Clinical Trial

Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH

Name: Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH — clinical trial record (NCT02716246)
Creator: DataLookout
License: https://creativecommons.org/licenses/by/4.0/

Recruiting Phase 2/3

View on ClinicalTrials.gov →

Summary

The main objective of this study is to evaluate the efficacy and safety of UX111 for the treatment of MPS IIIA.

Protocol Amendment History 2 changes

notable Primary completion pushed: 2027-07 -> 2029-03 2026-06-12

minor Completion pushed: 2027-07 -> 2029-03 2026-06-12

Trial Details

NCT Number	NCT02716246
Lead Sponsor	Ultragenyx Pharmaceutical Inc Collaborators: Abeona Therapeutics, Inc
Conditions	MPS IIIA, Sanfilippo Syndrome, Sanfilippo A, Mucopolysaccharidosis III
Enrollment	36 participants
Start Date	2016-04-25
Primary Completion	2029-03 (estimated)
Study Completion	2029-03 (estimated)
Updated on ClinicalTrials.gov	2026-07-17