Not applicableNot Yet RecruitingNCT07026565What this trial is testingPsychotherapy Group for Parents of Children With LCAWho this might be right forLeber Congenital Amaurosis (LCA) Instituto de Genética Ocular 40
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
Testing effectiveness (Phase 2)Study completedNCT03140969What this trial is testingStudy to Evaluate QR-110 in Leber's Congenital Amaurosis (LCA) Due to the c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 GeneWho this might be right forLeber's Congenital Amaurosis Laboratoires Thea 11
Not applicableStudy completedNCT02946879What this trial is testingLong-Term Follow-Up Gene Therapy Study for Leber Congenital Amaurosis OPTIRPE65 (Retinal Dystrophy Associated With Defects in RPE65)Who this might be right forLeber Congenital Amaurosis (LCA)Eye DiseasesEye Diseases, Hereditary+1 more MeiraGTx UK II Ltd 15
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
Not applicableStudy completedNCT02970266What this trial is testingGenetic Decryption of Leber Congenital Amaurosis (LCA) in a Large Cohort of Independent Families.Who this might be right forLeber Congenital Amaurosis Assistance Publique - Hôpitaux de Paris 659
Large-scale testing (Phase 3)Active Not RecruitingNCT03913143What this trial is testingEvaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)Who this might be right forLeber Congenital Amaurosis 10BlindnessLeber Congenital Amaurosis+7 more ProQR Therapeutics 36
Not applicableStudy completedNCT00422721What this trial is testingGenetic Study of Patients Suffering From Congenital Amaurosis of Leber or From an Early Severe Retinal DystrophyWho this might be right forAmaurosisRetinal Diseases Nantes University Hospital 360
Testing effectiveness (Phase 2)Study completedNCT00643747What this trial is testingSafety Study of RPE65 Gene Therapy to Treat Leber Congenital AmaurosisWho this might be right forRetinal Degeneration University College, London 12
Not applicableNot Yet RecruitingNCT06024057What this trial is testingAn Expanded Clinical Study Evaluating the AAV2-RPE65 Gene Therapy(LX101) in Patients With LCAWho this might be right forTo Evaluate the Scaling Clinical Study of AAV2-RPE65 Gene Therapy Agent (LX101) in Patients With Congenital Amaurosis (LCA) Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine 3
Early research (Phase 1)Active Not RecruitingNCT00481546What this trial is testingPhase I Trial of Gene Vector to Patients With Retinal Disease Due to RPE65 MutationsWho this might be right forAmaurosis of LeberRetinal Diseases University of Pennsylvania 15
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
Very early researchUnknownNCT03566147What this trial is testingTreatment of RP and LCA by Primary RPE TransplantationWho this might be right forLeber Congenital Amaurosis, Retinitis Pigmentosa Eyecure Therapeutics Inc. 30
Early research (Phase 1)Study completedNCT01014052What this trial is testingSafety/Proof of Concept Study of Oral QLT091001 in Subjects With Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due to Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT) MutationsWho this might be right forLCA (Leber Congenital Amaurosis)RP (Retinitis Pigmentosa) QLT Inc. 32
Testing effectiveness (Phase 2)Active Not RecruitingNCT01208389What this trial is testingPhase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2Who this might be right forLeber Congenital Amaurosis Spark Therapeutics, Inc. 12
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
Testing effectiveness (Phase 2)Active Not RecruitingNCT03920007What this trial is testingStudy of Subretinally Injected ATSN-101 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2DWho this might be right forLeber Congenital AmaurosisLCALCA1 Atsena Therapeutics Inc. 15
Testing effectiveness (Phase 2)Study completedNCT02781480What this trial is testingClinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA)Who this might be right forLeber Congenital Amaurosis MeiraGTx UK II Ltd 15
Not applicableStudy completedNCT04525261What this trial is testingNatural History of Patients With Inherited Retinal Diseases Due to Mutations in RPE65 GeneWho this might be right forLeber Congenital Amaurosis 2Retinitis Pigmentosa 20 University of Campania Luigi Vanvitelli 60
Testing effectiveness (Phase 2)Ended earlyNCT03913130What this trial is testingExtension Study to Study PQ-110-001 (NCT03140969)Who this might be right forLeber Congenital Amaurosis 10BlindnessLeber Congenital Amaurosis+7 more Laboratoires Thea 9