Phase 1 Study of GC1130A in Pediatric Patients With Sanfilippo Syndrome Type A (MPS IIIA)
- Registration Number
- NCT06567769
- Lead Sponsor
- GC Biopharma Corp
- Brief 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 pediatric patients with Sanfilippo Syndrome Type A (MPS IIIA).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 9
Inclusion Criteria
- Participants with documented MPS IIIA diagnosis
- Participants aged ≥ 24 months and ≤ 72 months
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 & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description Cohort 3 GC1130A - Cohort 2 GC1130A - Cohort 1 GC1130A -
- Primary Outcome Measures
Name Time Method Incidences and characteristics of adverse events up to 108 weeks
- Secondary Outcome Measures
Name Time Method Maximum concentration in cerebrospinal fluid (CSF) up to 104 weeks GC1130A PK parameters
Area under the concentration-time curve in CSF up to 104 weeks GC1130A PK parameters
Maximum concentration in serum up to 104 weeks GC1130A PK parameters
Area under the concentration-time curve in serum up to 104 weeks GC1130A PK parameters
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 serum up to 104 weeks Incidence of anti-drug and neutralizing antibodies of GC1130A in CSF 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
Trial Locations
- Locations (5)
National Center for Child Health and Development
🇯🇵Setagaya, Tokyo, Japan
UCSF Benioff Children's Hospital
🇺🇸Oakland, California, United States
University of Minnesota
🇺🇸Minneapolis, Minnesota, United States
Ajou University Medical Center
🇰🇷Suwon-si, Gyeongi-do, Korea, Republic of
Samsung Medical Center
🇰🇷Seoul-si, Korea, Republic of