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

NCT06567769 · Status: RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 9

Last updated 2025-08-28

No results posted yet for this 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).

Conditions

Interventions

DRUG

GC1130A

ICV injection

Sponsors & Collaborators

  • GC Biopharma Corp

    lead INDUSTRY

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
12 Months
Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-11-21
Primary Completion
2027-06-30
Completion
2027-06-30
FDA Drug
Yes

Countries

  • United States
  • Japan
  • South Korea

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06567769 on ClinicalTrials.gov