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Clinical Trials in the USA / NCT06567769
Active, not recruiting Phase 1

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

NCT06567769 · tracked via the Priya Life Science USA tracker
Sponsor
GC Biopharma Corp
Phase
Phase 1
Started
2024-11-21
Last updated
2026-09-08

Condition(s) studied

Sanfilippo Syndrome Type A

Investigational drug(s) / intervention(s)

GC1130A

GC1130A: ICV injection

Study 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

Sex
ALL
Min age
12 Months
Max age
18 Years
Healthy volunteers
No
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

Primary outcome measure(s)

Trial sites (4)

FacilityCityRegionStatus
UCSF Benioff Children's Hospital Oakland California
University of Minnesota Minneapolis Minnesota
Ajou University Medical Center Suwon Gyeongi-do
Samsung Medical Center Seoul South Korea

More GC Biopharma Corp trials in the USA

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06567769 on ClinicalTrials.gov ↗ ← All trials in the USA