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)

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).

Study Overview

• Status: Recruiting
• Conditions: Sanfilippo Syndrome Type A
• Intervention / Treatment: Drug: GC1130A

• Study Type: Interventional
• Enrollment (Estimated): 9
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: GC Biopharma Corp.; Phone Number: +82312609300; Email: mps3a_clinicaltrial@gccorp.com

Study Locations

• Japan
  • Tokyo
    • Setagaya City, Tokyo, Japan
      • Recruiting
      • National Center for Child Health and Development
      • Principal Investigator: Motomichi Kosuga, MD
• South Korea
  • Seoul, South Korea
    • Recruiting
    • Samsung Medical Center
    • Principal Investigator: Sung Yoon Cho, MD
    • Contact: Minji Im, MD; Phone Number: +82234103757; Email: mj33.im@samsung.com
  • Gyeongi-do
    • Suwon, Gyeongi-do, South Korea
      • Recruiting
      • Ajou University Medical Center
      • Contact: Bora Lim; Phone Number: +82312194404; Email: 115694@aumc.ac.kr
      • Principal Investigator: Young Bae Sohn, MD
• United States
  • California
    • Oakland, California, United States, 94609
      • Recruiting
      • UCSF Benioff Children's Hospital
      • Principal Investigator: Paul Harmatz, MD
      • Contact: Mariam Samara; Phone Number: 4785 510-428-3885; Email: Mariam.Samara@ucsf.edu
  • Minnesota
    • Minneapolis, Minnesota, United States, 55455
      • Recruiting
      • University of Minnesota
      • Contact: Melissa Fetterley; Phone Number: 612-672-5151; Email: Melissa.Fetterley@fairview.org
      • Contact: Brenda Diethelm-Okita; Phone Number: 612-625-1594; Email: dieth001@umn.edu
      • Principal Investigator: Chester Whitley, MD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Cohort 1	Drug: GC1130A; ICV injection
Experimental: Cohort 2	Drug: GC1130A; ICV injection
Experimental: Cohort 3	Drug: GC1130A; ICV injection

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Incidences and characteristics of adverse events	up to 108 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Maximum concentration in cerebrospinal fluid (CSF)	GC1130A PK parameters	up to 104 weeks
Area under the concentration-time curve in CSF	GC1130A PK parameters	up to 104 weeks
Maximum concentration in serum	GC1130A PK parameters	up to 104 weeks
Area under the concentration-time curve in serum	GC1130A PK parameters	up to 104 weeks
Change from baseline in CSF heparan sulfate concentration		up to 104 weeks
Change from baseline in serum heparan sulfate concentration		up to 104 weeks
Incidence of anti-drug and neutralizing antibodies of GC1130A in CSF		up to 104 weeks
Incidence of anti-drug and neutralizing antibodies of GC1130A in serum		up to 104 weeks
Change from baseline in raw scores in cognitive domain of Bayley Scales of Infant and Toddler Development Scores-3rd edition (BSID-III)		up to 104 weeks

Collaborators and Investigators

• Sponsor: GC Biopharma Corp
• Collaborators: Novel Pharma Inc.

Study record dates

Study Major Dates

• Study Start (Actual): November 21, 2024
• Primary Completion (Estimated): June 1, 2027
• Study Completion (Estimated): June 1, 2027

Study Registration Dates

• First Submitted: August 20, 2024
• First Submitted That Met QC Criteria: August 20, 2024
• First Posted (Actual): August 23, 2024

Study Record Updates

• Last Update Posted (Estimated): August 28, 2025
• Last Update Submitted That Met QC Criteria: August 21, 2025
• Last Verified: August 1, 2025

More Information

Terms related to this study

• Keywords: MPS IIIA; Mucopolysaccharidosis Type III Subtype A
• Additional Relevant MeSH Terms: Metabolism, Inborn Errors; Genetic Diseases, Inborn; Metabolic Diseases; Connective Tissue Diseases; Carbohydrate Metabolism, Inborn Errors; Lysosomal Storage Diseases; Mucinoses; Mucopolysaccharidoses; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Nutritional and Metabolic Diseases; Skin and Connective Tissue Diseases; Mucopolysaccharidosis III
• Other Study ID Numbers: GC1130_MPS3A_P0101

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No