Health Trials

Sanfilippo Syndrome Type A

1

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

Condition / disease
Location
Status: Recruiting

Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)

The purpose of this study is to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of recombinant human heparan N-sulfatase (rhHNS, GC1130A) administered via intracerebroventricular access device in patients with Sanfilippo Syndrome Type A (MPS IIIA).

Participants needed: 9
Trial details
Phase: Phase 1Age: 12-18Biological sex: AllType: InterventionalSponsor: GC Biopharma CorpUpdated: Aug 28, 2025Locations: 5
Eligibility criteria

Participants with documented MPS IIIA diagnosis [+1]

Participants with significant non-MPS IIIA related central nervous system impair... [+4]

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