Clinical Trials Directory

Trials / Conditions / Leigh Syndrome

Leigh Syndrome

11 registered clinical trials studyying Leigh Syndrome — 5 currently recruiting.

StatusTrialSponsorPhase
Not Yet RecruitingA Dose-ranging Study of TTI-0102 in Adults and Children With Leigh Syndrome Spectrum (LSS)
NCT06990984
Thiogenesis Therapeutics, Inc.Phase 2
Enrolling By InvitationSirolimus for Leigh Syndrome
NCT06843811
Matthew DemczkoPhase 2
WithdrawnA Study of the Natural Course of SURF1 Deficiency
NCT05277363
Taysha Gene Therapies, Inc.—
WithdrawnABI-009 (Nab-sirolimus) in Patients With Genetically-confirmed Leigh or Leigh-like Syndrome
NCT03747328
Aadi Bioscience, Inc.Phase 2
TerminatedA Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With
NCT04378075
PTC TherapeuticsPhase 2 / Phase 3
RecruitingThe International Registry for Leigh Syndrome
NCT03137355
The University of Texas Health Science Center, Houston—
CompletedA Dose-escalating Clinical Trial With KH176
NCT02544217
Khondrion BVPhase 1
CompletedLong-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome
NCT02352896
PTC TherapeuticsPhase 2
CompletedSafety and Efficacy Study of EPI-743 in Children With Leigh Syndrome
NCT01721733
PTC TherapeuticsPhase 2
RecruitingRare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
NCT01793168
Sanford Health—
RecruitingGlobal Registry and Natural History Study for Mitochondrial Disorders
NCT05554835
LMU Klinikum—