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Clinical trial

A Phase I/II Open Label, Single-dose, Gene Transfer Study of scAAV9.U1a.hSGSH (ABO-102) in Patients With Middle and Advanced Phases of MPS IIIA Disease

NCT04088734 is registered in ClinicalTrials.gov with Ultragenyx Pharmaceutical Inc as lead sponsor.

NCT NCT04088734
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Registry record

As filed in ClinicalTrials.gov.

Title
A Phase I/II Open Label, Single-dose, Gene Transfer Study of scAAV9.U1a.hSGSH (ABO-102) in Patients With Middle and Advanced Phases of MPS IIIA Disease
Lead sponsor
Ultragenyx Pharmaceutical Inc
Phase
Phase 1/2
Overall status
Terminated
Start date
September 18, 2019
Completion date
March 10, 2022
Enrollment
5 (actual)
Sites
3

Conditions

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

mps iiia, mucopolysaccharidosis iii, sanfilippo a, sanfilippo syndrome