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HGT-1410 intrathecal phase IIb study in pediatric early-stage MPS IIIA
NL-OMON40318Phase 2Active, not recruiting
- Sponsor
- Takeda
- Start
- Sep 2014
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
- Countries
- 1
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Overview
Phase 2, randomised, open-label trial of recombinant human heparan-N-sulfatase in mucopolysaccharidosis type III A (3 arms), sponsored by Takeda. Primary endpoint: Cognitive response over 48 weeks. Started Sep 2014; status: active, not recruiting.
02
Drugs
recombinant human heparan-N-sulfatase
03
Conditions
mucopolysaccharidosis type III A
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