Ignite Creation Date:
2024-05-06 @ 5:45 PM
Last Modification Date:
2024-10-26 @ 2:35 PM
Study NCT ID:
NCT05422482
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2024-04-12
First Post:
2022-05-31
Brief Title:
A Study to Evaluate the Safety Tolerability PK and PD of Intracerebroventricular GC1123 in Patients with MPS Ⅱ
Sponsor:
GC Biopharma Corp
Organization:
GC Biopharma Corp
Study Overview
Official Title:
An Open-Label Phase 1 and Extension Study to Evaluate the Safety Tolerability PK and PD of Intracerebroventricular GC1123 in Patients with MPS Ⅱ Who Have Central Nervous System Involvement and Are Receiving Treatment with Intravenous Drug
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2024-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
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 pharmacokinetics and pharmacodynamics of intracerebroventricular GC1123 in patients with MPS Ⅱ who have central nervous system involvement and are receiving treatment with intravenous drug
Detailed Description:
This study is designed as prospective open-label phase I and extension study Safety tolerability pharmacokinetic and pharmacodynamic properties of repeat-dose treatment of ICV-administered investigational product will be studied in patients undergoing standard treatments
Patients will undergo cerebrospinal fluid CSF reservoir device implantation surgery on their scalps and the reservoirs will be used to administer GC1123 to the cerebral ventricles monthly every 28 days The planned administering dose is 30 mg After the 2nd dose on the 6th patient Data and Safety Monitoring Boards DSMB will evaluate the safety and tolerability data of GC1123 The planned duration of the sutdy is total about 2 years phase I and extension
Patients will undergo cerebrospinal fluid CSF reservoir device implantation surgery on their scalps and the reservoirs will be used to administer GC1123 to the cerebral ventricles monthly every 28 days The planned administering dose is 30 mg After the 2nd dose on the 6th patient Data and Safety Monitoring Boards DSMB will evaluate the safety and tolerability data of GC1123 The planned duration of the sutdy is total about 2 years phase I and extension
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None