Ignite Creation Date:
2024-10-26 @ 3:36 PM
Last Modification Date:
2024-10-26 @ 3:36 PM
Study NCT ID:
NCT06528080
Status:
RECRUITING
Last Update Posted:
None
First Post:
2024-06-22
Brief Title:
Clinical Study of LY-M001 Injection in the Treatment of Adolescents With Type I Gaucher Disease
Sponsor:
None
Organization:
None
Study Overview
Official Title:
A Prospective Single-center Open-arm Single-arm Clinical Study to Evaluate the Safety and Efficacy of a Single Intravenous Infusion of LY-M001 Injection in Adolescents With Type I Gaucher Disease
Status:
RECRUITING
Status Verified Date:
2024-07
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 was to evaluate the safety tolerability efficacy immunogenicity PD and PK characteristics of LY-M001 injection in children with GD1 aged 12 years age 18 years This study mainly includes the main study stage and the long-term follow-up study stage
Detailed Description:
The study planned to enroll 6-9 patients with GD1 for 5 years with a total of 34 follow-up visits The main study period was 52 weeks and the long-term follow-up period was 53 weeks to 5 years after administration
In this study three dose groups were preset and the first subject was enrolled with 10 1013 vgkg as the initial dose the first dose group After the safety was determined by DLT observation subsequent subjects were enrolled The first dose group is scheduled to complete 3 patients with Gaucher disease type I and these 3 subjects will complete safety evaluation for at least 28 days after administration Based on safety and efficacy data it will be decided by SRC discussion to increase to the next dose group and 3 patients with Gaucher disease type I will be reenrolled in the next dose group In addition according to the safety data and effectiveness data of the first subject within 28 days the SRC comprehensively determines whether the DLT observation period needs to be extended
This is an open clinical study without blindness
In this study three dose groups were preset and the first subject was enrolled with 10 1013 vgkg as the initial dose the first dose group After the safety was determined by DLT observation subsequent subjects were enrolled The first dose group is scheduled to complete 3 patients with Gaucher disease type I and these 3 subjects will complete safety evaluation for at least 28 days after administration Based on safety and efficacy data it will be decided by SRC discussion to increase to the next dose group and 3 patients with Gaucher disease type I will be reenrolled in the next dose group In addition according to the safety data and effectiveness data of the first subject within 28 days the SRC comprehensively determines whether the DLT observation period needs to be extended
This is an open clinical study without blindness
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