Cystic Fibrosis
Conditions
Keywords
CF
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.
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.
Interventions
DRUGEsomeprazole
DRUGPlacebo
Sugar pill
Sponsors
Columbia University
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Caregiver, Investigator)
Eligibility
Sex/Gender
ALL
Age
18 Years to 65 Years
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)
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| Forced Expiratory Volume in One Second (FEV1) | 36 weeks | Forced Expiratory Volume in one second (FEV1) as measured by spirometry |
| Forced Vital Capacity (FVC) | 36 weeks | Forced Vital Capacity percent predicted |
| 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 |
Countries
United States
Participant flow
Pre-assignment details
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.
Participants by arm
| Arm | Count |
|---|---|
| Esomeprazole Esomeprazole 40 mg twice daily | 9 |
| Placebo A matching placebo (sugar pill) to esomeprazole 40 mg twice daily. | 8 |
| Total | 17 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Lost to Follow-up | 1 | 0 |
| Overall Study | Lung Transplant | 0 | 1 |
Baseline characteristics
| Characteristic | Esomeprazole | Placebo | Total |
|---|---|---|---|
| Age, Continuous | 35.72 years STANDARD_DEVIATION 9.6 | 32.81 years STANDARD_DEVIATION 5.84 | NA years |
| FEV1 | 58 percent predicted STANDARD_DEVIATION 19 | 46 percent predicted STANDARD_DEVIATION 21 | NA percent predicted |
| Forced Vital Capacity (FVC) | 74 Percent Predicted STANDARD_DEVIATION 20 | 71 Percent Predicted STANDARD_DEVIATION 16 | NA Percent Predicted |
| Number of exacerbations in the past two years | 4 Count of exacerbations STANDARD_DEVIATION 0 | 5.5 Count of exacerbations STANDARD_DEVIATION 1.4 | NA Count of exacerbations |
| Race and Ethnicity Not Collected | — | — | 0 Participants |
| Region of Enrollment United States | 9 Participants | 8 Participants | 17 Participants |
| Sex: Female, Male Female | 3 Participants | 2 Participants | 5 Participants |
| Sex: Female, Male Male | 6 Participants | 6 Participants | 12 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 9 | 0 / 8 |
| other Total, other adverse events | 0 / 9 | 0 / 8 |
| serious Total, serious adverse events | 0 / 9 | 0 / 8 |
Outcome results
Primary
Time to First Pulmonary Exacerbation
Time to need for oral or intravenous antibiotics for treatment of a pulmonary exacerbation is the primary outcome measure.
Time frame: 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.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Esomeprazole | Time to First Pulmonary Exacerbation | NA Weeks |
| Placebo | Time to First Pulmonary Exacerbation | NA Weeks |
Secondary
Forced Expiratory Volume in One Second (FEV1)
Forced Expiratory Volume in one second (FEV1) as measured by spirometry
Time frame: 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.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Esomeprazole | Forced Expiratory Volume in One Second (FEV1) | NA percentage of predicted |
| Placebo | Forced Expiratory Volume in One Second (FEV1) | NA percentage of predicted |
Secondary
Forced Vital Capacity (FVC)
Forced Vital Capacity percent predicted
Time frame: 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.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Esomeprazole | Forced Vital Capacity (FVC) | NA percentage of predicted |
| Placebo | Forced Vital Capacity (FVC) | NA percentage of predicted |
Secondary
Number of Exacerbations
Exacerbation defined as initiation of treatment with intravenous or oral antibiotics for 7 or more days based on respiratory symptoms
Time frame: 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.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Esomeprazole | Number of Exacerbations | NA Number of exacerbations |
| Placebo | Number of Exacerbations | NA Number of exacerbations |