Clinical Trials Register

Summary
EudraCT Number:	2011-004966-13
Sponsor's Protocol Code Number:	CQAW039A2208
National Competent Authority:	UK - MHRA
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2011-11-18
Trial results	View results

Index
A. PROTOCOL INFORMATION
B. SPONSOR INFORMATION
C. APPLICANT IDENTIFICATION
D. IMP IDENTIFICATION
D.8 INFORMATION ON PLACEBO
E. GENERAL INFORMATION ON THE TRIAL
F. POPULATION OF TRIAL SUBJECTS
G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
P. END OF TRIAL

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A. Protocol Information

A.1

Member State Concerned

UK - MHRA

A.2

EudraCT number

2011-004966-13

A.3

Full title of the trial

A double-blind, placebo-controlled, study examining the
effect of orally administered QAW039 on sputum
eosinophil levels and other efficacy outcomes in patients
with sputum eosinophilia and persistent asthma

A.3.1

Title of the trial for lay people, in easily understood, i.e. non-technical, language

A clinical trial looking at the effect of QAW039 in patients who have persistent asthma and a given type of white blood cells (eosinophils) in the mucus from their lungs

A.4.1

Sponsor's protocol code number

CQAW039A2208

A.7

Trial is part of a Paediatric Investigation Plan

A.8

EMA Decision number of Paediatric Investigation Plan

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	Novartis Pharma Services AG
B.1.3.4	Country	Switzerland
B.3.1 and B.3.2	Status of the sponsor	Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	Novartis Pharma Services AG
B.4.2	Country	Switzerland
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Novartis Pharmaceutical UK Ltd
B.5.2	Functional name of contact point	Medical Information Services
B.5.3	Address:
B.5.3.1	Street Address	Frimley Business Park, Frimley
B.5.3.2	Town/ city	Camberley, Surrey
B.5.3.3	Post code	GU16 7SR
B.5.3.4	Country	United Kingdom
B.5.4	Telephone number	4101276698370
B.5.5	Fax number	4101276698449
B.5.6	E-mail	medinfo.uk@novartis.com

D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.2	Product code	QAW039
D.3.4	Pharmaceutical form	Capsule
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Not established
D.3.9.1	CAS number	872365-14-5
D.3.9.2	Current sponsor code	QAW039
D.3.9.3	Other descriptive name	QAW039-NXA.001
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	25
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No
D.IMP: 2
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.2	Product code	QAW039
D.3.4	Pharmaceutical form	Capsule
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Not established
D.3.9.1	CAS number	872365-14-5
D.3.9.2	Current sponsor code	QAW039
D.3.9.3	Other descriptive name	QAW039-NXA.001
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	150
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No

D.8 Information on Placebo
D.8 Placebo: 1
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Capsule, hard
D.8.4	Route of administration of the placebo	Oral use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

sputum eosinophilia and moderate-to-severe asthma (GINA 2-5) incompletely controlled on current therapy

E.1.1.1

Medical condition in easily understood language

Asthma with a given type of white blood cells (eosinophils) in the mucus from the lungs

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	14.0
E.1.2	Level	LLT
E.1.2	Classification code	10062305
E.1.2	Term	Sputum eosinophils increased
E.1.2	System Organ Class	10022891 - Investigations

E.1.2 Medical condition or disease under investigation

E.1.2	Version	14.0
E.1.2	Level	LLT
E.1.2	Classification code	10003561
E.1.2	Term	Asthma, unspecified
E.1.2	System Organ Class	10038738 - Respiratory, thoracic and mediastinal disorders

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

To demonstrate a statistically significant reduction in sputum eosinophil levels in inadequately controlled, moderate-to-severe asthmatics (GINA 2- 5), with sputum eosinophilia after treatment with QAW039 for 12 weeks compared to placebo.

E.2.2

Secondary objectives of the trial

• To demonstrate that QAW039 provides significantly superior control of asthmatic symptoms as measured by the asthma control questionnaire (ACQ) compared to placebo.
• To assess safety and tolerability of QAW039 in this moderate-to-severe asthmatic population as compared to placebo.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Written informed consent must be obtained before any assessment is performed
2. Males and females of any race who are over the age of 18 years at the time informed consent is obtained.
3. Physician diagnosis of asthma, as per GINA guidelines and currently prescribed ICS or ICS-LABA therapy.
4. Patients who are demonstrated to have reversible airway obstruction, significant airflow variability or airway hyperresponsiveness (AHR) as defined in full protocol, or who have shown such responses in previous test(s) within the last five years.
5. An ACQ score ≥ 1.5 at randomisation or ≥ 1 exacerbations (as defined in protocol, requiring higher dose than the patient’s normal dose of OCS or IV corticosteroids for ≥ 3 days) in the past 12 months.
6. Patients currently on GINA step 2 to step 5 asthma therapies.
7. Sputum eosinophil count ≥ 2% at screening.

E.4

Principal exclusion criteria

1. Use of other investigational drugs at the time of enrollment, or within 30 days or 5 halflives to the time of enrollment, whichever is longer.
2. History of hypersensitivity to any of the study drugs or to drugs of similar chemical classes (CRTH2 antagonists).
3. History of long QT syndrome or whose QTc interval (Fridericia’s) is prolonged > 450 msec for males and > 470 msec for females at screening or baseline.
4. History of malignancy of any organ system (other than localized basal cell carcinoma of the skin), treated or untreated, within the past 5 years, regardless of whether there is evidence of local recurrence or metastases.
5. 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 (> 5 mIU/mL).
6. Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using effective methods of contraception as defined in full protocol during dosing of study treatment and for 5 days (5 half-lives) after treatment.
7. Acute illness other than asthma which, in the investigator’s opinion, may compromise the well-being of the patient or study endpoint assessments at the start of the study.
8. Patients who are considered unsuitable for inclusion by the assessing physician due to serious co-morbidities such as cancer, emphysema or significant bronchiectasis.
9. Recent (within 6 weeks of screening) or current lower respiratory tract infection.
10. Patients who have been hospitalized or required high-dose (>10mg prednisolone/day) oral corticosteroid (OCS) therapy within 6 weeks of the screening visit.
11. Patients with clinically significant laboratory abnormalities (not associated with the study indication) at screening as defined in full protocol.
12. Patients who have a clinically significant abnormality on a 12-lead ECG recorded within one month prior to or at screening.
13. Patients with a body mass index (BMI) < 17 or > 40 kg/m2.
14. Patients on maintenance immunotherapy who either began their immunotherapy regimen or had a clinically relevant change to their immunotherapy within 1 month prior to granting informed consent.
15. Use of immunosuppressive medication (except inhaled and topical corticosteroids and low dose (≤10mg prednisolone/day) oral corticosteroids) within 30 days before randomization into the study.
16. Use of Xolair (omalizumab) within 6 months before randomization into the study.
17. History of alcohol or other substance abuse.
18. A positive hepatitis B surface antigen or hepatitis C virus antibody, as determined by medical history and/or subject’s verbal report.
19. A positive human immunodeficiency virus test or is taking anti-retroviral medications, as determined by medical history and/or subject’s verbal report.
20. Patients on high-dose statin therapy, as defined in protocol.
21. Patients on statin therapy with a CK level >2 X ULN at screening.

E.5 End points

E.5.1

Primary end point(s)

Sputum eosinophils level

E.5.1.1

Timepoint(s) of evaluation of this end point

6 Weeks, 12 Weeks (end of active treatment period) and after a 6-week washout on placebo

E.5.2

Secondary end point(s)

Asthma Control Questionnaire (ACQ)

E.5.2.1

Timepoint(s) of evaluation of this end point

6 Weeks, 12 Weeks (end of active treatment period) and after a 6-week washout on placebo

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

E.6.2

Prophylaxis

E.6.3

Therapy

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

Yes

E.6.7

Pharmacodynamic

Yes

E.6.8

Bioequivalence

E.6.9

Dose response

E.6.10

Pharmacogenetic

Yes

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

Yes

E.7.3

Therapeutic confirmatory (Phase III)

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

Yes

E.8.1.1

Randomised

Yes

E.8.1.2

Open

E.8.1.3

Single blind

E.8.1.4

Double blind

Yes

E.8.1.5

Parallel group

Yes

E.8.1.6

Cross over

E.8.1.7

Other

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

E.8.2.2

Placebo

Yes

E.8.2.3

Other

E.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

Yes

E.8.4

The trial involves multiple sites in the Member State concerned

E.8.5

The trial involves multiple Member States

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

E.8.6.2

Trial being conducted completely outside of the EEA

E.8.7

Trial has a data monitoring committee

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

LVLS

E.8.9 Initial estimate of the duration of the trial

E.8.9.1

In the Member State concerned years

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

E.8.9.2

In all countries concerned by the trial months

E.8.9.2

In all countries concerned by the trial days

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

F.1.1.1

In Utero

F.1.1.2

Preterm newborn infants (up to gestational age < 37 weeks)

F.1.1.3

Newborns (0-27 days)

F.1.1.4

Infants and toddlers (28 days-23 months)

F.1.1.5

Children (2-11years)

F.1.1.6

Adolescents (12-17 years)

F.1.2

Adults (18-64 years)

Yes

F.1.3

Elderly (>=65 years)

Yes

F.2 Gender

F.2.1

Female

Yes

F.2.2

Male

Yes

F.3 Group of trial subjects

F.3.1

Healthy volunteers

F.3.2

Patients

Yes

F.3.3

Specific vulnerable populations

Yes

F.3.3.1

Women of childbearing potential not using contraception

F.3.3.2

Women of child-bearing potential using contraception

Yes

F.3.3.3

Pregnant women

F.3.3.4

Nursing women

F.3.3.5

Emergency situation

F.3.3.6

Subjects incapable of giving consent personally

F.3.3.7

Others

F.4 Planned number of subjects to be included

F.4.1

In the member state

F.4.2

For a multinational trial

F.4.2.1

In the EEA

F.4.2.2

In the whole clinical trial

F.5

Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)

G. Investigator Networks to be involved in the Trial

N. Review by the Competent Authority or Ethics Committee in the country concerned

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2011-12-20

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2011-12-16

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2013-06-25

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