Ignite Creation Date:
2024-05-05 @ 11:31 AM
Last Modification Date:
2024-10-26 @ 9:09 AM
Study NCT ID:
NCT00076557
Status:
TERMINATED
Last Update Posted:
2007-04-04
First Post:
2004-01-26
Brief Title:
Safety of a New Type of Treatment Called Gene Transfer for the Treatment of Severe Hemophilia B
Sponsor:
Avigen
Organization:
Avigen
Study Overview
Official Title:
A Phase I Safety Study in Subjects With Severe Hemophilia B Factor IX Deficiency Using Adeno-Associated Viral Vector to Deliver the Gene for Human Factor IX Into the Liver
Status:
TERMINATED
Status Verified Date:
2004-01
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
In this study a modified virus called adeno-associated virus AAV will be used to transfer a normal gene for human clotting factor IX into patients with severe hemophilia B AAV human Factor IX vector Gene therapy is a very new medical technique being used in a number of clinical studies for diseases such as cancer and cystic fibrosis At this time the US Food and Drug Administration has approved no gene transfer products for commercial use To date 8 subjects have received AAV vector in the muscle for a hemophilia B trial by intramuscular injection and to date 6 subjects have been treated with AAV vector in the current hemophilia B liver trial Eleven cystic fibrosis subjects have received AAV vector into their nasal sinuses or lungs to date In this study AAV human Factor IX vector will be injected into the liver using a catheter inserted into a large blood vessel called the proper hepatic artery or the right hepatic artery
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None