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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Prescreening Study to Identify Potential Stargardt Participants for ACDN-01 Clinical Trials (STARPATH)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Hereditary, Juvenile Macular Degeneration, Stargardt Disease 1, Stargardt disease, cone-rod dystrophy, eye disorder, hereditary disease, inherited retinal dystrophy, macular degeneration, retinal degeneration, retinal disorderRecruitingNot stated2026-10-09NCT06445322
Natural History Study of Advanced RCDCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Hereditary, Rod-Cone Dystrophy, cone-rod dystrophy, eye disorder, hereditary disease, inherited retinal dystrophy, retinal degeneration, retinal disorder, retinitis pigmentosaRecruitingNot applicable2026-10-08NCT07854899
Rod and Cone Mediated Function in Retinal DiseaseCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Hereditary, Stargardt disease, eye disorder, hereditary disease, inherited retinal dystrophy, macular degeneration, retinal degeneration, retinal disorder, retinitis pigmentosaRecruitingNot stated2026-10-08NCT02617966
Familial Mediterranean Fever and Related Disorders: Genetics and Disease CharacteristicsBody Temperature Changes, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Fever, Headache, Hypertrophy, Hypohidrosis, Neurologic Manifestations, Pain, Pathological Conditions, Anatomical, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, Skin and Connective Tissue Diseases, Splenomegaly, eye disorder, familial Mediterranean fever, hereditary disease, hereditary periodic fever syndrome, hereditary skin disorder, inherited retinal dystrophy, retinal degeneration, retinal disorder, retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis, and migraine headache syndrome, skin disorder, sweat gland disorderRecruitingNot stated2026-10-08NCT00001373
Safety Evaluation Study of Pitavastatin PLGA Nano-particle for Retinitis PigmentosaCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Hereditary, eye disorder, hereditary disease, inherited retinal dystrophy, retinal degeneration, retinal disorder, retinitis pigmentosaRecruitingPhase 12026-10-07NCT07774975
Study to Evaluate ACDN-01 in ABCA4-related Stargardt Retinopathy (STELLAR)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Hereditary, Juvenile Macular Degeneration, Stargardt Disease 1, Stargardt disease, cone-rod dystrophy, eye disorder, hereditary disease, inherited retinal dystrophy, macular degeneration, retinal degeneration, retinal disorderRecruitingPhase 1 / Phase 22026-10-07NCT06467344
Phase I/II Open-Label Trial of UGX202 for Advanced Retinitis PigmentosaCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Hereditary, eye disorder, hereditary disease, inherited retinal dystrophy, retinal degeneration, retinal disorder, retinitis pigmentosaNot yet recruitingPhase 12026-10-02NCT07847723
A Study of AAV5-hRKp.RPGR for the Treatment of Japanese Participants With X-linked 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 32026-09-25NCT05926583
A Phase 1/2 Study in Healthy Volunteers and Participants With Autosomal Dominant Retinitis Pigmentosa (RHO-adRP)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Hereditary, eye disorder, hereditary disease, inherited retinal dystrophy, retinal degeneration, retinal disorder, retinitis pigmentosa, retinitis pigmentosa 4RecruitingPhase 1 / Phase 22026-09-24NCT07408232
Oral Metformin for Treatment of ABCA4 RetinopathyCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Hereditary, Stargardt disease, eye disorder, hereditary disease, inherited retinal dystrophy, macular degeneration, retinal degeneration, retinal disorderActive, not recruitingPhase 1 / Phase 22026-09-24NCT04545736
Cell Collection to Study Eye DiseasesCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Hereditary, age-related macular degeneration, eye disorder, hereditary disease, inherited retinal dystrophy, late-onset retinal degeneration, macular degeneration, retinal degeneration, retinal disorder, retinitis pigmentosa, vitelliform macular dystrophyRecruitingNot stated2026-09-22NCT01432847
The Effects of Disulfiram (Antabuse®) on Visual Acuity in Patients With Retinal DegenerationAlcohol Use Disorder, Chemically-Induced Disorders, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Hereditary, Stargardt disease, age-related macular degeneration, alcohol-related disorders, eye disorder, hereditary disease, inherited retinal dystrophy, macular degeneration, psychiatric disorder, retinal degeneration, retinal disorder, retinitis pigmentosa, substance-related disorderRecruitingPhase 12026-09-16NCT06319872
Open-Label Study to Evaluate the Safety, Tolerability, and Efficacy of Gene Therapy (SPVN20) in Subjects With Rod-Cone DystrophyCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Hereditary, Rod-Cone Dystrophy, cone-rod dystrophy, eye disorder, hereditary disease, inherited retinal dystrophy, retinal degeneration, retinal disorder, retinitis pigmentosaRecruitingPhase 1 / Phase 22026-09-08NCT07807111
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
Study to Evaluate the Long-Term Safety of Ultevursen in Participants 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 disorderNot yet recruitingPhase 22026-09-01NCT07796646
Activating Spinal Circuits to Improve Walking, Balance, Strength, and Reduce SpasticityCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Hereditary, Muscle Hypertonia, Muscle Spasticity, Neurologic Manifestations, Neuromuscular Manifestations, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, cone-rod dystrophy, eye disorder, hereditary disease, inherited retinal dystrophy, muscle tissue disorder, musculoskeletal system disorder, nervous system disorder, retinal degeneration, retinal disorderActive, not recruitingNot applicable2026-09-01NCT05429736
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
Long-term Follow-up Study in Subjects Who Received Voretigene Neparvovec-rzyl (AAV2-hRPE65v2)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Hereditary, Leber congenital amaurosis, eye disorder, hereditary disease, inherited retinal dystrophy, retinal degeneration, retinal disorder, retinitis pigmentosaActive, not recruitingNot stated2026-08-19NCT03602820
Restoration of Central Vision With PRIMA in Patients With Photoreceptor DegenerationCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Hereditary, Stargardt disease, eye disorder, hereditary disease, inherited retinal dystrophy, macular degeneration, retinal degeneration, retinal disorder, retinitis pigmentosaRecruitingNot applicable2026-08-17NCT07266584
Virtual Reality Mobility Assessment of Functional Vision in Retinal DiseaseCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Hereditary, cone-rod dystrophy, eye disorder, hereditary disease, inherited retinal dystrophy, retinal degeneration, retinal disorderRecruitingPhase 12026-08-10NCT04289571
Global Registry and Natural History Study for Mitochondrial DisordersAbnormalities, Multiple, Barth syndrome, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cardiovascular Abnormalities, Cerebral Small Vessel Diseases, Chronic Disease, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Epilepsies, Myoclonic, Epilepsy, Generalized, Epileptic Syndromes, Kearns-Sayre syndrome, Leber hereditary optic neuropathy, Leigh syndrome, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, MELAS syndrome, MERRF syndrome, NARP syndrome, Neurologic Manifestations, Nutritional and Metabolic Diseases, Ocular Motility Disorders, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Pearson syndrome, Pyruvate Metabolism, Inborn Errors, Signs and Symptoms, X-linked disease, ataxia neuropathy spectrum, brain disorder, cardiomyopathy, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, coenzyme Q10 deficiency, congenital heart disease, cranial nerve neuropathy, epilepsy, eye disorder, heart disorder, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, inborn mitochondrial metabolism disorder, inborn mitochondrial myopathy, inherited retinal dystrophy, maternally-inherited diabetes and deafness, metabolic disease, mitochondrial encephalomyopathy, mitochondrial neurogastrointestinal encephalomyopathy, muscle tissue disorder, musculoskeletal system disorder, myelodysplastic syndrome, nervous system disorder, neuromuscular disease, ophthalmoplegia, palsy, progressive external ophthalmoplegia, progressive myoclonus epilepsy, retinal degeneration, retinal disorder, retinitis pigmentosa, spinocerebellar ataxia with epilepsy, vascular disorder, very long chain acyl-CoA dehydrogenase deficiencyRecruitingNot stated2026-08-07NCT05554835
FIGHT-RP 1 Extension StudyCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Hereditary, eye disorder, hereditary disease, inherited retinal dystrophy, retinal degeneration, retinal disorder, retinitis pigmentosaActive, not recruitingPhase 12026-08-07NCT03999021
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
Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital AmaurosisCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Hereditary, Leber congenital amaurosis, Meckel syndrome, type 4, congenital stationary night blindness autosomal dominant 1, enhanced S-cone syndrome, eye disorder, hereditary disease, inherited retinal dystrophy, retinal degeneration, retinal disorder, retinitis pigmentosaActive, not recruitingPhase 1 / Phase 22026-07-31NCT05203939
Safety of a Single, Intravitreal Injection of 6.0M jCell (Famzeretcel) in Retinitis Pigmentosa (RP)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Hereditary, eye disorder, hereditary disease, inherited retinal dystrophy, retinal degeneration, retinal disorder, retinitis pigmentosaActive, not recruitingPhase 22026-07-30NCT06912633