Trial Outcomes & Findings for Genotype-tailored Treatment of Symptomatic Acid-Reflux in Children With Uncontrolled Asthma (NCT NCT03015610)
NCT ID: NCT03015610
Last Updated: 2025-09-09
Results Overview
The ACQ considers a broad set of common indicators of asthma control including use of bronchodilators, cough, nocturnal symptoms, level of activity, and pulmonary function. Scores range between 0 (totally controlled) and 6 (severely uncontrolled). Reported is the change from week 0 to week 26; a negative change value indicates an improvement in asthma control.
COMPLETED
PHASE3
41 participants
Week 0 (baseline) to week 26
2025-09-09
Participant Flow
Participant milestones
| Measure |
Placebo
Participants will receive oral blinded matched placebo once daily.
|
Genotype-guided Lansoprazole
Participants will receive oral blinded commercially available lansoprazole once daily with a dose appropriate for the participant's metabolizer phenotype.
|
|---|---|---|
|
Overall Study
STARTED
|
17
|
18
|
|
Overall Study
COMPLETED
|
16
|
13
|
|
Overall Study
NOT COMPLETED
|
1
|
5
|
Reasons for withdrawal
| Measure |
Placebo
Participants will receive oral blinded matched placebo once daily.
|
Genotype-guided Lansoprazole
Participants will receive oral blinded commercially available lansoprazole once daily with a dose appropriate for the participant's metabolizer phenotype.
|
|---|---|---|
|
Overall Study
Lost to Follow-up
|
1
|
5
|
Baseline Characteristics
Genotype-tailored Treatment of Symptomatic Acid-Reflux in Children With Uncontrolled Asthma
Baseline characteristics by cohort
| Measure |
Placebo
n=17 Participants
Participants will receive oral blinded matched placebo once daily.
|
Genotype-guided Lansoprazole
n=18 Participants
Participants will receive oral blinded commercially available lansoprazole once daily with a dose appropriate for the participant's metabolizer phenotype.
|
Total
n=35 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
9.8 years
STANDARD_DEVIATION 3.6 • n=5 Participants
|
10.8 years
STANDARD_DEVIATION 3.3 • n=7 Participants
|
10.3 years
STANDARD_DEVIATION 3.5 • n=5 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=5 Participants
|
11 Participants
n=7 Participants
|
17 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
11 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
18 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
14 Participants
n=5 Participants
|
15 Participants
n=7 Participants
|
29 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
9 Participants
n=5 Participants
|
11 Participants
n=7 Participants
|
20 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
6 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
2 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
17 Participants
n=5 Participants
|
18 Participants
n=7 Participants
|
35 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Week 0 (baseline) to week 26Population: Participants with data collected at both timepoints.
The ACQ considers a broad set of common indicators of asthma control including use of bronchodilators, cough, nocturnal symptoms, level of activity, and pulmonary function. Scores range between 0 (totally controlled) and 6 (severely uncontrolled). Reported is the change from week 0 to week 26; a negative change value indicates an improvement in asthma control.
Outcome measures
| Measure |
Placebo
n=15 Participants
Participants will receive oral blinded matched placebo once daily.
|
Genotype-guided Lansoprazole
n=13 Participants
Participants will receive oral blinded commercially available lansoprazole once daily with a dose appropriate for the participant's metabolizer phenotype.
|
|---|---|---|
|
Change in Asthma Control Questionnaire (ACQ) From Screening Through Week 26
|
-0.5 score on a scale
Standard Deviation 0.5
|
-0.6 score on a scale
Standard Deviation 0.9
|
SECONDARY outcome
Timeframe: Week 0 (baseline) to week 26Population: Participants with data collected at both timepoints.
A 10-item tool that has been validated in children in the assessment of gastroesophageal reflux disease related symptoms such as chest/abdominal pain, pain/choking with eating, swallowing dysfunction, regurgitation and nausea. It assesses symptom frequency and severity from the previous 7-days on an 8-point scale with 0 and 7 indicating the least and greatest severity, respectively. The total score ranges from 0 to 70, where a higher score indicates greater GERD symptom severity. Reported is the change from week 0 to week 26.
Outcome measures
| Measure |
Placebo
n=15 Participants
Participants will receive oral blinded matched placebo once daily.
|
Genotype-guided Lansoprazole
n=14 Participants
Participants will receive oral blinded commercially available lansoprazole once daily with a dose appropriate for the participant's metabolizer phenotype.
|
|---|---|---|
|
Change in GERD (Gastroesophageal Reflux Disease) Symptom Assessment Questionnaire Score (GSAS) From Screening Through Week 26
|
-32.3 score on a scale
Standard Deviation 18.7
|
23.4 score on a scale
Standard Deviation 21.4
|
SECONDARY outcome
Timeframe: Week 0 (baseline) to week 26Population: Participants with data collected at both timepoints.
Questionnaire measures changes in asthma control. Each of the 11 items are scored on a 4-point Likert scale to assess frequency (not at all, 1 to 3 days, 4 to 7 days, and 8 to 14 days) and severity (not applicable, mild, moderate, and severe). The adjusted overall score ranges from 0 (worst possible symptoms) to 1 (no symptoms). Reported is the change from week 0 to week 26.
Outcome measures
| Measure |
Placebo
n=12 Participants
Participants will receive oral blinded matched placebo once daily.
|
Genotype-guided Lansoprazole
n=9 Participants
Participants will receive oral blinded commercially available lansoprazole once daily with a dose appropriate for the participant's metabolizer phenotype.
|
|---|---|---|
|
Change in Asthma Symptom Utility Index (ASUI) From Screening Through Week 26
|
0 score on a scale
Standard Deviation 0.2
|
-0.1 score on a scale
Standard Deviation 0.3
|
SECONDARY outcome
Timeframe: Up to week 26An exacerbation will be defined per the recommendations of the NIH Asthma Exacerbation Taskforce and will be defined as a worsening of asthma requiring the use of a systemic corticosteroid (at least 3 days of prednisolone/ prednisone or ≥1 days of dexamethasone) to prevent asthma worsening.
Outcome measures
| Measure |
Placebo
n=17 Participants
Participants will receive oral blinded matched placebo once daily.
|
Genotype-guided Lansoprazole
n=18 Participants
Participants will receive oral blinded commercially available lansoprazole once daily with a dose appropriate for the participant's metabolizer phenotype.
|
|---|---|---|
|
Annualized Rate of Asthma Exacerbations
|
0 exacerbations per participant per year
Interval 0.0 to 0.0
|
0 exacerbations per participant per year
Interval 0.0 to 2.0
|
SECONDARY outcome
Timeframe: Up to week 26A study EPAC will be present if the participant meets any of the following criteria, (1) addition of systemic corticosteroid medication for asthma or (2) any unscheduled encounter to a non-study related health care provider (phone contact, ED, urgent care, hospital) for asthma symptoms.
Outcome measures
| Measure |
Placebo
n=17 Participants
Participants will receive oral blinded matched placebo once daily.
|
Genotype-guided Lansoprazole
n=18 Participants
Participants will receive oral blinded commercially available lansoprazole once daily with a dose appropriate for the participant's metabolizer phenotype.
|
|---|---|---|
|
Annualized Rate of Episodes of Poor Asthma Control (EPAC)
|
0 episodes per participant per year
Interval 0.0 to 0.0
|
0 episodes per participant per year
Interval 0.0 to 2.08
|
SECONDARY outcome
Timeframe: Up to week 26Participants/Caregivers will be asked to report diagnoses of RTI that occurred during the treatment period using interval reporting. RTI will include: (1) bronchitis, (2) otitis, (3) pneumonia.
Outcome measures
| Measure |
Placebo
n=17 Participants
Participants will receive oral blinded matched placebo once daily.
|
Genotype-guided Lansoprazole
n=18 Participants
Participants will receive oral blinded commercially available lansoprazole once daily with a dose appropriate for the participant's metabolizer phenotype.
|
|---|---|---|
|
Annualized Rate of Respiratory Tract Infection (RTI)
|
4.16 RTIs per participant per year
Interval 2.26 to 12.0
|
4.0 RTIs per participant per year
Interval 1.86 to 4.16
|
SECONDARY outcome
Timeframe: Week -2 (screening) to week 26Population: Participants with data collected at both timepoints.
Forced Expiratory Volume in 1 Second (FEV1) measurement (mean change in FEV1 percent predicted from screening).
Outcome measures
| Measure |
Placebo
n=14 Participants
Participants will receive oral blinded matched placebo once daily.
|
Genotype-guided Lansoprazole
n=12 Participants
Participants will receive oral blinded commercially available lansoprazole once daily with a dose appropriate for the participant's metabolizer phenotype.
|
|---|---|---|
|
Change in Lung Function Testing From Screening Through Week 26
|
4.4 percentage of predicted value
Standard Deviation 16.1
|
-3.7 percentage of predicted value
Standard Deviation 17.6
|
Adverse Events
Placebo
Genotype-guided Lansoprazole
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Placebo
n=17 participants at risk
participants will receive oral blinded matched placebo once daily
matched placebo: these participants will receive a one-time dose of lansoprazole followed by PK analysis and then once daily placebo for 24 weeks
|
Genotype-guided Lansoprazole
n=18 participants at risk
participants will receive oral blinded commercially available lansoprazole once daily with a dose appropriate for the participant's metabolizer phenotype
commercially available lansoprazole: these participants will receive a one-time dose of lansoprazole followed by PK analysis and then once daily lansoprazole for 24 weeks
|
|---|---|---|
|
Nervous system disorders
Headaches
|
64.7%
11/17 • 24 weeks
|
83.3%
15/18 • 24 weeks
|
|
Gastrointestinal disorders
Nausea
|
47.1%
8/17 • 24 weeks
|
50.0%
9/18 • 24 weeks
|
|
Gastrointestinal disorders
Bloating
|
17.6%
3/17 • 24 weeks
|
5.6%
1/18 • 24 weeks
|
|
Gastrointestinal disorders
Diarrhea
|
47.1%
8/17 • 24 weeks
|
38.9%
7/18 • 24 weeks
|
|
Gastrointestinal disorders
Constipation
|
47.1%
8/17 • 24 weeks
|
16.7%
3/18 • 24 weeks
|
|
Gastrointestinal disorders
Flatulence
|
64.7%
11/17 • 24 weeks
|
55.6%
10/18 • 24 weeks
|
|
Skin and subcutaneous tissue disorders
Skin rash
|
23.5%
4/17 • 24 weeks
|
22.2%
4/18 • 24 weeks
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place