Investigational Drug Targets Cystic Fibrosis Mutation and Regulator

Trial Objective

Our researchers are studying glycerol phenylbutyrate (GPBA), also known as RAVICTI®, to determine how safe it is and how well people tolerate it in the treatment of cystic fibrosis (CF).

Investigational Drug Targets Cystic Fibrosis Mutation and Regulator

Pancreatic enzymes are needed to release the active molecules of GPBA, which is an oil of phenylbutyrate, a drug used to treat urea-cycle disorders. This study will determine if GPBA (RAVICTI®) improves the function of a specific gene mutation (F508del) for a protein called the cystic fibrosis transmembrane conductance regulator (CFTR). The study also will measure how typical pancreatic enzyme replacement therapy (PERT) affects absorption of active phenylbutyrate.

RAVICTI® is the brand name of a GPBA drug approved by the Food and Drug Administration (FDA) for the treatment of urea-cycle disorders. RAVICTI® is not approved for treatment of cystic fibrosis, and its use in this study is investigational.

Enrollment

This trial is active and currently recruiting.

How to Participate

For more information, call 303.270.2321 or complete the form below.

Who Can Participate

 Adults with cystic fibrosis (CF) who have documentation of two copies of the F508del cystic fibrosis transmembrane conductance regulator (CFTR) mutation, a forced expiratory volume during the first second of air exhaled (FEV1) equal to or greater than 30 percent, and who take pancreatic enzyme replacement therapy (PERT) may be eligible to participate.


Estimated Time Commitment

5 visits totalling up to 21 hours

Trial Location

National Jewish Main Campus, Denver, CO

Compensation

Provided

Trial Sponsors

Food and Drug Administration and Cystic Fibrosis Foundation Therapeutics, Inc., with research support from  Horizon Pharma

Age

18+


Gender

Any Gender


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