| Lenmeldy (atidarsagene autotemcel) / Kyowa Kirin |
2019-002636-82: A clinical trial to evaluate the effect of OTL-200, a gene therapy treatment in patients with Late Juvenile (LJ) Metachromatic Leukodystrophy (MLD) |
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| Not yet recruiting | 3 | 6 | Europe | OTL-200, Dispersion for infusion | Orchard Therapeutics (Europe) Limited, Orchard Therapeutics (Europe) Limited | Metachromatic Leukodystrophy, Metachromatic Leukodystrophy, is an inherited genetic disease affecting mainly babies and young children, and more rarely also affecting adolescents and adults., Body processes [G] - Genetic Phenomena [G05] | | | | |
| Active, not recruiting | 3 | 6 | Europe | OTL-200 | Orchard Therapeutics, Ospedale San Raffaele | Lysosomal Storage Diseases, Metachromatic Leukodystrophy | 03/25 | 03/31 | | |
| Active, not recruiting | 2 | 10 | Europe | OTL-200, Previously GSK2696274 | Orchard Therapeutics, Ospedale San Raffaele | Lysosomal Storage Disease, Metachromatic Leukodystrophy | 04/22 | 01/26 | | |
| Active, not recruiting | 1/2 | 20 | Europe | OTL-200 Gene Therapy, Previously GSK2696274 | Orchard Therapeutics, Ospedale San Raffaele | Lysosomal Storage Disease, Metachromatic Leukodystrophy | 04/18 | 03/25 | | |
NCT02559830: Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy |
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| Recruiting | 1/2 | 50 | RoW | transduced CD34+ hematopoietic stem cell | Shenzhen Second People's Hospital, Shenzhen University, Guangzhou Women and Children's Medical Center | Metachromatic Leukodystrophy, Adrenoleukodystrophy | 10/25 | 10/25 | | |
