Ignite Creation Date:
2024-05-06 @ 7:53 PM
Last Modification Date:
2024-10-26 @ 3:16 PM
Study NCT ID:
NCT06178432
Status:
NOT_YET_RECRUITING
Last Update Posted:
2023-12-21
First Post:
2023-11-30
Brief Title:
Evaluation of the Safety Tolerability and Efficacy of Gene Therapy Drug for Late Onset Pompe Disease LOPD
Sponsor:
Huashan Hospital
Organization:
Huashan Hospital
Study Overview
Official Title:
A Single-arm Open-label Single-dose Study to Evaluate the Safety Tolerability and Efficacy of CRG003 Injection in the Treatment of Late Onset Pompe Disease
Status:
NOT_YET_RECRUITING
Status Verified Date:
2023-12
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:
This is a single-center single-arm open-label single-dose treatment clinical study to evaluate the safety tolerability and efficacy of CRG003 injection in participants with late onset Pompe disease LOPD with a long-term follow-up period of 5 years
CRG003 BBM-G102 injection is an adeno-associated virus AAV gene therapy product for treating Pompe disease to stably express active GAA enzyme in the liver on a long-term basis after the injection
CRG003 BBM-G102 injection is an adeno-associated virus AAV gene therapy product for treating Pompe disease to stably express active GAA enzyme in the liver on a long-term basis after the injection
Detailed Description:
None
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