Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR-TIGET)

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Trial	Conditions	Status	Phase	Updated	More info	NCT ID
A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome	Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cytopenia, Hemic and Lymphatic Diseases, Primary Immunodeficiency Diseases, Wiskott-Aldrich syndrome, X-linked disease, blood coagulation disease, hematologic disorder, hemorrhagic disease, hereditary disease, immune system disorder, inborn error of immunity, inherited blood coagulation disorder, leukocyte disorder, leukopenia, lymphopenia	Active, not recruiting	Phase 3	2025-09-29		NCT03837483
OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD)	Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hereditary Central Nervous System Demyelinating Diseases, Leukoencephalopathies, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, Sulfatidosis, brain disorder, central nervous system disorder, demyelinating disease, hereditary disease, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, metachromatic leukodystrophy, nervous system disorder, sphingolipidosis	Active, not recruiting	Phase 3	2025-09-05		NCT04283227

Clinical trials