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

This study has been completed.
Sponsor:
Collaborator:
Takeda Pharmaceuticals North America, Inc.
Information provided by:
University of Arkansas
ClinicalTrials.gov Identifier:
NCT00706004
First received: June 25, 2008
Last updated: March 14, 2011
Last verified: March 2011
  Purpose

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 Intervention
Constipation
Cystic Fibrosis
Drug: lubiprostone

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Short Term Safety and Efficacy of Lubiprostone in Adults With Cystic Fibrosis

Resource links provided by NLM:


Further study details as provided by University of Arkansas:

Primary Outcome Measures:
  • Number of Spontaneous Bowel Movements Per Week [ Time Frame: 2-week run-in period, 2-weeks of treatment, 4-weeks of treatment ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Patient Assessment of Constipation Symptoms [ Time Frame: 2-week run-in period, 2 weeks of treatment, 4 weeks of treatment ] [ Designated as safety issue: No ]
    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 [ Time Frame: 2-week run-in period, 2 weeks of treatment, 4 weeks of treatment ] [ Designated as safety issue: No ]
    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 [ Time Frame: baseline, 2 weeks of treatment, 4 weeks of treatment ] [ Designated as safety issue: Yes ]
  • Self Reported Adverse Effects at Each Study Visit [ Time Frame: During entire study period ] [ Designated as safety issue: Yes ]
    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 [ Time Frame: baseline, 4 weeks ] [ Designated as safety issue: Yes ]
  • Serum Chloride [ Time Frame: baseline and 4 weeks ] [ Designated as safety issue: Yes ]
  • Serum Potassium [ Time Frame: baseline and 4 weeks ] [ Designated as safety issue: Yes ]
  • Serum Bicarb [ Time Frame: baseline and 4 weeks ] [ Designated as safety issue: Yes ]
  • Serum BUN [ Time Frame: baseline and 4 weeks ] [ Designated as safety issue: Yes ]
  • Serum Creatinine [ Time Frame: baseline and 4 weeks ] [ Designated as safety issue: Yes ]
  • AST [ Time Frame: baseline and 4 weeks ] [ Designated as safety issue: Yes ]
  • ALT [ Time Frame: baseline and 4 weeks ] [ Designated as safety issue: Yes ]
  • Serum Calcium [ Time Frame: baseline and 4 weeks ] [ Designated as safety issue: Yes ]
  • Serum Magnesium [ Time Frame: baseline and 4 weeks ] [ Designated as safety issue: Yes ]
  • Serum Phosphate [ Time Frame: baseline and 4 weeks ] [ Designated as safety issue: Yes ]
  • Serum Glucose [ Time Frame: baseline and 4 weeks ] [ Designated as safety issue: Yes ]
  • Serum Vitamin D [ Time Frame: baseline and 4 weeks ] [ Designated as safety issue: No ]
  • Serum Vitamin A [ Time Frame: baseline and 4 weeks ] [ Designated as safety issue: No ]
  • Serum Vitamin E [ Time Frame: baseline and 4 weeks ] [ Designated as safety issue: No ]
  • Serum Prealbumin [ Time Frame: baseline and 4 weeks ] [ Designated as safety issue: No ]
  • Serum Albumin [ Time Frame: baseline and 4 weeks ] [ Designated as safety issue: No ]

Enrollment: 9
Study Start Date: July 2008
Study Completion Date: December 2009
Primary Completion Date: December 2009 (Final data collection date for primary outcome measure)
Intervention Details:
    Drug: lubiprostone
    lubiprostone 24 microgram capsule taken twice daily for 4 weeks
    Other Name: Brand name: Amitiza
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

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00706004

Locations
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.
Investigators
Principal Investigator: Catherine E. O'Brien, Pharm.D. University of Arkansas
  More Information

No publications provided

Responsible Party: Catherine E. O'Brien, University of Arkansas for Medical Sciences
ClinicalTrials.gov Identifier: NCT00706004     History of Changes
Other Study ID Numbers: 76992
Study First Received: June 25, 2008
Results First Received: February 16, 2011
Last Updated: March 14, 2011
Health Authority: United States: Institutional Review Board

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

Additional relevant MeSH terms:
Constipation
Cystic Fibrosis
Fibrosis
Digestive System Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Lung Diseases
Pancreatic Diseases
Pathologic Processes
Respiratory Tract Diseases
Signs and Symptoms
Signs and Symptoms, Digestive

ClinicalTrials.gov processed this record on October 23, 2014