Ignite Creation Date:
2025-12-24 @ 11:10 PM
Ignite Modification Date:
2026-01-01 @ 4:07 PM
Study NCT ID:
NCT05399069
Status:
UNKNOWN
Last Update Posted:
2022-11-09
First Post:
2022-05-11
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Safety and Tolerability of VGR-R01 in Patients With Bietti Crystalline Dystrophy
Sponsor:
Beijing Tongren Hospital
Organization:
Study Overview
Official Title:
An Open-Label Safety Study of Single-dose VGR-R01 in Patients With Bietti Crystalline Dystrophy
Status:
UNKNOWN
Status Verified Date:
2022-04
Last Known Status:
RECRUITING
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:
An Open-Label, Non-Randomized, Uncontrolled, Single-dose Pilot Study of VGR-R01 in Patients with Bietti Crystalline Dystrophy.
Detailed Description:
VGR-R01 is a novel AAV vector carrying the human CYP4v2 coding sequence. This study is intended to evaluate the safety and tolerability of a single subretinal administration of VGR-R01. All subjects will undergo 365(±7) days of safety observation and will be encouraged to enroll in an extension study to evaluate the long-term safety of VGR-R01 for a total of five 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?: