To assess the efficacy and safety of QAW039 when added to existing asthma therapy in patients with uncontrolled severe asthma

What we are studying

The purpose of this study is to determine: 1) if QAW039 works better than placebo when taken with your usual asthma medicines, 2) if QAW039 reduce the number of asthma attacks you have, improve your asthma symptoms, and improve your quality of life, 3) if you have any side effects when you are taking the study medication. About 846 patients with severe asthma aged 12 years and older will join in this study at about 200 study sites in countries all over the world. Patients will be separated into two groups based on their eosinophil (a type of blood cell) count. About two thirds of patients out of the 846 patients in the study will be in one group, and the other third will be in the second group. About 127 patients out of the 846 patients in this study (15%) will be 12 to 17 years old. All patients will receive placebo during the study. There is about a 2 in 3 chance (66%) that you will receive QAW039 study medicine in addition to placebo during the study. There is about a 1 in 3 chance (33%) that you will receive only placebo during the entire study. A placebo is used to make sure that the changes you report are not happening just by chance. Study medication will be provided to you in bottles. If you take part in this study you will need to take 2 tablets of study medication every day in the morning for the entire study. You will take one tablet from one bottle and one tablet from the other bottle every morning between 6:00 a.m. and 10:00 a.m., with a liquid, with or without food, except on the morning of your office visits. The morning of your office visits you will not take any study medication until after you have been seen by the Study Doctor. If at any time during the study the doctor feels that it would not be in your best interest to continue, you will be taken out of the study. Novartis as a sponsor of this study has a right to stop this study and withdraw you from the study at any time. You and your doctor will not know what you have been given, but your doctor can find out what you were taking if there is an emergency. In addition to usual daily asthma medication, you will also be allowed to use an inhaler of so- called rescue medication (for example: salbutamol or albuterol). You be able to use it whenever you need to, for the rest of the study. If you join the study you will be asked to come to the doctor’s office/clinic/study site 11 times over about 60 weeks (14 months). Most study visits will take about 1 to 2 hours, but 4 visits will take more than 2 hours, so that tests can be done about 2 hours after you have taken study medicine at the office/clinic/study site. You will also have 6 telephone call “visits” from the study coordinator or Study Doctor.

Who is Eligible

  • Genders:
    • Men
    • Women
  • Races:
    • White
    • African American
    • Asian
    • American Indian or Alaska Native
    • Native Hawaiian or Pacific Islander
    • Other
  • All Ethnicities
  • Ages 12+

Eligibility Criteria

  • Diagnosed with asthma for at least 24 months prior to Visit 1
  • Willing to complete an electronic patient diary
  • Weight more than 66 pounds
  • 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
  • Doses must have been stable for at least 4 weeks prior to Visit 1
  • Breathing between 40% - 80% of the predicted normal value for the patient after withholding bronchodilators
  • Willing to complete several questionnaires
  • History of 2 or more asthma exacerbations within the 12 months prior to Visit 1
  • Willing to adhere to study requirements and medications
  • Not using other investigational drugs within 30 days of Visit 1
  • Does not have clinically significant ECG abnormality such as sustained ventricular tachycardia or clinically significant second or third degree AV block without a pacemaker
  • No history of cancer treated or untreated with the exception of local basal cell carcinoma of the skin
  • Not pregnant or nursing (lactating)
  • No use of tobacco products within the 6 month period prior to Visit 1
  • No history of chronic lung disease other than asthma including chronic obstructive pulmonary disease; bronchiectasis; sarcoidosis; interstitial lung disease; cystic fibrosis; and tuberculosis
  • No history of myocardial infarction within 12 months of Visit 1
  • No history of alcohol or drug abuse within 12 months prior to Visit 1

What is involved

  • Informed consent or assent (once)
  • Demographics (once)
  • Medical history (once)
  • Pulmonary history: Medical History (once)
  • Asthma exacerbation history (once)
  • Smoking history (once)
  • Prior/Concomitant medication (11 times)
  • Review and record surgeries and procedures (17 times)
  • Physical examination (3 times)
  • Abbreviated physical examination (8 times)
  • Height (twice)
  • Weight (3 times)
  • Vital signs (11 times)
  • Electrocardiograms (twice)
  • Electrocardiograms (Pre-dose) (9 times)
  • Electrocardiograms (2h-post dose) (4 times)
  • Pregnancy test—serum (once)
  • Urinalysis (dipstick) performed at study site (10 times)
  • Blood for hematology and chemistry (10 times)
  • PK blood sample (Pre-dose) (5 times)
  • PK blood sample (2h-post dose) (4 times)
  • Blood for RAST test (once)
  • Blood sample for IgE (3 times)
  • Telephone contact with patient (6 times)
  • Serious adverse event recording (continuous)
  • Adverse event recording (continuous)
  • Asthma exacerbation recording (17 times)
  • Spirometry (twice)
  • 1St Pre-dose Spirometry - 45 min pre-dose (9 times)
  • 2Nd pre-dose Spirometry - 15 min pre-dose (9 times)
  • Reversibility test (spirometry after administration of short-acting beta- agonist [SABA]) (6 times)
  • Airway hyper-reactivity test ***optional test if reversibility is not met *** (once)
  • Exhaled Nitric Oxide Fraction (FeNO) (4 times)
  • Train and assess patient’s ability to use eDiary/e-PEF and dispense (once)
  • Ediary/Epef Download and review eDiary/ePEF compliance prior to randomizing the patient (twice)
  • Download and review eDiary/ePEF entries (8 times)
  • ACQ-5 in clinic (11 times)
  • AQLQ+12 in clinic (11 times)
  • EQ-5D in clinic (11 times)
  • PGIC in clinic (11 times)
  • Dispense rescue SABA to be used on an as-needed basis
  • Dispense study drug (8 times)
  • Dispense patient card (once)

Compensation

$1,225 for completing all study visits. You will be paid for each complete visit if you withdraw for any reason from the study. Visit 1, 201, 202, 205, 299: $100. Visit 101, 199, 203, 204, 206, 207: $75. 6 Telephone calls: $25 each. Follow-up visit $50.

Contact Information

Study Coordinator
Bob Hmieleski
Email
[email protected]
Phone
-8550
Principal Investigator
Dr. Eugene Bleecker

Disclaimer: The information on this website is for general informational purposes only and SHOULD NOT be relied upon as a substitute for sound professional medical advice, evaluation or care from your physician or other qualified health care provider.