QAW039 long term safety study in uncontrolled asthma patients
Research type
Research Study
Full title
A 2-treatment period, randomised, placebo-controlled, multicentre parallel-group study to assess the safety of QAW039 when added to existing asthma therapy in GINA steps 3, 4 and 5 patients with uncontrolled asthma.
IRAS ID
231209
Contact name
Shuaib Nasser
Contact email
Sponsor organisation
Novartis Pharma AG
Eudract number
2016-001560-11
Clinicaltrials.gov Identifier
Duration of Study in the UK
4 years, 8 months, 24 days
Research summary
Summary of Research
Asthma presents a major global health burden. Despite existing therapies, there is still significant unmet medical need in asthma, with an estimated 300 million people affected worldwide. Annual worldwide deaths have been estimated at 250,000 (Masoli et al, 2004).Currently there is a need for well tolerated, easily administered, anti-inflammatory therapies. QAW039 is an experimental medicine being tested for the treatment of patients with asthma. The purpose of this study is to obtain long-term safety data for QAW039 with 150mg/450mg when compared with placebo, when added to GINA steps 3, 4 and 5 standard of care asthma therapy in adults or adolescents (12 years or older) with moderate to severe asthma as many asthma patients will receive asthma treatments for a long period of time as part of their care.
40 UK participants will be recruited from approximately 10 sites. Many patients will be identified from other QAW039 phase III trials i.e. CQAW039A2307 or the patients will have never participated in a QAW039 clinical trial. Approximately 2800 patients are expected to complete other QAW039 Phase 3 studies globally and be eligible for participation in the CQAW039A2315 study. It is anticipated that 60-75% of these eligible patients will provide consent and participate in this long term safety study. It is anticipated total sample size of the study is 1896 to 2314 patients.
Consenting participants will enter a double blind treatment period 1 (52 weeks long) for QAW039 150mg once daily, QAW039 450mg once daily or placebo once daily and the participants will be randomised in a 3:3:1 ratio. Patients will continue into an optional treatment period 2 for 104 weeks(single blind only) on the same treatment as given in treatment period 1.
Summary of Results
In the present study CQAW039A2315, QAW039 (both doses) was well tolerated, with a safety profile similar to that of placebo. The overall safety profile was comparable for patients who were newly recruited or sourced from previous Phase 3 studies.
The exploratory analysis showed an overall reduction in the rates of moderate-to-severe asthma exacerbations and increase in time to first moderate-to-severe asthma exacerbation for QAW039 treatment groups versus placebo for both the high eosinophils subpopulation and overall population.
The LS mean treatment difference in pre-dose FEV1 indicated improvements for the two QAW039 treatment doses over placebo at Week 52 for both high eosinophils subpopulation and overall population.
Since the study was early terminated and no multiplicity adjustment was performed for the exploratory analyses, these efficacy results should be interpreted with caution.REC name
East of England - Cambridge Central Research Ethics Committee
REC reference
17/EE/0361
Date of REC Opinion
31 Oct 2017
REC opinion
Further Information Favourable Opinion