Health TrialsbyHealthPanel

Sanfilippo A

2

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

Condition / disease
Location
Status: Not yet recruiting

MPS IIIA Disease Monitoring Program

The main objective of this observational study is to evaluate the long-term safety and effectiveness of rebisufligene etisparvovec for 10 years after rebisufligene etisparvovec administration.

Participants needed: 55
Trial details
Biological sex: AllType: ObservationalSponsor: Ultragenyx Pharmaceutical IncUpdated: Oct 2, 2026
Eligibility criteria

Confirmed diagnosis of MPS IIIA [+3]

Presence or history of any condition that, in the view of the Investigator, woul...

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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: Sep 22, 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]

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