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

Sex: All

Ages: 12 Months – 18 Years

Healthy volunteers: No

Phase: PHASE1

Enrollment: 9

Sponsor: GC Biopharma Corp

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 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 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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View on ClinicalTrials.gov

Source: ClinicalTrials.gov (NCT06567769). StuddyBuddy aggregates publicly available trial information.