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
Not applicableActive Not RecruitingNCT06642051What this trial is testingSafety of the Sonablate HIFU System for the Ablation of Incompetent Veins of the PeripheryWho this might be right forChronic Venous Insufficiency, CVIVenous MalformationsVenous Leg Ulcers+3 more Sonablate 30
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
Not applicableLooking for participantsNCT05355415What this trial is testingAdaptive Optics Imaging of Outer Retinal DiseasesWho this might be right forRetinal DegenerationAge-Related Macular DegenerationRetinitis Pigmentosa+7 more Food and Drug Administration (FDA) 100
Not applicableNot Yet RecruitingNCT07313085What this trial is testingMechanisms of Fecal Incontinence in Wolfram SyndromeWho this might be right forWolfram SyndromeFecal Incontinence Hôpital Necker-Enfants Malades 20
Large-scale testing (Phase 3)Study completedNCT01203436What this trial is testingSupplemental Therapeutic Oxygen for Prethreshold Retinopathy of PrematurityWho this might be right forInfant, NewbornInfant, Low Birth WeightInfant, Small for Gestational Age+3 more NICHD Neonatal Research Network 649
Testing effectiveness (Phase 2)Looking for participantsNCT06789445What this trial is testingInvestigate the Safety of OpCT-001 in Adults Who Have Primary Photoreceptor Disease (CLARICO)Who this might be right forPrimary Photoreceptor DiseaseRetinitis Pigmentosa (RP)Usher Syndrome+6 more BlueRock Therapeutics 54
Testing effectiveness (Phase 2)Study completedNCT00500812What this trial is testingA Safety Study for Cethrin (BA-210) in the Treatment of Acute Thoracic and Cervical Spinal Cord InjuriesWho this might be right forSpinal Cord Injury Vertex Pharmaceuticals Incorporated 48
Testing effectiveness (Phase 2)Active Not RecruitingNCT04355689What this trial is testingSafety and Efficacy of NPI-001 Tablets for RP Associated With Usher SyndromeWho this might be right forUsher Syndromes Nacuity Pharmaceuticals, Inc. 49
Not applicableStudy completedNCT02965534What this trial is testingEvaluation of a Night Spectacle Correction Concerning an Improvement of Mesopic Vision QualityWho this might be right forMyopia-Night Blindness University of Applied Sciences Jena 100
Not applicableLooking for participantsNCT03988764What this trial is testingMonogenic Diabetes Misdiagnosed as Type 1Who this might be right forDiabetes Mellitus, Type 1Monogenic DiabetesNeonatal Diabetes+4 more McGill University Health Centre/Research Institute of the McGill University Health Centre 5,000
Not applicableEnrolling By InvitationNCT00341874What this trial is testingGenetic Analyses of Nonsyndromic and Syndromic Deafness in PakistanWho this might be right forHearing Disorder National Institute on Deafness and Other Communication Disorders (NIDCD) 24,000
Testing effectiveness (Phase 2)UnknownNCT04940572What this trial is testingEfficacy Study of Daily Administration of VPA in Patients Affected by Wolfram SyndromeWho this might be right forWolfram Syndrome Centre d'Etude des Cellules Souches 23
Not applicableWithdrawnNCT01302327What this trial is testingGLP Analogs for Diabetes in Wolfram Syndrome PatientsWho this might be right forDiabetes Mellitus Associated With Genetic SyndromeWolfram Syndrome Hadassah Medical Organization
Testing effectiveness (Phase 2)Looking for participantsNCT06627179What this trial is testingStudy to Evaluate Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A GeneWho this might be right forRetinitis Pigmentosa (RP)Usher Syndrome Type 2Deaf Blind+4 more Laboratoires Thea 81
Not applicableLooking for participantsNCT06475651What this trial is testingCharacterization and Contribution of Genome-wide DNA Methylation (DNA Methylation Episignatures) in Rare Diseases With Prenatal OnsetWho this might be right forRare Fetal Genetic DiseasesCongenital Malformation Assistance Publique - Hôpitaux de Paris 63
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
Post-approval studies (Phase 4)Ended earlyNCT00634972What this trial is testingEfficient Study of ACULAR in Inhibiting Proliferative Retinopathy in PrematurityWho this might be right forRetinopathy of PrematurityRetinal DetachmentBlindness University of South Alabama 83
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