Ignite Creation Date:
2025-12-24 @ 9:24 PM
Ignite Modification Date:
2025-12-25 @ 7:09 PM
Study NCT ID:
NCT06178432
Status:
NOT_YET_RECRUITING
Last Update Posted:
2023-12-21
First Post:
2023-11-30
Is Possible Gene Therapy:
True
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Evaluation of the Safety, Tolerability and Efficacy of Gene Therapy Drug for Late Onset Pompe Disease (LOPD)
Sponsor:
Huashan Hospital
Organization:
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:
False
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?: