Effect of Proton Pump Inhibitors on CF Pulmonary Exacerbations

• Sponsor: Columbia University (Other)
• Overall Status: Completed
• CT.gov ID: NCT01983774
• Collaborator: (none)
• Enrollment: 21
• Location: 1
• Arms: 2
• Duration (Months): 42
• Patients Per Site Per Month: 0.5

Study Details

Study Description

Brief Summary

Gastroesophageal reflux disease (GERD) is a common problem in cystic fibrosis (CF). It may lead to worsening lung function and more respiratory infections for a person with CF. This study will look at treating GERD with a medication, esomeprazole. The medication stops stomach acid from being made. The study will see if there are fewer respiratory infections and improved lung function in patients with CF when taking esomeprazole.

Condition or Disease	Intervention/Treatment	Phase
Cystic Fibrosis	Drug: Esomeprazole; Drug: Placebo	Phase 2

Detailed Description

This is a randomized, placebo controlled intervention study in patients with CF who have a history of frequent exacerbations. Treatment duration is 6 months.

Study Design

• Study Type: Interventional
• Actual Enrollment: 21 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Triple (Participant, Care Provider, Investigator)
• Primary Purpose: Treatment
• Official Title: Effect of Proton Pump Inhibitors on CF Exacerbations
• Study Start Date: Feb 1, 2008
• Actual Primary Completion Date: Aug 1, 2011
• Actual Study Completion Date: Aug 1, 2011

Arms and Interventions

Arm	Intervention/Treatment
Placebo Comparator: Placebo A matching placebo (sugar pill) to esomeprazole 40mg twice daily	Drug: Placebo; Sugar pill
Active Comparator: Esomeprazole Esomeprazole 40mg twice daily	Drug: Esomeprazole; Other Names: Nexium

Outcome Measures

Primary Outcome Measures

1. Time to First Pulmonary Exacerbation [36 weeks]

   Time to need for oral or intravenous antibiotics for treatment of a pulmonary exacerbation is the primary outcome measure.

Secondary Outcome Measures

1. Forced Expiratory Volume in One Second (FEV1) [36 weeks]

   Forced Expiratory Volume in one second (FEV1) as measured by spirometry

2. Forced Vital Capacity (FVC) [36 weeks]

   Forced Vital Capacity percent predicted

3. Number of Exacerbations [36 weeks]

   Exacerbation defined as initiation of treatment with intravenous or oral antibiotics for 7 or more days based on respiratory symptoms

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years to 65 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

1. Cystic fibrosis

2. Age > 18 years

3. Stable maintenance medical regimen during the previous 6 weeks.

4. Negative pregnancy test (women of childbearing potential) at both screening and baseline visits.

5. Women of childbearing potential must be using medically acceptable contraception.

6. At least two respiratory exacerbations per year requiring oral and/or intravenous antibiotics for each of the two years prior to study entry, but no more than 4 exacerbations requiring intravenous antibiotics during either of those years.

Exclusion Criteria:

1. Previous anti-reflux or peptic ulcer surgery,

2. Use of proton pump inhibitor (PPI) within the past two weeks

3. Pulmonary exacerbation requiring antibiotics within the previous 2 weeks

4. Parenteral hyperalimentation

5. Cigarette smoking

6. Treatment with azoles, iron, anti-coagulants, digitalis

7. Use of any investigative drugs within the previous month.

8. Use of over the counter acid suppressor agents (not including acid neutralizers)

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Columbia University Cystic Fibrosis Center	New York	New York	United States	10003

Sponsors and Collaborators

• Columbia University

Investigators

• Principal Investigator: Emily DiMango, MD, Columbia University

Study Documents (Full-Text)

None provided.

More Information

Additional Information:

• Results Reference

Publications

None provided.

• Responsible Party: Emily DiMango, MD, Associate Professor, Columbia University
• ClinicalTrials.gov Identifier: NCT01983774
• Other Study ID Numbers: AAAC5640
• First Posted: Nov 14, 2013
• Last Update Posted: Feb 17, 2022
• Last Verified: Jan 1, 2022

• Keywords provided by Emily DiMango, MD, Associate Professor, Columbia University: CF
• Additional relevant MeSH terms: Cystic Fibrosis; Esomeprazole

Study Results

Participant Flow

Pre-assignment Detail	Twenty-one participants were assessed for eligibility. Of those, four were excluded (two did not meet inclusion criteria and two declined participation). Seventeen participants were randomized.

Arm/Group Title	Esomeprazole	Placebo
Arm/Group Description	Esomeprazole 40mg twice daily Esomeprazole	A matching placebo (sugar pill) to esomeprazole 40mg twice daily Placebo: Sugar pill
Period Title: Overall Study
STARTED	9	8
COMPLETED	8	7
NOT COMPLETED	1	1

Baseline Characteristics

Arm/Group Title	Esomeprazole	Placebo	Total
Arm/Group Description	Esomeprazole 40 mg twice daily	A matching placebo (sugar pill) to esomeprazole 40 mg twice daily.	Total of all reporting groups
Overall Participants	9	8	17
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	35.72 (9.6)	32.81 (5.84)	NA (NA)
Sex: Female, Male (Count of Participants)
Female	3 33.3%	2 25%	5 29.4%
Male	6 66.7%	6 75%	12 70.6%
Race and Ethnicity Not Collected (Count of Participants)
Count of Participants [Participants]	0 0%
Region of Enrollment (Count of Participants)
United States	9 100%	8 100%	17 100%
FEV1 (percent predicted) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [percent predicted]	58 (19)	46 (21)	NA (NA)
Forced Vital Capacity (FVC) (Percent Predicted) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [Percent Predicted]	74 (20)	71 (16)	NA (NA)
Number of exacerbations in the past two years (Count of exacerbations) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [Count of exacerbations]	4 (0)	5.5 (1.4)	NA (NA)

Outcome Measures

1. Primary Outcome

Title	Time to First Pulmonary Exacerbation
Description	Time to need for oral or intravenous antibiotics for treatment of a pulmonary exacerbation is the primary outcome measure.
Time Frame	36 weeks

Outcome Measure Data

Analysis Population Description

Values per arm for time to first pulmonary exacerbation cannot be reported. The data set was destroyed and no longer exists. The only available results exist as a figure within the study publication.

Arm/Group Title	Esomeprazole	Placebo
Arm/Group Description	Esomeprazole 40mg twice daily Esomeprazole	A matching placebo (sugar pill) to esomeprazole 40mg twice daily Placebo: Sugar pill
Measure Participants	9	8
Mean (Standard Deviation) [Weeks]	NA (NA)	NA (NA)

2. Secondary Outcome

Title	Forced Expiratory Volume in One Second (FEV1)
Description	Forced Expiratory Volume in one second (FEV1) as measured by spirometry
Time Frame	36 weeks

Outcome Measure Data

Analysis Population Description

Values per arm for FEV1 cannot be reported. The data set was destroyed and no longer exists. The only available results exist as a figure within the study publication.

Arm/Group Title	Esomeprazole	Placebo
Arm/Group Description	Esomeprazole 40mg twice daily	A matching placebo (sugar pill) to esomeprazole 40mg twice daily
Measure Participants	9	8
Mean (Standard Deviation) [percentage of predicted]	NA (NA)	NA (NA)

3. Secondary Outcome

Title	Forced Vital Capacity (FVC)
Description	Forced Vital Capacity percent predicted
Time Frame	36 weeks

Outcome Measure Data

Analysis Population Description

Values per arm for FVC cannot be reported. The data set was destroyed and no longer exists. The only available results exist as a figure within the study publication.

Arm/Group Title	Esomeprazole	Placebo
Arm/Group Description	Esomeprazole 40mg twice daily Esomeprazole	A matching placebo (sugar pill) to esomeprazole 40mg twice daily Placebo: Sugar pill
Measure Participants	9	8
Mean (Standard Deviation) [percentage of predicted]	NA (NA)	NA (NA)

4. Secondary Outcome

Title	Number of Exacerbations
Description	Exacerbation defined as initiation of treatment with intravenous or oral antibiotics for 7 or more days based on respiratory symptoms
Time Frame	36 weeks

Outcome Measure Data

Analysis Population Description

Values per arm for number of exacerbations cannot be reported. The data set was destroyed and no longer exists. The only available results exist in the study publication.

Arm/Group Title	Esomeprazole	Placebo
Arm/Group Description	Esomeprazole 40mg twice daily Esomeprazole	A matching placebo (sugar pill) to esomeprazole 40mg twice daily Placebo: Sugar pill
Measure Participants	9	8
Mean (Standard Deviation) [Number of exacerbations]	NA (NA)	NA (NA)

Adverse Events

Arm/Group Title	Esomeprazole		Placebo
Arm/Group Description	Esomeprazole 40mg twice daily Esomeprazole		A matching placebo (sugar pill) to esomeprazole 40mg twice daily Placebo: Sugar pill
All Cause Mortality
	Esomeprazole		Placebo
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/9 (0%)		0/8 (0%)
Serious Adverse Events
	Esomeprazole		Placebo
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/9 (0%)		0/8 (0%)
Other (Not Including Serious) Adverse Events
	Esomeprazole		Placebo
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/9 (0%)		0/8 (0%)

Limitations/Caveats

Study findings are limited by small sample size without adequate power to detect significant differences between participants treated with esomeprazole compared with placebo.

More Information

Certain Agreements

All Principal Investigators ARE employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.