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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
An Optimised GA Interventional Trial (Opti-GAIN) to Test if Treatment With CTx001 is Safe and Works for People With Geographic Atrophy (GA)Geographic Atrophy Secondary to Age-related Macular DegenerationRecruitingPhase 1 / Phase 22026-10-08NCT07392255
A Non-Interventional Study Observing Short-Term Progression in Geographic Atrophy (GA)Geographic Atrophy, eye disorder, macular degeneration, retinal degeneration, retinal disorderRecruitingNot stated2026-10-08NCT07144137
A Study Investigating the Efficacy and Safety of Intravitreal (IVT) Injections of Vonaprument (Formerly ANX007) in Participants With Dry Age-Related Macular Degeneration (AMD) With Geographic Atrophy (GA)Geographic Atrophy, eye disorder, macular degeneration, retinal degeneration, retinal disorderActive, not recruitingPhase 32026-10-06NCT06510816
A Study of AAVB-039 in Participants With Stargardt Disease (STGD1)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Hereditary, Stargardt disease, eye disorder, hereditary disease, macular degeneration, retinal degeneration, retinal disorderRecruitingPhase 1 / Phase 22026-09-30NCT07161544
A Study to Test Different Doses of BI 1584862 in People With a Distinct Form of Age-related Macular Degeneration (AMD) Called Geographic AtrophyGeographic Atrophy, eye disorder, macular degeneration, retinal degeneration, retinal disorderActive, not recruitingPhase 22026-09-30NCT06769048
ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisiseye disorder, retinal degeneration, retinal disorder, retinoschisisRecruitingPhase 32026-09-29NCT05878860
Safety and Tolerability of Subretinally Injected OPGx-BEST1 in Patients With Best Vitelliform Macular Dystrophy (BVMD) or Autosomal-Recessive Bestrophinopathy (ARB)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Hereditary, autosomal recessive bestrophinopathy, eye disorder, hereditary disease, macular degeneration, retinal degeneration, retinal disorder, vitelliform macular dystrophy, vitelliform macular dystrophy 2RecruitingPhase 1 / Phase 22026-09-24NCT07185256
Study of ALK-001 on the Progression of Stargardt DiseaseCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Hereditary, Stargardt disease, eye disorder, hereditary disease, macular degeneration, retinal degeneration, retinal disorderRecruitingPhase 32026-09-18NCT07419334
A Study Investigating Subcutaneously Administered Pozelimab in Combination With Cemdisiran or Cemdisiran Alone in Adult Participants With Geographic AtrophyGeographic Atrophy, eye disorder, macular degeneration, retinal degeneration, retinal disorderRecruitingPhase 32026-09-18NCT06541704
An Observational Study of the Progression of Intermediate Age-Related Macular Degenerationage-related macular degeneration, eye disorder, macular degeneration, retinal degeneration, retinal disorderActive, not recruitingNot stated2026-09-16NCT05300724
Universal Rare Gene Study: A Registry and Natural History Study of Retinal Dystrophies Associated With Rare Disease-Causing Genetic VariantsCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Hereditary, eye disorder, hereditary disease, inherited retinal dystrophy, retinal degeneration, retinal disorder, retinitis pigmentosaRecruitingNot stated2026-09-08NCT05589714
A Study of the Safety and Tolerability of ASP7317 in Senior Adults Who Are Losing Their Clear, Sharp Central Vision Due to Geographic Atrophy Secondary to Dry Age-related Macular DegenerationGeographic Atrophy, age-related macular degeneration, eye disorder, macular degeneration, retinal degeneration, retinal disorderRecruitingPhase 12026-09-03NCT03178149
A Study to Learn How Stargardt-type Eye Conditions Progress in Children and AdultsCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Hereditary, Stargardt disease, eye disorder, hereditary disease, macular degeneration, retinal degeneration, retinal disorderRecruitingNot stated2026-08-25NCT07425574
Binocular iPad Sub-StudyNeurologic Manifestations, Pathological Conditions, Signs and Symptoms, Sensation Disorders, Signs and Symptoms, amblyopia, brain disorder, central nervous system disorder, eye disorder, nervous system disorder, vision disorderActive, not recruitingNot applicable2026-08-25NCT02365090
A Study to Investigate the Safety of OpCT-001 in Adults Who Have Primary Photoreceptor Disease (CLARICO)Abnormalities, Multiple, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Deaf-Blind Disorders, Deafness, Ear Diseases, Eye Diseases, Hereditary, Hearing Loss, Sensorineural, Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Rod-Cone Dystrophy, Sensation Disorders, Signs and Symptoms, Usher syndrome, blindness (disorder), cone-rod dystrophy, eye disorder, hearing disorder, hearing loss disorder, hereditary disease, inherited retinal dystrophy, nervous system disorder, otorhinolaryngologic disease, retinal degeneration, retinal disorder, retinitis pigmentosa, vision disorderRecruitingPhase 1 / Phase 22026-08-19NCT06789445
Intravitreal (IVT) Pozelimab for Geographic Atrophy (GA) in Adult ParticipantsGeographic Atrophy, eye disorder, macular degeneration, retinal degeneration, retinal disorderRecruitingPhase 12026-08-05NCT07230834
A Phase 3 Study Of OCU400 Gene Therapy for the Treatment Of Retinitis PigmentosaCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Hereditary, eye disorder, hereditary disease, inherited retinal dystrophy, retinal degeneration, retinal disorder, retinitis pigmentosaActive, not recruitingPhase 32026-07-31NCT06388200
A Phase 2/3 Trial to Assess the Efficacy and Safety of OCU410ST for Stargardt DiseaseCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Hereditary, Stargardt disease, eye disorder, hereditary disease, macular degeneration, retinal degeneration, retinal disorderActive, not recruitingPhase 2 / Phase 32026-07-31NCT05956626
Rate of Progression in USH2A-related Retinal DegenerationUsher syndrome type 2A, retinitis pigmentosa 39Active, not recruitingNot stated2026-07-23NCT03146078
Study to Evaluate Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A GeneAbnormalities, Multiple, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Deaf-Blind Disorders, Deafness, Ear Diseases, Eye Abnormalities, Eye Diseases, Hereditary, Hearing Loss, Sensorineural, Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Sensation Disorders, Signs and Symptoms, Usher syndrome, Usher syndrome type 2, blindness (disorder), eye disorder, hearing disorder, hearing loss disorder, hereditary disease, inherited retinal dystrophy, nervous system disorder, otorhinolaryngologic disease, retinal degeneration, retinal disorder, retinitis pigmentosa, vision disorderActive, not recruitingPhase 22026-07-09NCT06627179
A Phase 2 Open-label Study to Evaluate the Safety of Laruparetigene Zovaparvovec Administered Bilaterally in Male Participants With X-Linked Retinitis PigmentosaEye Diseases, Hereditary, eye disorder, retinal degeneration, retinal disorderActive, not recruitingPhase 22026-06-25NCT07174726
An Observational Study in Children and Adults With Stargardt DiseaseCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Hereditary, Stargardt disease, eye disorder, hereditary disease, macular degeneration, retinal degeneration, retinal disorderActive, not recruitingNot stated2026-06-16NCT06591806
Oral N-acetylcysteine for Retinitis PigmentosaAbnormalities, Multiple, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Deaf-Blind Disorders, Deafness, Ear Diseases, Eye Diseases, Hereditary, Hearing Loss, Sensorineural, Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Sensation Disorders, Signs and Symptoms, Usher syndrome, blindness (disorder), eye disorder, hearing disorder, hearing loss disorder, hereditary disease, inherited retinal dystrophy, nervous system disorder, otorhinolaryngologic disease, retinal degeneration, retinal disorder, retinitis pigmentosa, vision disorderActive, not recruitingPhase 32026-06-15NCT05537220
A Repeat-Dose, Open-Label, Two Arm Safety and Efficacy Study of Two Doses of VP-001 Administered Intravitreally in Participants With Confirmed PRPF31 Mutation-Associated Retinal Dystrophy, Including Participants Previously Treated With VP001Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Hereditary, eye disorder, hereditary disease, inherited retinal dystrophy, retinal degeneration, retinal disorder, retinitis pigmentosa 11Active, not recruitingPhase 1 / Phase 22026-05-29NCT06852963
Promising ROd-cone DYstrophy Gene therapYCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Hereditary, cone-rod dystrophy, eye disorder, hereditary disease, inherited retinal dystrophy, retinal degeneration, retinal disorder, retinitis pigmentosaActive, not recruitingPhase 1 / Phase 22026-05-15NCT05748873