Page last updated: July 29, 2025
Page reviewed by: Dr. Cara O’Neill, FAAP
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Clinical Trial Summary
Study: Phase 1 Study of GC1130A in Pediatric Patients With Sanfilippo Syndrome Type A (MPS IIIA)
Trial Listing: Read this clinical trial’s information on ClinicalTrials.gov, for more details including contact information to reach out to the study sites, full inclusion and exclusion criteria. We encourage you to check the ClinicalTrials.gov link frequently for updates on possible new study sites and changes in enrollment status or criteria.
Study opened: November 2024 began enrolling
Study design: A Phase 1, Open-label, Ascending Dose Study
Number of participants: 9 (estimated), ages 12 months to 18 years old
Study status: Recruiting
Route of administration: ICV ERT (intracerebral ventricular enzyme replacement therapy)
Study length: 2 years
Study sites: USA (University of Minnesota and UCSF Benioff Children’s Hospital in Oakland, CA); Korea (Samsung Medical Center in Seoul-si and Ajou University Medical Center in Suwon-si, Gyeongi-do); and Japan (National Center for Child Health and Development in Setagaya, Tokyo)
Brief Summary of Trial
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 pediatric patients with Sanfilippo Syndrome Type A (MPS IIIA).
You may also find information about this and other clinical trials for Sanfilippo Syndrome in the downloadable .PDF created by Cure Sanfilippo Foundation. See the link below to access.
Trial Updates & Information
February 2025
GC Biopharma shares research updates on rare disorder drugs at the WORLDSymposium 2025. Read more.
November 2024
GC Biopharma announced that the first patient in the U.S. has been dosed in multinational clinical trial with GC1130A for Sanfillippo syndrome type A (MPS IIIA). Read the announcement.
Early November 2024
As anticipated from the presentation during Cure Sanfilippo Foundation’s ADVANCE 2024 conference, the clinical trial of GC1130A enzyme replacement therapy for Sanfilippo Type A is NOW LISTED as “RECRUITING” on ClinicalTrials.gov.
August 2024
The clinical trial was published on ClinicalTrials.gov on Aug. 23, 2024.
In August 2023, Korea Biomedical Review published the following article, “GC Biopharma starts process for Sanfilippo syndrome treatment’s Japanese clinical trial.”
June 2024
GC1130A receives Fast Track Designation by the FDA. This designation allows for more frequent interactions between the company and FDA to streamline drug development for serious conditions with unmet treatment need.
Earlier regulatory milestones include:
- 2024: European Medicines Agency (EMA) granted GC1130A Orphan Drug Disease status.
- 2023: FDA granted GC1130A Rare Pediatric Disease Designation (RPDD) and Orphan Drug Disease (ODD)
May 2024
In May 2024, GC Biopharma/Novel Pharma issued a press release announcing its Sanfilippo Syndrome treatment had obtained FDA IND Approval.
August 2023
SooKyung Shin, R. Ph, M.S, and Dong-Kyu Jin, MD, PhD, of GC Biopharma/Novel Pharma presented to the community during Cure Sanfilippo Foundation’s ADVANCE 2023 conference. Their presentation can be viewed below.