Not applicableStudy completedNCT04805268What this trial is testingA Single Case Study of HypermetabolismWho this might be right forHypermetabolism Due to Defect in MitochondriaLuft Disease The Hospital for Sick Children 1
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 participantsNCT01793168What this trial is testingRare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at SanfordWho this might be right forRare DisordersUndiagnosed DisordersDisorders of Unknown Prevalence+340 more Sanford Health 20,000
Large-scale testing (Phase 3)Study completedNCT05162768What this trial is testingStudy to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD)Who this might be right forMitochondrial MyopathiesMitochondrial PathologyMitochondrial DNA Mutation+5 more Stealth BioTherapeutics Inc. 102
Post-approval studies (Phase 4)Not Yet RecruitingNCT07531251What this trial is testingPh3b/4 Trial in Patients With Barth SyndromeWho this might be right forBarth Syndrome Stealth BioTherapeutics Inc. 48