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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
A Study to Evaluate the Safety and Tolerability of QR-1123 in Subjects With Autosomal Dominant Retinitis Pigmentosa Due to the P23H Mutation in the RHO GeneCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Diseases, Hereditary, Neurologic Manifestations, Pathological Conditions, Signs and Symptoms, Sensation Disorders, Signs and Symptoms, congenital stationary night blindness autosomal dominant 1, contractures, pterygia, and spondylocarpotarsal fusion syndrome 1A, eye disorder, hereditary disease, inherited retinal dystrophy, nervous system disorder, retinal degeneration, retinal disorder, retinitis, retinitis pigmentosa, vision disorderActive, not recruitingPhase 1 / Phase 22022-05-06NCT04123626
A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Eye Abnormalities, Eye Diseases, Hereditary, Leber congenital amaurosis, Leber congenital amaurosis 10, Leber hereditary optic neuropathy, Meckel syndrome, type 4, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pathological Conditions, Signs and Symptoms, Sensation Disorders, Signs and Symptoms, blindness (disorder), cranial nerve neuropathy, eye disorder, hereditary disease, hereditary optic atrophy, inborn mitochondrial metabolism disorder, inherited neurodegenerative disorder, metabolic disease, nervous system disorder, neurodegenerative disease, optic atrophy, optic nerve disorder, retinal disorder, vision disorderActive, not recruitingPhase 2 / Phase 32022-03-17NCT03913143