E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
lung function effects of two doses of indacaterol acetate, 75 μg and 150 μg, in pediatric asthma patients 6-11 years old |
|
E.1.1.1 | Medical condition in easily understood language |
pediatric asthma patients 6-11 years old |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Respiratory Tract Diseases [C08] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10003553 |
E.1.2 | Term | Asthma |
E.1.2 | System Organ Class | 10038738 - Respiratory, thoracic and mediastinal disorders |
|
E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
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 |
|
E.2.2 | Secondary objectives of the trial |
Systemic exposure to indacaterol in plasma |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal 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. |
|
E.4 | Principal 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. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Primary Outcome Measure:
• Trough FEV1 [Time Frame: 2 weeks] [Designated as safety issue: No]
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. |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
|
E.5.2 | Secondary end point(s) |
Secondary Outcome Measures:
• Systemic exposure to indacaterol in plasma [Time Frame: 2 weeks]
[Designated as safety issue: No]
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.
• Asthma Control as assessed by pediatric interviewer-administered Asthma
Control Questionnaire [Time Frame: 2 weeks] [Designated as safety
issue: No]
Asthma Control as assessed by pediatric interviewer-administered
Asthma Control Questionnaire (ACQ-IA) score at week 2 for
indacaterol acetate 75 μg and 150 μg o.d.
• FEV1 and FVC [Time Frame: 2 weeks] [Designated as safety issue: No]
FEV1 and FVC at 30 minutes and 1-hour post dose at week 2 for
indacaterol acetate 75 μg and 150 μg o.d.
• Rescue medication usage [Time Frame: 2 weeks] [Designated as safety
issue: No]
Rescue medication usage over 2 weeks of treatment as determined
by patient diary data for indacaterol acetate 75 μg and 150 μg o.d.
• Symptoms as recorded by patient e-diary [Time Frame: 2 weeks]
[Designated as safety issue: No]
Symptoms as recorded by patient e-diary for indacaterol acetate 75
μg and 150 μg o.d.
• Pre-dose morning and evening PEF [Time Frame: 2 weeks] [Designated
as safety issue: No]
Pre-dose morning and evening PEF over 2 weeks of treatment as
determined by electronic peak flow meter data. |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
|
E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Yes |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 2 |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 5 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 25 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Belgium |
Colombia |
Croatia |
Germany |
Guatemala |
Hungary |
Philippines |
Russian Federation |
Slovakia |
South Africa |
Turkey |
|
E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
|
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial days | 4 |