Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)
A Phase 1, Open-label, Ascending Dose Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics and Pharmacodynamics of Recombinant Human Heparan N-Sulfatase (rhHNS, GC1130A) Via Intracerebroventricular Access Device in Patients With Sanfilippo Syndrome Type A (MPS IIIA)
GC Biopharma Corp
9 participants
Nov 21, 2024
INTERVENTIONAL
Conditions
Summary
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).
Eligibility
Inclusion Criteria2
- Participants with documented MPS IIIA diagnosis
- Participants aged ≥ 12 months and ≤ 18 years
Exclusion Criteria5
- Participants with significant non-MPS IIIA related central nervous system impairment
- Participants with previous complication from intraventricular drug administration
- Participants with contraindications for MRI scans and for neurosurgery
- Participants that received treatment with any investigational drug or a device intended as a treatment for MPS IIIA within 30 days or 5 half-lives prior to the study
- Participants that received a hematopoietic stem cell or bone marrow transplant or received gene therapy
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Interventions
ICV injection
Locations(5)
View Full Details on ClinicalTrials.gov
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NCT06567769