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Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
Safety of a New Type of Treatment Called Gene Transfer for the Treatment of Severe Hemophilia B
NCTID
NCT00076557
(View at clinicaltrials.gov)
Description
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 U.S. 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).
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Development Status
Inactive
Indication
Hemophilia B
Disease Ontology Term
DOID:12259
Compound Name
AAV2-hFIX16
Sponsor
Avigen
Funder Type
Industry
Recruitment Status
Terminated
Enrollment Count
15
Results Posted
Not Available
Therapy Information
Target Gene/Variant
F9
Therapy Type
Gene transfer
Therapy Route
In-vivo
Mechanism of Action
Functional gene replacement
Route of Administration
Hepatic artery infusion
Drug Product Type
Viral vector
Target Tissue/Cell
Hepatocyte
Delivery System
Viral transduction
Vector Type
AAV2
Editor Type
Dose 1
8E10 vg/kg
Dose 2
4E11 vg/kg
Dose 3
2E12 vg/kg
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1, Phase2
Submit Date
2004-01-26
Completion Date
Last Update
2007-04-04
Participation Criteria
Eligible Age
>=18 Years
Standard Ages
Adult, Older adult
Sexes Eligible for Study
MALE
Locations
No.of Trial Sites
3
Locations
United States
Regulatory Information
Has US IND
True
FDA Designations
Orphan Drug Designation
Recent Updates
Avigen suspended enrollment in May 2004; transferred AAV-based product rights to Genzyme Corporation in December 2005, LTFU was sponsored by CHOP from 2009-2013, then Spark Therapeutics afterwards
Resources/Links
Clinical Publications
Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B
Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response
News and Press Releases
Company Discontinues Trial of Hemophilia Gene Therapy
SEC Form 10-K: Avigen, Inc. FY2003
SEC Form 10-K: Avigen, Inc. FY2004
SEC Form 10-K: Avigen, Inc. FY2005
Preclinical Publications
Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy
Related NCTID
Long Term Follow-Up: NCT00515710