Short Term Safety and Efficacy of Lubiprostone in Adults With Cystic Fibrosis

Short Term Safety and Efficacy of Lubiprostone in Adults With Cystic Fibrosis

The purpose of this study is to determine the short term safety and effectiveness of lubiprostone when used for constipation in adults with cystic fibrosis.

Cystic fibrosis (CF) affects about 30,000 people in the United States. It is caused by an absent or dysfunctional protein called CF transmembrane conductance regulator (CFTR) which functions as a chloride channel. Lubiprostone is a medication indicated for constipation that activates type 2 chloride channels (ClC-2) and has the potential to be an effective treatment for constipation in adults with CF. It has the potential to correct the underlying disorder by utilizing a chloride channel whose activity does not depend on CFTR. This project is a prospective open-label pilot study to examine the safety and effectiveness of lubiprostone when used in adults with CF with constipation. The specific aims are as follows: 1) Determine the effectiveness of lubiprostone for constipation in participants with CF, and 2) Determine the short term safety of lubiprostone in adults with CF. Data will also be collected to generate further information about the effect of lubiprostone on nutritional status.

Inclusion Criteria:

• Men and women of all races
• 18 years of age or older at time of enrollment
• Diagnosis of cystic fibrosis
• History of constipation, as defined by the Rome III criteria, OR the requirement of chronic scheduled doses of a laxative, OR a score ≥1.5 on the Patient assessment of constipation symptoms (PAC-SYM)15 survey administered during the screening visit, OR a score of less than 3 on the Bristol Stool Scale (BSS) at the screening visit.

Exclusion Criteria:

• Current gastrointestinal (GI) obstruction
• History of GI obstruction requiring hospitalization within six months of enrollment
• Pregnancy or breastfeeding
• Hypersensitivity to lubiprostone or any of its components
• Serum creatinine >1.8 mg/dL at last annual visit
• Clinically significant liver disease, defined as aspartate aminotransferase (AST) or alanine aminotransferase (ALT) >3X upper limit of normal
• History of frequent hospital admissions for CF exacerbations (≥4 in the last 6 months)
• Currently registered on a lung transplant waiting list
• Women of capable of child-bearing who either refuse a pregnancy test, or are incapable or unwilling to use contraception during the protocol, or both.
• Any other condition, in the opinion of the investigators, that interferes with the ability of the participant to comply with study requirements, confers significant risk to the participant, or limits the ability of the participant to complete the study