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Immune modulation with methotrexate to reduce alloimmune response to laronidase in infants with severe MPS I before HSCT
2015-003031-35Phase 3Terminated
- Sponsor
- Manchester University NHS Foundation Trust
- Start
- Oct 2015
- Modality
- Orthosteric Ligands → Classical Binding Small Molecules → Small Molecules, Metabolically Activated Prodrugs → Prodrugs/Conditional Activator Small Molecules → Small Molecules, Nucleic Acid-Directed Small Molecules → Small Molecules
- Countries
- 1
01
Overview
Phase 3, open-label, single-arm trial of methotrexate in mucopolysaccharidosis type I, sponsored by Manchester University NHS Foundation Trust. Primary endpoint: Peak anti-laronidase IgG titre. Started Oct 2015; status: terminated.
02
Drugs
methotrexate
03
Conditions
mucopolysaccharidosis type I
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