Large-scale testing (Phase 3)Study completedNCT03406104What this trial is testingRESCUE and REVERSE Long-term Follow-upWho this might be right forLeber Hereditary Optic Neuropathy GenSight Biologics 62
Testing effectiveness (Phase 2)Study completedNCT02064569What this trial is testingSafety Evaluation of Gene Therapy in Leber Hereditary Optic Neuropathy (LHON) PatientsWho this might be right forLeber Hereditary Optic Neuropathy GenSight Biologics 19
Early research (Phase 1)Study completedNCT01064505What this trial is testingSafety Study of a Single IVT Injection of QPI-1007 in Chronic Optic Nerve Atrophy and Recent Onset NAION PatientsWho this might be right forOptic AtrophyNon-arteritic Anterior Ischemic Optic Neuropathy Quark Pharmaceuticals 48
Not applicableNot Yet RecruitingNCT07075887What this trial is testingOptic Nerve Head Evaluation Through Multimodal Blood Flow Analysis: a Prospective Observational, Multi-center StudyWho this might be right forNeuro-OphthalmologyOptic NeuropathiesOptic Atrophy, Hereditary, Leber+3 more Barboni 70
Not applicableLooking for participantsNCT06682819What this trial is testingMetabolomics Analysis According to the Retinal Nerve Fiber Layer in Patients With NOHL Mutations (MétabOCT)Who this might be right forHealthy SubjectsLeber Hereditary Optic Neuropathy Hôpital Necker-Enfants Malades 90
Large-scale testing (Phase 3)Active Not RecruitingNCT00999609What this trial is testingSafety and Efficacy Study in Subjects With Leber Congenital AmaurosisWho this might be right forInherited Retinal Dystrophy Due to RPE65 MutationsLeber Congenital Amaurosis Spark Therapeutics, Inc. 31
Early research (Phase 1)Study completedNCT00516477What this trial is testingSafety Study in Subjects With Leber Congenital AmaurosisWho this might be right forLeber Congenital Amaurosis Spark Therapeutics, Inc. 12
Testing effectiveness (Phase 2)Study completedNCT02693119What this trial is testingA Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide Topical Ophthalmic Solution for Treatment of Leber's Hereditary Optic NeuropathyWho this might be right forLeber's Hereditary Optic Neuropathy Stealth BioTherapeutics Inc. 12
Testing effectiveness (Phase 2)Looking for participantsNCT07303296What this trial is testingEfficacy and Safety Study of Bilateral IVT Injection of GS010 at Two Dose Levels in LHON PatientsWho this might be right forLeber Hereditary Optic Disease GenSight Biologics 14
Not applicableStudy completedNCT01892943What this trial is testingLeber Hereditary Optic Neuropathy (LHON) Historical Case Record SurveyWho this might be right forLeber Hereditary Optic Neuropathy (LHON) Santhera Pharmaceuticals 306
Not applicableLooking for participantsNCT02435940What this trial is testingInherited Retinal Degenerative Disease RegistryWho this might be right forEye Diseases HereditaryRetinal DiseaseAchromatopsia+25 more Foundation Fighting Blindness 20,000
Early research (Phase 1)Study completedNCT00821340What this trial is testingClinical Trial of Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 MutationsWho this might be right forLeber Congenital Amaurosis Hadassah Medical Organization 3
Testing effectiveness (Phase 2)Study completedNCT01496040What this trial is testingClinical Gene Therapy Protocol for the Treatment of Retinal Dystrophy Caused by Defects in RPE65Who this might be right forLeber Congenital Amaurosis Nantes University Hospital 9
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
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
Testing effectiveness (Phase 2)UnknownNCT02176733What this trial is testingTrial of Cyclosporine in the Acute Phase of Leber Hereditary Optic NeuropathyWho this might be right forLeber Hereditary Optic Neuropathy University Hospital, Angers 12
Large-scale testing (Phase 3)Looking for participantsNCT04912843What this trial is testingGene Therapy Clinical Trial for the Treatment Of Leber's HereDitary Optic NeuropathyWho this might be right forLeber's Hereditary Optic Neuropathy (LHON) Wuhan Neurophth Biotechnology Limited Company 102
Not applicableStudy completedNCT02796274What this trial is testingHistorical Case Record Survey of Visual Acuity Data From Patients With Leber's Hereditary Optic Neuropathy (LHON)Who this might be right forLeber's Hereditary Optic Neuropathy (LHON) Santhera Pharmaceuticals 219
Very early researchActive Not RecruitingNCT06088992What this trial is testingLeber Congenital Amaurosis Inherited Blindness of Gene Therapy Trial(LIGHT)Who this might be right forLeber Congenital Amaurosis Xinhua Hospital, Shanghai Jiao Tong University School of Medicine 9
Large-scale testing (Phase 3)Looking for participantsNCT06891443What this trial is testingStudy to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)Who this might be right forLeber Congenital Amaurosis 10BlindnessLeber Congenital Amaurosis+7 more Laboratoires Thea 32