Wednesday, March 10, 2010

Plans to Issue New FOA

The National Heart, Lung, and Blood Institute (NHLBI) and the Canadian Institutes of Health Research (CIHR) expect to issue a new Funding Opportunity Announcement (FOA) June 2010 with the expected receipt date for applications to be October 2010. The future plans are to find new participants to participate in the Pediatric Heart Network (PHN), a cooperative network of pediatric cardiovascular clinical research centers that includes a data coordinating center.

Congenital cardiovascular malformations affect approximately 40,000 infants in the U.S. each year leading the cause of infant deaths. The incidence of congenital heart disease is at least triple that of childhood cancers, and is substantially greater than pediatric AIDS. In the past 25 years, fewer than 40 randomized clinical trials have been carried out in patients with congenital or acquired heart disease.

The major barriers to doing clinical studies in pediatric heart diseases concern the small numbers of individuals with a particular congenital heart defect at any one center, differences in treatment approaches between centers, absence of systematic centralized databases, and a lack of resources to nationally coordinate collaborative efforts. Additional barriers relate to parents that are unaware of studies, or live a distance from a research site, or just lack familiarity with what happens during a study.

The network approach attempts to address these barriers by offering an effective flexible way to study adequate numbers of patients with uncommon diseases. This is accomplished by using a common infrastructure to recruit, monitor, and follow patients whose conditions need to be characterized in a standardized fashion.

For example, Vanderbilt Children’s Hospital a member of the Pediatric Heart Network and participating in a PHN study on the cardiac side effects of the rare disorder known as Marfan Syndrome. Marfan syndrome is an inherited disorder caused by a mutation in the gene that controls certain connective tissues. The malady is characterized by elongated bones and ligaments, and the eyes, lungs, and the wall of the aorta can be affected. A specific study ongoing at Vanderbilt Children’s Hospital is comparing two drugs, Atenolol and Losartan to see which one is better at slowing the speed of aortic enlargement.

PHN has completed a few studies on the Marfan syndrome with several studies still ongoing and more in the pipeline. The Marfan trial is 3 years old and about three quarters completed with 22 sites nationwide participating. Vanderbilt has 13 patients in the study with each patient participating for three years.