Ignite Creation Date:
2024-05-06 @ 4:00 PM
Last Modification Date:
2024-10-26 @ 2:01 PM
Study NCT ID:
NCT04836377
Status:
TERMINATED
Last Update Posted:
2023-08-29
First Post:
2021-03-23
Brief Title:
A Long-Term Follow-up Study of Subjects With Gaucher Disease Who Previously Received AVR-RD-02
Sponsor:
AVROBIO
Organization:
AVROBIO
Study Overview
Official Title:
A Long-Term Follow-up Study of Subjects With Gaucher Disease Who Previously Received AVR-RD-02
Status:
TERMINATED
Status Verified Date:
2022-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
This study was voluntarily terminated due to a business decision not to proceed and not due to any safety or efficacy issue
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This is a multicenter multinational long-term follow-up study to assess the long-term safety and durability of effect of AVR-RD-02 treatment in subjects who previously received AVR-RD-02 treatment single dose administration
Detailed Description:
Subjects who received AVR-RD-02 in the preceding treatment study and who meet all eligibility criteria may participate in the AVRO-RD-02-LTF01 study For individual participating subjects the Baseline visit for the AVRO-RD-02-LTF01 study will coincide with the subjects last visit in the preceding treatment study whenever feasible Subjects will be asked to return for study visits at approximately 6-month intervals for the first 4 years of the study and then annually thereafter for 10 years for a total follow-up of 14 years in this study during which time periodic safety and efficacy assessments will be performed to assess measures of safety engraftment and clinical response after AVR-RD-02 treatment
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
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