Spark Therapeutics, Inc. | ClinicalTrials HQ

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Trial	Conditions	Status	Phase	Updated	More info	NCT ID
Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2	Eye Diseases, Hereditary, Leber congenital amaurosis, eye disorder, retinal disorder	Active, not recruiting	Phase 1 / Phase 2	2025-04-29		NCT01208389
Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis	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 pigmentosa	Active, not recruiting	Phase 3	2025-04-23		NCT00999609
Long-Term Safety and Efficacy of Spark-Sponsored Gene Therapies in Males With Hemophilia A	Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, blood coagulation disease, coagulation protein disease, hematologic disorder, hemophilia A, hemorrhagic disease, hereditary disease, inherited blood coagulation disorder	Active, not recruiting	Not stated	2024-11-29		NCT03432520
A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)	Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, disorder of glycogen metabolism, glycogen storage disease II, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal storage disease, metabolic disease, nervous system disorder	Active, not recruiting	Phase 1 / Phase 2	2024-11-27		NCT04093349