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).
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.