drugset
drugset / Trial / NCT06567769

Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)

NCT06567769 ↗

Phase 1 Recruiting 9 enrolled GC Biopharma Corp
Non-randomizedSequentialOpen-labelTreatment

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).

Timeline

Start
2024-11-21
Primary completion
2027-06
Completion
2027-06

Drugs

EvaluationDrugModalityDoseRoute
Subject GC1130A Protein / enzyme biologic — Other

Indications

mucopolysaccharidosis type 3A