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Open-label extension of intrathecal HGT-1410 in pediatric MPS IIIA
NL-OMON45009Phase 2Active, not recruiting
- Sponsor
- Takeda
- Start
- Sep 2015
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
- Countries
- 1
01
Overview
Phase 2, open-label trial of recombinant human heparan-N-sulfatase in mucopolysaccharidosis type III A (2 arms), sponsored by Takeda. Primary endpoint: Long-term safety. Started Sep 2015; status: active, not recruiting.
02
Drugs
recombinant human heparan-N-sulfatase
03
Conditions
mucopolysaccharidosis type III A
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