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

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

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.

Study Overview

• Status: Completed
• Conditions: Asthma
• Intervention / Treatment: Drug: Indacaterol acetate 75 μg; Drug: Indacaterol acetate 150 μg

• Study Type: Interventional
• Enrollment (Actual): 79
• Phase: Phase 2

Contacts and Locations

Study Locations

• Belgium
  • Brugge, Belgium, 8000
    • Novartis Investigative Site
  • Brussel, Belgium, 1020
    • Novartis Investigative Site
• Colombia
  • Bogota, Colombia
    • Novartis Investigative Site
  • Bucaramanga, Colombia
    • Novartis Investigative Site
• Croatia
  • Slavonski Brod, Croatia, 35000
    • Novartis Investigative Site
  • Zagreb, Croatia, 10000
    • Novartis Investigative Site
• Germany
  • Hamm, Germany, D 59063
    • Novartis Investigative Site
  • Mannheim, Germany, 68161
    • Novartis Investigative Site
  • Rosenheim, Germany, 83026
    • Novartis Investigative Site
• Guatemala
  • Guatemala City, Guatemala, 01011
    • Novartis Investigative Site
  • Guatemala City, Guatemala, 01015
    • Novartis Investigative Site
  • GTM
    • Guatemala City, GTM, Guatemala, 01010
      • Novartis Investigative Site
• Hungary
  • Budapest, Hungary, 1083
    • Novartis Investigative Site
  • Szeged, Hungary, 6720
    • Novartis Investigative Site
  • Szigetvar, Hungary, 7900
    • Novartis Investigative Site
  • Pest
    • Torokbalint, Pest, Hungary, 2045
      • Novartis Investigative Site
• Philippines
  • Manila, Philippines, 1008
    • Novartis Investigative Site
  • Quezon City, Philippines, 1113
    • Novartis Investigative Site
  • Metro Manila
    • Manila, Metro Manila, Philippines, 1000
      • Novartis Investigative Site
• Russian Federation
  • Saint Petersburg, Russian Federation, 199106
    • Novartis Investigative Site
  • St.Petersburg, Russian Federation, 196240
    • Novartis Investigative Site
• Slovakia
  • SVK
    • Nove Zamky, SVK, Slovakia, 94001
      • Novartis Investigative Site
  • Slovak Republic
    • Nitra, Slovak Republic, Slovakia, 949 01
      • Novartis Investigative Site
• South Africa
  • Cape Town, South Africa, 7531
    • Novartis Investigative Site
  • Cape Town, South Africa, 7700
    • Novartis Investigative Site
  • Mpumalanga
    • Middelburg, Mpumalanga, South Africa, 1050
      • Novartis Investigative Site
  • Western Cape
    • Panorama, Western Cape, South Africa, 7500
      • Novartis Investigative Site
• Turkey
  • Adana, Turkey, 01330
    • Novartis Investigative Site
  • Ankara, Turkey, 06100
    • Novartis Investigative Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 years to 11 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: Indacaterol acetate 75 μg o.d.; Indacaterol acetate 75 μg o.d. delivered via Concept1 inhaler	Drug: Indacaterol acetate 75 μg; Indacaterol acetate 75 μg o.d. delivered via Concept1 inhaler; Other Names: QAB149
Active Comparator: Indacaterol acetate 150 μg o.d.; Indacaterol acetate 150 μg o.d. delivered via Concept1 inhaler	Drug: Indacaterol acetate 150 μg; Indacaterol acetate 150 μg o.d. delivered via Concept1 inhaler; Other Names: QAB149

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Novartis Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Actual): April 18, 2017
• Primary Completion (Actual): June 19, 2019
• Study Completion (Actual): July 17, 2019

Study Registration Dates

• First Submitted: September 1, 2016
• First Submitted That Met QC Criteria: September 1, 2016
• First Posted (Estimate): September 8, 2016

Study Record Updates

• Last Update Posted (Actual): January 27, 2020
• Last Update Submitted That Met QC Criteria: January 15, 2020
• Last Verified: January 1, 2020

More Information

Terms related to this study

• Keywords: indacaterol acetate, asthma, GINA 2015, pediatrics
• Additional Relevant MeSH Terms: Respiratory Tract Diseases; Immune System Diseases; Lung Diseases; Hypersensitivity, Immediate; Bronchial Diseases; Lung Diseases, Obstructive; Respiratory Hypersensitivity; Hypersensitivity; Asthma
• Other Study ID Numbers: CQMF149G2202; 2016-002113-21 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Undecided

IPD Plan Description

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No