Evaluating a new treatment for children with Sanfilippo Syndrome Type A
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)
This study is testing a new drug for young children with Sanfilippo Syndrome Type A to see if it is safe and helps improve their condition.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 9 (estimated) |
| Ages | 12 Months to 18 Years |
| Sex | All |
| Sponsor | GC Biopharma Corp Industry-sponsored |
| Locations | 5 sites (Oakland, California and 4 other locations) |
| Trial ID | NCT06567769 on ClinicalTrials.gov |
What this trial studies
This study aims to assess the safety, tolerability, efficacy, pharmacokinetics, and pharmacodynamics of a new drug called GC1130A, which is administered through an intracerebroventricular access device in pediatric patients diagnosed with Sanfilippo Syndrome Type A. The trial will involve children aged between 24 to 72 months who have a confirmed diagnosis of this rare genetic disorder. The study will monitor how well the drug works and its effects on the patients over time.
Who should consider this trial
Good fit: Ideal candidates for this study are children aged 2 to 6 years with a documented diagnosis of Sanfilippo Syndrome Type A.
Not a fit: Patients with significant non-MPS IIIA related central nervous system impairment or those who have received certain prior treatments may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly improve the quality of life and health outcomes for children suffering from Sanfilippo Syndrome Type A.
How similar studies have performed: While this approach is novel for Sanfilippo Syndrome Type A, similar studies in other mucopolysaccharidosis conditions have shown promising results.
Eligibility criteria
Show full inclusion / exclusion criteria
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
Where this trial is running
Oakland, California and 4 other locations
- UCSF Benioff Children's Hospital — Oakland, California, United States (Recruiting)
- University of Minnesota — Minneapolis, Minnesota, United States (Recruiting)
- National Center for Child Health and Development — Setagaya City, Tokyo, Japan (Recruiting)
- Ajou University Medical Center — Suwon, Gyeongi-do, South Korea (Recruiting)
- Samsung Medical Center — Seoul, South Korea (Recruiting)
Study contacts
- Study coordinator: GC Biopharma Corp.
- Email: mps3a_clinicaltrial@gccorp.com
- Phone: +82312609300
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.