Ignite Creation Date:
2024-10-26 @ 3:38 PM
Last Modification Date:
2024-10-26 @ 3:38 PM
Study NCT ID:
NCT06567769
Status:
NOT_YET_RECRUITING
Last Update Posted:
None
First Post:
2024-08-20
Brief Title:
Phase 1 Study of GC1130A in Pediatric Patients With Sanfilippo Syndrome Type A MPS IIIA
Sponsor:
None
Organization:
None
Study Overview
Official 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
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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 pediatric patients with Sanfilippo Syndrome Type A MPS IIIA
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None