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)
Status: Recruiting
Location: See all (5) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1
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 patients with Sanfilippo Syndrome Type A (MPS IIIA).
Eligibility
Participation Requirements
Sex: All
Minimum Age: 1
Maximum Age: 18
Healthy Volunteers: f
View:
• Participants with documented MPS IIIA diagnosis
• Participants aged ≥ 12 months and ≤ 18 years
Locations
United States
California
UCSF Benioff Children's Hospital
RECRUITING
Oakland
Minnesota
University of Minnesota
RECRUITING
Minneapolis
Other Locations
Japan
National Center for Child Health and Development
RECRUITING
Setagaya City
Republic of Korea
Samsung Medical Center
RECRUITING
Seoul
Ajou University Medical Center
RECRUITING
Suwon
Contact Information
Primary
GC Biopharma Corp.
mps3a_clinicaltrial@gccorp.com
+82312609300
Time Frame
Start Date: 2024-11-21
Estimated Completion Date: 2027-06
Participants
Target number of participants: 9
Treatments
Experimental: Cohort 1
Experimental: Cohort 2
Experimental: Cohort 3
Related Therapeutic Areas
Sponsors
Leads: GC Biopharma Corp