Status: Recruiting
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
The main objective of this study is to evaluate the efficacy and safety of UX111 for the treatment of MPS IIIA.
Participants needed: 36
Trial details
Phase: Phase 2, Phase 3Biological sex: AllType: InterventionalSponsor: Ultragenyx Pharmaceutical IncUpdated: Jun 11, 2026Locations: 5
Eligibility criteria
No detectable or significantly reduced SGSH enzyme activity by leukocyte assay,... [+4]
Inability to participate in the clinical evaluation as determined by PI [+29]