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Intrathecal rhHNS dose-escalation study in Sanfilippo syndrome type A
NL-OMON36295Phase 2Active, not recruiting
- Sponsor
- Takeda
- Start
- Sep 2010
- 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 endpoint: Safety of intrathecal rhHNS administration. Started Sep 2010; 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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