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Phase I/II trial of idursulfase beta (BHP001) in mucopolysaccharidosis type II
jRCT1091220350Phase 2Completed
- Sponsor
- Torayuki Okuyama
- Start
- Jun 2018
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of Idursulfase beta in mucopolysaccharidosis type 2, sponsored by Torayuki Okuyama. Primary endpoint: Heparan sulfate concentration in cerebrospinal fluid. Started Jun 2018; status: completed.
02
Drugs
Idursulfase beta
03
Conditions
mucopolysaccharidosis type 2
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