Short Term Safety and Efficacy of Lubiprostone in Adults With Cystic Fibrosis
Primary Purpose
Constipation, Cystic Fibrosis
Status
Completed
Phase
Not Applicable
Locations
United States
Study Type
Interventional
Intervention
lubiprostone
Sponsored by
About this trial
This is an interventional treatment trial for Constipation focused on measuring constipation, cystic fibrosis, lubiprostone, Constipation in adults with cystic fibrosis
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
Sites / Locations
- University of Arkansas for Medical Sciences
Outcomes
Primary Outcome Measures
Number of Spontaneous Bowel Movements Per Week
Secondary Outcome Measures
Patient Assessment of Constipation Symptoms
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
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
Self Reported Adverse Effects at Each Study Visit
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
Serum Chloride
Serum Potassium
Serum Bicarb
Serum BUN
Serum Creatinine
AST
ALT
Serum Calcium
Serum Magnesium
Serum Phosphate
Serum Glucose
Serum Vitamin D
Serum Vitamin A
Serum Vitamin E
Serum Prealbumin
Serum Albumin
Full Information
NCT ID
NCT00706004
First Posted
June 25, 2008
Last Updated
March 14, 2011
Sponsor
University of Arkansas
Collaborators
Takeda Pharmaceuticals North America, Inc.
1. Study Identification
Unique Protocol Identification Number
NCT00706004
Brief Title
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
2. Study Status
Record Verification Date
March 2011
Overall Recruitment Status
Completed
Study Start Date
July 2008 (undefined)
Primary Completion Date
December 2009 (Actual)
Study Completion Date
December 2009 (Actual)
3. Sponsor/Collaborators
Name of the Sponsor
University of Arkansas
Collaborators
Takeda Pharmaceuticals North America, Inc.
4. Oversight
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Constipation, Cystic Fibrosis
Keywords
constipation, cystic fibrosis, lubiprostone, Constipation in adults with cystic fibrosis
7. Study Design
Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
9 (Actual)
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
lubiprostone
Other Intervention Name(s)
Brand name: Amitiza
Intervention Description
lubiprostone 24 microgram capsule taken twice daily for 4 weeks
Primary Outcome Measure Information:
Title
Number of Spontaneous Bowel Movements Per Week
Time Frame
2-week run-in period, 2-weeks of treatment, 4-weeks of treatment
Secondary Outcome Measure Information:
Title
Patient Assessment of Constipation Symptoms
Description
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).
Time Frame
2-week run-in period, 2 weeks of treatment, 4 weeks of treatment
Title
Bristol Stool Scale Score
Description
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.
Time Frame
2-week run-in period, 2 weeks of treatment, 4 weeks of treatment
Title
Body Mass Index
Time Frame
baseline, 2 weeks of treatment, 4 weeks of treatment
Title
Self Reported Adverse Effects at Each Study Visit
Description
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.
Time Frame
During entire study period
Title
Serum Sodium
Time Frame
baseline, 4 weeks
Title
Serum Chloride
Time Frame
baseline and 4 weeks
Title
Serum Potassium
Time Frame
baseline and 4 weeks
Title
Serum Bicarb
Time Frame
baseline and 4 weeks
Title
Serum BUN
Time Frame
baseline and 4 weeks
Title
Serum Creatinine
Time Frame
baseline and 4 weeks
Title
AST
Time Frame
baseline and 4 weeks
Title
ALT
Time Frame
baseline and 4 weeks
Title
Serum Calcium
Time Frame
baseline and 4 weeks
Title
Serum Magnesium
Time Frame
baseline and 4 weeks
Title
Serum Phosphate
Time Frame
baseline and 4 weeks
Title
Serum Glucose
Time Frame
baseline and 4 weeks
Title
Serum Vitamin D
Time Frame
baseline and 4 weeks
Title
Serum Vitamin A
Time Frame
baseline and 4 weeks
Title
Serum Vitamin E
Time Frame
baseline and 4 weeks
Title
Serum Prealbumin
Time Frame
baseline and 4 weeks
Title
Serum Albumin
Time Frame
baseline and 4 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Catherine E. O'Brien, Pharm.D.
Organizational Affiliation
University of Arkansas
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of Arkansas for Medical Sciences
City
Little Rock
State/Province
Arkansas
ZIP/Postal Code
72205
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Short Term Safety and Efficacy of Lubiprostone in Adults With Cystic Fibrosis
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