Ignite Creation Date:
2024-05-06 @ 7:52 PM
Last Modification Date:
2024-10-26 @ 3:15 PM
Study NCT ID:
NCT06162338
Status:
RECRUITING
Last Update Posted:
2023-12-08
First Post:
2023-11-20
Brief Title:
A Study of the Safety and Preliminary Efficacy of LY-M001 Injection in the Treatment of Adult Patients With Gaucher Disease Type I
Sponsor:
He Huang
Organization:
Zhejiang University
Study Overview
Official Title:
A Prospective Single-center Open-arm Single-arm Study of the Safety and Preliminary Efficacy of Single Intravenous Infusion Administration of LY-M001 Injection in the Treatment of Adult Patients With Gaucher Disease Type I
Status:
RECRUITING
Status Verified Date:
2023-10
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:
LY-M001
Brief Summary:
This is a prospective single-center open single-arm single-dose intravenous infusion study to evaluate the safety and initial efficacy pharmacodynamic characteristics immunogenicity biodistribution and viral shedding of LY-M001 injectionThis study mainly includes the main study stage and the long-term follow-up study stage
Detailed Description:
This study included the screening period weeks -8 to days -2 the baseline period days -1 the treatment and safety observation period days 0 to 28 and the short-term follow-up period weeks 5 from day 29 to week 38 Participants eligible for the screening period will be admitted to the study center for a single LY-M001 treatment and a short-term follow-up period after the end of the treatment and safety observation period Participants who complete all follow-up during the main study phase or who withdraw early from the study are required to complete all assessments required for the End of Study EOS visitThe study included up to three adult Gaucher disease type I subjects at preset dose group levelsWith 50 1012 vgkg as the initial effective dose first dose group 1 to 2 subjects are expected to be included The first dose group was enrolled by sentinel method and the first subject in this group was observed for at least 28 days after receiving LY-M001 dose-limited toxicity DLT observation period to enroll the next subject
Participants enrolled in the long-term follow-up study were those who completed the main study or withdrew early and the duration of the long-term follow-up study and the main study lasted for a total of 5 years
Participants enrolled in the long-term follow-up study were those who completed the main study or withdrew early and the duration of the long-term follow-up study and the main study lasted for a total of 5 years
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