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Trial intelligence / Profile preview
OTL-203 versus standard-of-care allo-HSCT in Hurler syndrome
NL-OMON59828Phase 3Active, not recruiting
- Sponsor
- Orchard Therapeutics
- Start
- Sep 2024
- Modality
- Bone Marrow HSCs → Hematopoietic Stem Cells → Adult Stem Cells → Stem Cell Therapies → Cell Therapies, Peripheral Blood HSCs → Hematopoietic Stem Cells → Adult Stem Cells → Stem Cell Therapies → Cell Therapies, Lentiviral Vectors → Retroviral Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Countries
- 1
01
Overview
Phase 3, open-label trial of OTL-203 in mucopolysaccharidosis type I (2 arms), sponsored by Orchard Therapeutics. Primary endpoint: Event-free survival at Year 2. Started Sep 2024; status: active, not recruiting.
02
Drugs
OTL-203
03
Conditions
mucopolysaccharidosis type I
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