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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
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
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
A 24-Month Trial of NPI-001 for the Preservation of Photoreceptors in Retinitis Pigmentosa Associated With Usher SyndromeAbnormalities, 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 disorderNot yet recruitingPhase 32026-07-17NCT07710196
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
24-Month Trial of NPI-001 for the Preservation of Photoreceptors in Retinitis Pigmentosa Associated With Usher SyndromeAbnormalities, 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 disorderNot yet recruitingPhase 32026-07-01NCT07290530
Stem Cell Ophthalmology Treatment Study IIAbnormalities, Multiple, CNS demyelinating autoimmune disease, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cranial Nerve Injuries, Craniocerebral Trauma, Deaf-Blind Disorders, Deafness, Doyne honeycomb retinal dystrophy, Ear Diseases, Eye Diseases, Hereditary, Geographic Atrophy, Hearing Loss, Sensorineural, Leber hereditary optic neuropathy, Neurologic Manifestations, Nutritional and Metabolic Diseases, Optic Atrophy, Autosomal Dominant, Optic Neuropathy, Ischemic, Pathological Conditions, Signs and Symptoms, Sensation Disorders, Signs and Symptoms, Stargardt disease, Usher syndrome, Vision, Low, acute transverse myelitis, age-related macular degeneration, autoimmune disease, autoimmune disorder of the nervous system, blindness (disorder), cardiovascular disorder, cone dystrophy, cone-rod dystrophy, cranial nerve neuropathy, demyelinating disease, eye disorder, glaucoma, hearing disorder, hearing loss disorder, hereditary disease, hereditary optic atrophy, immune system disorder, inborn mitochondrial metabolism disorder, inherited neurodegenerative disorder, inherited retinal dystrophy, injury, macular degeneration, metabolic disease, nervous system disorder, nervous system injury, neurodegenerative disease, neuromyelitis optica, nutritional deficiency disease, nutritional disorder, ocular hypertension, optic atrophy, optic nerve disorder, optic neuritis, otorhinolaryngologic disease, retinal degeneration, retinal disorder, retinitis pigmentosa, traumatic optic neuropathy, vascular disorder, vision disorder, vitamin A deficiency, vitamin deficiency disorder, wet macular degenerationRecruitingNot applicable2026-06-29NCT03011541
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
Inherited Retinal Degenerative Disease RegistryAbnormalities, Multiple, Bardet-Biedl syndrome, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Charcot-Marie-Tooth disease, Chronic Disease, Ciliopathies, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Deaf-Blind Disorders, Deafness, Disease Attributes, Dyslipidemias, Ear Diseases, Eye Diseases, Hereditary, Goldmann-Favre syndrome, Hearing Loss, Sensorineural, Juvenile Macular Degeneration, Kearns-Sayre syndrome, Leber congenital amaurosis, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Neurologic Manifestations, Neuronal Ceroid-Lipofuscinoses, Nutritional and Metabolic Diseases, Ocular Motility Disorders, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Peroxisomal Disorders, Rod-Cone Dystrophy, Sensation Disorders, Signs and Symptoms, Stargardt disease, Usher syndrome, X-linked disease, abetalipoproteinemia, achromatopsia, achromatopsia 3, adult Refsum disease, blindness (disorder), brain disorder, cardiomyopathy, cardiovascular disorder, central areolar choroidal dystrophy, central nervous system disorder, central nervous system malformation, choroideremia, color vision disorder, cone dystrophy, cone-rod dystrophy, congenital stationary night blindness, cranial nerve neuropathy, enhanced S-cone syndrome, eye disorder, fundus albipunctatus, hearing disorder, hearing loss disorder, heart disorder, hereditary disease, hereditary motor and sensory neuropathy, hypobetalipoproteinemia, hypolipoproteinemia, hypothalamic disorder, inborn errors of metabolism, inborn mitochondrial metabolism disorder, inborn mitochondrial myopathy, inherited neurodegenerative disorder, inherited retinal dystrophy, juvenile neuronal ceroid lipofuscinosis, lysosomal lipid storage disorder, macular degeneration, metabolic disease, muscle tissue disorder, musculoskeletal system disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, ophthalmoplegia, optic choroid disorder, ornithine aminotransferase deficiency, otorhinolaryngologic disease, palsy, peripheral nervous system disorder, polyneuropathy, progressive external ophthalmoplegia, retinal degeneration, retinal disorder, retinitis pigmentosa, retinitis punctata albescens, retinoschisis, uveal disorder, vision disorder, vitelliform macular dystrophyRecruitingNot stated2026-05-19NCT02435940
Adaptive Optics Imaging of Outer Retinal DiseasesAbnormalities, 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, age-related macular degeneration, blindness (disorder), cone dystrophy, cone-rod dystrophy, eye disorder, hearing disorder, hearing loss disorder, hereditary disease, inherited retinal dystrophy, late-onset retinal degeneration, macular degeneration, nervous system disorder, otorhinolaryngologic disease, retinal degeneration, retinal disorder, retinitis pigmentosa, vision disorderRecruitingNot stated2026-05-08NCT05355415
Observational Study to Investigate the Short-term Effects of Transcorneal Electrical Stimulation on Visual PerformanceAbnormalities, 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), 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 disorderRecruitingNot stated2026-05-01NCT07548944
Natural History of Photoreceptor Degeneration in USH1B: Clinical Parameters and Validation of Functional Vision Tests in MYO7AAbnormalities, 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 disorderRecruitingNot stated2026-02-10NCT07278843
A Study to Determine the Long-Term Safety, Tolerability and Biological Activity of SAR421869 in Patients With Usher Syndrome Type 1BAbnormalities, 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 22026-01-13NCT02065011
Study of Subretinally Injected AAVB-081 in Patients With Usher Syndrome Type IB (USH1B) 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, Usher syndrome type 1B, 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 disorderRecruitingPhase 1 / Phase 22025-05-02NCT06591793
Natural History Study of Usher Syndrome ( Light4Deaf )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, 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 disorderRecruitingNot stated2020-12-14NCT04665726