Phase 3 N=24 Phase III Trial of Coenzyme Q10 in Mitochondrial Disease
Mitochondrial Diseases
Primary: McMaster Gross Motor Function (GMFM 88) — -0.002; -0.12 units on a scale — p=0.95
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Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.
Phase 2 N=53 RTA 408 Capsules in Patients With Mitochondrial Myopathy - MOTOR
MItochondrial Myopathies
Primary: Change of Peak Workload (in Watts/kg) During Exercise Testing — 0.0090; 0.020; -0.0300; 0.1050 Watts/kg — p=0.7321
Phase 2 N=30 Safety, Tolerability, Efficacy of MTP-131 for Treatment of Mitochondrial Disease in Subjects From the MMPOWER Study
Primary Mitochondrial Disease
Primary: Distance Walked on the 6-minute Walk Test (6MWT) by Visit — 394.1; 378.2 meters
Phase 2 N=36 Safety, Tolerability, and Efficacy of MTP-131 for the Treatment of Mitochondrial Myopathy
Mitochondrial Myopathy
Primary: Change in Distance Walked (Meters) on the 6-minute Walk Test (6MWT) — 13.5; 36.5; 64.5; 20.4 meters
N/A N=55 Evaluating Mitochondrial Dysfunction in Patients With Neurofibromatosis Type 1
Neurofibromatosis 1
Primary: Mitochondrial Respiration Efficiency (as Measured by OCR). — 67.7167; 53.7000; 73.3358; 68.5789 picomole/minute — p=<0.01
Phase 1 N=32 A Dose-escalating Clinical Trial With KH176
MELAS · LHON · Leigh Syndrome
Primary: SAD: Change From Baseline in ECG Results by Timepoint: Corrected QT Interval According to Fridericia's Formula (QTcF) — 386.2; 395.0; 385.8; 399.3 msec
Phase 2 N=36 Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease
Inherited Mitochondrial Disease, Including Leigh Syndrome
Primary: Change From Baseline in Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Sections I-IV — -0.3; 0.1; -0.6; 0.0 units on a scale — p=0.1875
Phase 2 N=20 EPI-743 for Metabolism or Mitochondrial Disorders
Mitochondrial Disease · Neurology · Myopathy
Primary: Newcastle Paediatric Mitochondrial Disease Scale (NPMDS) Part I-III, Baseline — 23.9; 20.4 Units on a scale
Phase 3 N=101 A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease
Inherited Mitochondrial Disease
Primary: Number of Participants With Treatment-emergent Adverse Events (TEAEs) — 91 Participants
Phase 2 N=212 An Efficacy and Safety Study of 24 Week Treatment With Mavodelpar (REN001) in Primary Mitochondrial Myopathy Patients
Primary Mitochondrial Myopathy
Primary: Change in Distance Walked During a 12 Minute Walk Test — 26.75; 30.89 meters
Phase 2 N=11 Mitochondria and Chronic Kidney Disease
Hemodialysis-Induced Symptom · Mitochondrial Diseases
Primary: Phosphocreatine (PCR) Recovery Time After Knee Extension Assessed by 31 Phosphorus Magnetic Resonance Spectroscopy (31P-MRS) — 60.59; 62.66 seconds
N/A N=35 Mitochondrial Function in Circulating Cells and Muscle Tissue
Healthy · Obesity · Insulin Sensitivity
Primary: Mitochondrial Function in Circulating Cells and Muscle Tissue in Young Women — 26.48; 30.46; 120.04; 140.43 pmol O2/min per 400K cells
N/A N=14 Impacts of Mitochondrial-targeted Antioxidant on Peripheral Artery Disease Patients
Peripheral Arterial Disease · Peripheral Artery Disease
Primary: Endothelial Function — 6.13; 3.78 % dilation
N/A N=110 Mitochondrial Stress, Brain Imaging, and Epigenetics
Mitochondrial Diseases
Primary: Average TSST-induced Elevation in Cortisol — 37.44; 33.86; 42.83; 45.42 ng*min/mL
Phase 2 N=61 Heart Rate Variability in Response to Metformin Challenge
Fibromyalgia · Mitochondrial Diseases · Movement Disorders
Primary: Heart Rate Variability (Time Domain) — 0.92; 0.83 msec/msec — p=0.77
Phase 3 N=12 Betaine and Peroxisome Biogenesis Disorders
Peroxisome Biogenesis Disorders
Primary: Peroxisome Biochemical Functions as Measured by Plasma Very Long Chain Fatty Acid — 0.180; 0.188 ratio
Phase 2 N=16 Effect of a Wide Spectrum Nutritional Supplement on Mitochondrial Function in Children With Autism Spectrum Disorder
Autism Spectrum Disorder
Primary: Change in Mitochondrial Activity in Study Patients: Citrate Synthase — 40.56; 29.09; 27.41; 37.78 nmoles/min/mg protein
Phase 2 N=40 The Use of a Mitochondrial Enhancement Treatment in Bipolar Disorder
Bipolar Depression
Primary: The 25-Item Hamilton Depression Rating Scale. — 23.3; 22.1; 17.5; 18.5 units on a scale
Phase 2 N=41 A Phase 2 Study to Evaluate the Impact of MTP-131 (Bendavia™) on Skeletal Muscle Function in Elderly
Skeletal Muscle Mitochondrial Dysfunction in the Elderly
Primary: Change From Baseline in ATPmax (Maximal ATP Synthetic Rate) — 0.17; 0.12; 0.09; 0.06 mM/sec
Phase 4 N=22 Responses to Influenza Vaccine in Patients With Mitochondrial Disorders (MELAS)
MELAS Syndrome
Primary: Clinical Safety of TIV Vaccine — 4; 0; 0; 0 Participants
Phase 2 N=39 Effects of Acipimox on Mitochondrial Function in Obesity
Abdominal Obesity · Insulin Resistance · Hypertriglyceridemia
Primary: Change From Baseline in Phosphocreatine Recovery (ViPCr) at 6-months — 1.7; 1.6 mM/s — p=0.97
Phase 2 N=27 Study of Idebenone in the Treatment of Mitochondrial Encephalopathy Lactic Acidosis & Stroke-like Episodes
MELAS Syndrome
Primary: Mean Change in Cerebral Lactate Concentration (as Measured by Magnetic Resonance Spectroscopy) — -0.09; 0.16; -0.49 IU
Phase 1 N=14 Treatment Development for Glucose Transporter Type I Deficiency Syndrome (G1D)
Glucose Transporter Type 1 Deficiency Syndrome · GLUT1 Deficiency Syndrome
Primary: Number of Participants With Reduction in Spike-wave Fraction of the EEG Recording Time — 13 Participants
Phase 2 N=11 Trial of Oral Glutamine on Mitochondrial Function in CKD
Cardiovascular Disease · Sarcopenia · Endothelial Dysfunction
Primary: Muscle Mitochondrial Function — 0.88; 0.90 mM ATP/s — p=0.15
Phase 2 N=15 A Pilot Trial of Triheptanoin for People With Amyotrophic Lateral Sclerosis (PALS)
ALS
Primary: ALS Functional Rating Scale-revised Version (ALSFRS-R) Slope — -0.39; -0.57 points per month — p=0.32
Phase 2 N=13 High Protein Diet in Patients With Long-chain Fatty Acid Oxidation Disorders
Very Long-chain Acyl-CoA Dehydrogenase Deficiency · Trifunctional Protein Deficiency · Carnitine Palmitoyltransferase 2 Deficiency
Primary: Energy Expenditure — 2045; 2343 kcal
Phase 2 N=38 HSCT for High Risk Inherited Inborn Errors
Adrenoleukodystrophy · Metachromatic Leukodystrophy · Globoid Cell Leukodystrophy
Primary: Number of Patients With Donor Cell Engraftment — 26 Participants
N/A N=20 Hepatic Mitochondrial Function in Youth
Hepatic Steatosis · Bariatric Surgery Candidate · Adolescent Obesity
Primary: Change in Percentage of Liver Fat Per MRI — -13.93 percentage of liver fat
Phase 2 N=1 Nicotinamide Riboside on Mitochondrial Function in Li-Fraumeni Syndrome
Cancer · Skin Fibroblasts · Muscle Weakness
Primary: Change in PCr Recovery Tc Measurement From Baseline to 12 Week NR Supplementation Using the 31P-MRS Skeletal Muscle Submaximal Exercise. — 60; 73 Seconds
Phase 4 N=12 Growth and Development Study of Alglucosidase Alfa
Pompe Disease · Glycogen Storage Disease Type II (GSD-II) · Acid Maltase Deficiency Disease
Primary: Recumbent Height/Length of Participants in Centimeters (cm) — 80.4; 93.8; 67.7; 91.1 cm