Not applicableLooking for participantsNCT05250375What this trial is testingNatural History Study of Mitochondrial MyopathyWho this might be right forPrimary Mitochondrial Disease Children's Hospital of Philadelphia 1,300
Not applicableUnknownNCT05910151What this trial is testingSelective Screening of Children for Hereditary Metabolic Diseases by Tandem Mass Spectrometry in KazakhstanWho this might be right forPropionic/Methylmalonic AcidemiasMaple Syrup Urine DiseaseCitrullinemia+26 more West Kazakhstan Medical University 2,250
Not applicableUnknownNCT06025903What this trial is testingAssessing the Role of Mitochondrial Dysfunction in Primary Progressive Multiple SclerosisWho this might be right forMultiple Sclerosis IRCCS San Raffaele 140
Not applicableStudy completedNCT03917212What this trial is testingEnergy Metabolism in Branched-chain Organic AcidemiasWho this might be right forEnergy MetabolismMetabolic SyndromeInsulin Resistance German Diabetes Center 57
Not applicableWithdrawnNCT05277363What this trial is testingThe Natural Course of SURF1 DeficiencyWho this might be right forLeigh Syndrome Taysha Gene Therapies, Inc.
Testing effectiveness (Phase 2)Study completedNCT04513002What this trial is testingAtaxia-telangiectasia: Treating Mitochondrial Dysfunction With a Novel Form of AnaplerosisWho this might be right forAtaxia Telangiectasia The University of Queensland 30
Not applicableStudy completedNCT03979482What this trial is testingSkeletal Muscle Mitochondrial Abnormalities and the Metabolic Syndrome in PAHWho this might be right forPulmonary Artery Hypertension Laval University 30
Not applicableActive Not RecruitingNCT03655223What this trial is testingEarly Check: Expanded Screening in NewbornsWho this might be right forSpinal Muscular AtrophyFragile X SyndromeFragile X - Premutation+182 more RTI International 30,000
Testing effectiveness (Phase 2)Ended earlyNCT01681368What this trial is testingBirinapant for Advanced Ovarian, Fallopian Tube, and Peritoneal CancerWho this might be right forEpithelial Ovarian CancerPeritoneal NeoplasmsFallopian Tube Neoplasms National Cancer Institute (NCI) 11
Testing effectiveness (Phase 2)Study completedNCT02805790What this trial is testingSafety, Tolerability, Efficacy of MTP-131 for Treatment of Mitochondrial Disease in Subjects From the MMPOWER StudyWho this might be right forPrimary Mitochondrial Disease Stealth BioTherapeutics Inc. 30
Large-scale testing (Phase 3)Ended earlyNCT03323749What this trial is testingA Trial to Evaluate Safety and Efficacy of Elamipretide Primary Mitochondrial Myopathy Followed by Open-Label ExtensionWho this might be right forPrimary Mitochondrial Myopathy Stealth BioTherapeutics Inc. 218
Not applicableStudy completedNCT05017818What this trial is testingA Retrospective Study of Subjects With Thymidine Kinase 2 DeficiencyWho this might be right forThymidine Kinase 2 Deficiency Zogenix MDS, Inc. 61
Not applicableLooking for participantsNCT06890520What this trial is testingNeurometabolic Profile of Individuals With Primary Mitochondrial DiseaseWho this might be right forPrimary Mitochondrial Disease Children's Hospital of Philadelphia 30
Early research (Phase 1)Looking for participantsNCT06340685What this trial is testingTriheptanoin for Children With Primary-Specific Pyruvate Dehydrogenase Complex (PDC) DeficiencyWho this might be right forPyruvate Dehydrogenase Complex Deficiency Jirair Krikor Bedoyan 6
Not applicableLooking for participantsNCT04602325What this trial is testingSystemic Biomarkers of Brain Injury From HyperammonemiaWho this might be right forUrea Cycle DisorderOrganic AcidemiaMaple Syrup Urine Disease+3 more Children's National Research Institute 24
Early research (Phase 1)Looking for participantsNCT05569122What this trial is testingApplying pGz in Mitochondrial DiseaseWho this might be right forMitochondrial MyopathiesMitochondrial Diseases Children's Hospital of Philadelphia 90
Testing effectiveness (Phase 2)Ended earlyNCT04641962What this trial is testingEvaluate ASP0367 in Participants With Primary Mitochondrial MyopathyWho this might be right forPrimary Mitochondrial Myopathy Astellas Pharma Inc 34
Large-scale testing (Phase 3)Looking for participantsNCT06451757What this trial is testingKHENERFIN Study: A Trial to Evaluate the Efficacy and Safety of Sonlicromanol in Primary Mitochondrial DiseasesWho this might be right forMitochondrial DiseasesMaternally Inherited Diabetes and Deafness (MIDD)Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like Episodes (MELAS)+1 more Khondrion BV 220
Not applicableStudy completedNCT03048617What this trial is testingAn Observational Study of Patients With Primary Mitochondrial Disease (SPIMM-300)Who this might be right forPrimary Mitochondrial Disease Stealth BioTherapeutics Inc. 215
Testing effectiveness (Phase 2)Study completedNCT02367014What this trial is testingSafety, Tolerability, and Efficacy of MTP-131 for the Treatment of Mitochondrial MyopathyWho this might be right forMitochondrial Myopathy Stealth BioTherapeutics Inc. 36