Efficacy and Safety of Indacaterol Acetate Delivered Via the Concept1 Inhalation Device in Children Greater or Equal to 6 and Less Than 12 Years of Age With Asthma

Study Overview
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Brief Summary
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This study is designed to explore lung function effects of two doses of indacaterol acetate, 75 μg and 150 μg, in pediatric asthma patients 6-11 years old, and to compare the systemic exposure to indacaterol in plasma with historical data in adults, to identify an appropriate dose to Phase III evaluation.

Official Title
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A Multicenter, Randomized, Double-blind, Active-controlled, 2 Week Treatment, Parallel-group Study to Assess the Efficacy and Safety of Indacaterol Acetate Delivered Via the Concept1 Inhalation Device in Children Greater or Equal to 6 and Less Than 12 Years of Age With Asthma

Conditions
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Asthma

Intervention / Treatment
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* Drug: Indacaterol acetate 75 μg
* Drug: Indacaterol acetate 150 μg


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Male and female children ≥ 6 years and < 12 years with confirmed diagnosis of asthma for at least 1 year prior to study enrollment. Written informed consent by parent(s)/legal guardian(s) and depending upon their age and local requirements a consent or assent for the patient. Patients receiving daily treatment with a stable low dose Inhaled Corticosteroid (ICS) (with or without additional controller), or patients receiving daily treatment with a stable mid-dose ICS (monotherapy or together with LTRA) for at least 4 weeks prior to Screening, and able to tolerate fluticasone propionate 100 μg b.i.d. inhaler starting at Visit 1 (or soon after). Patients with a pre-bronchodilator FEV1 ≥ 50% and ≤ 90% of the predicted normal value for the patient at the start and end of Run-in (Visits 101 and 199). Patients who demonstrate an increase in FEV1 of 12% within 30 minutes after administration of 400 μg salbutamol/360 μg albuterol (or equivalent dose) at Visit 101. All patients must perform a reversibility test at Visit 101. Exclusion Criteria: Patients taking a mid-dose ICS (per GINA guidelines) in combination with LABA or any patient taking high-dose ICS. Evidence of unstable disease within 4 weeks prior to Screening (Visit 1). Patients who have had an asthma attack/exacerbation requiring systemic steroids (SCS) or hospitalization or emergency room visit within 3 months prior to Visit 1 (Screening) or more than 3 separate exacerbations in the 12 months preceding Visit 1. Suspected or documented bacterial or viral infection of the upper or lower respiratory tract, sinus or middle ear that is not resolved within 4 weeks of Screening (Visit 1). Prior intubation for asthma.

Ages Eligible for Study
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Minimum Age: 6 Years
Maximum Age: 11 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: Indacaterol acetate 75 μg o.d.<br>Indacaterol acetate 75 μg o.d. delivered via Concept1 inhaler | Drug: Indacaterol acetate 75 μg<br>* Indacaterol acetate 75 μg o.d. delivered via Concept1 inhaler<br>* Other names: QAB149;|
| Active Comparator: Indacaterol acetate 150 μg o.d.<br>Indacaterol acetate 150 μg o.d. delivered via Concept1 inhaler | Drug: Indacaterol acetate 150 μg<br>* Indacaterol acetate 150 μg o.d. delivered via Concept1 inhaler<br>* Other names: QAB149;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Trough Forced Expiratoty Volume in 1 Second (FEV1) | Change from baseline in pre-dose trough FEV1 after 2 weeks of treatment with indacaterol acetate 75 μg o.d and 150 μg o.d. The primary endpoint is change from baseline in pre-dose trough FEV1 (mL) after 2 weeks of treatment. The pre-dose trough FEV1 (mL) is defined as the mean of the two FEV1 (mL), values measured at -45 min and -15 min pre-dose. | 2 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Systemic Exposure to Indacaterol in Plasma | Systemic exposure to indacaterol in plasma following sparse pharmacokinetic (PK) sampling on Day 1 and Day 14 after inhalation of indacaterol acetate 75 μg and 150 μg. | day 1, day 14 | 
| Asthma Control as Assessed by Pediatric Interviewer-administered Asthma Control Questionnaire | Asthma Control as assessed by pediatric interviewer-administered Asthma Control Questionnaire (ACQ-IA) score at week 2 (mean change). A decrease in the score shows an improvement. The scale ranges from 0 (no symptoms) to 6 (severe symptoms every day). Results are given as a change as compared from baseline | 2 weeks | 
| Pre-dose Morning and Evening Peak Expiratoty Flow (PEF) | Pre-dose morning and evening PEF over 2 weeks of treatment as determined by electronic peak flow meter data (mean change) | 2 weeks | 
| Rescue Medication Usage (Mean Daiily Number of Puffs) | Rescue medication usage over 2 weeks of treatment as determined by patient diary data for indacaterol acetate 75 μg and 150 μg o.d.Results given as mean change of puffs of rescue medication. | 2 weeks | 
| Rescue Medication Usage (Percentage of Rescue Medication Free Days) | Rescue medication usage over 2 weeks of treatment as determined by patient diary data for indacaterol acetate 75 μg and 150 μg o.d. | 2 weeks | 
| Forced Expiratoty Volume in 1 Second (FEV1) and Forced Vital Capacity (FVC) | FEV1 and FVC at 30 minutes and 1-hour post dose at week 2 for indacaterol acetate 75 μg and 150 μg o.d. | 2 weeks | 
| Symptoms as Recorded by Patient E-diary (Mean Total Daily Symptom Score) | Symptoms as recorded by patient e-diary for indacaterol acetate 75 μg and 150 μg o.d. (mean change) The scale rages from 0 (no problem) - 4 (very severe problems). | 2 weeks | 
| Symptoms as Recorded by Patient E-diary (Percentage of Asthma Symptoms Free Days) | Symptoms as recorded by patient e-diary for indacaterol acetate 75 μg and 150 μg o.d. (mean change) | 2 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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indacaterol acetate, asthma, GINA 2015, pediatrics