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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00706004
Recruitment Status : Completed
First Posted : June 27, 2008
Results First Posted : April 18, 2011
Last Update Posted : April 18, 2011
Takeda Pharmaceuticals North America, Inc.
Information provided by:
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.

Condition or disease Intervention/treatment Phase
Constipation Cystic Fibrosis Drug: lubiprostone Not Applicable

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.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 9 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Short Term Safety and Efficacy of Lubiprostone in Adults With Cystic Fibrosis
Study Start Date : July 2008
Actual Primary Completion Date : December 2009
Actual Study Completion Date : December 2009

Resource links provided by the National Library of Medicine

Intervention Details:
  • Drug: lubiprostone
    lubiprostone 24 microgram capsule taken twice daily for 4 weeks
    Other Name: Brand name: Amitiza

Primary Outcome Measures :
  1. Number of Spontaneous Bowel Movements Per Week [ Time Frame: 2-week run-in period, 2-weeks of treatment, 4-weeks of treatment ]

Secondary Outcome Measures :
  1. Patient Assessment of Constipation Symptoms [ Time Frame: 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).

  2. Bristol Stool Scale Score [ Time Frame: 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.

  3. Body Mass Index [ Time Frame: baseline, 2 weeks of treatment, 4 weeks of treatment ]
  4. Self Reported Adverse Effects at Each Study Visit [ Time Frame: 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.

  5. Serum Sodium [ Time Frame: baseline, 4 weeks ]
  6. Serum Chloride [ Time Frame: baseline and 4 weeks ]
  7. Serum Potassium [ Time Frame: baseline and 4 weeks ]
  8. Serum Bicarb [ Time Frame: baseline and 4 weeks ]
  9. Serum BUN [ Time Frame: baseline and 4 weeks ]
  10. Serum Creatinine [ Time Frame: baseline and 4 weeks ]
  11. AST [ Time Frame: baseline and 4 weeks ]
  12. ALT [ Time Frame: baseline and 4 weeks ]
  13. Serum Calcium [ Time Frame: baseline and 4 weeks ]
  14. Serum Magnesium [ Time Frame: baseline and 4 weeks ]
  15. Serum Phosphate [ Time Frame: baseline and 4 weeks ]
  16. Serum Glucose [ Time Frame: baseline and 4 weeks ]
  17. Serum Vitamin D [ Time Frame: baseline and 4 weeks ]
  18. Serum Vitamin A [ Time Frame: baseline and 4 weeks ]
  19. Serum Vitamin E [ Time Frame: baseline and 4 weeks ]
  20. Serum Prealbumin [ Time Frame: baseline and 4 weeks ]
  21. Serum Albumin [ Time Frame: baseline and 4 weeks ]

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

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To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00706004

United States, Arkansas
University of Arkansas for Medical Sciences
Little Rock, Arkansas, United States, 72205
Sponsors and Collaborators
University of Arkansas
Takeda Pharmaceuticals North America, Inc.
Principal Investigator: Catherine E. O'Brien, Pharm.D. University of Arkansas

Responsible Party: Catherine E. O'Brien, University of Arkansas for Medical Sciences Identifier: NCT00706004     History of Changes
Other Study ID Numbers: 76992
First Posted: June 27, 2008    Key Record Dates
Results First Posted: April 18, 2011
Last Update Posted: April 18, 2011
Last Verified: March 2011

Keywords provided by University of Arkansas:
cystic fibrosis
Constipation in adults with cystic fibrosis

Additional relevant MeSH terms:
Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Signs and Symptoms, Digestive
Signs and Symptoms
Chloride Channel Agonists
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action