Asthma
Conditions
Brief summary
The objectives of this study are to evaluate the efficacy, tolerability and pharmacokinetics of 2 doses of MAP0010 (Unit Dose Budesonide) in asthmatic children/adolescents.
Interventions
DRUGMAP0010 low dose
a single dose of MAP0010 low dose delivered by nebulization twice daily for 6 weeks
a single dose of MAP0010 high dose delivered by nebulization twice daily for 6 weeks
DRUGPlacebo
Placebo delivered by nebulization twice daily for 6 weeks
Sponsors
MAP Pharmaceuticals, Inc., a wholly owned subsidiary of Allergan
Allergan
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Caregiver, Investigator)
Eligibility
Sex/Gender
ALL
Age
1 Years to 18 Years
Healthy volunteers
No
Inclusion criteria
* Male or female asthmatic children/adolescents * 1 to 18 years of age * FEV1 greater than or equal to 50% predicted normal (where obtainable) * Stable but symptomatic * Diagnosis of asthma (per NIH criteria) at least 3 months before screening OR documented exacerbation or worsening of asthma or symptoms suggestive of asthma including nocturnal asthma, within 6 months of screening OR documented SAB use more than or at least once for symptom relief during the 4 days of run in with a total symptom score greater than or equal to 1 OR greater than or equal to 1 night disturbed due to asthma symptoms in previous month.
Exclusion criteria
* Any other significant childhood illness. * Participated in any investigational clinical trial within the 30 days prior to screening. * Use of any corticosteroid within 2 weeks of screening. * Use of oral corticosteroid within 30 days of screening or prolonged use of oral corticosteroids within 12 weeks of screening. * Use of inhaled long acting bronchodilators. * Presumptive or documented history of upper or lower respiratory infection within 2 weeks before screening. * Any history of acute or severe asthma attack requiring ICU admission or ventilatory support. * History suggestive (or diagnosis) of other concomitant lung disease.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Daytime Composite Symptom Score | baseline, week 6 | The individual symptoms at each time point to be monitored are: cough, wheeze, and shortness of breath. The individual symptoms were scored using a four point scale: 0=no symptoms; 1=mild symptoms; 2=moderate symptoms; 3=severe symptoms Daily composite symptom score is based on the average of the individual symptom scores for a day. Daytime composite symptom score is defined as average of the last 5 days' daily composite symptom scores within the last 5 days immediately preceding the end day of that week. The range for the daytime composite symptom score is 0 (no symptoms) to 3 (severe symptoms). A negative change indicates an improvement of symptoms and a positive change indicates a worsening of symptoms. |
| Change From Baseline in Nighttime Composite Symptom Score | baseline, week 6 | The individual symptoms at each time point to be monitored are: cough, wheeze, and shortness of breath. The individual symptoms were scored using a four point scale: 0=no symptoms; 1=mild symptoms; 2=moderate symptoms; 3=severe symptoms Nightly composite symptom score is based on the average of the individual symptom scores for the night. Nightime composite symptom score is defined as average of the last 5 days' nightly composite symptom scores within the last 5 nights immediately preceding the end day of that week. The range for the nighttime composite symptom score is 0 (no symptoms) to 3 (severe symptoms). A negative change indicates an improvement of symptoms and a positive change indicates a worsening of symptoms. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in FEV1% Predicted | baseline, week 6 | The forced expiratory volume in 1 second (FEV1) is the amount forced of air exhaled in 1 second. The percent predicted is calculated for age, gender, and height. Subjects had to perform at least 3 acceptable maneuvers into a spirometer. An increase indicates an improvement (a greater volume of air expired). |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| MAP0010 High Dose a single dose of MAP0010 high dose delivered by nebulization twice daily for 6 weeks | 68 |
| MAP0010 Low Dose a single dose of MAP0010 low dose delivered by nebulization twice daily for 6 weeks | 65 |
| Placebo Placebo delivered by nebulization twice daily for 6 weeks | 65 |
| Total | 198 |
Baseline characteristics
| Characteristic | MAP0010 High Dose | MAP0010 Low Dose | Placebo | Total |
|---|---|---|---|---|
| Age, Continuous | 10.2 years STANDARD_DEVIATION 4.76 | 10.5 years STANDARD_DEVIATION 4.75 | 10.2 years STANDARD_DEVIATION 4.52 | 10.3 years STANDARD_DEVIATION 4.68 |
| Sex: Female, Male Female | 27 Participants | 25 Participants | 20 Participants | 72 Participants |
| Sex: Female, Male Male | 41 Participants | 40 Participants | 45 Participants | 126 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — | — / — |
| other Total, other adverse events | 21 / 69 | 10 / 67 | 20 / 69 |
| serious Total, serious adverse events | 0 / 69 | 0 / 67 | 0 / 69 |
Outcome results
Primary
Change From Baseline in Daytime Composite Symptom Score
The individual symptoms at each time point to be monitored are: cough, wheeze, and shortness of breath. The individual symptoms were scored using a four point scale: 0=no symptoms; 1=mild symptoms; 2=moderate symptoms; 3=severe symptoms Daily composite symptom score is based on the average of the individual symptom scores for a day. Daytime composite symptom score is defined as average of the last 5 days' daily composite symptom scores within the last 5 days immediately preceding the end day of that week. The range for the daytime composite symptom score is 0 (no symptoms) to 3 (severe symptoms). A negative change indicates an improvement of symptoms and a positive change indicates a worsening of symptoms.
Time frame: baseline, week 6
Population: Patients with available data at specified time points are included in the analysis population.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| MAP0010 High Dose | Change From Baseline in Daytime Composite Symptom Score | Baseline | 3.1 units on a scale | Standard Deviation 1.9 |
| MAP0010 High Dose | Change From Baseline in Daytime Composite Symptom Score | Change from Baseline at week 6 | -0.7 units on a scale | Standard Deviation 1.8 |
| MAP0010 Low Dose | Change From Baseline in Daytime Composite Symptom Score | Baseline | 2.8 units on a scale | Standard Deviation 1.75 |
| MAP0010 Low Dose | Change From Baseline in Daytime Composite Symptom Score | Change from Baseline at week 6 | -1.4 units on a scale | Standard Deviation 1.48 |
| Placebo | Change From Baseline in Daytime Composite Symptom Score | Baseline | 2.6 units on a scale | Standard Deviation 1.59 |
| Placebo | Change From Baseline in Daytime Composite Symptom Score | Change from Baseline at week 6 | -0.6 units on a scale | Standard Deviation 1.63 |
Primary
Change From Baseline in Nighttime Composite Symptom Score
The individual symptoms at each time point to be monitored are: cough, wheeze, and shortness of breath. The individual symptoms were scored using a four point scale: 0=no symptoms; 1=mild symptoms; 2=moderate symptoms; 3=severe symptoms Nightly composite symptom score is based on the average of the individual symptom scores for the night. Nightime composite symptom score is defined as average of the last 5 days' nightly composite symptom scores within the last 5 nights immediately preceding the end day of that week. The range for the nighttime composite symptom score is 0 (no symptoms) to 3 (severe symptoms). A negative change indicates an improvement of symptoms and a positive change indicates a worsening of symptoms.
Time frame: baseline, week 6
Population: Patients with available data at specified time points are included in the analysis population.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| MAP0010 High Dose | Change From Baseline in Nighttime Composite Symptom Score | Baseline | 2.7 units on a scale | Standard Deviation 1.96 |
| MAP0010 High Dose | Change From Baseline in Nighttime Composite Symptom Score | Change from Baseline at week 6 | -0.6 units on a scale | Standard Deviation 2 |
| MAP0010 Low Dose | Change From Baseline in Nighttime Composite Symptom Score | Baseline | 2.4 units on a scale | Standard Deviation 1.75 |
| MAP0010 Low Dose | Change From Baseline in Nighttime Composite Symptom Score | Change from Baseline at week 6 | -1.2 units on a scale | Standard Deviation 1.42 |
| Placebo | Change From Baseline in Nighttime Composite Symptom Score | Change from Baseline at week 6 | -0.4 units on a scale | Standard Deviation 1.6 |
| Placebo | Change From Baseline in Nighttime Composite Symptom Score | Baseline | 2.4 units on a scale | Standard Deviation 1.49 |
Secondary
Change From Baseline in FEV1% Predicted
The forced expiratory volume in 1 second (FEV1) is the amount forced of air exhaled in 1 second. The percent predicted is calculated for age, gender, and height. Subjects had to perform at least 3 acceptable maneuvers into a spirometer. An increase indicates an improvement (a greater volume of air expired).
Time frame: baseline, week 6
Population: Patients with available data at specified time points are included in the analysis population.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| MAP0010 High Dose | Change From Baseline in FEV1% Predicted | Baseline | 74.8 percentage of predicted FEV1 | Standard Deviation 11.45 |
| MAP0010 High Dose | Change From Baseline in FEV1% Predicted | Change from Baseline at 6 weeks | 6.3 percentage of predicted FEV1 | Standard Deviation 9.87 |
| MAP0010 Low Dose | Change From Baseline in FEV1% Predicted | Baseline | 73.8 percentage of predicted FEV1 | Standard Deviation 13.1 |
| MAP0010 Low Dose | Change From Baseline in FEV1% Predicted | Change from Baseline at 6 weeks | 6.3 percentage of predicted FEV1 | Standard Deviation 7.73 |
| Placebo | Change From Baseline in FEV1% Predicted | Baseline | 65.9 percentage of predicted FEV1 | Standard Deviation 7.22 |
| Placebo | Change From Baseline in FEV1% Predicted | Change from Baseline at 6 weeks | 5.9 percentage of predicted FEV1 | Standard Deviation 8.73 |