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Phase IIb intrathecal HGT-1410 in pediatric early-stage MPS IIIA
2013-003450-24Phase 2Completed
- Sponsor
- Takeda
- Start
- Nov 2013
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
- Countries
- 1
01
Overview
Phase 2, randomised, open-label trial of recombinant human heparan-N-sulfatase in mucopolysaccharidosis type III A (2 arms), sponsored by Takeda. Primary endpoint: Cognitive developmental quotient response over 48 weeks. Started Nov 2013; status: completed.
02
Drugs
recombinant human heparan-N-sulfatase
03
Conditions
mucopolysaccharidosis type III A
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