Clinical Trials List
Protocol NumberCQAW039A2314
2016-01-01 - 2019-05-31
Phase III
Terminated5
Study ended1
ICD-10J45.52
Severe persistent asthma with status asthmaticus
ICD-9493.01
Extrinsic asthma with status asthmaticus
A 52-week, multicenter, randomized, double-blind, placebocontrolled study to assess the efficacy and safety of QAW039 when added to existing asthma therapy in patients with uncontrolled severe asthma
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Trial Applicant
NOVARTIS (TAIWAN) CO., LTD.
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Sponsor
Novartis
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Trial scale
Multi-Regional Multi-Center
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Update
2025/08/20
Investigators and Locations
Co-Principal Investigator
- Wei- Chang Huang Division of Thoracic Medicine
- 王俊隆 Division of Thoracic Medicine
- Pin-Kuei Fu Division of Thoracic Medicine
- 覃俊士 Division of Thoracic Medicine
- Ming -Cheng Chan Division of Thoracic Medicine
The Actual Total Number of Participants Enrolled
0 Terminated
The Actual Total Number of Participants Enrolled
0 Terminated
Co-Principal Investigator
- Tzuen-Ren Hsiue Division of Thoracic Medicine
The Actual Total Number of Participants Enrolled
0 Terminated
Co-Principal Investigator
- 張志豪 Division of Thoracic Medicine
- Cheng-Ta Yang Division of Thoracic Medicine
- 胡漢忠 Division of Thoracic Medicine
- 莊立邦 Division of Thoracic Medicine
- Shih-Wei Lin Division of Thoracic Medicine
The Actual Total Number of Participants Enrolled
0 Terminated
Co-Principal Investigator
The Actual Total Number of Participants Enrolled
0 Terminated
Audit
CRO
Co-Principal Investigator
- Bing-Ru Wu Division of Thoracic Medicine
- 梁信杰 Division of Thoracic Medicine
- 廖偉志 Division of Thoracic Medicine
- Yu-Chao Lin Division of Thoracic Medicine
- Shuo-Chueh Chen Division of Thoracic Medicine
- 許武輝 Division of Thoracic Medicine
- Chia-Hsiang Li Division of Thoracic Medicine
- Chen Chia-Hung Division of Thoracic Medicine
- Chih-Ching Yen Division of Thoracic Medicine
- Hsu Wu-Huei Division of Thoracic Medicine
- Chih-Yen Tu Division of Thoracic Medicine
- 程味兒 Division of Thoracic Medicine
The Actual Total Number of Participants Enrolled
2 Study ended
Condition/Disease
uncontrolled severe asthma
Objectives
In patients with severe asthma and high eosinophil counts (≥250 cells/µl)
receiving SoC asthma therapy, to demonstrate the efficacy (as measured
by rate of moderate-to-severe asthma exacerbations) of at least one dose
level of QAW039 (150 mg or 450 mg once daily), compared with placebo,
at the end of the 52-week active-treatment epoch.
In patients with severe asthma receiving SoC asthma therapy, to
demonstrate the efficacy (as measured by rate of moderate-to-severe
asthma exacerbations) of at least one dose level of QAW039 (150 mg or
450 mg once daily), compared with placebo, at the end of the 52-week
active-treatment epoch.
Test Drug
QAW039
Active Ingredient
QAW039
Dosage Form
tablet
Dosage
150, 450
Endpoints
Efficacy assessments:
Asthma exacerbations
Asthma Quality of Life Questionnaire for 12 years and older
(AQLQ+12)
Asthma Control Questionnaire (ACQ-5)
Spirometry (Pre-dose FEV1)
Safety assessments:
History and physical examination
Vital signs
Hematology
Blood chemistry including liver function tests, metabolic panels,
amylase, lipase and hsCRP.
CK-MB and Troponin I (in response to CK results outside of the
normal range)
HbA1c (collected at screening only)
Urinalysis
Pregnancy test (females of childbearing potential)
ECG
Adverse events including serious adverse events
Asthma exacerbations
Asthma Quality of Life Questionnaire for 12 years and older
(AQLQ+12)
Asthma Control Questionnaire (ACQ-5)
Spirometry (Pre-dose FEV1)
Safety assessments:
History and physical examination
Vital signs
Hematology
Blood chemistry including liver function tests, metabolic panels,
amylase, lipase and hsCRP.
CK-MB and Troponin I (in response to CK results outside of the
normal range)
HbA1c (collected at screening only)
Urinalysis
Pregnancy test (females of childbearing potential)
ECG
Adverse events including serious adverse events
Inclution Criteria
Written informed consent must be obtained within 14 days prior to or
at Visit 1 before any assessment is performed including any
adjustment to asthma medication.
Male and female patients aged ≥12 years.
Patients must have a diagnosis of asthma (according to GINA 2015)
for a period of at least 24 months prior to Visit 1.
Patients have been treated with high-dose inhaled corticosteroids
plus a LABA (or alternate therapy: montelukast or theophylline or
tiotropium) with or without maintenance oral corticosteroids for at
least 3 months prior to Visit 1(See GINA 2015 for the definition of
high dose ICS). The doses must have been stable for at least 4
weeks prior to Visit 1.
FEV1 of ≥40% and ≤80% of the predicted normal value for the
patient, after withholding bronchodilators at Visit 1 and Visit 101.
Demonstration of inadequate control of asthma based on an ACQ
score ≥1.5 at Visit 1.
A history of 2 or more asthma exacerbations within the 12 months
prior to Visit 1 that required either:
Treatment with systemic corticosteroids (tablets, suspension or
injection)
OR
Hospitalization (defined as an inpatient stay or >24-hour stay in
an observation area in the emergency room of other equivalent
facility.
A clinical diagnosis of asthma supported by at least one of the
following:
An increase of ≥12% and ≥200 ml in FEV1 within 30 minutes after
administration of 400 mcg of salbutamol/albuterol (or equivalent
dose) prior to randomization. Spacer devices are not permitted
during reversibility testing. All patients must perform a
reversibility test at Visit 1. If reversibility is not demonstrated at
Visit 1*, the following historical information may be used:
Documented evidence of reversibility that was performed
according to ATS/ERS guidelines (ATS/ERS 2005) with the 2
years prior to Visit 1. Where a patient is assessed as eligible
based on historical evidence of reversibility, a copy of the original
printed spirometry report with relevant spirometry tracings must
be available as source documentation.
Documented evidence of a positive airways hyper-reactivity
(AHR) test result within the 2 years prior to or at Visit 101,
defined as a provoked fall in FEV1 of 20% by methacholine at ≤8
mg/ml (or histamine ≤10 mg/ml or acetylcholine <20 mg/mL)
when not on ICS or ≤16 mg/ml or histamine ≤20 mg/ml or
acetylcholine <40 mg/mL) on ICS therapy performed according to
ATS/ERS guidelines
at Visit 1 before any assessment is performed including any
adjustment to asthma medication.
Male and female patients aged ≥12 years.
Patients must have a diagnosis of asthma (according to GINA 2015)
for a period of at least 24 months prior to Visit 1.
Patients have been treated with high-dose inhaled corticosteroids
plus a LABA (or alternate therapy: montelukast or theophylline or
tiotropium) with or without maintenance oral corticosteroids for at
least 3 months prior to Visit 1(See GINA 2015 for the definition of
high dose ICS). The doses must have been stable for at least 4
weeks prior to Visit 1.
FEV1 of ≥40% and ≤80% of the predicted normal value for the
patient, after withholding bronchodilators at Visit 1 and Visit 101.
Demonstration of inadequate control of asthma based on an ACQ
score ≥1.5 at Visit 1.
A history of 2 or more asthma exacerbations within the 12 months
prior to Visit 1 that required either:
Treatment with systemic corticosteroids (tablets, suspension or
injection)
OR
Hospitalization (defined as an inpatient stay or >24-hour stay in
an observation area in the emergency room of other equivalent
facility.
A clinical diagnosis of asthma supported by at least one of the
following:
An increase of ≥12% and ≥200 ml in FEV1 within 30 minutes after
administration of 400 mcg of salbutamol/albuterol (or equivalent
dose) prior to randomization. Spacer devices are not permitted
during reversibility testing. All patients must perform a
reversibility test at Visit 1. If reversibility is not demonstrated at
Visit 1*, the following historical information may be used:
Documented evidence of reversibility that was performed
according to ATS/ERS guidelines (ATS/ERS 2005) with the 2
years prior to Visit 1. Where a patient is assessed as eligible
based on historical evidence of reversibility, a copy of the original
printed spirometry report with relevant spirometry tracings must
be available as source documentation.
Documented evidence of a positive airways hyper-reactivity
(AHR) test result within the 2 years prior to or at Visit 101,
defined as a provoked fall in FEV1 of 20% by methacholine at ≤8
mg/ml (or histamine ≤10 mg/ml or acetylcholine <20 mg/mL)
when not on ICS or ≤16 mg/ml or histamine ≤20 mg/ml or
acetylcholine <40 mg/mL) on ICS therapy performed according to
ATS/ERS guidelines
Exclusion Criteria
Use of other investigational drugs within 5 half-lives of enrollment, or
within 30 days until the expected pharmacodynamic effect has
returned to baseline, whichever is longer.
Subjects who have participated in another trial of QAW039 (i.e.
subject received study medication [active (QAW039), placebo or
other].
Patients with a resting QTcF (Fridericia) ≥450 msec (male) or ≥460
msec (female) at Visit 1 or at Visit 101.
Patients with a history of malignancy of any organ, treated or
untreated, whether or not there is evidence of local recurrence of metastases, with the exception of local basal cell carcinoma of the
skin.
Pregnant or nursing (lactating) women, where pregnancy is defined
as the state of a female after conception and until the termination of
gestation, confirmed by a positive hCG laboratory test.
Women of child-bearing potential, defined as all women
physiologically capable of becoming pregnant, unless they are using
effective methods of contraception during dosing of study treatment.
Patients who have a clinically significant laboratory abnormality at
Visit 1 or Visit 101
Patients with serious co-morbidities including, but not limited to,
neurodegenerative diseases, rheumatoid arthritis and other
autoimmune diseases.
Patients on >20 mg of simvastatin, > 40 mg of atorvastatin, >40 mg
of pravastatin, or >2 mg of pitavastatin. Statin doses less than or
equal to these doses as well as other statins will be permitted during
the study.
Patients on any statin therapy with a CK level >2 X ULN at Visit 1.
within 30 days until the expected pharmacodynamic effect has
returned to baseline, whichever is longer.
Subjects who have participated in another trial of QAW039 (i.e.
subject received study medication [active (QAW039), placebo or
other].
Patients with a resting QTcF (Fridericia) ≥450 msec (male) or ≥460
msec (female) at Visit 1 or at Visit 101.
Patients with a history of malignancy of any organ, treated or
untreated, whether or not there is evidence of local recurrence of metastases, with the exception of local basal cell carcinoma of the
skin.
Pregnant or nursing (lactating) women, where pregnancy is defined
as the state of a female after conception and until the termination of
gestation, confirmed by a positive hCG laboratory test.
Women of child-bearing potential, defined as all women
physiologically capable of becoming pregnant, unless they are using
effective methods of contraception during dosing of study treatment.
Patients who have a clinically significant laboratory abnormality at
Visit 1 or Visit 101
Patients with serious co-morbidities including, but not limited to,
neurodegenerative diseases, rheumatoid arthritis and other
autoimmune diseases.
Patients on >20 mg of simvastatin, > 40 mg of atorvastatin, >40 mg
of pravastatin, or >2 mg of pitavastatin. Statin doses less than or
equal to these doses as well as other statins will be permitted during
the study.
Patients on any statin therapy with a CK level >2 X ULN at Visit 1.
The Estimated Number of Participants
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Taiwan
7 participants
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Global
864 participants
