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This purpose of this multi-center international study is to investigate the safety and therapeutic benefit of fenretinide (LAU-7b) in people with cystic fibrosis, a rare progressive genetic disease. Researchers believe that LAU-7b, a derivative of vitamin A, may preserve lung function in patients with cystic fibrosis by reducing persistent inflammation and to improve the lungs’ capacity to defend against resistant bacteria.
This study is open to participants of both genders, ages 18 and up, who have cystic fibrosis with a history of exacerbation.
Males and females: Ages 18 years and older
In this study, participants will be randomly assigned to take either the study drug or a placebo, in capsule form, 3 times a day for 21 days, followed by a 7-day break. This regimen is repeated for a total of 6 cycles with visits for blood work and physical examinations at intervals throughout the study period.
Duration: About 7 months
Study Description at National Institutes of Health
Pulmonary Medicine Research
Joseph Pilewski, MD
For more information about the study or enrollment, please contact:
Clinical Research Nurse Coordinator
Adrienne DeRicco, RN, BSN, CCRC
Children's Hospital's main campus is located in the Lawrenceville neighborhood. Our main hospital address is:
UPMC Children’s Hospital of Pittsburgh
One Children’s Hospital Way
4401 Penn Ave.
Pittsburgh, PA 15224
In addition to the main hospital, Children's has many convenient locations in other neighborhoods throughout the greater Pittsburgh region.
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Interested in giving to Children's Hospital? Visit Children's Hospital of Pittsburgh Foundation's website to make a donation online.