| ClinicalTrials.gov ID Number | Intervention/ Study Focus | Mitochondrial Disorder | Principal Investigator | Status |
|---|---|---|---|---|
| NCT02616484 | Evaluation of treatment with the investigational drug dichloroacetate (DCA) in children 6 months to 17 years with deficiency of the pyruvate dehydrogenase complex (PDC) | Pyruvate Dehydrogenase Complex Deficiency (PDCDP | Dr. Peter Stacpoole, pws@ufl.edu | Recruiting |
| NCT03048617 | An Observational Study of Patients With Primary Mitochondrial Disease (SPIMM-300) (RePOWER) NOTE: The site list will grow as Stealth activates each location on a rolling basis. Stay tuned for more information in late March. | People ages 16-65 with primary mitochondrial disease | Stealth BioTherapeutics | Recruiting |
| NCT02652767 | Efficacy Study of GS010 for the Treatment of Vision Loss up to 6 Months From Onset in LHON Due to the ND4 Mutation (RESCUE) | LHON | GenSight Biologics | Not recruiting |
| NCT02473445 | A Long-Term Extension Study of RP103-MITO-001 to Assess RP103 in Children With Inherited Mitochondrial Disease
| Children between 2-17 years of age with genetically diagnosed mitochondrial disease | Raptor Pharmaceuticals | Ongoing but not recruiting |
| NCT02435940 | Inherited Retinal Degenerative Disease Registry
| Kearns-Sayre Syndrome and other inherited retinal degenerative diseases
| Foundation Fighting Blindness Clinical Research Institute | Recruiting |
| NCT02427178 | MNGIE Allogeneic Hematopoietic Stem Cell Transplant Safety Study (MASS)
| Patients 5 – 55 years of age who have been identified as having MNGIE by genetic testing and/or reduced thymidine phosphorylase levels | Columbia University | Recruiting |
| NCT02398201 | A Study of Bezafibrate in Mitochondrial Myopathy
| Adult patients 18-64 years of age with confirmed mt.3243G>A mutation and evidence of myopathy
| Newcastle-upon-Tyne Hospitals NHS Trust | Recruiting |
| NCT02805790 | A Study to Evaluate the Safety, Tolerability, and Efficacy of Subcutaneous Injections of Elamipretide (MTP-131) in Subjects With Genetically Confirmed Mitochondrial Disease Previously Treated in the Stealth BioTherapeutics SPIMM-201 Study (MMPOWER-2) | Patients with genetically confirmed mitochondrial disease | Stealth BioTherapeutics | Ongoing but not recruiting |
| NCT02053766 | Anesthesia in Patients With Mitochondrial Disease | Children up to 17 years old | The University of Texas Health Science Center, Houston | Recruiting |
| NCT02348125
| Does clinical treatment of mitochondrial dysfunction impact Autism Spectrum Disorder? | Children between 3-12 years of age with a diagnosed Autism Spectrum Disorder | Drexel University | Recruiting |
| NCT02311257 | Survey on Supplement Use in Mitochondrial Disease | (1) NAMDC Clinical Registry or RDCRN Contact Registry participant; and (2) Diagnosis of a mitochondrial disease confirmed by either electron transport chain abnormalities or molecular testing.
| University of South Florida | Completed |
| NCT02284334 | Glycemic Index in Mitochondrial Disease
| Patients 7-65 years of age with mitochondrial diseases, from existing observational cohort study and/or CHOP Genetics/Metabolism clinic
| University of Pennsylvania/ Children’s Hospital of Philadelphia | Completed |
| NCT02255422
| RTA 408 Capsules in Patients With Mitochondrial Myopathy – MOTOR | Adult patients 18-75 with mitochondrial myopathy and genetically-identified disease | Reata Pharmaceuticals AbbVie | Recruiting |
| NCT02367014 | Bendavia (MTP-131) in people ages 16-65 years with Mitochondrial Myopathy
| Adult patients 16-65 years with mitochondrial myopathy and genetically-identified disease | Stealth BioTherapeutics | Completed |
| NCT02161848 | MRI Study – Chronic Progressive External Ophthalmoplegia (CPEO)
| Danish patients between 18-80 years of age with verified single large-scale mtDNA deletions and chronic progressive external ophthalmoplegia.
| Rigshospitalet, Denmark | Completed |
| NCT02161380 | Safety Study of Adeno-associated virus vector for LHON gene therapy | LHON due to G11778A mutation (LHON GTT) and 15-60 years of age | University of Miami | Recruiting |
| NCT02154711 | MRI muscle phenotyping in mitochondrial disease | Genetically diagnosed mitochondrial disease and healthy controls | University of Pennsylvania | Recruiting |
| NCT02104336 | Phase 2 Study of EPI-743 in Children With Pearson Syndrome | Patients with Pearson Syndrome up to 18 years of age with a genetically confirmed diagnosis | Edison Pharma | Terminated |
| NCT02064569 | Safety evaluation of gene therapy GS010 in LHON patients | LHON due to G11778A mutation – 18+ years of age | GenSight Biologics – Paris, France | Ongoing but not recruiting |
| NCT02053766 | Anesthesia in Patients with Mitochondrial Disease | Pediatric patient diagnosed with mitochondrial dysfunction based on modified Walker criteria and is undergoing procedure >1 hour | University of Texas Health Science Center, Houston | Recruiting |
| NCT02023866 | Open-Label, Dose-Escalating Study to Assess Safety, Tolerability, Efficacy, PK and PD of RP103 in Children With Inherited Mitochondrial Disease | Genetically diagnosed mitochondrial disease | Raptor Pharma | Completed |
| NCT02003170 | Etiology and early diagnosis of neurodevelop-mental disorders | Children with neurodevelop-mental delays who present to Arkansas Children’s Outpatient Clinics | University of Arkansas | Recruiting |
| NCT02000284 | Mitochondrial Dysfunction in Autism Spectrum Disorders | Mitochondrial Disease, Autism Spectrum Disorder, Developmental Delay or Typically Developing Children | Arkansas Children’s Hospital | Recruiting |
| NCT01886378 | Phase 2 open-label study evaluating safety and clinical effect of UX007 (Triheptanoin) in patients with long-chain fatty acid oxidation disorders (LC-FAOD) | Confirmed diagnosis of CPT II, VLCAD, LCHAD, or TFP deficiency and at least 6 months of age | Ultragenyx | Completed |
| NCT01803906 | Tissue Sample Study for Mitochondrial Disorders
| Patients of all ages, race, gender with known or suspected mitochondrial disorders and their carrier relatives (if requested).
| Columbia University | Enrolling by invitation only |
| NCT01780168 | NIH MINI Study (Metabolism, Infection and Immunity in Inborn Errors of Metabolism | Urea Cycle Disorders, Fatty Acid Oxidation, Organic Acidemias, Mitochondrial Disease
| National Human Genome Research Institute | Recruiting |
| NCT01694953 | Natural History Study of MNGIE | 5+ years old with Thymidine Phosphorylase defect | Columbia University | Recruiting |
| NCT01694940 | Patient contact registry and tissue biorepository | Possible or known mitochondrial disorders | NAMDC/ Columbia University | Recruiting |
| NCT01642056 | EPI-743 for Metabolism or Mitochondrial Disorders
| Children between 2 and 11 years of age who have metabolic or mitochondrial problems. | National Human Genome Research Institute (NHGRI) | Recruiting |
| NCT01532791 | Natural History Study – Mitochondrial Disease | 6 years and older with documented mtDNA point mutations | Columbia University | Recruiting |
| NCT01461304 | Triheptanoin (C7) for Fatty Acid Oxidation Disorders and Glycogen Storage Diseases | VLCAD, CPT1, CPT2, TFP, LCHAD, Glycogen Storage Diseases, Pyruvate Carboxylase | University of Pittsburgh Ultragenyx | Expanded access is currently available for this treatment. |
| NCT01148550 | Longitudinal Study of Mitochondrial Hepatopathies (MITOHEP) | Hepatic respiratory chain or fatty acid oxidation disorders | National Institute of Diabetes and Digestive and Kidney Diseases | Recruiting |
| NCT02154711 | Magnetic Resonance Imaging (MRI) Muscle Phenotyping in Mitochondrial Disease (Currently recruiting) | The purpose of the study is to use a new research imaging technique, a kind of magnetic resonance imaging (MRI), to measure important metabolic features of muscle, including mitochondrial function, in people with mitochondrial disease and in healthy individuals ages 18-65. | University of Pennsylvania | Recruiting |
| NCT01694940 | North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC) | Mitochondrial Disorders | Mitochondrial Genetic Disorders | Mitochondrial Diseases | Disorder of Mitochondrial Respiratory Chain Complexes | Deletion and Duplication of Mitochondrial DNA | Columbia University | National Institute of Neurological Disorders and Stroke (NINDS) | Recruiting |
| NCT02809170 | The Effect of Arginine and Citrulline Supplementation on Endothelial Dysfunction in Mitochondrial Diseases | Mitochondrial Disease | Tawam Hospital | Recruiting |
| NCT02053766 | Anesthesia in Patients With Mitochondrial Disease | Mitochondrial Disease | The University of Texas Health Science Center, Houston | Recruiting |
| NCT02745938 | GDF-15 as a Biomarker for Mitochondrial Disease | Mitochondrial Disease | Metabolic Myopathy | Muscular Dystrophy | Rigshospitalet, Denmark | Recruiting |
| NCT02920671 | Muscle OXPHOS and Nutrient Homeostasis | Mitochondrial Diseases | Obesity | University of Pennsylvania | Recruiting |
| NCT02000284 | Mitochondrial Dysfunction in Autism Spectrum Disorder | Autism Spectrum Disorder | Autism | Mitochondrial Disease | Developmental Delay | University of Arkansas | Jane Botsford Johnson Foundation | Arkansas Biosciences Institute (ABI) | UT Health Science Center at San Antonio | St. Christopher’s Hospital for Children | Recruiting |
| NCT02398201 | A Study of Bezafibrate in Mitochondrial Myopathy | Mitochondrial Disease | Newcastle-upon-Tyne Hospitals NHS Trust | Newcastle University | Recruiting |
| NCT01642056 | EPI-743 for Metabolism or Mitochondrial Disorders | Undiagnosed Diseases | Metabolic Disease | Neurology | Myoptahy | Oxidation/Reduction | Mitochondrial Disorders | National Human Genome Research Institute (NHGRI) | National Institutes of Health Clinical Center (CC) | Recruiting |
| NCT02909400 | The KHENERGY Study | Mitochondrial Diseases | Mitochondrial Myopathies | Mitochondrial Encephalomyopathies | MELAS | MIDD | Khondrion BV | Radboud Center for Mitochondrial Medicine (RCMM) | Radboud University | Recruiting |
| NCT03056794 | Natural History and Advanced Genetic Study of Pyruvate Dehydrogenase Complex Deficiencies | Pyruvate Dehydrogenase Complex Deficiency Disease | University Hospitals Cleveland Medical Center | Rare Diseases Clinical Research Network | National Institute of Neurological Disorders and Stroke (NINDS) | Recruiting |
| NCT02985710 | Assessment of Small Fiber Neuropathy in Rare Diseases Using Sudoscan | Small Fiber Neuropathy | Fabry Disease | Ehlers Danlos Syndrome | Mitochondrial Disease | Massachusetts General Hospital | Recruiting |
| NCT01498263 | Inherited Diseases, Caregiving, and Social Networks | Alzheimer’s Disease | Inborn Errors of Metabolism | Mitochondrial Disorders | Undiagnosed Diseases | National Human Genome Research Institute (NHGRI) | National Institutes of Health Clinical Center (CC) | Recruiting |
| NCT01439854 | Study of Dapagliflozin on Mitochondrial Dysfunction and Impaired Insulin Signaling/Action | Insulin Sensitivity | Multiple Mitochondrial Dysfunctions Syndrome | The University of Texas Health Science Center at San Antonio | National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | Recruiting |
| NCT01793168 | Rare Disease Patient Registry & Natural History Study – Coordination of Rare Diseases at Sanford | Please see page for the list of disorders. | Sanford Health | National Ataxia Foundation | International WAGR Syndrome Association | 4p- Support Group | ML4 Foundation | Cornelia de Lange Syndrome Foundation | Stickler Involved People | Kawasaki Disease Foundation | Klippel-Feil Syndrome Alliance | Klippel-Feil Syndrome Freedom | Hyperacusis Research Limited | Hypersomnia Foundation | Kabuki Syndrome Network | Kleine-Levin Syndrome Foundation | Leiomyosarcoma Direct Research Foundation | Marinesco-Sjogren Syndrome Support Group | Mucolipidosis Type IV Foundation | People with Narcolepsy 4 People with Narcolepsy (PWN4PWN) | Soft Bones Incorporated | Recruiting |
| NCT02838979 | Trial of Oral Glutamine on Mitochondrial Function in CKD | Cardiovascular Disease | Sarcopenia | Endothelial Dysfunction | Muscle Mitochondrial Function | Kidney Disease | University of Washington | New York Medical College | Emory University | Vanderbilt University | National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | Recruiting |
| NCT02895789 | Oxidative Capacity and Exercise Tolerance in Ambulatory SMA | Spinal Muscular Atrophy Type 3 | Mitochondrial Myopathy | Columbia University | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | Recruiting |
| NCT02596698 | Mitochondrial Dysfunction, Inflammation, and White Matter Integrity in Youth With Mood Disorders | Depression | Bipolar Disorder | Bipolar Disorder Not Otherwise Specified | Unspecified Mood Disorder | University of Minnesota – Clinical and Translational Science Institute | Ontario Mental Health Foundation | Recruting |
| NCT02457702 | Mitochondrial Function in Patients With Severe Liver Disease | Hepatic Insufficiency | Hypoalbuminemia | University of Texas Southwestern Medical Center | National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | National Institutes of Health (NIH) | Recruiting |
| NCT02549703 | Mitochondrial Dysfunction and Disease Progression | Clinically Isolated Syndrome | Relapsing-Remitting Multiple Sclerosis | Secondary Progressive Multiple Sclerosis | Primary Progressive Multiple Sclerosis | Icahn School of Medicine at Mount Sinai | Columbia University | The New York Stem Cell Foundation | Recruiting |
| NCT02517307 | Fatty Acid Oxidation Defects and Insulin Sensitivity | Very Long-chain Acyl-CoA Dehydrogenase Deficiency | Trifunctional Protein Deficiency | Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency | Medium-chain Acyl-CoA Dehydrogenase Deficiency | Normal Volunteers | Oregon Health and Science University | Recruiting |
| NCT02255435 | RTA 408 Capsules in Patients With Friedreich’s Ataxia – MOXIe | Friedreich Ataxia | Reata Pharmaceuticals, Inc. | AbbVie | Friedreich’s Ataxia Research Alliance | Recruiting |
| NCT03137355 | The Leigh Syndrome Registry | Leigh Syndrome | Leigh Disease | Leigh’s Necrotizing Encephalopathy | Subacute Necrotizing Encephalomyopathy | Subacute Necrotizing Encephalomyelopathy | The University of Texas Health Science Center, Houston | Recruiting |
| NCT03059420 | Genetic Studies of Strabismus, Congenital Cranial Dysinnervation Disorders (CCDDs), and Their Associated Anomalies | Congenital Fibrosis of Extraocular Muscles | Duane Retraction Syndrome | Duane Radial Ray Syndrome | Mobius Syndrome | Brown Syndrome | Marcus Gunn Syndrome | Strabismus Congenital | Horizontal Gaze Palsy | Horizontal Gaze Palsy With Progressive Scoliosis | Facial Palsy | Facial Paresis, Hereditary, Congenital | Third Nerve Palsy | Fourth Nerve Palsy | Sixth Nerve Palsy | Synkinesis | Ocular Motility Disorders | Levator-Medial Rectus Synkinesis | Athabaskan Brainstem Dysgenesis | Tongue Paralysis | Ninth Nerve Disorder | Fifth Nerve Palsy | Seventh Nerve Palsy | Eleventh Nerve Disorder | Twelfth Nerve Disorder | Vagus Nerve Paralysis | Moebius Sequence | Boston Children’s Hospital | Howard Hughes Medical Institute | National Eye Institute (NEI) | Recruiting |
| NCT03153293 | Safety and Efficacy Study of Gene Therapy for The Treatment of Leber’s Hereditary Optic Neuropathy | Leber Hereditary Optic Neuropathy | Huazhong University of Science and Technology | Shiyan Taihe Hospital | Not Recruiting |
| NCT01694953 | The Natural History Study of Mitochondrial NeuroGastroIntestinal Encephalopathy (MNGIE) | Mitochondrial NeuroGastroIntestinal Encephalopathy (MNGIE) | Columbia University | National Institute of Neurological Disorders and Stroke (NINDS) | Recruiting |
| NCT03011541 | FA Clinical Outcome Measures | Friedreich Ataxia | Neuro-Degenerative Disease | Children’s Hospital of Philadelphia | Friedreich’s Ataxia Research Alliance | University of Rochester | Recruiting |
| NCT02771379 | Post Authorisation Safety Study With Raxone in LHON Patients | Leber’s Hereditary Optic Neuropathy (LHON) | Santhera Pharmaceuticals | Recruiting |
| NCT03011541 | Stem Cell Ophthalmology Treatment Study II | Retinal Disease | Age-Related Macular Degeneration | Retinitis Pigmentosa | Stargardt Disease | Optic Neuropathy | Nonarteritic Ischemic Optic Neuropathy | Optic Atrophy | Optic Nerve Disease | Glaucoma | Leber Hereditary Optic Neuropathy | MD Stem Cells | Recruiting |
| NCT02635269 | Fat and Sugar Metabolism During Exercise in Patients With Metabolic Myopathy | Metabolism, Inborn Errors | Lipid Metabolism, Inborn Errors | Carbohydrate Metabolism, Inborn Errors | Long-Chain 3-Hydroxyacyl-CoA Dehydrogenase Deficiency | Glycogenin-1 Deficiency (Glycogen Storage Disease Type XV) | Carnitine Palmitoyl Transferase 2 Deficiency | VLCAD Deficiency | Medium-chain Acyl-CoA Dehydrogenase Deficiency | Multiple Acyl-CoA Dehydrogenase Deficiency | Carnitine Transporter Deficiency | Neutral Lipid Storage Disease | Glycogen Storage Disease Type II | Glycogen Storage Disease Type III | Glycogen Storage Disease Type IV | Glycogen Storage Disease Type V | Muscle Phosphofructokinase Deficiency | Phosphoglucomutase 1 Deficiency | Phosphoglycerate Mutase Deficiency | Phosphoglycerate Kinase Deficiency | Phosphorylase Kinase Deficiency | Beta Enolase Deficiency | Lactate Dehydrogenase Deficiency | Glycogen Synthase Deficiency | Rigshospitalet, Denmark | Recruiting |
| NCT02840669 | A Study to Characterize the Cardiac Phenotype of Individuals With Friedreich’s Ataxia (CARFA Study) | Friedreich’s Ataxia | Adverum Biotechnologies, Inc. | Annapurna Therapeutics SAS, a wholly owned subsidiary of Adverum Biotechnologies | Weill Medical College of Cornell University | Recruiting |
| NCT02316314 | Characterization of the Cardiac Phenotype of Friedreich’s Ataxia (FRDA) | Friedreich’s Ataxia | Weill Medical College of Cornell University | Recruiting |
| NCT01599286 | Short-term Outcome of N-Carbamylglutamate in the Treatment of Acute Hyperammonemia | Propionic Acidemia, Type I and/or Type II | Methylmalonic Acidemia | Carbamoyl-Phosphate Synthase I Deficiency Disease | Ornithine Carbamoyltransferase Deficiency | Mendel Tuchman | Children’s Research Institute | Boston Children’s Hospital | University Hospitals Cleveland Medical Center | University of California, Los Angeles | Children’s Hospital of Philadelphia | Stanford University | Icahn School of Medicine at Mount Sinai | University of Pittsburgh | Children’s Hospital Colorado | Recruiting |
| NCT03120013 | Rehabilitative Trial With Cerebello-Spinal tDCS in Neurodegenerative Ataxia | Ataxia, Cerebellar | Cerebellar Ataxia | Spinocerebellar Ataxias | Ataxia, Spinocerebellar | Spinocerebellar Ataxia Type 1 | Spinocerebellar Ataxia Type 2 | Spinocerebellar Ataxia 3 | Spinocerebellar Degenerations | Friedreich Ataxia | Ataxia With Oculomotor Apraxia | Multiple System Atrophy | Azienda Ospedaliera Spedali Civili di Brescia | Recruiting |
| NCT02660112 | (+) Epicatechin to Treat Friedreich’s Ataxia | Friedreich’s Ataxia | Ralitza Gavrilova | Cardero Therapeutics, Inc. | Mayo Clinic | Recruiting |
| NCT02796274 | Historical Case Record Survey of Visual Acuity Data From Patients With Leber’s Hereditary Optic Neuropathy (LHON) | Leber’s Hereditary Optic Neuropathy (LHON) | Santhera Pharmaceuticals | Recruiting |
| NCT02774005 | Study to Assess the Efficacy and Safety of Raxone in LHON Patients | Leber’s Hereditary Optic Neuropathy (LHON) | Santhera Pharmaceuticals | Not Recruiting |
| NCT02497534 | Biomarkers in Friedreich’s Ataxia | Friedreich’s Ataxia | University of Florida | Children’s Miracle Network | National Institutes of Health (NIH) | Recruiting |
| NCT02705547 | Rosuvastatin (Crestor) in Friedreich Ataxia | Friedreich’s Ataxia | Children’s Hospital of Philadelphia | Friedreich’s Ataxia Research Alliance | Recruiting |
| NCT02424435 | Methylprednisolone Treatment of Friedreich Ataxia | Friedreich’s Ataxia | Children’s Hospital of Philadelphia | Friedreich’s Ataxia Research Alliance | Recruiting |
| NCT01921868 | An Open-label Study of the Effects of Acetyl-L-Carnitine on Cardiovascular Outcomes in Friedreich’s Ataxia | Friedreich’s Ataxia | University of South Florida | Recruiting |
| NCT03011203 | Acute Nutritional Ketosis in GSD IIIa | Glycogen Storage Disease IIIA | University Medical Center Groningen | University of the Faroe Islands | University of Oxford | Stichting Stofwisselkracht | Stichting Metakids | Recruiting |
| NCT02923063 | Exercise Study Testing Enhanced Energetics of Muscle Mitochondria in CKD | Moderate-severe Chronic Kidney Disease Not Treated With Dialysis | Non-insulin Dependent Diabetic Kidney Disease | University of Washington | Recruiting |
| NCT00004568 | Study of Inherited Neurological Disorders | Ataxia | Motor Neuron Disease | Muscular Disease | Muscular Dystrophy | Peripheral Nervous System Disease | National Institute of Neurological Disorders and Stroke (NINDS) | National Institutes of Health Clinical Center (CC) | Recruiting |
| NCT02427178 | MNGIE Allogeneic Hematopoietic Stem Cell Transplant Safety Study | Mitochondrial Neurogastrointestinal Encephalomyopathy (MNGIE) | Michio Hirano | Cornell University | National Institute of Neurological Disorders and Stroke (NINDS) | Columbia University | Recruiting |
| NCT03111719 | Human Brown Adipose Tissue and Mitochondrial Respiration | Mitochondrial Metabolism Disorders | Adipose Tissue, Brown | Aging | Maastricht University Medical Center | Ministry of Education, Culture and Science | Recruiting |
| NCT02696044 | Treatment of Mitochondrial Dysfunction in Rett Syndrome With Triheptanoin | Rett Syndrome | Emory University | Ultragenyx Pharmaceutical Inc | Recruiting |
| NCT01625663 | Heart and Muscle Metabolism in Barth Syndrome | Barth Syndrome | Washington University School of Medicine | University of Florida | Recruiting |
| NCT02697201 | Dynamics of Muscle Mitochondria in Type 2 Diabetes (DYNAMMO T2D) | Insulin Resistance | The Cleveland Clinic | Recruiting |
| NCT02700698 | Mitochondrial Function in Circulating Cells and Muscle Tissue | Healthy | Obesity | Insulin Sensitivity | Women | University of Arkansas | Arkansas Children’s Hospital Research Institute | Recruiting |
| NCT00727974 | Genetic Analysis of Children With Cyclic Vomiting Syndrome (CVS) and Migraines | Vomiting | Migraines | Medical College of Wisconsin | Children’s Hospital and Health System Foundation, Wisconsin | Recruiting |
| NCT01629459 | Resistance Exercise in Barth Syndrome | Barth Syndrome | Washington University School of Medicine | Recruiting | NCT04378075 | A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy | Mitochondrial Diseases | Drug Resistant Epilepsy | PTC Therapeutics | Not Yet Recruiting |