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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Long-term Follow-Up for RGX-121Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Death, Death, Sudden, Infant Death, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Skin and Connective Tissue Diseases, Sudden Infant Death, X-linked disease, connective tissue disorder, cutaneous mucinosis, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, inherited neurodegenerative disorder, intellectual disability, lysosomal storage disease, metabolic disease, mucopolysaccharidosis, mucopolysaccharidosis type 2, nervous system disorderActive, not recruitingNot stated2026-08-19NCT04597385
RGX-314 Gene Therapy Administered in the Suprachoroidal Space for Participants With Diabetic Retinopathy (DR) With and Without Center Involved-Diabetic Macular Edema (CI-DME)Diabetes Complications, cardiovascular disorder, diabetes mellitus, diabetic peripheral angiopathy, diabetic retinopathy, endocrine system disorder, eye disorder, retinal disorder, vascular disorderActive, not recruitingPhase 22026-08-19NCT04567550
Long-Term Follow-Up Study of RGX-314 Administered in the Suprachoroidal Space for Participants With and Without Diabetic RetinopathyDiabetes Complications, cardiovascular disorder, diabetes mellitus, diabetic peripheral angiopathy, diabetic retinopathy, endocrine system disorder, eye disorder, retinal disorder, vascular disorderEnrolling by invitationPhase 22026-08-05NCT05296447
Long-Term Follow-Up Study of RGX-314 Administered in the Suprachoroidal Space for Participants With nAMDNeovascular Age-Related Macular Degeneration (nAMD)Enrolling by invitationPhase 22026-08-05NCT05210803
Pivotal 2 Study of RGX-314 Gene Therapy in Participants With nAMDEye Diseases, Hereditary, Metaplasia, Neovascularization, Pathologic, Pathologic Processes, Pathological Conditions, Signs and Symptoms, age-related macular degeneration, choroidal neovascularization, eye disorder, macular degeneration, optic choroid disorder, retinal degeneration, retinal disorder, uveal disorder, wet macular degenerationActive, not recruitingPhase 32026-08-03NCT05407636
AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne muscular dystrophy, Muscular Disorders, Atrophic, X-linked disease, hereditary disease, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neuromuscular diseaseActive, not recruitingPhase 2 / Phase 32026-07-21NCT05693142
AFFINITY BEYOND: Anti-AAV8 Antibody Assessment Study of Males With DMDCongenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne muscular dystrophy, Muscular Disorders, Atrophic, X-linked disease, hereditary disease, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neuromuscular diseaseActive, not recruitingNot stated2026-07-07NCT05683379
RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Death, Death, Sudden, Infant Death, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Skin and Connective Tissue Diseases, Sudden Infant Death, X-linked disease, connective tissue disorder, cutaneous mucinosis, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, inherited neurodegenerative disorder, intellectual disability, lysosomal storage disease, metabolic disease, mucopolysaccharidosis, mucopolysaccharidosis type 2, nervous system disorderActive, not recruitingPhase 32026-06-04NCT07236606
Long Term Follow-Up for RGX-111Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Mucopolysaccharidosis I, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, connective tissue disorder, cutaneous mucinosis, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal storage disease, metabolic disease, mucopolysaccharidosisEnrolling by invitationNot stated2026-04-29NCT06103487
Pivotal 1 Study of ABBV-RGX-314 (Also Known as RGX-314) Gene Therapy Administered Via Subretinal Delivery One Time in Participants With nAMDEye Diseases, Hereditary, Metaplasia, Neovascularization, Pathologic, Pathologic Processes, Pathological Conditions, Signs and Symptoms, age-related macular degeneration, choroidal neovascularization, eye disorder, macular degeneration, optic choroid disorder, retinal degeneration, retinal disorder, uveal disorder, wet macular degenerationActive, not recruitingPhase 2 / Phase 32026-04-28NCT04704921
Long Term Follow-up for RGX-202Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne muscular dystrophy, Muscular Disorders, Atrophic, X-linked disease, hereditary disease, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neuromuscular diseaseEnrolling by invitationNot stated2026-03-24NCT06491927
RGX-314 Gene Therapy Administered in the Suprachoroidal Space for Participants With Neovascular Age-Related Macular Degeneration (nAMD)Neovascular Age-Related Macular Degeneration (nAMD)Active, not recruitingPhase 22026-02-27NCT04514653
Phase 2 Trial of RGX-314 in Adults With Center Involved - Diabetic Macular Edema (CI - DME)diabetic macular edemaRecruitingPhase 22025-04-24NCT06942520
CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Death, Death, Sudden, Infant Death, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Skin and Connective Tissue Diseases, Sudden Infant Death, X-linked disease, connective tissue disorder, cutaneous mucinosis, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, inherited neurodegenerative disorder, intellectual disability, lysosomal storage disease, metabolic disease, mucopolysaccharidosis, mucopolysaccharidosis type 2, nervous system disorderActive, not recruitingPhase 2 / Phase 32025-01-28NCT03566043