Not applicableStudy completedNCT01625663What this trial is testingHeart and Muscle Metabolism in Barth SyndromeWho this might be right forBarth Syndrome Duke University 64
Large-scale testing (Phase 3)Looking for participantsNCT06056297What this trial is testingMavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious InfectionsWho this might be right forNeutropenia X4 Pharmaceuticals 176
Not applicableStudy completedNCT01194141What this trial is testingExercise Training in Barth SyndromeWho this might be right forBarth Syndrome Washington University School of Medicine 4
Not applicableStudy completedNCT01629459What this trial is testingResistance Exercise in Barth SyndromeWho this might be right forBarth Syndrome Washington University School of Medicine 12
Not applicableEnrolling By InvitationNCT03655223What 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
Not applicableLooking for participantsNCT05554835What this trial is testingGlobal Registry and Natural History Study for Mitochondrial DisordersWho this might be right forMitochondrial DiseasesKearns-Sayre SyndromeMIDD+15 more LMU Klinikum 6,000
Not applicableAvailableNCT04689360What this trial is testingAn Intermediate Size Expanded Access Protocol of ElamipretideWho this might be right forMitochondrial DiseasesBarth Syndrome Stealth BioTherapeutics Inc.
Large-scale testing (Phase 3)Study completedNCT03098797What this trial is testingA Trial to Evaluate Safety, Tolerability and Efficacy of Elamipretide in Subjects With Barth SyndromeWho this might be right forBarth Syndrome Stealth BioTherapeutics Inc. 12
Not applicableLooking for participantsNCT01694940What this trial is testingNorth American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)Who this might be right forMitochondrial DisordersMitochondrial Genetic DisordersMitochondrial Diseases+2 more Columbia University 1,000
Not applicableNo Longer AvailableNCT01461304What this trial is testingCompassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy MetabolismWho this might be right forVery Long-chain acylCoA Dehydrogenase (VLCAD) DeficiencyCarnitine Palmitoyltransferase Deficiencies (CPT1, CPT2)Mitochondrial Trifunctional Protein Deficiency+5 more Jerry Vockley, MD, PhD