RECRUITINGPhase 1INTERVENTIONAL
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)
About This 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 patients with Sanfilippo Syndrome Type A (MPS IIIA).
Who May Be Eligible (Plain English)
Who May Qualify:
- Participants with documented MPS IIIA diagnosis
- Participants aged ≥ 12 months and ≤ 18 years
Who Should NOT Join This Trial:
- 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
Always talk to your doctor about whether this trial is right for you.
Original Eligibility Criteria
View original clinical language
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
Treatments Being Tested
DRUG
GC1130A
ICV injection
Locations (5)
UCSF Benioff Children's Hospital
Oakland, California, United States
University of Minnesota
Minneapolis, Minnesota, United States
National Center for Child Health and Development
Setagaya City, Tokyo, Japan
Ajou University Medical Center
Suwon, Gyeongi-do, South Korea
Samsung Medical Center
Seoul, South Korea