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Clinical trial

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)

NCT06567769 is registered in ClinicalTrials.gov with GC Biopharma Corp as lead sponsor.

NCT NCT06567769
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Registry record

As filed in ClinicalTrials.gov.

Title
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)
Lead sponsor
GC Biopharma Corp
Phase
Phase 1
Overall status
Active, not recruiting
Start date
November 21, 2024
Completion date
June 30, 2027
Enrollment
9 (estimated)
Sites
4

Conditions

As the registry lists them; a condition with its own page is linked.

sanfilippo syndrome type a