The inherited blood coagulation disorder hemophilia A results from deficiency in the expression or function of FVIII. The MiniAdF VIII vector is a """"""""gutless"""""""" adenovirus vector designed to restore production of human FVIII by delivering the entire FVIII cDNA to somatic cells. Nonclinical pharmacology, toxicology and biodistribution studies have been performed. Importantly, physiological levels of FVIII were produced in vivo, and these levels persisted for an extended period of time, resulting in phenotypic correction in hemophilic mice and dogs. The objective of this clinical study is to evaluate through dose escalation the safety of intravenous infusion of MiniAdFVIII vector in severe hemophilia A patients without inhibitors. Additional objectives of this study are as follows: (1) to evaluate through dose escalation the ability of an intravenous infusion of MiniAdFVIlI vector to produce circulating, functional levels of FVIII, (2) to evaluate the effect of MiniAdFVIIl vector therapy on the frequency and severity of bleeding events following defined dose escalation, (3) to evaluate immunologic responses following the administration of MiniAdF VIII vector by monitoring anti-adenoviral and anti-FVIII antibody titers in blood, and (4) to determine the functional FVIII expression profile by measuring the level, time course, and duration of functional and circulating FVIII.

Proposed Commercial Applications

NOT AVAILABLE

Agency
National Institute of Health (NIH)
Institute
National Heart, Lung, and Blood Institute (NHLBI)
Type
Small Business Innovation Research Grants (SBIR) - Phase II (R44)
Project #
5R44HL063477-03
Application #
6527227
Study Section
Special Emphasis Panel (ZRG1-HEM-2 (01))
Program Officer
Link, Rebecca P
Project Start
1999-08-09
Project End
2003-04-29
Budget Start
2002-09-30
Budget End
2003-04-29
Support Year
3
Fiscal Year
2002
Total Cost
$25,000
Indirect Cost
Name
Corautus Genetics, Inc.
Department
Type
DUNS #
City
Atlanta
State
GA
Country
United States
Zip Code
30308