Ignite Creation Date:
2025-12-24 @ 1:04 PM
Ignite Modification Date:
2025-12-27 @ 10:00 PM
Study NCT ID:
NCT06488261
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-07-22
First Post:
2023-11-20
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Long Term Follow-Up for Safety of AVR-RD-02
Sponsor:
John Bernat
Organization:
Study Overview
Official Title:
A Long-Term Follow-Up Safety Study of Subjects With Gaucher Disease Who Previously Received AVR-RD-02
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2025-07
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 goal of this observational study is to assess the safety and tolerability 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 (NCT04145037) and who meet all eligibility criteria may participate. Subjects will be asked to return for study visits annually for 14 years to assess measures of safety and persistence of AVR-RD-02 treatment.
Study Oversight
Has Oversight DMC:
False
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?:
False
Is an FDA AA801 Violation?: