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Open-label extension of intrathecal rhHNS for Sanfilippo syndrome type A
NL-OMON45126Phase 2Active, not recruiting
- Sponsor
- Takeda
- Start
- Mar 2011
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
- Countries
- 1
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Overview
Phase 2, open-label, single-arm trial of recombinant human heparan-N-sulfatase in mucopolysaccharidosis type III A, sponsored by Takeda. Primary endpoints: Long-term safety of intrathecal rhHNS and Safety, tolerability, and patency of implanted intrathecal drug delivery device. Started Mar 2011; status: active, not recruiting.
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Drugs
recombinant human heparan-N-sulfatase
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Conditions
mucopolysaccharidosis type III A
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