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Condition: Mitochondrial Genetic Disorders Synced from ClinicalTrials.gov · July 29, 2026

Recruiting studies: Mitochondrial Genetic Disorders

Studies currently listed as recruiting on the public registry for this search term. Each page explains the study in plain language, with locations and the study team's contact details. Clinicians: generate a printable list for your patient →

Efficacy and Safety of the Treatment of Pyruvate Dehydrogenase Deficiency Patients With Glycerol Phenylbutyrate (RAVICTI
Phase 2 · 1 location(s) · Assistance Publique - Hôpitaux de Paris · NCT06887777Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy
Phase 2 · 1 location(s) · Lexeo Therapeutics · NCT07721025A Study to Learn More About the Effects and Long-Term Safety of Omaveloxolone (BIIB141) in Children and Teens With Fried
Phase 3 · 34 location(s) · Biogen · NCT06953583The NIH MINI Study: Metabolism, Infection, and Immunity in Inborn Errors of Metabolism
Phase N/A · 1 location(s) · National Human Genome Research Institute (NHGRI) · NCT01780168KHENERFIN Study: A Trial to Evaluate the Efficacy and Safety of Sonlicromanol in Primary Mitochondrial Diseases
Phase 3 · 10 location(s) · Khondrion BV · NCT06451757Electroencephalogram in Patients With Friedreich's Ataxia for the Study of the Structural and Functional Connectome.
NA · 1 location(s) · IRCCS Eugenio Medea · NCT07095062Friedreich Ataxia Nerve Ultrasund
Phase N/A · 1 location(s) · Centre Hospitalier Universitaire de Nice · NCT07508631Effect of MitoQ on Platelet Function and Reactive Oxygen Species Generation in Patients With Sickle Cell Anemia
NA · 5 location(s) · University of Pittsburgh · NCT04109820Triheptanoin for Children With Primary-Specific Pyruvate Dehydrogenase Complex (PDC) Deficiency
Phase 1 · 1 location(s) · Jirair Krikor Bedoyan · NCT06340685Natural History and Advanced Genetic Study of Pyruvate Dehydrogenase Complex Deficiencies
Phase N/A · 1 location(s) · University of Pittsburgh · NCT03056794Hepatic Histopathology in Urea Cycle Disorders
Phase N/A · 2 location(s) · Baylor College of Medicine · NCT04908319Patient-reported, Health Economic and Psychosocial Outcomes in Friedreich Ataxia
Phase N/A · 6 location(s) · German Center for Neurodegenerative Diseases (DZNE) · NCT05943002Efficacy of Stabilometric Platform to Improve Standing Balance in Patients With Friedreich's Ataxia
NA · 1 location(s) · IRCCS Eugenio Medea · NCT06692296New Non-invasive Modalities for Assessing Retinal Structure and Function
NA · 1 location(s) · Randy Kardon · NCT03475173Clinical Trial in Patients With Barth Syndrome- 4TAZPower
Phase 4 · 3 location(s) · Stealth BioTherapeutics Inc. · NCT07531251Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study
Phase N/A · 34 location(s) · Friedreich's Ataxia Research Alliance · NCT06016946A Study to Learn More About the Long-Term Safety of BIIB141 (Omaveloxolone) in Participants With Friedreich's Ataxia Who
Phase N/A · 14 location(s) · Biogen · NCT06623890Drug Repurposing for Mitochondrial Disorders Using iPSCs Derived Neural Cells
Phase N/A · 2 location(s) · Charite University, Berlin, Germany · NCT06967831Ganciclovir Resistant/Refractory Cytomegalovirus Infection in SOT Recipients and HSCT Patients
Phase N/A · 23 location(s) · IRCCS Azienda Ospedaliero-Universitaria di Bologna · NCT05234723The International Registry for Leigh Syndrome
Phase N/A · 1 location(s) · The University of Texas Health Science Center, Houston · NCT03137355Characterization of Patients With Cardiomyopathy to Identify Critical Patients Candidates for Cardiac Transplantation
Phase N/A · 3 location(s) · IRCCS Azienda Ospedaliero-Universitaria di Bologna · NCT06813443A Longitudinal Natural History Study of OPA1-Associated Autosomal-Dominant Optic Atrophy
Phase N/A · 1 location(s) · Ludwig-Maximilians - University of Munich · NCT07729982A Multiple Ascending Dose Study of DT-216P2 in Patients With Friedreich's Ataxia
Phase 1/Phase 2 · 3 location(s) · Design Therapeutics, Inc. · NCT06874010Global Registry and Natural History Study for Mitochondrial Disorders
Phase N/A · 33 location(s) · LMU Klinikum · NCT05554835North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)
Phase N/A · 17 location(s) · Columbia University · NCT01694940A Study of SGT-212 Gene Therapy in Friedreich's Ataxia
Phase 1 · 3 location(s) · Solid Biosciences Inc. · NCT07180355Gene Therapy Clinical Trial for the Treatment Of Leber's HereDitary Optic Neuropathy
Phase 2/Phase 3 · 1 location(s) · Wuhan Neurophth Biotechnology Limited Company · NCT04912843Clinical Course Of Disease In Participants With FA-CM
Phase N/A · 19 location(s) · Lexeo Therapeutics · NCT06865482Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Phase N/A · 2 location(s) · Sanford Health · NCT01793168Study of N-acetylcysteine in the Treatment of Patients With the m.3243A>G Mutation and Low Brain Glutathione Levels
Phase 1 · 1 location(s) · Michio Hirano, MD · NCT05241262
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This page is independently generated by Eichor from the public ClinicalTrials.gov record and re-synced daily. It is not the sponsor's official website unless claimed. Nothing here is medical advice; eligibility is always determined by the study team — talk to your own doctor first.