Clinical trial NCT00706004: Short Term Safety and Efficacy of Lubiprostone in Adults With Cystic Fibrosis
Official title: Short Term Safety and Efficacy of Lubiprostone in Adults With Cystic Fibrosis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Constipation
Cystic Fibrosis
Keywords: constipation
cystic fibrosis
lubiprostone
Constipation in adults with cystic fibrosis
Lead sponsor: University of Arkansas
Sponsor class: OTHER
Collaborators: name: Takeda Pharmaceuticals North America, Inc.
First posted: 2008-06-27
Enrollment: 9
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility criteria: 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

Primary outcomes:
measure: Number of Spontaneous Bowel Movements Per Week
timeFrame: 2-week run-in period, 2-weeks of treatment, 4-weeks of treatment
Secondary outcomes:
measure: Patient Assessment of Constipation Symptoms
timeFrame: 2-week run-in period, 2 weeks of treatment, 4 weeks of treatment
measure: Bristol Stool Scale Score
timeFrame: 2-week run-in period, 2 weeks of treatment, 4 weeks of treatment
measure: Body Mass Index
timeFrame: baseline, 2 weeks of treatment, 4 weeks of treatment
measure: Self Reported Adverse Effects at Each Study Visit
timeFrame: During entire study period
measure: Serum Sodium
timeFrame: baseline, 4 weeks
measure: Serum Chloride
timeFrame: baseline and 4 weeks
measure: Serum Potassium
timeFrame: baseline and 4 weeks
measure: Serum Bicarb
timeFrame: baseline and 4 weeks
measure: Serum BUN
timeFrame: baseline and 4 weeks
measure: Serum Creatinine
timeFrame: baseline and 4 weeks
measure: AST
timeFrame: baseline and 4 weeks
measure: ALT
timeFrame: baseline and 4 weeks
measure: Serum Calcium
timeFrame: baseline and 4 weeks
measure: Serum Magnesium
timeFrame: baseline and 4 weeks
measure: Serum Phosphate
timeFrame: baseline and 4 weeks
measure: Serum Glucose
timeFrame: baseline and 4 weeks
measure: Serum Vitamin D
timeFrame: baseline and 4 weeks
measure: Serum Vitamin A
timeFrame: baseline and 4 weeks
measure: Serum Vitamin E
timeFrame: baseline and 4 weeks
measure: Serum Prealbumin
timeFrame: baseline and 4 weeks
measure: Serum Albumin
timeFrame: baseline and 4 weeks
Other outcomes:
Locations:
city: Little Rock
country: United States
facility: University of Arkansas for Medical Sciences
state: Arkansas
Study officials:
affiliation: University of Arkansas
name: Catherine E. O'Brien, Pharm.D.
role: PRINCIPAL_INVESTIGATOR
References: