Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)
Recruiting
Phase 1
Interventional Study
Sanfilippo Syndrome Type A
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
At a Glance
- Age
- 12 Months – 18
- Sex
- Any
- Trial phase
- Phase 1
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 9 (estimated)
- Sponsor
- GC Biopharma Corp · Industry
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About This Trial
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).
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
* Participants with documented MPS IIIA diagnosis
* Participants aged ≥ 12 months and ≤ 18 years
Exclusion Criteria:
* Participants with significant non-MPS IIIA related central nervous system impairment
* Participants with previous complication from intraventricular drug admi…
Contacts