Trial Outcomes & Findings for Effect of Proton Pump Inhibitors on CF Pulmonary Exacerbations (NCT NCT01983774)

Results Overview

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

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

21 participants

Primary outcome timeframe

36 weeks

Results posted on

2022-02-17

Participant Flow

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.

Participant milestones

Participant milestones
Measure	Esomeprazole Esomeprazole 40mg twice daily Esomeprazole	Placebo A matching placebo (sugar pill) to esomeprazole 40mg twice daily Placebo: Sugar pill
Overall Study STARTED	9	8
Overall Study COMPLETED	8	7
Overall Study NOT COMPLETED	1	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Esomeprazole Esomeprazole 40mg twice daily Esomeprazole	Placebo A matching placebo (sugar pill) to esomeprazole 40mg twice daily Placebo: Sugar pill
Overall Study Lung Transplant	0	1
Overall Study Lost to Follow-up	1	0

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Esomeprazole n=9 Participants Esomeprazole 40 mg twice daily	Placebo n=8 Participants A matching placebo (sugar pill) to esomeprazole 40 mg twice daily.	Total n=17 Participants Total of all reporting groups
Age, Continuous	35.72 years STANDARD_DEVIATION 9.6 • n=9 Participants	32.81 years STANDARD_DEVIATION 5.84 • n=8 Participants	NA years STANDARD_DEVIATION NA • n=17 Participants
Sex: Female, Male Female	3 Participants n=9 Participants	2 Participants n=8 Participants	5 Participants n=17 Participants
Sex: Female, Male Male	6 Participants n=9 Participants	6 Participants n=8 Participants	12 Participants n=17 Participants
Race and Ethnicity Not Collected	—	—	0 Participants Race and Ethnicity were not collected from any participant.
Region of Enrollment United States	9 Participants n=9 Participants	8 Participants n=8 Participants	17 Participants n=17 Participants
FEV1	58 percent predicted STANDARD_DEVIATION 19 • n=9 Participants	46 percent predicted STANDARD_DEVIATION 21 • n=8 Participants	NA percent predicted STANDARD_DEVIATION NA • n=17 Participants
Forced Vital Capacity (FVC)	74 Percent Predicted STANDARD_DEVIATION 20 • n=9 Participants	71 Percent Predicted STANDARD_DEVIATION 16 • n=8 Participants	NA Percent Predicted STANDARD_DEVIATION NA • n=17 Participants
Number of exacerbations in the past two years	4 Count of exacerbations STANDARD_DEVIATION 0 • n=9 Participants	5.5 Count of exacerbations STANDARD_DEVIATION 1.4 • n=8 Participants	NA Count of exacerbations STANDARD_DEVIATION NA • n=17 Participants

PRIMARY outcome

Timeframe: 36 weeks

Population: 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.

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

Outcome measures

Outcome measures
Measure	Esomeprazole n=9 Participants Esomeprazole 40mg twice daily Esomeprazole	Placebo n=8 Participants A matching placebo (sugar pill) to esomeprazole 40mg twice daily Placebo: Sugar pill
Time to First Pulmonary Exacerbation	NA Weeks Standard Deviation NA Time to first pulmonary exacerbation for esomeprazole arm cannot be reported. The data set was destroyed and no longer exists. The only available results exist as a figure within the study publication.	NA Weeks Standard Deviation NA Time to first pulmonary exacerbation for placebo arm cannot be reported. The data set was destroyed and no longer exists. The only available results exist as a figure within the study publication.

SECONDARY outcome

Timeframe: 36 weeks

Population: 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.

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

Outcome measures

Outcome measures
Measure	Esomeprazole n=9 Participants Esomeprazole 40mg twice daily Esomeprazole	Placebo n=8 Participants A matching placebo (sugar pill) to esomeprazole 40mg twice daily Placebo: Sugar pill
Forced Expiratory Volume in One Second (FEV1)	NA percentage of predicted Standard Deviation NA Values for the esomeprazole arm cannot be reported. The data set was destroyed and no longer exists. The only available results exist as a figure within the study publication.	NA percentage of predicted Standard Deviation NA Values for the placebo arm cannot be reported. The data set was destroyed and no longer exists. The only available results exist as a figure within the study publication.

SECONDARY outcome

Timeframe: 36 weeks

Population: 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.

Forced Vital Capacity percent predicted

Outcome measures

Outcome measures
Measure	Esomeprazole n=9 Participants Esomeprazole 40mg twice daily Esomeprazole	Placebo n=8 Participants A matching placebo (sugar pill) to esomeprazole 40mg twice daily Placebo: Sugar pill
Forced Vital Capacity (FVC)	NA percentage of predicted Standard Deviation NA Values for the esomeprazole arm cannot be reported. The data set was destroyed and no longer exists. The only available results exist as a figure within the study publication.	NA percentage of predicted Standard Deviation NA Values for the placebo arm cannot be reported. The data set was destroyed and no longer exists. The only available results exist as a figure within the study publication.

SECONDARY outcome

Timeframe: 36 weeks

Population: 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.

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

Outcome measures

Outcome measures
Measure	Esomeprazole n=9 Participants Esomeprazole 40mg twice daily Esomeprazole	Placebo n=8 Participants A matching placebo (sugar pill) to esomeprazole 40mg twice daily Placebo: Sugar pill
Number of Exacerbations	NA Number of exacerbations Standard Deviation NA Values for the esomeprazole arm cannot be reported. The data set was destroyed and no longer exists. The only available results exist as listed in the study publication.	NA Number of exacerbations Standard Deviation NA Values for the placebo arm cannot be reported. The data set was destroyed and no longer exists. The only available results exist as listed in the study publication.

Adverse Events

Esomeprazole

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Emily DiMango, MD

Columbia University

Phone: 212-305-0631

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place