Fundación Cardio Infantil - Instituto de Cardiología

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Trial	Conditions	Status	Phase	Updated	More info	NCT ID
A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT)	Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Skin and Connective Tissue Diseases, 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 disorder	Active, not recruiting	Phase 3	2026-10-02		NCT04573023
Safety, Tolerability and Efficacy of ICV AX 250 Treatment in MPS IIIB -OLE	Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Mucopolysaccharidosis III, 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, mucopolysaccharidosis, mucopolysaccharidosis type 3B	Enrolling by invitation	Phase 4	2023-08-09		NCT05492799

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