Ignite Creation Date:
2024-10-26 @ 3:43 PM
Last Modification Date:
2024-10-26 @ 3:43 PM
Study NCT ID:
NCT06650319
Status:
ENROLLING_BY_INVITATION
Last Update Posted:
None
First Post:
2024-10-15
Brief Title:
A Clinical Study to Evaluate the Safety and Efficacy of LY-M003 Injection in Patients with Wilson Disease
Sponsor:
None
Organization:
None
Study Overview
Official Title:
A Prospective Single-center Open Single-arm Single-dose Clinical Study Evaluating the Safety Tolerability and Efficacy of LY-M003 Injection in Adults with Wilson Disease
Status:
ENROLLING_BY_INVITATION
Status Verified Date:
2024-09
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:
WD
Brief Summary:
Wilsons disease WD also known as Wilsons disease is a rare autosomal recessive metabolic disorder caused by a mutation of the copper transport ATPase β ATP7B gene located on the long arm of chromosome 13 13q143 This leads to accumulation of copper ions in multiple organs such as liver brain and kidney resulting in organ involvement In this study rAAV8 as the carrier of gene therapy products After a single intravenous infusion ATP7B protein can be specifically transduced to the target organ liver and expressed in hepatocytes for a long time
Detailed Description:
This study adopted a prospective single-center open single-arm single-dose clinical design to evaluate the safety tolerability efficacy immunogenicity PD and PK characteristics of LY-M003 injection in WD patients including the main study phase and the long-term follow-up study phase
The main study phase consisted of a screening period weeks -8 to days -2 a baseline period days -1 a treatment and safety observation period days 1-28 and a short-term follow-up period weeks 5 starting from days 29 to 52 Subjects eligible for the screening period will be admitted to the study Center for a single LY-M003 treatment and will enter the short-term follow-up period after the end of the treatment and safety observation period and complete all follow-up during the main study phase Subjects who withdraw early from the study will be required to complete all assessments required for the End of Study EOS visit
Subjects who complete the main study phase or withdraw early from the study will enter the long-term follow-up study phase to obtain long-term evaluation data After the completion of the 5-year long-term follow-up in this study based on the obtained safety and efficacy data of this product it will be discussed by the investigators and partners to determine whether further extension of follow-up time is necessary
The main study phase consisted of a screening period weeks -8 to days -2 a baseline period days -1 a treatment and safety observation period days 1-28 and a short-term follow-up period weeks 5 starting from days 29 to 52 Subjects eligible for the screening period will be admitted to the study Center for a single LY-M003 treatment and will enter the short-term follow-up period after the end of the treatment and safety observation period and complete all follow-up during the main study phase Subjects who withdraw early from the study will be required to complete all assessments required for the End of Study EOS visit
Subjects who complete the main study phase or withdraw early from the study will enter the long-term follow-up study phase to obtain long-term evaluation data After the completion of the 5-year long-term follow-up in this study based on the obtained safety and efficacy data of this product it will be discussed by the investigators and partners to determine whether further extension of follow-up time is necessary
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