Ignite Creation Date:
2024-05-06 @ 6:49 PM
Last Modification Date:
2024-10-26 @ 2:55 PM
Study NCT ID:
NCT05793307
Status:
RECRUITING
Last Update Posted:
2023-06-06
First Post:
2023-03-20
Brief Title:
Evaluation of the Safety and Efficacy of Infantile-onset Pompe Disease Gene Therapy Drug
Sponsor:
GeneCradle Inc
Organization:
GeneCradle Inc
Study Overview
Official Title:
A Multi-centered Single Arm Open Labeled Study to Evaluate the Safety and Efficacy of an Adeno-associated Virus Vector Expressing the Human Acid Alpha-glucosidase GAA Transgene Intravenous Injection in Patients With Infantile-onset Pompe Disease
Status:
RECRUITING
Status Verified Date:
2023-06
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 study is being conducted to evaluate the safety and effectiveness of GC301 adeno-associated virus vector expressing codon-optimized human acid alpha-glucosidase GAA as potential gene therapy for Pompe disease Patients diagnosed with infantile-onset Pompe disease who are younger than 6 months old will be studied
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