NCT06567769

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)

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 9 (estimated)
• Sponsor: GC Biopharma Corp · Industry
• Sex: All
• Age: 12 Months – 18 Years
• Healthy volunteers: Not accepted

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).

Conditions

• Sanfilippo Syndrome Type A

Interventions

Type	Name	Description
DRUG	GC1130A	ICV injection

Timeline

• Start date: 2024-11-21
• Primary completion: 2027-06-01
• Completion: 2027-06-01
• First posted: 2024-08-23
• Last updated: 2025-08-28

Locations

5 sites across 3 countries: United States, Japan, South Korea

Regulatory

• FDA-regulated drug study