Ignite Creation Date:
2024-05-06 @ 7:58 PM
Last Modification Date:
2024-10-26 @ 3:18 PM
Study NCT ID:
NCT06207552
Status:
RECRUITING
Last Update Posted:
2024-07-09
First Post:
2023-12-18
Brief Title:
Evaluation of the Safety Tolerability and Efficacy of a Gene Therapy Drug for the Treatment of Pediatric Fabry Disease
Sponsor:
Childrens Hospital of Fudan University
Organization:
Childrens Hospital of Fudan University
Study Overview
Official Title:
A Single-arm Open Label Single-dose Clinical Study to Evaluate the Safety Tolerability and Efficacy of BBM-F101 Injection in the Treatment of Pediatric Fabry Disease
Status:
RECRUITING
Status Verified Date:
2024-07
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:
This is a single-arm open label single-dose clinical study to evaluate the safety tolerability and efficacy of BBM-F101 injection in the pediatric Fabry disease participants up to 52 weeks after infusion and the long-term safety and efficacy of BBM-F101 injection up to 5 years after infusion
BBM-F101 injection is an adeno-associated virus AAV gene therapy product for the treatment of pediatric Fabry disease
BBM-F101 injection is an adeno-associated virus AAV gene therapy product for the treatment of pediatric Fabry disease
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