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

Cystic Fibrosis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00706004
• Other study ID #: 76992
• Status: Completed
• Phase: N/A
• First received: June 25, 2008
• Last updated: March 14, 2011
• Start date: July 2008
• Est. completion date: December 2009

Study information

• Verified date: March 2011
• Source: University of Arkansas
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Institutional Review Board
• Study type: Interventional

Clinical Trial 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.

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 information / eligibility

• Status: Completed
• Enrollment: 9
• Est. completion date: December 2009
• Est. primary completion date: December 2009
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

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

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Constipation
• Cystic Fibrosis
• Fibrosis

Intervention

Drug:
• lubiprostone
lubiprostone 24 microgram capsule taken twice daily for 4 weeks

Locations

Country	Name	City	State
United States	University of Arkansas for Medical Sciences	Little Rock	Arkansas

Sponsors (2)

Lead Sponsor	Collaborator
University of Arkansas	Takeda Pharmaceuticals North America, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Spontaneous Bowel Movements Per Week		2-week run-in period, 2-weeks of treatment, 4-weeks of treatment	No
Secondary	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).	2-week run-in period, 2 weeks of treatment, 4 weeks of treatment	No
Secondary	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.	2-week run-in period, 2 weeks of treatment, 4 weeks of treatment	No
Secondary	Body Mass Index		baseline, 2 weeks of treatment, 4 weeks of treatment	Yes
Secondary	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.	During entire study period	Yes
Secondary	Serum Sodium		baseline, 4 weeks	Yes
Secondary	Serum Chloride		baseline and 4 weeks	Yes
Secondary	Serum Potassium		baseline and 4 weeks	Yes
Secondary	Serum Bicarb		baseline and 4 weeks	Yes
Secondary	Serum BUN		baseline and 4 weeks	Yes
Secondary	Serum Creatinine		baseline and 4 weeks	Yes
Secondary	AST		baseline and 4 weeks	Yes
Secondary	ALT		baseline and 4 weeks	Yes
Secondary	Serum Calcium		baseline and 4 weeks	Yes
Secondary	Serum Magnesium		baseline and 4 weeks	Yes
Secondary	Serum Phosphate		baseline and 4 weeks	Yes
Secondary	Serum Glucose		baseline and 4 weeks	Yes
Secondary	Serum Vitamin D		baseline and 4 weeks	No
Secondary	Serum Vitamin A		baseline and 4 weeks	No
Secondary	Serum Vitamin E		baseline and 4 weeks	No
Secondary	Serum Prealbumin		baseline and 4 weeks	No
Secondary	Serum Albumin		baseline and 4 weeks	No