baseline ECG recording consists of a triplicate recording (3 recordings of a least 3 complexes within a 5 minute window) prior to dosing on Day 1. For the evaluation the average of the 3 recordings will be taken as baseline. Other ECG recordings will be singlet (one recording of at least 3 complexes) prior to dosing, or around the expected Cmax on Day 1 or 7 for the multiple- dose part.	Baseline (pre-dose Day 1), Day1, Day 2, Day 3, Day 4, Day 5, Day 6, Day 7, Follow-up
MAD: Change From Baseline in ECG Results by Time Point: P Wave-Q Wave Interval (PQ Interval)	The baseline ECG recording consists of a triplicate recording (3 recordings of a least 3 complexes within a 5 minute window) prior to dosing on Day 1. For the evaluation of the PQ interval the average of the 3 recordings will be taken as baseline.	Baseline (pre-dose Day 1), Day1, Day 2, Day 3, Day 4, Day 5, Day 6, Day 7, Follow-up
MAD: Change From Baseline in ECG Results by Time Point: the Interval That Denotes Depolarization of the Ventricles, Between the Beginning of the Q Wave and the End of the S Wave (QRS Interval)	The baseline ECG recording consists of a triplicate recording (3 recordings of a least 3 complexes within a 5 minute window) prior to dosing on Day 1. For the evaluation the average of the 3 recordings will be taken as baseline. Other ECG recordings will be singlet (one recording of at least 3 complexes) prior to dosing, or around the expected Cmax on Day 1 or 7 for the multiple- dose part.	Baseline (pre-dose Day 1), Day1, Day 2, Day 3, Day 4, Day 5, Day 6, Day 7, Follow-up
MAD: Change From Baseline in ECG Results by Time Point: QT Interval	The baseline ECG recording consists of a triplicate recording (3 recordings of a least 3 complexes within a 5 minute window) prior to dosing on Day 1. For the evaluation the average of the 3 recordings will be taken as baseline. Other ECG recordings will be singlet (one recording of at least 3 complexes) prior to dosing, or around the expected Cmax on Day 1 or 7 for the multiple- dose part.	Baseline (pre-dose Day 1), Day1, Day 2, Day 3, Day 4, Day 5, Day 6, Day 7, Follow-up
SAD: Change From Baseline in ECG Results by Time Point: PQ Interval	The baseline ECG recording consists of a triplicate recording (3 recordings of a least 3 complexes within a 5 minute window) prior to dosing on Day 1. For the evaluation the average of the 3 recordings will be taken as baseline.	Pre-dose, Day 1, Day 7
SAD: Change From Baseline in ECG Results by Time Point: QRS Interval	The baseline ECG recording consists of a triplicate recording (3 recordings of a least 3 complexes within a 5 minute window) prior to dosing on Day 1. For the evaluation the average of the 3 recordings will be taken as baseline.	Pre-dose, Day 1, Day 7
SAD: Change From Baseline in ECG Results by Time Point: QT Interval	The baseline ECG recording consists of a triplicate recording (3 recordings of a least 3 complexes within a 5 minute window) prior to dosing on Day 1. For the evaluation the average of the 3 recordings will be taken as baseline.	Pre-dose, Day 1, Day 7
SAD: Change From Baseline in ECG Results by Time Point: QTcB Interval	The baseline ECG recording consists of a triplicate recording (3 recordings of a least 3 complexes within a 5 minute window) prior to dosing on Day 1. For the evaluation the average of the 3 recordings will be taken as baseline.	Pre-dose, Day1, Day 7
Terminal Elimination Half-life (T1/2) of KH176 Over 24 Hours: SAD	Plasma concentrations of KH176 were analysed after single dose administration of doses 10mg, 30mg, 100mg (with and without food), 300mg, 800mg, 2000mg b.i.d.	Pre-dose (5 min before dosing) and 0.5, 1, 1.5, 2, 3, 6, 8, 12, and 24 hours post-dose
Terminal Elimination Half-life (T1/2) of KH183: SAD	Plasma concentrations of KH183 were analysed after single dose administration of doses 10mg, 30mg, 100mg (with and without food) , 300mg, 800mg, 2000mg b.i.d.	Pre-dose (5 min before dosing) and 0.5, 1, 1.5, 2, 3, 6, 8, 12, and 24 hours post-dose
Maximum Concentration (Cmax) of KH176: SAD Group	Plasma concentrations of KH176 were analysed after single dose administration of doses 10mg, 30mg, 100mg (with and without food), 300mg, 800mg, 2000mg b.i.d.	Pre-dose (5 min before dosing) and 0.5, 1, 1.5, 2, 3, 6, 8, 12, and 24 hours post-dose
Maximum Concentration (Cmax) of KH183 Over 24 Hours: SAD	Plasma concentrations of KH183 were analysed after single dose administration of doses 10mg, 30mg, 100mg (with and without food) , 300mg, 800mg, 2000mg b.i.d.	Pre-dose (5 min before dosing) and 0.5, 1, 1.5, 2, 3, 6, 8, 12, and 24 hours post-dose
Maximum Concentration (Cmax) of KH183 (Active Metabolite of KH176): MAD Group	Plasma concentrations of KH183 were analysed after multiple-dose administration of doses of 100, 200 and 400 mg b.i.d.: Cmax was obtained directly from the concentration-time data.	Predose at Day 1, 2, 4, 7, and post-dose at Day 1 and Day 7 at 0.5, 1, 1.5, 2, 3, 4, 6, 8, 12 hours
Maximum Concentration (Cmax) of KH176: MAD	Plasma concentrations of KH176 were analysed after multiple-dose administration of doses of 100, 200 and 400 mg b.i.d.	pre-dose at Day 1, 2, 4, 7, and post-dose at Day 1 and Day 7 at 0.5, 1, 1.5, 2, 3, 4, 6, 8, 12 hours
Time to Maximum Concentration (Tmax) of KH176 Over 24 Hours: SAD	Plasma concentrations of KH176 were analysed after single dose administration of doses 10mg, 30mg, 100mg (with and without food), 300mg, 800mg, 2000mg b.i.d.	Pre-dose (5 min before dosing) and 0.5, 1, 1.5, 2, 3, 6, 8, 12, and 24 hours post-dose
Time to Reach Peak Plasma Concentration (Tmax) of KH183: SAD	Plasma concentrations of KH183 were analysed after single dose administration of doses 10mg, 30mg, 100mg (with and without food), 300mg, 800mg, 2000mg b.i.d.	Pre-dose (5 min before dosing) and 0.5, 1, 1.5, 2, 3, 6, 8, 12, and 24 hours post-dose
Time to Maximum Concentration (Tmax) of KH176 at Day 1, Day 7: MAD Group	Plasma concentrations of KH176 were analysed after multiple-dose administration of doses of 100, 200 and 400 mg b.i.d.	pre-dose at Day 1, 2, 4, 7, and post-dose at Day 1 and Day 7 at 0.5, 1, 1.5, 2, 3, 4, 6, 8, 12 hours
Time to Maximum Concentration (Tmax) of KH183 (Active Metabolite of KH176): MAD Group	Plasma concentrations of KH183 were analysed after multiple-dose administration of doses of 100, 200 and 400 mg b.i.d.. tmax was obtained directly from the concentration-time data.	Predose at Day 1, 2, 4, 7, and post-dose at Day 1 and Day 7 at 0.5, 1, 1.5, 2, 3, 4, 6, 8, 12 hours
Accumulation Factor (Racc) of KH176 Over 7 Days: MAD Group	Plasma concentrations of KH176 were analysed after multiple-dose administration of doses of 100, 200 and 400 mg b.i.d.. Racc was calculated as follows: AUCtau day 7/ AUCtau day 1.	Pre-dose at Day 1, 2, 4, 7, and post-dose at Day 1 and Day 7 at 0.5, 1, 1.5, 2, 3, 4, 6, 8, 12 hours
Accumulation Factor (Racc) of KH183 (Active Metabolite of KH176): MAD Group	Plasma concentrations of KH183 were analysed after multiple-dose administration of doses of 100, 200 and 400 mg b.i.d.: Accumulation factor was calculated as follows: AUCtau day 7/ AUCtau day 1.	Predose at Day 1, 2, 4, 7, and post-dose at Day 1 and Day 7 at 0.5, 1, 1.5, 2, 3, 4, 6, 8, 12 hours
Area Under the Plasma Concentration Versus Time Curve (AUClast) of KH183: SAD Group	Plasma concentrations of KH183 were analysed after single dose administration of doses 10mg, 30mg, 100mg (with and without food) , 300mg, 800mg, 2000mg b.i.d.	Pre-dose (5 min before dosing) and 0.5, 1, 1.5, 2, 3, 6, 8, 12, and 24 hours post-dose
Area Under the Plasma Concentration Versus Time Curve (AUClast) of KH176: SAD Group	Plasma concentrations of KH176 were analysed after single dose administration of doses 10mg, 30mg, 100mg (with and without food), 300mg, 800mg, 2000mg b.i.d.	pre-dose (5 min before dosing) and 0.5, 1, 1.5, 2, 3, 6, 8, 12, and 24 hours post-dose
Area Under the Plasma Concentration-time Curve (AUCtau) of KH176: MAD Group:	Plasma concentrations of KH176 were analysed after multiple-dose administration of doses of 100, 200 and 400 mg b.i.d.	Pre-dose at Day 1, 2, 4, 7, and post-dose at Day 1 and Day 7 at 0.5, 1, 1.5, 2, 3, 4, 6, 8, 12 hours
Area Under the Plasma Concentration-time Curve During a Dose Interval (AUCtau) of KH183 (Active Metabolite of KH176): MAD Group	Plasma concentrations of KH183 were analysed after multiple-dose administration of doses of 100, 200 and 400 mg b.i.d.	Predose at Day 1, 2, 4, 7, and post-dose at Day 1 and Day 7 at 0.5, 1, 1.5, 2, 3, 4, 6, 8, 12 hours
Area Under the Plasma Concentration-time Curve From Time Zero Until Infinity (AUCl0-inf) of KH183: SAD	Plasma concentrations of KH183 were analysed after single dose administration of doses 10mg, 30mg, 100mg (with and without food) , 300mg, 800mg, 2000mg b.i.d.	Pre-dose (5 min before dosing) and 0.5, 1, 1.5, 2, 3, 6, 8, 12, and 24 hours post-dose
Area Under the Plasma Concentration-time Curve From Time Zero Until Infinity (AUCl0-inf) of KH176: SAD	Plasma concentrations of KH176 were analysed after single dose administration of doses 10mg, 30mg, 100mg (with and without food) , 300mg, 800mg, 2000mg b.i.d.	Pre-dose (5 min before dosing) and 0.5, 1, 1.5, 2, 3, 6, 8, 12, and 24 hours post-dose
KH176: Percentage of Administered Dose Excreted in Urine: SAD	Urine concentrations of KH176 were analysed after single dose administration of doses 10mg, 30mg, 100mg (with and without food), 300mg, 800mg, 2000mg b.i.d.	24 hours post-dose
KH183: Percentage of Administered Dose Excreted in Urine: SAD	Urine concentrations of KH176 were analysed after single dose administration of doses 10mg, 30mg, 100mg (with and without food), 300mg, 800mg, 2000mg b.i.d.	24 hours post-dose
KH176 + KH183: Percentage of Administered Dose Excreted in Urine: SAD	Urine concentrations of KH176 were analysed after single dose administration of doses 10mg, 30mg, 100mg (with and without food), 300mg, 800mg, 2000mg b.i.d.	24 hours post-dose
KH176: Percentage of Administered Dose Excreted in Urine: MAD	Urine concentrations of KH176 were analysed after multiple-dose administration of doses of 100, 200 and 400 mg b.i.d.	Day 7 post dose
KH183: Percentage of Administered Dose Excreted in Urine: MAD	Urine concentrations of KH183 were analysed after multiple-dose administration of doses of 100, 200 and 400 mg b.i.d.	Post dose Day 7
KH173 + KH183: Percentage of Administered Dose Excreted in Urine: MAD	Urine concentrations of KH176 and KH183 were analysed after multiple-dose administration of doses of 100, 200 and 400 mg b.i.d.	Day 7 Post dose

Collaborators and Investigators

• Sponsor: Khondrion BV
• Collaborators: Drug Research Unit Ghent, Belgium

Publications and helpful links

General Publications

• Koene S, Spaans E, Van Bortel L, Van Lancker G, Delafontaine B, Badilini F, Beyrath J, Smeitink J. KH176 under development for rare mitochondrial disease: a first in man randomized controlled clinical trial in healthy male volunteers. Orphanet J Rare Dis. 2017 Oct 16;12(1):163. doi: 10.1186/s13023-017-0715-0.

Study record dates

Study Major Dates

• Study Start: May 1, 2015
• Primary Completion (Actual): September 1, 2015
• Study Completion (Actual): October 1, 2015

Study Registration Dates

• First Submitted: April 19, 2015
• First Submitted That Met QC Criteria: September 4, 2015
• First Posted (Estimate): September 9, 2015

Study Record Updates

• Last Update Posted (Actual): October 18, 2021
• Last Update Submitted That Met QC Criteria: October 15, 2021
• Last Verified: February 1, 2016

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Brain Diseases, Metabolic; Brain Diseases, Metabolic, Inborn; Pyruvate Metabolism, Inborn Errors; Mitochondrial Diseases; Leigh Disease; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Protective Agents; Antioxidants; 6-hydroxy-2,5,7,8-tetramethylchroman-2-carboxylic acid
• Other Study ID Numbers: KH176-101