Health Trials

MPS IIIA

1

Review clinical trials related to MPS IIIA. Use filters to narrow results by trial status, phase, treatment, biological sex and sponsor.

Condition / disease
Location
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]

Read more