Home›Trial›NCT02716246
🔬NCT02716246Phase 2Phase 3RECRUITING

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

Study Summary

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

Conditions
MPS IIIASanfilippo SyndromeSanfilippo AMucopolysaccharidosis III
Interventions / Drugs
UX111Prophylactic Immunomodulatory (IM) TherapyOptimized Prophylactic IM TherapyAdjuvant IM Therapy
View on ClinicalTrials.gov ↗
Sponsor
RARE
Ultragenyx Pharmaceutical
Rare Disease
C63/100
Study Details
Enrollment36 participants
Primary Completion—
Lead SponsorUltragenyx Pharmaceutical Inc
Data Retrieved2026-04-11 04:25:35.045817+00:00
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