Trial Profile

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

Phase of Trial: Phase I

Latest Information Update: 27 May 2025

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At a glance

25 Nov 2024 According to a GC Biopharma media release, the trial will be conducted at two to three institutions in the US including University of California San Francisco (UCSF) Benioff Childrens Hospitals, Samsung Medical Center and Ajou University Hospital in Korea, and one institution in Japan.
25 Nov 2024 According to a GC Biopharma media release, first patient has been dosed in this study.
29 Oct 2024 Status changed from not yet recruiting to recruiting.

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