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

Not Applicable

Completed

Conditions: Constipation
Cystic Fibrosis

Registration Number: NCT00706004

Lead Sponsor: University of Arkansas

Brief Summary: 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.

Detailed Description: 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.

Recruitment & Eligibility

Status: COMPLETED

Sex: All

Target Recruitment: 9

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

Study & Design

Study Type: INTERVENTIONAL

Study Design: SINGLE_GROUP

Primary Outcome Measures

Name	Time	Method
Number of Spontaneous Bowel Movements Per Week	2-week run-in period, 2-weeks of treatment, 4-weeks of treatment

Secondary Outcome Measures

Name	Time	Method
Patient Assessment of Constipation Symptoms	2-week run-in period, 2 weeks of treatment, 4 weeks of treatment	The Patient Assessment of Constipation - Symptom (PAC-SYM) survey is a 1-page 12-item tool that measures a patient's assessment of constipation symptoms. The items are in Likert scale format and address the severity of stool, rectal and abdominal symptoms over the past 2 weeks. Items are scored on a scale of 0 to 4, with 4 indicating the most severe. To compute the overall score, the scores of the non-missing items are summed and this is divided by the total number of non-missing items (overall score range, 0 to 4).
Bristol Stool Scale Score	2-week run-in period, 2 weeks of treatment, 4 weeks of treatment	The Bristol Stool Scale is a scale used to rate the consistency of stool. Stool types are accompanied by a written description. There are seven types of stool that are scored from 1 to 7. A score of 1 or 2 indicates constipation; a score of 6 or 7, diarrhea.
Body Mass Index	baseline, 2 weeks of treatment, 4 weeks of treatment
Self Reported Adverse Effects at Each Study Visit	During entire study period	Adverse effects are problems reported by each study subject that they experienced during this clinical trial. Examples are headache and nausea. Study subjects were asked at each visit while on study drug to report any adverse affects that had occurred since the last visit.
Serum Sodium	baseline, 4 weeks
Serum Chloride	baseline and 4 weeks
Serum Potassium	baseline and 4 weeks
Serum Bicarb	baseline and 4 weeks
Serum BUN	baseline and 4 weeks
Serum Creatinine	baseline and 4 weeks
AST	baseline and 4 weeks
ALT	baseline and 4 weeks
Serum Calcium	baseline and 4 weeks
Serum Magnesium	baseline and 4 weeks
Serum Phosphate	baseline and 4 weeks
Serum Glucose	baseline and 4 weeks
Serum Vitamin D	baseline and 4 weeks
Serum Vitamin A	baseline and 4 weeks
Serum Vitamin E	baseline and 4 weeks
Serum Prealbumin	baseline and 4 weeks
Serum Albumin	baseline and 4 weeks