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rhHNS in Pediatric Early Stage MPS IIIA
NCT02060526Phase 2Completed
- Sponsor
- Takeda
- Start
- Feb 2014
- Primary completion
- Jun 2016
- Modality
- Replacement Enzymes
- Countries
- 8
01
Overview
Phase 2, randomised, open-label trial of recombinant human heparan-N-sulfatase in Sanfilippo syndrome type A (3 arms), sponsored by Takeda. Primary endpoint: Number of Participants With Overall Response Using Bayley Scales of Infant Development Assessment Third Edition at Week 48. Started Feb 2014; status: completed.
02
Drugs
recombinant human heparan-N-sulfatase
03
Conditions
Sanfilippo syndrome type A
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