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Phase 1 Open-label Ascending Dose Study of GC1130A in MPS IIIA (Sanfilippo Syndrome Type A)
jRCT2031240514Phase 1Completed
- Sponsor
- GC Biopharma Corp
- Start
- Dec 2024
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
01
Overview
Phase 1, open-label, single-arm trial of recombinant human heparan-N-sulfatase in mucopolysaccharidosis type III A, sponsored by GC Biopharma Corp. Primary endpoint: Incidence and characteristics of adverse events. Started Dec 2024; status: completed.
02
Drugs
recombinant human heparan-N-sulfatase
03
Conditions
mucopolysaccharidosis type III A
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