Trial Outcomes & Findings for Short Term Safety and Efficacy of Lubiprostone in Adults With Cystic Fibrosis (NCT NCT00706004)

NCT ID: NCT00706004

Last Updated: 2011-04-18

Results Overview

• Recruitment status: COMPLETED
• Study phase: NA
• Target enrollment: 9 participants
• Primary outcome timeframe: 2-week run-in period, 2-weeks of treatment, 4-weeks of treatment
• Results posted on: 2011-04-18

Participant Flow

Participant milestones

Measure	Lubiprostone 24 micrograms twice daily
Overall Study STARTED	9
Overall Study COMPLETED	7
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Measure	Lubiprostone 24 micrograms twice daily
Overall Study Did not meet eligibility criteria	2

Baseline Characteristics

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

Baseline characteristics by cohort

Measure	Lubiprostone n=7 Participants 24 micrograms twice daily
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	7 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age Continuous	26 years STANDARD_DEVIATION 5.4 • n=5 Participants
Sex: Female, Male Female	4 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants
Region of Enrollment United States	7 participants n=5 Participants

PRIMARY outcome

Timeframe: 2-week run-in period, 2-weeks of treatment, 4-weeks of treatment

Population: Participants who completed the study

Outcome measures

Measure	Lubiprostone 24 Mcg Twice Daily n=7 Participants Participants served as their own controls
Number of Spontaneous Bowel Movements Per Week Baseline period	9.7 bowel movements Standard Deviation 5.7
Number of Spontaneous Bowel Movements Per Week 2 weeks	10.8 bowel movements Standard Deviation 5.1
Number of Spontaneous Bowel Movements Per Week 4 weeks	8.8 bowel movements Standard Deviation 2.9

SECONDARY outcome

Timeframe: 2-week run-in period, 2 weeks of treatment, 4 weeks of treatment

Population: Participants who completed the study

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).

Outcome measures

Measure	Lubiprostone 24 Mcg Twice Daily n=7 Participants Participants served as their own controls
Patient Assessment of Constipation Symptoms Baseline	1.18 scores on a scale Standard Deviation 0.56
Patient Assessment of Constipation Symptoms 2 weeks	0.54 scores on a scale Standard Deviation 0.27
Patient Assessment of Constipation Symptoms 4 weeks	0.44 scores on a scale Standard Deviation 0.36

SECONDARY outcome

Timeframe: 2-week run-in period, 2 weeks of treatment, 4 weeks of treatment

Population: Participants who completed the study

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.

Outcome measures

Measure	Lubiprostone 24 Mcg Twice Daily n=7 Participants Participants served as their own controls
Bristol Stool Scale Score Baseline	3.4 scores on a scale Standard Deviation 1.1
Bristol Stool Scale Score 4 weeks	3.3 scores on a scale Standard Deviation 1.1
Bristol Stool Scale Score 2 weeks	3.6 scores on a scale Standard Deviation 0.8

SECONDARY outcome

Timeframe: baseline, 2 weeks of treatment, 4 weeks of treatment

Population: Participants who completed the study

Outcome measures

Measure	Lubiprostone 24 Mcg Twice Daily n=7 Participants Participants served as their own controls
Body Mass Index Baseline	24.0 kg/m^2 Standard Deviation 2.8
Body Mass Index 2 weeks	24.0 kg/m^2 Standard Deviation 2.5
Body Mass Index 4 weeks	24.3 kg/m^2 Standard Deviation 2.5

SECONDARY outcome

Timeframe: During entire study period

Population: Participants who completed the study

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.

Outcome measures

Measure	Lubiprostone 24 Mcg Twice Daily n=7 Participants Participants served as their own controls
Self Reported Adverse Effects at Each Study Visit 2 weeks	5 participants
Self Reported Adverse Effects at Each Study Visit 4 weeks	3 participants

SECONDARY outcome

Timeframe: baseline, 4 weeks

Population: Participants who completed the study

Outcome measures

Measure	Lubiprostone 24 Mcg Twice Daily n=7 Participants Participants served as their own controls
Serum Sodium Baseline	137.4 nmol/L Standard Deviation 2.8
Serum Sodium 4 weeks	139.2 nmol/L Standard Deviation 3.6

SECONDARY outcome

Timeframe: baseline and 4 weeks

Population: Participants who completed the study

Outcome measures

Measure	Lubiprostone 24 Mcg Twice Daily n=7 Participants Participants served as their own controls
Serum Chloride Baseline	103.0 nmol/L Standard Deviation 3.9
Serum Chloride 4 weeks	104.3 nmol/L Standard Deviation 3.4

SECONDARY outcome

Timeframe: baseline and 4 weeks

Population: Participants who completed the study

Outcome measures

Measure	Lubiprostone 24 Mcg Twice Daily n=7 Participants Participants served as their own controls
Serum Potassium Baseline	4.4 nmol/L Standard Deviation 0.4
Serum Potassium 4 weeks	4.4 nmol/L Standard Deviation 0.2

SECONDARY outcome

Timeframe: baseline and 4 weeks

Population: Participants who completed the study

Outcome measures

Measure	Lubiprostone 24 Mcg Twice Daily n=7 Participants Participants served as their own controls
Serum Bicarb Baseline	23.8 nmol/L Standard Deviation 4.0
Serum Bicarb 4 weeks	25.2 nmol/L Standard Deviation 3.3

SECONDARY outcome

Timeframe: baseline and 4 weeks

Population: Participants who completed the study

Outcome measures

Measure	Lubiprostone 24 Mcg Twice Daily n=7 Participants Participants served as their own controls
Serum BUN Baseline	13.0 mg/dL Standard Deviation 4.6
Serum BUN 4 weeks	12.7 mg/dL Standard Deviation 3.3

SECONDARY outcome

Timeframe: baseline and 4 weeks

Population: Participants who completed the study

Outcome measures

Measure	Lubiprostone 24 Mcg Twice Daily n=7 Participants Participants served as their own controls
Serum Creatinine Baseline	0.79 mg/dL Standard Deviation 0.15
Serum Creatinine 4 weeks	0.84 mg/dL Standard Deviation 0.10

SECONDARY outcome

Timeframe: baseline and 4 weeks

Population: Participants who completed the study

Outcome measures

Measure	Lubiprostone 24 Mcg Twice Daily n=7 Participants Participants served as their own controls
AST Baseline	20.6 U/L Standard Deviation 11.1
AST 4 weeks	17.5 U/L Standard Deviation 3.9

SECONDARY outcome

Timeframe: baseline and 4 weeks

Population: Participants who completed the study

Outcome measures

Measure	Lubiprostone 24 Mcg Twice Daily n=7 Participants Participants served as their own controls
ALT Baseline	25.1 U/L Standard Deviation 16.7
ALT 4 weeks	23.8 U/L Standard Deviation 14.5

SECONDARY outcome

Timeframe: baseline and 4 weeks

Population: Participants who completed the study

Outcome measures

Measure	Lubiprostone 24 Mcg Twice Daily n=7 Participants Participants served as their own controls
Serum Calcium Baseline	9.3 mg/dL Standard Deviation 0.3
Serum Calcium 4 weeks	9.3 mg/dL Standard Deviation 0.2

SECONDARY outcome

Timeframe: baseline and 4 weeks

Population: Participants who completed the study

Outcome measures

Measure	Lubiprostone 24 Mcg Twice Daily n=7 Participants Participants served as their own controls
Serum Magnesium Baseline	2.0 mg/dL Standard Deviation 0.3
Serum Magnesium 4 weeks	2.0 mg/dL Standard Deviation 0.2

SECONDARY outcome

Timeframe: baseline and 4 weeks

Population: Participants who completed the study

Outcome measures

Measure	Lubiprostone 24 Mcg Twice Daily n=7 Participants Participants served as their own controls
Serum Phosphate Baseline	3.8 mg/dL Standard Deviation 0.6
Serum Phosphate 4 weeks	3.7 mg/dL Standard Deviation 0.5

SECONDARY outcome

Timeframe: baseline and 4 weeks

Population: Participants who completed the study

Outcome measures

Measure	Lubiprostone 24 Mcg Twice Daily n=7 Participants Participants served as their own controls
Serum Glucose Baseline	150.3 mg/dL Standard Deviation 101.9
Serum Glucose 4 weeks	131.8 mg/dL Standard Deviation 83.3

SECONDARY outcome

Timeframe: baseline and 4 weeks

Population: Participants who completed the study

Outcome measures

Measure	Lubiprostone 24 Mcg Twice Daily n=7 Participants Participants served as their own controls
Serum Vitamin D Baseline	25.3 ng/mL Standard Deviation 15.9
Serum Vitamin D 4 weeks	25.3 ng/mL Standard Deviation 18.4

SECONDARY outcome

Timeframe: baseline and 4 weeks

Population: Participants who completed the study

Outcome measures

Measure	Lubiprostone 24 Mcg Twice Daily n=7 Participants Participants served as their own controls
Serum Vitamin A Baseline	41.3 ug/dL Standard Deviation 14.6
Serum Vitamin A 4 weeks	42.7 ug/dL Standard Deviation 23.3

SECONDARY outcome

Timeframe: baseline and 4 weeks

Population: Participants who completed the study

Outcome measures

Measure	Lubiprostone 24 Mcg Twice Daily n=7 Participants Participants served as their own controls
Serum Vitamin E Baseline	6.6 mg/L Standard Deviation 3.4
Serum Vitamin E 4 weeks	7.7 mg/L Standard Deviation 6.1

SECONDARY outcome

Timeframe: baseline and 4 weeks

Population: Participants who completed the study

Outcome measures

Measure	Lubiprostone 24 Mcg Twice Daily n=7 Participants Participants served as their own controls
Serum Prealbumin Baseline	22.6 mg/dL Standard Deviation 3.3
Serum Prealbumin 4 weeks	21.5 mg/dL Standard Deviation 6.1

SECONDARY outcome

Timeframe: baseline and 4 weeks

Population: Participants who completed the study

Outcome measures

Measure	Lubiprostone 24 Mcg Twice Daily n=7 Participants Participants served as their own controls
Serum Albumin Baseline	4.3 g/dL Standard Deviation 0.3
Serum Albumin 4 weeks	4.3 g/dL Standard Deviation 0.2

Adverse Events

Lubiprostone

Serious events: 0 serious events

Other events: 6 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Lubiprostone n=7 participants at risk 24 micrograms twice daily
Gastrointestinal disorders Nausea	28.6% 2/7 • Number of events 3
Gastrointestinal disorders Abdominal bloating	14.3% 1/7 • Number of events 1
Gastrointestinal disorders Abdominal cramping	14.3% 1/7 • Number of events 2
Gastrointestinal disorders Heartburn	14.3% 1/7 • Number of events 1
General disorders Headache	14.3% 1/7 • Number of events 2
General disorders Migraine	14.3% 1/7 • Number of events 1
General disorders Insomnia	14.3% 1/7 • Number of events 2
Nervous system disorders Dizziness	14.3% 1/7 • Number of events 1
Respiratory, thoracic and mediastinal disorders Cough	14.3% 1/7 • Number of events 1
Respiratory, thoracic and mediastinal disorders Transient chest tightness	14.3% 1/7 • Number of events 1

Additional Information

Catherine O'Brien

University of Arkansas for Medical Sciences

Phone: 501-364-1828

Email: obriencatherinee@uams.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place