AsthmaPhase 3NCT02563067

Study of Efficacy and Safety of QAW039 in Patients With Severe Asthma Inadequately Controlled With Standard of Care Asthma Treatment.

A Phase 3 study testing QAW039 in people with Asthma, run by Novartis Pharmaceuticals at 163 sites in 19 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Trial summary

This study aimed to determine the efficacy and safety of QAW039 and QAW039 450 mg compared to placebo, when added to GINA (Global Initiative for Asthma) steps 4 and 5 standard-of- care (SoC) asthma therapy (GINA 2016) in the following two populations: patient with inadequately controlled severe asthma and high… Read more about this study

Who is it for?: Ages 12 years and older
Conditions the study is for: Asthma
What is being tested?: QAW039
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 877 participants
Where: 163 sitesin 19 countries
Study dates: 3 Dec 2015to 2 Aug 2019
Phase: Phase 3
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 12 years and older
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

Written informed consent.
Male and female patients aged ≥12 years.
A diagnosis of severe asthma, uncontrolled on GINA 4/5 asthma medication.
Evidence of airway reversibility or airway hyper- reactivity.
FEV1 ≤80% of the predicted normal value for patients aged ≥18 years; FEV1 of ≤90% for patients aged 12 to <18 years
An ACQ score ≥1.5
A history of 2 or more asthma exacerbations within the 12 months prior to entering the study.

Who cannot join

Use of other investigational drugs within 5 half-lives of study entry, or within 30 days, whichever is longer.
Subjects who have participated in another trial of QAW039.
A QTcF (Fridericia) ≥450 msec (male) or ≥460 msec (female).
History of malignancy with the exception of local basal cell carcinoma of the skin.
Pregnant or nursing (lactating) women.
Serious co-morbidities.
Patients on >20 mg of simvastatin, > 40 mg of atorvastatin, >40 mg of pravastatin, or >2 mg of pitavastatin.

Trial locations and contacts

163 sites in 19 countries.

Country

City, postcode or hospital

163 sites in 19 countries

Italy21 sites

Novartis Investigative Site
Acquaviva delle Fonti, BA, 70021
Novartis Investigative Site
Ancona, AN, 60126
Novartis Investigative Site
Bari, BA, 70124
Novartis Investigative Site
Brescia, BS, 25123
Novartis Investigative Site
Cagliari, Sicily, 09042
Novartis Investigative Site
Catania, CT, 95125
Novartis Investigative Site
Catanzaro, CZ, 88100
Novartis Investigative Site
Cona, FE, 44100
Novartis Investigative Site
Foggia, FG, 71100
Novartis Investigative Site
Legnago, VR, 37045
Novartis Investigative Site
Messina, ME, 98125
Novartis Investigative Site
Milan, 20100
Novartis Investigative Site
Milan, 20142
Novartis Investigative Site
Padova, PD, 35128
Novartis Investigative Site
Pisa, PI, 56124
Novartis Investigative Site
Reggio Emilia, 42123
Novartis Investigative Site
Roma, RM, 00168
Novartis Investigative Site
Salerno, 84100
Novartis Investigative Site
Siena, SI, 53100
Novartis Investigative Site
Torino, TO, 10128
Novartis Investigative Site
Verona, VR, 37126

Other trials in Italy

United States18 sites

Novartis Investigative Site
Colorado Springs, Colorado, 80907
Novartis Investigative Site
Dallas, Texas, 75230
Novartis Investigative Site
Denver, Colorado, 80206
Novartis Investigative Site
Edmond, Oklahoma, 73034
Novartis Investigative Site
Greenville, South Carolina, 29615
Novartis Investigative Site
Houston, Texas, 77005
Novartis Investigative Site
Little Rock, Arkansas, 72209
Novartis Investigative Site
Louisville, Kentucky, 40215
Novartis Investigative Site
New York, New York, 10016
Novartis Investigative Site
Overland Park, Kansas, 66210
Novartis Investigative Site
Philadelphia, Pennsylvania, 19115
Novartis Investigative Site
Plano, Texas, 75093
Novartis Investigative Site
Richmond, Virginia, 23220
Novartis Investigative Site
Richmond, Virginia, 23225
Novartis Investigative Site
San Jose, California, 95117
Novartis Investigative Site
Stockton, California, 95207
Novartis Investigative Site
The Bronx, New York, 10459
Novartis Investigative Site
Winter Park, Florida, 32789

Other trials in United States

Argentina18 sites

Novartis Investigative Site
Berazategui, Buenos Aires, 1888
Novartis Investigative Site
Buenos Aires, Ciudad Autonoma de Bs As, C1425FVH
Novartis Investigative Site
Buenos Aires, C1012AAR
Novartis Investigative Site
Buenos Aires, C1125ABE
Novartis Investigative Site
CABA, Buenos Aires, C1122AAK
Novartis Investigative Site
CABA, Buenos Aires, C1425BEN
Novartis Investigative Site
CABA, Buenos Aires, C1426ABP
Novartis Investigative Site
Lanus, Buenos Aires, B8000XAV
Novartis Investigative Site
Mar del Plata, Buenos Aires, 7600
Novartis Investigative Site
Mendoza, 5500
Novartis Investigative Site
Mendoza, M5500CBA
Novartis Investigative Site
Ranelagh, Partido de Berazate, Buenos Aires, 1884
Novartis Investigative Site
Rosario, Santa Fe Province, S2000AII
Novartis Investigative Site
Rosario, Santa Fe Province, S2000BRH
Novartis Investigative Site
San Miguel de Tucumán, Tucumán Province, 4000
Novartis Investigative Site
Santa Fe, Rosario, S2000DBS
Novartis Investigative Site
Santa Fe, S3000FIL
Novartis Investigative Site
Santa Rosa, La Pampa Province, 6300

Other trials in Argentina

Japan16 sites

Novartis Investigative Site
Bunkyo Ku, Tokyo, 113-8431
Novartis Investigative Site
Chikushino-shi, Fukuoka, 818-8502
Novartis Investigative Site
Chuo Ku, Tokyo, 104-0031
Novartis Investigative Site
Fukuoka, Fukuoka, 811-1394
Novartis Investigative Site
Hamamatsu, Shizuoka, 431-3192
Novartis Investigative Site
Hiroshima, Hiroshima, 734-8530
Novartis Investigative Site
Kagoshima, Kagoshima-ken, 890 8520
Novartis Investigative Site
Kasuga, Fukuoka, 816-0813
Novartis Investigative Site
Kishiwada, Osaka, 596-8501
Novartis Investigative Site
Naka-gun, Ibaraki, 319-1113
Novartis Investigative Site
Sakaidechō, Kagawa-ken, 762-8550
Novartis Investigative Site
Setagaya-Ku, Tokyo, 157-0072
Novartis Investigative Site
Shinagawa-ku, Tokyo, 142-8666
Novartis Investigative Site
Takamatsu, Kagawa-ken, 761-8073
Novartis Investigative Site
Toshima Ku, Tokyo, 170 0003
Novartis Investigative Site
Yokohama, Kanagawa, 232 0024

Other trials in Japan

Spain13 sites

Novartis Investigative Site
Barcelona, Catalonia, 08036
Novartis Investigative Site
Girona, 17005
Novartis Investigative Site
Guadalajara, 19002
Novartis Investigative Site
Jerez de la Frontera, Cadiz, 11407
Novartis Investigative Site
Las Palmas de Gran Canaria, 35010
Novartis Investigative Site
Lugo, Galicia, 27003
Novartis Investigative Site
Madrid, 28041
Novartis Investigative Site
Madrid, 28046
Novartis Investigative Site
Málaga, Andalusia, 29009
Novartis Investigative Site
Palma de Mallorca, Balearic Islands, 07120
Novartis Investigative Site
Sabadell, Catalonia, 08208
Novartis Investigative Site
Santiago de Compostela, 15706
Novartis Investigative Site
Zaragoza, 50009

Other trials in Spain

India12 sites

Novartis Investigative Site
Ahmedabad, Gujarat, 380 008
Novartis Investigative Site
Ahmedabad, Gujarat, 380 060
Novartis Investigative Site
Bangalore, Karnataka, 560004
Novartis Investigative Site
Bangalore, Karnataka, 560099
Novartis Investigative Site
Coimbatore, Tamil Nadu, 641 045
Novartis Investigative Site
Hyderabad, Telangana, 500082
Novartis Investigative Site
Jaipur, Rajasthan, 302001
Novartis Investigative Site
Jaipur, Rajasthan, 302039
Novartis Investigative Site
Lucknow, Uttar Pradesh, 226006
Novartis Investigative Site
Madurai, Tamil Nadu, 625 020
Novartis Investigative Site
Nagpur, Maharashtra, 440015
Novartis Investigative Site
Pune, Maharashtra, 411014

Other trials in India

Russia11 sites

Novartis Investigative Site
Izhevsk, 426061
Novartis Investigative Site
Moscow, 105077
Novartis Investigative Site
Moscow, 109544
Novartis Investigative Site
Moscow, 115478
Novartis Investigative Site
N.Novgorod, 603126
Novartis Investigative Site
Novosibirsk, 630091
Novartis Investigative Site
Saint Petersburg, 194354
Novartis Investigative Site
Saint Petersburg, 197022
Novartis Investigative Site
Sestroretsk, 197706
Novartis Investigative Site
Stavropol, 355000
Novartis Investigative Site
Yekaterinburg, 620035

Other trials in Russia

Slovakia10 sites

Novartis Investigative Site
Bardejov, Slovak Republic, 085 01
Novartis Investigative Site
Bojnice, Slovak Republic, 972 01
Novartis Investigative Site
Bratislava, 826 06
Novartis Investigative Site
Dúbravka, Bratislava Region, 84102
Novartis Investigative Site
Komárno, 945 01
Novartis Investigative Site
Košice, 040 01
Novartis Investigative Site
Levice, 934 01
Novartis Investigative Site
Liptovský Hrádok, Slovak Republic, 033 01
Novartis Investigative Site
Spišská Nová Ves, 052 01
Novartis Investigative Site
Vyšné Hágy, 5984

Other trials in Slovakia

Czechia7 sites

Novartis Investigative Site
Brno, 615 00
Novartis Investigative Site
Jindřichův Hradec, Czech Republic, 377 01
Novartis Investigative Site
Karlovy Vary, 360 17
Novartis Investigative Site
Mladá Boleslav, 293 50
Novartis Investigative Site
Plzen-Bory, 305 99
Novartis Investigative Site
Teplice, Czech Republic, 415 01
Novartis Investigative Site
Teplice, CZE, 415 01

Other trials in Czechia

Canada5 sites

Novartis Investigative Site
Montreal, Quebec, H3G 1L5
Novartis Investigative Site
Montreal, Quebec, H4J 1C5
Novartis Investigative Site
Québec, G1V 4W2
Novartis Investigative Site
Vancouver, British Columbia, V5Z 1M9
Novartis Investigative Site
Vancouver BC, British Columbia, V6Z 1Y6

Other trials in Canada

Israel5 sites

Novartis Investigative Site
Haifa, 3525408
Novartis Investigative Site
Jerusalem, 91031
Novartis Investigative Site
Jerusalem, 91120
Novartis Investigative Site
Petah Tikva, 49100
Novartis Investigative Site
Rehovot, 76100

Other trials in Israel

Greece4 sites

Novartis Investigative Site
Athens, GR, 115 27
Novartis Investigative Site
Athens, 12462
Novartis Investigative Site
Athens, 175 62
Novartis Investigative Site
Thessaloniki, GR, 570 10

Other trials in Greece

Malaysia4 sites

Novartis Investigative Site
Batu Caves, 68100
Novartis Investigative Site
Kota Bharu, Kelantan, 15586
Novartis Investigative Site
Kuantan, Pahang, 25100
Novartis Investigative Site
Taiping, Perak, 34000

Other trials in Malaysia

Netherlands4 sites

Novartis Investigative Site
Amsterdam, 1105 AZ
Novartis Investigative Site
Arnhem, 6815 AD
Novartis Investigative Site
Leeuwarden, 8934 AD
Novartis Investigative Site
Leiden, 2333 ZA

Other trials in Netherlands

Lebanon3 sites

Novartis Investigative Site
Beirut, 166378
Novartis Investigative Site
El Achrafiyé, 166830
Novartis Investigative Site
El Chouf, LBN, 1503201002

Other trials in Lebanon

Mexico3 sites

Novartis Investigative Site
Guadalajara, Jalisco, 44130
Novartis Investigative Site
Guadalajara Jalisco, Jalisco, 44220
Novartis Investigative Site
Zapopan, Jalisco, 45200

Other trials in Mexico

Serbia3 sites

Novartis Investigative Site
Belgrade, 11000
Novartis Investigative Site
Belgrade, 11070
Novartis Investigative Site
Kragujevac, 34000

Other trials in Serbia

South Africa3 sites

Novartis Investigative Site
Mowbray, 7700
Novartis Investigative Site
Panorama, 7500
Novartis Investigative Site
Tygerberg, Cape Town, 7505

Other trials in South Africa

Taiwan3 sites

Novartis Investigative Site
Taichung, Taiwan ROC, 40201
Novartis Investigative Site
Taichung, 40447
Novartis Investigative Site
Taichung, 40705

Other trials in Taiwan

About this study

Official title: A 52-week, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy and Safety of QAW039 When Added to Existing Asthma Therapy in Patients With Uncontrolled Severe Asthma.

patient with inadequately controlled severe asthma and high eosinophil counts (eosinophil count at Visit 1 ≥250 cells/ µl) (sub-population)
patients with inadequately controlled severe asthma (overall study population) Inadequate control is defined as partly controlled or uncontrolled asthma (GINA 2016)

Read the detailed description

This study used a randomized, multicenter, double-blind, placebo-controlled parallel-group design in which QAW039 (150 mg and 450 mg) or placebo was added to GINA steps 4 and 5 asthma therapy.

The study included:

Screening period of up to 2 weeks to assess eligibility;
Run-in period of approximately 2 weeks and a maximum of 6 weeks on placebo to collect baseline data for efficacy variables and compliance with the Electronic Peak Flow/ eDiary device. Upon completion of the run-in period, all patients who met the eligibility criteria were randomized to one of three treatments: QAW039 150 mg or QAW039 450 mg or placebo once daily in a ratio of 1:1:1.
Treatment period of 52 weeks (assessment period for all Primary and Secondary Outcome Measures). Clinic visits were scheduled approximately 4 weeks after randomization and then at approximately 8-week intervals during the active-treatment period. Phone calls occurred at specified time points between visits occurring at 8-week intervals. Patients who had successfully completed 52 weeks of treatment in this study were offered an optional participation in a safety study (CQAW039A2315).
Follow-up period of 4 weeks, investigational and drug-free, following the last dose of study drug. A follow-up visit occurred approximately 4 weeks (i.e., approximately 30 days) following the last dose of study therapy to complete safety assessments and pregnancy testing (if applicable).

The follow-up period applied to all patients except those patients who had entered the optional safety study (CQAW039A2315) directly after the Week 52 study visit

Conditions: Asthma
Being tested: QAW039, Placebo
Keywords: Asthma, QAW039
Medical subject headings: Asthma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

QAW039Drug
QAW039 150 mg once daily
QAW039Drug
QAW039 450 mg once daily
PlaceboDrug
Placebo once daily

Study groups in NCT02563067
Study group	Role	What they receive
QAW039 150 mg	Experimental	QAW039 150 mg once daily Receives: Drug: QAW039
QAW039 450 mg	Experimental	QAW039 450 mg once daily Receives: Drug: QAW039
Placebo	Placebo comparator	Placebo once daily Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

Rate of Moderate-to-severe Asthma Exacerbations During the 52-week Treatment Period in High Eosinophils Subpopulation
A severe asthma exacerbation is defined as treatment with 'rescue' systemic corticosteroids for greater than or equal to 3 days and hospitalization; or treatment with 'rescue' systemic corticosteroids for greater than or equal to 3 days and emergency department visit (greater than 24 hours*); or death due to asthma.
A moderate asthma exacerbation is defined as treatment with 'rescue' systemic corticosteroids for greater than or equal to 3 days either as an outpatient or in emergency department visits (Emergency department visit less than or equal to 24 hours). The high eosinophils subpopulation consists of all patients with blood eosinophil count ≥ 250 cells/μL at baseline.
Measured: 52 weeks
Rate of Moderate-to-severe Asthma Exacerbations During the 52-week Treatment Period in Overall Population
A severe asthma exacerbation is defined as treatment with 'rescue' systemic corticosteroids for greater than or equal to 3 days and hospitalization; or treatment with 'rescue' systemic corticosteroids for greater than or equal to 3 days and emergency department visit (greater than 24 hours*); or death due to asthma.
A moderate asthma exacerbation is defined as treatment with 'rescue' systemic corticosteroids for greater than or equal to 3 days either as an outpatient or in emergency department visits (Emergency department visit less than or equal to 24 hours).
Measured: 52 weeks

Secondary outcomes (6)

Change From Baseline to Week 52 in Asthma Quality of Life Questionnaire for Participants 12 Years and Older (AQLQ+12) Score in High Eosinophils Subpopulation
AQLQ is a 32-item instrument administered as a self-assessment. AQLQ+12 is a modified version of AQLQ developed to measure functional impairments of participants aged 12-70 years. It is divided into 4 domains: activity limitation, symptoms, emotional function, and environmental stimuli. Participants were asked to recall their experiences during the last 2 weeks and respond to each question on a 7-point scale (1=severe impairment, 7=no impairment), where higher scores indicated "better quality of life." Overall AQLQ+12 score is the mean of all 32 responses. The high eosinophils subpopulation consists of all patients with blood eosinophil count ≥ 250 cells/μL at baseline.
Measured: Baseline, Week 52
Change From Baseline to Week 52 in Asthma Control Questionnaire-5 (ACQ-5) Score in High Eosinophils Subpopulation
The ACQ-5 is a five-item, self-completed questionnaire, which is used as a measure of asthma control of a participant. The five questions (concerning nocturnal awakening, waking in the morning, activity limitation, shortness of breath and wheeze) enquire about the frequency and/or severity of symptoms over the previous week. The response options for all these questions range from zero (no impairment/limitation) to six (total impairment/ limitation) scale. The ACQ-5 score is the average of the individual item scores and ranges from 0 to 6. Higher scores indicates worsening of condition. The high eosinophils subpopulation consists of all patients with blood eosinophil count ≥ 250 cells/μL at baseline.
Measured: Baseline, Week 52
Change From Baseline to Week 52 in Pre-dose Forced Expiratory Volume in 1 Second (FEV1) in High Eosinophils Subpopulation
Forced Expiratory Volume in one second (FEV1) is calculated as the volume of air forcibly exhaled in one second as measured by a spirometer. Baseline is defined as the last available FEV1 measurement taken prior to the first dose of randomized study drug. The high eosinophils subpopulation consists of all patients with blood eosinophil count ≥ 250 cells/μL at baseline.
Measured: Baseline, Week 52
Change From Baseline to Week 52 in Asthma Quality of Life Questionnaire for Participants 12 Years and Older (AQLQ+12) Score in Overall Population
AQLQ is a 32-item instrument administered as a self-assessment. AQLQ+12 is a modified version of AQLQ developed to measure functional impairments of participants aged 12-70 years. It is divided into 4 domains: activity limitation, symptoms, emotional function, and environmental stimuli. Participants were asked to recall their experiences during the last 2 weeks and respond to each question on a 7-point scale (1=severe impairment, 7=no impairment), where higher scores indicated "better quality of life." Overall AQLQ+12 score is the mean of all 32 responses.
Measured: Baseline, Week 52
Change From Baseline to Week 52 in Asthma Control Questionnaire-5 (ACQ-5) Score in Overall Population
The ACQ-5 is a five-item, self-completed questionnaire, which is used as a measure of asthma control of a participant. The five questions (concerning nocturnal awakening, waking in the morning, activity limitation, shortness of breath and wheeze) enquire about the frequency and/or severity of symptoms over the previous week. The response options for all these questions range from zero (no impairment/limitation) to six (total impairment/ limitation) scale. The ACQ-5 score is the average of the individual item scores and ranges from 0 to 6. Higher scores indicates worsening of condition.
Measured: Baseline, Week 52
Change From Baseline to Week 52 in Pre-dose Forced Expiratory Volume in 1 Second (FEV1) in Overall Population
Forced Expiratory Volume in one second (FEV1) is calculated as the volume of air forcibly exhaled in one second as measured by a spirometer. Baseline is defined as the last available FEV1 measurement taken prior to the first dose of randomized study drug.
Measured: Baseline, Week 52

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

3 Dec 20152 Aug 2019

First posted29 Sep 2015 (estimated)
Study start3 Dec 2015
Primary completion4 Jul 2019
Study completion2 Aug 2019

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (1)Papers the sponsor has linked to this study.
Other trials for AsthmaStudies of the same condition, open and under way.
More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 3
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Double blind
Who is masked: Participant, Investigator
Enrolment: 877 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02563067
Sponsor's protocol ID: CQAW039A2314
Other IDs: 2015-003172-67, 2015
Status last verified: Apr 2021
First submitted: 28 Sep 2015
Last updated: 3 May 2021
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: Undecided: 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

Publications

Brightling CE, Gaga M, Inoue H, Li J, Maspero J, Wenzel S, Maitra S, Lawrence D, Brockhaus F, Lehmann T, Brindicci C, Knorr B, Bleecker ER. Effectiveness of fevipiprant in reducing exacerbations in patients with severe asthma (LUSTER-1 and LUSTER-2): two phase 3 randomised controlled trials. Lancet Respir Med. 2021 Jan;9(1):43-56. doi: 10.1016/S2213-2600(20)30412-4. Epub 2020 Sep 24.
PubMed 32979986

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

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Study at a glance

From the registry record, last updated by the sponsor on 3 May 2021. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A 52-week, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy and Safety of QAW039 When Added to Existing Asthma Therapy in Patients With Uncontrolled Severe Asthma.
ClinicalTrials.gov ID: NCT02563067
Acronym / sponsor ID: CQAW039A2314
Conditions: Asthma
Keywords: Asthma, QAW039
Phase: Phase 3
Study type: Interventional
Recruitment status: Completed
Enrolment: 877 participants (actual)
Lead sponsor: Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
Study start: 3 Dec 2015 (actual)
Primary completion: 4 Jul 2019 (actual)
Study completion: 2 Aug 2019 (actual)
First posted: 29 Sep 2015 (estimated)
Results: Posted 3 May 2021

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

patient with inadequately controlled severe asthma and high eosinophil counts (eosinophil count at Visit 1 ≥250 cells/ µl) (sub-population)
patients with inadequately controlled severe asthma (overall study population) Inadequate control is defined as partly controlled or uncontrolled asthma (GINA 2016)

Detailed description

This study used a randomized, multicenter, double-blind, placebo-controlled parallel-group design in which QAW039 (150 mg and 450 mg) or placebo was added to GINA steps 4 and 5 asthma therapy.

The study included:

Screening period of up to 2 weeks to assess eligibility;
Run-in period of approximately 2 weeks and a maximum of 6 weeks on placebo to collect baseline data for efficacy variables and compliance with the Electronic Peak Flow/ eDiary device. Upon completion of the run-in period, all patients who met the eligibility criteria were randomized to one of three treatments: QAW039 150 mg or QAW039 450 mg or placebo once daily in a ratio of 1:1:1.
Treatment period of 52 weeks (assessment period for all Primary and Secondary Outcome Measures). Clinic visits were scheduled approximately 4 weeks after randomization and then at approximately 8-week intervals during the active-treatment period. Phone calls occurred at specified time points between visits occurring at 8-week intervals. Patients who had successfully completed 52 weeks of treatment in this study were offered an optional participation in a safety study (CQAW039A2315).
Follow-up period of 4 weeks, investigational and drug-free, following the last dose of study drug. A follow-up visit occurred approximately 4 weeks (i.e., approximately 30 days) following the last dose of study therapy to complete safety assessments and pregnancy testing (if applicable).

The follow-up period applied to all patients except those patients who had entered the optional safety study (CQAW039A2315) directly after the Week 52 study visit

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Double blind (participant, investigator)
Number of arms: 3

Arm	Type	Receives
QAW039 150 mg	Experimental	Drug: QAW039QAW039 150 mg once daily
QAW039 450 mg	Experimental	Drug: QAW039QAW039 450 mg once daily
Placebo	Placebo comparator	Drug: PlaceboPlacebo once daily

Who may qualify

Ages: 12 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (7 listed)

Written informed consent.
Male and female patients aged ≥12 years.
A diagnosis of severe asthma, uncontrolled on GINA 4/5 asthma medication.
Evidence of airway reversibility or airway hyper- reactivity.
FEV1 ≤80% of the predicted normal value for patients aged ≥18 years; FEV1 of ≤90% for patients aged 12 to <18 years

Key exclusion criteria (7 listed)

Use of other investigational drugs within 5 half-lives of study entry, or within 30 days, whichever is longer.
Subjects who have participated in another trial of QAW039.
A QTcF (Fridericia) ≥450 msec (male) or ≥460 msec (female).
History of malignancy with the exception of local basal cell carcinoma of the skin.
Pregnant or nursing (lactating) women.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
QAW039QAW039 150 mg once daily	Drug	QAW039 150 mg
QAW039QAW039 450 mg once daily	Drug	QAW039 450 mg
PlaceboPlacebo once daily	Drug	Placebo

Primary outcome

Rate of Moderate-to-severe Asthma Exacerbations During the 52-week Treatment Period in High Eosinophils Subpopulation
Time frame: 52 weeks
A severe asthma exacerbation is defined as treatment with 'rescue' systemic corticosteroids for greater than or equal to 3 days and hospitalization; or treatment with 'rescue' systemic corticosteroids for greater than or equal to 3 days and emergency department visit (greater than 24 hours*); or death due to asthma. A moderate asthma exacerbation is defined as treatment with 'rescue' systemic corticosteroids for greater than or equal to 3 days either as an outpatient or in emergency department visits (Emergency department visit less than or equal to 24 hours). The high eosinophils subpopulation consists of all patients with blood eosinophil count ≥ 250 cells/μL at baseline.
Rate of Moderate-to-severe Asthma Exacerbations During the 52-week Treatment Period in Overall Population
Time frame: 52 weeks
A severe asthma exacerbation is defined as treatment with 'rescue' systemic corticosteroids for greater than or equal to 3 days and hospitalization; or treatment with 'rescue' systemic corticosteroids for greater than or equal to 3 days and emergency department visit (greater than 24 hours*); or death due to asthma. A moderate asthma exacerbation is defined as treatment with 'rescue' systemic corticosteroids for greater than or equal to 3 days either as an outpatient or in emergency department visits (Emergency department visit less than or equal to 24 hours).

6 secondary outcomes

Change From Baseline to Week 52 in Asthma Quality of Life Questionnaire for Participants 12 Years and Older (AQLQ+12) Score in High Eosinophils Subpopulation
Time frame: Baseline, Week 52
Change From Baseline to Week 52 in Asthma Control Questionnaire-5 (ACQ-5) Score in High Eosinophils Subpopulation
Time frame: Baseline, Week 52
Change From Baseline to Week 52 in Pre-dose Forced Expiratory Volume in 1 Second (FEV1) in High Eosinophils Subpopulation
Time frame: Baseline, Week 52
Change From Baseline to Week 52 in Asthma Quality of Life Questionnaire for Participants 12 Years and Older (AQLQ+12) Score in Overall Population
Time frame: Baseline, Week 52
Change From Baseline to Week 52 in Asthma Control Questionnaire-5 (ACQ-5) Score in Overall Population
Time frame: Baseline, Week 52
Change From Baseline to Week 52 in Pre-dose Forced Expiratory Volume in 1 Second (FEV1) in Overall Population
Time frame: Baseline, Week 52

Results and safety

Results posted to the registry on 3 May 2021, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
QAW039 150 mg	296	277
QAW039 450 mg	294	268
Placebo	287	267

Primary outcome: Rate of Moderate-to-severe Asthma Exacerbations During the 52-week Treatment Period in High Eosinophils Subpopulation

Time frame: 52 weeks. Least squares mean (95% Confidence Interval). Unit: Events/year

Measure	QAW039 150 mg n = 198	QAW039 450 mg n = 196	Placebo n = 193
Value	0.73 (0.57 to 0.94)	0.76 (0.59 to 0.99)	1.06 (0.84 to 1.33)

QAW039 150 mg vs Placebo: Rate Ratio: 0.69 (95% CI 0.50 to 0.96), p = 0.059, negative binomial regression model
QAW039 450 mg vs Placebo: Rate Ratio: 0.72 (95% CI 0.52 to 1.01), p = 0.114, negative binomial regression model

Population analysed: Full Analysis Set (FAS) in high eosinophils subpopulation (count ≥ 250 cells/μL)

Primary outcome: Rate of Moderate-to-severe Asthma Exacerbations During the 52-week Treatment Period in Overall Population

Time frame: 52 weeks. Least squares mean (95% Confidence Interval). Unit: Events/year

Measure	QAW039 150 mg n = 296	QAW039 450 mg n = 293	Placebo n = 287
Value	0.76 (0.62 to 0.92)	0.70 (0.57 to 0.87)	0.93 (0.76 to 1.12)

QAW039 150 mg vs Placebo: Rate Ratio: 0.82 (95% CI 0.62 to 1.07), p = 0.369, negative binomial regression model
QAW039 450 mg vs Placebo: Rate Ratio: 0.76 (95% CI 0.58 to 1.00), p = 0.348, negative binomial regression model

Population analysed: Full Analysis Set (FAS), including all randomized patients who received at least one dose of study drug.

6 secondary outcomes are reported in the full results.

Adverse events

Time frame: Adverse events (AEs) are presented from first dose of study treatment until last dose of study treatment plus 7 days post treatment. Serious AEs (including the All-Cause Mortality data table) are presented from first dose of study treatment until last dose of study treatment plus 30 days post treatment, up to maximum duration of 56 weeks.. Other adverse events reported at 4% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
QAW039 150 mg	20 of 296 (6.8%)	181 of 296 (61.1%)	0 of 296
QAW039 450 mg	20 of 293 (6.8%)	183 of 293 (62.5%)	1 of 293
Placebo	18 of 287 (6.3%)	180 of 287 (62.7%)	1 of 287

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 3 May 2021.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 32979986Brightling CE, Gaga M, Inoue H, Li J, Maspero J, Wenzel S, Maitra S, Lawrence D, Brockhaus F, Lehmann T, Brindicci C, Knorr B, Bleecker ER. Effectiveness of fevipiprant in reducing exacerbations in patients with severe asthma (LUSTER-1 and LUSTER-2): two phase 3 randomised controlled trials. Lancet Respir Med. 2021 Jan;9(1):43-56. doi: 10.1016/S2213-2600(20)30412-4. Epub 2020 Sep 24.
Individual participant dataSharing planned: Undecided. 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

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

The record lists no contact. Use the official record to reach the sponsor.

163 sites in 19 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing QAW039 in people with Asthma, run by Novartis Pharmaceuticals at 163 sites in 19 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 3 May 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 163 sites in Italy, United States, Argentina, Japan, Spain and 14 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 3 May 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Rate of Moderate-to-severe Asthma Exacerbations During the 52-week Treatment Period in High Eosinophils Subpopulation (time frame: 52 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02563067, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 May 2021.