Cystic Fibrosis Treatment with LAU-7b: The APPLAUD Study – Phase II

Study of the Efficacy and Safety of LAU-7b in the Treatment of Cystic Fibrosis in Adults

Protocol Description

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. 

Study Level: Phase II

Eligibility Criteria

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.
Visits: 8
Duration: About 7 months

Status: Open to Enrollment

Source(s) of Support

Laurent Pharmaceuticals

Primary Investigator

Joseph Pilewski, MD

Contact Information

For more information about the study or enrollment, please contact:
Clinical Research Nurse Coordinator
Adrienne DeRicco, RN, BSN, CCRC