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Clinical trial

Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA

NCT02716246 is registered in ClinicalTrials.gov with Ultragenyx Pharmaceutical Inc as lead sponsor.

NCT NCT02716246
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Registry record

As filed in ClinicalTrials.gov.

Title
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA
Lead sponsor
Ultragenyx Pharmaceutical Inc
Phase
Phase 2/3
Overall status
Recruiting
Start date
April 25, 2016
Completion date
March 31, 2029
Enrollment
36 (estimated)
Sites
5

Conditions

As the registry lists them; a condition with its own page is linked.

mps iiia, mucopolysaccharidosis iii, sanfilippo a, sanfilippo syndrome