A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT
A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT
Active, not recruiting · NCT06149403 · Registry updated 2025-12-22Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Mucopolysaccharidosis I, 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 | Active, not recruiting | Phase 3 | 2025-12-22 | NCT06149403 |
1 results · Page 1 of 1
Organizations
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| Organizations | Linked trials |
|---|---|
| Orchard Therapeutics | 5 |
5 results · Page 1 of 1
Research sites
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| Research sites | City | Country | Linked trials |
|---|---|---|---|
| Manchester University NHS Foundation Trust Blood and Marrow Transplant Programme, Royal Manchester Children's Hospital | Manchester | United Kingdom | 1 |
| Ospedale San Raffaele | Milan | Italy | 1 |
| Princess Maxima Center | Utrecht | Netherlands | 1 |
| UMC Utrecht | Utrecht | Netherlands | 30 |
| University of Minnesota, Pediatrics | Minneapolis | United States | 1 |