Cystic Fibrosis Research

The pediatric research program at the Children's Hospital of Philadelphia is among the largest in the country, ranking second in National Institutes of Health funding. The Cystic Fibrosis Center at The Children's Hospital of Philadelphia is no exception. The Center is affiliated with the Institute for Human Gene Therapy, devoted to the multidisciplinary study of gene therapy, with a special focus on the treatment of human disease.

The Cystic Fibrosis Center is currently involved in many exciting breakthrough research studies. A partial list and description of some of our studies is presented below. Please contact our Research Coordinator, Michael Donahue, CRNP at 215-590-3749, if you are interested in participating in any of the studies or would like to obtain more information.

  • Meroprenem
  • Bone health
  • Gene therapy perspectives
  • Nutrition
  • ESCF
  • Steatorrhea
  • Synagis study


Evaluation of the efficacy, safety, tolerance, and pharmacokinetics of meropenem for the treatment of acute pulmonary exacerbations in patients with cystic fibrosis.

A multi-centered trial testing the effectiveness of a new antibiotic named meropenem in treating patients with a pulmonary exacerbation. The objective is to evaluate the efficacy, safety, tolerance, and pharmacokinetics of intravenous meropenem in patients with cystic fibrosis experiencing an acute pulmonary exacerbation.

Bone Health

A multi-centered study originated at The Children's Hospital of Philadelphia to study bone mineralization in patients with cystic fibrosis. Some people with cystic fibrosis (CF) have poor bone mineralization, a measure of the strength and health of bones. In order to discover how common this problem is in people with CF and what factors may lead to poor bone health, we are inviting people with CF between the ages of 8 and 25 to participate in a bone health study.

Subjects will answer questions about physical activity, food intake and family medical history. We will measure their bone age, bone density, height, weight and other easy measurements. They will provide us with a blood and urine sample on the morning of the study visit and will be asked to keep a 3-day record of their food intake at home. Through participation in this study, subjects will help doctors and other health professionals learn more about whether there are certain ways in which poor bone health can be prevented and how it can be treated.

Gene Therapy Perspectives

Consumer perspectives on the promise of CF gene therapy through parent interviews

A funded multi-center study from the NIH, which will examine attitudes of gene therapy for cystic fibrosis.


Nutritional status and pulmonary disease in children with CF

A multi-centered study originated at The Children's Hospital of Philadelphia to understand how dietary intake and growth affect the lung disease of young children with cystic fibrosis and to use what we learn through this study to improve the care of children with cystic fibrosis. This study is currently not enrolling new patients.


Epidemiological study of CF

A multi-center, longitudinal follow-up study of patients with cystic fibrosis for monitoring pulmonary function, pulmonary exacerbations, and the safety of long-term treatment with Pulmozyme® (dornase alfa, recombinant) inhalation solution.

A multi-center study investigating the long-term effects of Pulmozyme® on the pulmonary function of patients with cystic fibrosis.


Novel approach for qualifying steatorrhea

Quantifying fat malabsorption in people with cystic fibrosis and pancreatic disease is important for guiding nutritional intervention for good growth and development and monitoring therapy with pancreatic enzymes. The 72-hour stool and diet collection is the widely accepted test for this purpose. Unfortunately, this test requires three days of consuming and documenting a high fat diet and collection of the entire stool output.

Many people and families with cystic fibrosis and health care providers find this test unappealing. Furthermore, since few clinical labs routinely conduct this test, stool collections have to be shipped to distant labs resulting in delayed reporting of results. Therefore, despite the clinically important information provided by the 72-hour stool and diet collection, it is under-utilized or avoided in many clinical care and research settings. There is an urgent need for a new diagnostic test to assess the presence and severity of pancreatic-based dietary fat malabsorption that is more convenient, accurate and acceptable than the 72-hour stool collection.

The aim of this study is to develop an acceptable, accurate, reliable and specific alternative test to the 72-hour stool and diet collection to diagnose malabsorption due to pancreatic insufficiency. Our proposed method, the 'Malabsorption Blood Test' utilizes two naturally occurring fats: Pentadecanoic fatty acid (PA) and Triheptadecanoin (THA), a triglyceride containing three heptadecanoic acids (HA), as markers of dietary fat absorption.

After a study meal containing a dose of the two special fats, PA, being a free fatty acid is absorbed independent of pancreatic enzymes, whereas THA requires hydrolysis by pancreatic lipase before absorption. Therefore the difference in serum levels of the two fats after an oral dose will reflect pancreatic-based fat malabsorption. Using mathematical modeling techniques, this difference will be used to derive a formula for quantifying the degree of fat malabsorption due to pancreatic insufficiency in people with cystic fibrosis, and potentially other diseases.

Synagis Study

A Phase IV study of the safety of Synagis (Palivizumab) for prophylaxis of respiratory syncytial virus disease in children with cystic fibrosis

A multi-center research study evaluating the safety of Synagis when used in patients with cystic fibrosis.

Reviewed on April 06, 2014