Condition: Sanfilippo Syndrome Type A · Sponsor: GC Biopharma Corp
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).
This description comes directly from the study's public registry record.
GC Biopharma Corp. · +82312609300 · mps3a_clinicaltrial@gccorp.com
Always discuss trial participation with your own doctor first.
| 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 |
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Source record: clinicaltrials.gov/study/NCT06567769