Trial Outcomes & Findings for 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 (NCT NCT02892019)

NCT ID: NCT02892019

Last Updated: 2020-01-27

Results Overview

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.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 79 participants
• Primary outcome timeframe: 2 weeks
• Results posted on: 2020-01-27

Participant Flow

randomized set : 80 participants (41 and 39) Full analysis set : 79 participants (41 and 38) safety set : 79 participants (41 and 38) per protocol set : 35 participants (35 and 34)

Participant milestones

Measure	Indacaterol Acetate 150 μg o.d. Indacaterol acetate 150 μg o.d. delivered via Concept1 inhaler	Indacaterol Acetate 75 μg o.d. Indacaterol acetate 75 μg o.d. delivered via Concept1 inhaler
Overall Study STARTED	41	39
Overall Study COMPLETED	40	36
Overall Study NOT COMPLETED	1	3

Reasons for withdrawal

Measure	Indacaterol Acetate 150 μg o.d. Indacaterol acetate 150 μg o.d. delivered via Concept1 inhaler	Indacaterol Acetate 75 μg o.d. Indacaterol acetate 75 μg o.d. delivered via Concept1 inhaler
Overall Study randomized but not treated	0	1
Overall Study Withdrawal by Subject	0	1
Overall Study Adverse Event	1	1

Baseline Characteristics

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

Baseline characteristics by cohort

Measure	Indacaterol Acetate 150 μg o.d. n=41 Participants Indacaterol acetate 150 μg o.d. delivered via Concept1 inhaler	Indacaterol Acetate 75 μg o.d. n=39 Participants Indacaterol acetate 75 μg o.d. delivered via Concept1 inhaler	Total n=80 Participants Total of all reporting groups
Age, Continuous	9.3 years STANDARD_DEVIATION 1.47 • n=5 Participants	9.1 years STANDARD_DEVIATION 1.58 • n=7 Participants	9.2 years STANDARD_DEVIATION 1.51 • n=5 Participants
Sex: Female, Male Female	13 Participants n=5 Participants	15 Participants n=7 Participants	28 Participants n=5 Participants
Sex: Female, Male Male	28 Participants n=5 Participants	24 Participants n=7 Participants	52 Participants n=5 Participants
Race/Ethnicity, Customized Caucasian	17 Participants n=5 Participants	17 Participants n=7 Participants	34 Participants n=5 Participants
Race/Ethnicity, Customized Black	3 Participants n=5 Participants	3 Participants n=7 Participants	6 Participants n=5 Participants
Race/Ethnicity, Customized Asian	1 Participants n=5 Participants	3 Participants n=7 Participants	4 Participants n=5 Participants
Race/Ethnicity, Customized Native American	3 Participants n=5 Participants	3 Participants n=7 Participants	6 Participants n=5 Participants
Race/Ethnicity, Customized Unknown	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race/Ethnicity, Customized Other	16 Participants n=5 Participants	13 Participants n=7 Participants	29 Participants n=5 Participants

PRIMARY outcome

Timeframe: 2 weeks

Population: full analysis set

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.

Outcome measures

Measure	Indacaterol Acetate 150 μg o.d. n=41 Participants Indacaterol acetate 150 μg o.d. delivered via Concept1 inhaler	Indacaterol Acetate 75 μg o.d. n=38 Participants Indacaterol acetate 75 μg o.d. delivered via Concept1 inhaler
Trough Forced Expiratoty Volume in 1 Second (FEV1)	0.171 L Standard Deviation 0.1664	0.212 L Standard Deviation 0.2521

SECONDARY outcome

Timeframe: day 1, day 14

Population: safety set

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.

Outcome measures

Measure	Indacaterol Acetate 150 μg o.d. n=41 Participants Indacaterol acetate 150 μg o.d. delivered via Concept1 inhaler	Indacaterol Acetate 75 μg o.d. n=38 Participants Indacaterol acetate 75 μg o.d. delivered via Concept1 inhaler
Systemic Exposure to Indacaterol in Plasma Day 1 <=-2 hours	0.0 pg/mL Standard Deviation 0.0	0.0 pg/mL Standard Deviation 0.0
Systemic Exposure to Indacaterol in Plasma Day 1, 15 minutes	204.1 pg/mL Standard Deviation 98.27	73.7 pg/mL Standard Deviation 39.55
Systemic Exposure to Indacaterol in Plasma Day 1, 1 hour	125.8 pg/mL Standard Deviation 63.13	43.9 pg/mL Standard Deviation 20.84
Systemic Exposure to Indacaterol in Plasma Day 14 <=-2 hours	80.9 pg/mL Standard Deviation 32.51	46.7 pg/mL Standard Deviation 29.61
Systemic Exposure to Indacaterol in Plasma Day 14, 15 minutes	393.1 pg/mL Standard Deviation 182.11	137.3 pg/mL Standard Deviation 47.43
Systemic Exposure to Indacaterol in Plasma Day 14, 1 hour	255.5 pg/mL Standard Deviation 117.67	103.0 pg/mL Standard Deviation 37.88

SECONDARY outcome

Timeframe: 2 weeks

Population: full analysis set

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

Outcome measures

Measure	Indacaterol Acetate 150 μg o.d. n=41 Participants Indacaterol acetate 150 μg o.d. delivered via Concept1 inhaler	Indacaterol Acetate 75 μg o.d. n=38 Participants Indacaterol acetate 75 μg o.d. delivered via Concept1 inhaler
Asthma Control as Assessed by Pediatric Interviewer-administered Asthma Control Questionnaire	-0.375 score on a scale Standard Deviation 0.3796	-0.270 score on a scale Standard Deviation 0.6341

SECONDARY outcome

Timeframe: 2 weeks

Population: full analysis set

Pre-dose morning and evening PEF over 2 weeks of treatment as determined by electronic peak flow meter data (mean change)

Outcome measures

Measure	Indacaterol Acetate 150 μg o.d. n=41 Participants Indacaterol acetate 150 μg o.d. delivered via Concept1 inhaler	Indacaterol Acetate 75 μg o.d. n=38 Participants Indacaterol acetate 75 μg o.d. delivered via Concept1 inhaler
Pre-dose Morning and Evening Peak Expiratoty Flow (PEF) Evening PEF	29.7 L/min Standard Deviation 25.57	15.2 L/min Standard Deviation 28.58
Pre-dose Morning and Evening Peak Expiratoty Flow (PEF) Morning PEF	29.6 L/min Standard Deviation 26.41	14.7 L/min Standard Deviation 29.38

SECONDARY outcome

Timeframe: 2 weeks

Population: full analysis set

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.

Outcome measures

Measure	Indacaterol Acetate 150 μg o.d. n=41 Participants Indacaterol acetate 150 μg o.d. delivered via Concept1 inhaler	Indacaterol Acetate 75 μg o.d. n=38 Participants Indacaterol acetate 75 μg o.d. delivered via Concept1 inhaler
Rescue Medication Usage (Mean Daiily Number of Puffs)	-0.19 number of puffs Standard Deviation 1.479	-0.22 number of puffs Standard Deviation 0.930

SECONDARY outcome

Timeframe: 2 weeks

Population: full analysis set

Rescue medication usage over 2 weeks of treatment as determined by patient diary data for indacaterol acetate 75 μg and 150 μg o.d.

Outcome measures

Measure	Indacaterol Acetate 150 μg o.d. n=41 Participants Indacaterol acetate 150 μg o.d. delivered via Concept1 inhaler	Indacaterol Acetate 75 μg o.d. n=38 Participants Indacaterol acetate 75 μg o.d. delivered via Concept1 inhaler
Rescue Medication Usage (Percentage of Rescue Medication Free Days)	7.4 percentage Standard Deviation 22.37	3.1 percentage Standard Deviation 19.42

SECONDARY outcome

Timeframe: 2 weeks

Population: full analysis set

FEV1 and FVC at 30 minutes and 1-hour post dose at week 2 for indacaterol acetate 75 μg and 150 μg o.d.

Outcome measures

Measure	Indacaterol Acetate 150 μg o.d. n=41 Participants Indacaterol acetate 150 μg o.d. delivered via Concept1 inhaler	Indacaterol Acetate 75 μg o.d. n=38 Participants Indacaterol acetate 75 μg o.d. delivered via Concept1 inhaler
Forced Expiratoty Volume in 1 Second (FEV1) and Forced Vital Capacity (FVC) 30 minutes	0.234 L Standard Deviation 0.1786	0.224 L Standard Deviation 0.2459
Forced Expiratoty Volume in 1 Second (FEV1) and Forced Vital Capacity (FVC) 1 hour	0.224 L Standard Deviation 0.1725	0.258 L Standard Deviation 0.2451

SECONDARY outcome

Timeframe: 2 weeks

Population: full analysis set

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).

Outcome measures

Measure	Indacaterol Acetate 150 μg o.d. n=41 Participants Indacaterol acetate 150 μg o.d. delivered via Concept1 inhaler	Indacaterol Acetate 75 μg o.d. n=38 Participants Indacaterol acetate 75 μg o.d. delivered via Concept1 inhaler
Symptoms as Recorded by Patient E-diary (Mean Total Daily Symptom Score)	-0.18 score on a scale Standard Deviation 0.695	0.08 score on a scale Standard Deviation 0.365

SECONDARY outcome

Timeframe: 2 weeks

Population: full analysis set

Symptoms as recorded by patient e-diary for indacaterol acetate 75 μg and 150 μg o.d. (mean change)

Outcome measures

Measure	Indacaterol Acetate 150 μg o.d. n=41 Participants Indacaterol acetate 150 μg o.d. delivered via Concept1 inhaler	Indacaterol Acetate 75 μg o.d. n=38 Participants Indacaterol acetate 75 μg o.d. delivered via Concept1 inhaler
Symptoms as Recorded by Patient E-diary (Percentage of Asthma Symptoms Free Days)	12.3 percentage Standard Deviation 21.25	4.4 percentage Standard Deviation 26.60

Adverse Events

QAB149 150 ug OD

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

QAB149 75 ug OD

Serious events: 0 serious events

Other events: 13 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	QAB149 150 ug OD n=41 participants at risk QAB149 150 ug OD	QAB149 75 ug OD n=38 participants at risk QAB149 75 ug OD
Cardiac disorders Defect conduction intraventricular	0.00% 0/41 • 2 years AE additional description	2.6% 1/38 • 2 years AE additional description
Gastrointestinal disorders Nausea	2.4% 1/41 • 2 years AE additional description	2.6% 1/38 • 2 years AE additional description
Gastrointestinal disorders Vomiting	0.00% 0/41 • 2 years AE additional description	2.6% 1/38 • 2 years AE additional description
Infections and infestations Conjunctivitis	0.00% 0/41 • 2 years AE additional description	2.6% 1/38 • 2 years AE additional description
Infections and infestations Influenza	0.00% 0/41 • 2 years AE additional description	5.3% 2/38 • 2 years AE additional description
Infections and infestations Rhinitis	0.00% 0/41 • 2 years AE additional description	2.6% 1/38 • 2 years AE additional description
Infections and infestations Upper respiratory tract infection	0.00% 0/41 • 2 years AE additional description	7.9% 3/38 • 2 years AE additional description
Injury, poisoning and procedural complications Limb injury	0.00% 0/41 • 2 years AE additional description	2.6% 1/38 • 2 years AE additional description
Investigations Blood glucose increased	0.00% 0/41 • 2 years AE additional description	2.6% 1/38 • 2 years AE additional description
Investigations Peak expiratory flow rate decreased	0.00% 0/41 • 2 years AE additional description	2.6% 1/38 • 2 years AE additional description
Nervous system disorders Dizziness	2.4% 1/41 • 2 years AE additional description	0.00% 0/38 • 2 years AE additional description
Respiratory, thoracic and mediastinal disorders Asthma	2.4% 1/41 • 2 years AE additional description	5.3% 2/38 • 2 years AE additional description
Respiratory, thoracic and mediastinal disorders Dysphonia	0.00% 0/41 • 2 years AE additional description	2.6% 1/38 • 2 years AE additional description

Additional Information

Study Director

Novartis Pharmaceuticals

Phone: 862-778-8300

Email: novartis.email@novartis.com

Results disclosure agreements

• Principal investigator is a sponsor employee The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (ie, data from all sites) in the clinical trial.
• Publication restrictions are in place

Restriction type: OTHER