E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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E.1.1.1 | Medical condition in easily understood language |
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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 | 20.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10003553 |
E.1.2 | Term | Asthma |
E.1.2 | System Organ Class | 10038738 - Respiratory, thoracic and mediastinal disorders |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
Treatment Period 1 (double-blind, 52-week treatment period):
In patients with moderate-to-severe asthma receiving SoC asthma therapy, to evaluate the long-term safety of QAW039 (150 mg once daily and 450 mg once daily), compared with placebo, as assessed by:
-treatment emergent adverse events (AEs);
-treatment emergent serious adverse events (SAEs); and
-study treatment discontinuations due to treatment emergent AEs.
Treatment Period 1 and Treatment Period 2 combined:
In patients with moderate-to-severe-asthma receiving SoC asthma therapy, to evaluate the long-term safety of QAW039 (150 mg once daily and 450 mg once daily), compared with placebo, as assessed by:
-treatment emergent AEs
-treatment emergent SAEs; and
-study treatmentdiscontinuations due to treatment emergent AEs. |
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E.2.2 | Secondary objectives of the trial |
Treatment Period 1 (double-blind, 52-week treatment period):
In patients with moderate-to-severe asthma receiving SoC asthma therapy, to evaluate the long-term safety of QAW039 (150 mg once daily and 450 mg once daily), compared with placebo, as assessed by:
-rate of patients with at least 1 treatment emergent AE by primary system organ class;
-rate of treatment emergent patient deaths and patient hospitalizations due to an asthma exacerbation.
Treatment Period 1 and Treatment Period 2 combined:
In patients with moderate-to-severe-asthma receiving SoC asthma therapy, to evaluate the long-term safety of QAW039 (150 mg once daily and 450 mg once daily), compared with placebo, as assessed by:
-rate of patients with at least 1 treatment emergent AE by primary system organ class; and
-rate of treatment emergent patient deaths and patient hospitalizations due to an asthma exacerbation. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Patients completing a prior Phase 3 study of QAW039:
- Informed consent and assent (if applicable).
-Completion of the Treatment Period (on blinded study drug) of a prior Phase 3 study of QAW039.
-Patient is able to safely continue into the study as judged by the investigator.
Patients who have not previously participated in a study of QAW039:
-Written informed consent.
-Male and female patients aged ≥12 years.
-A diagnosis of asthma, uncontrolled on GINA 3/4/5 asthma medication. -Evidence of airway reversibility or airway hyper- reactivity.
-FEV1 of ≤85% of the predicted normal value.
-An ACQ score ≥1.5 prior to entering the study.
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E.4 | Principal exclusion criteria |
Patients completing a prior phase 3 study of QAW039:
- Pregnant or nursing (lactating) women.
- Women of child-bearing potential unless they are using basic methods of contraception during dosing of study drug
- Patients who did not complete the Treatment Period on blinded study drug of the prior QAW039 study they participated in.
- Inability to comply with all study requirements.
- Patient who experienced a serious and drug-related AE in the prior QAW039 study they participated in.
Patients who have not previously participated in a study of QAW039:
-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 greater than 20 mg of simvastatin
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E.5 End points |
E.5.1 | Primary end point(s) |
-treatment emergent adverse events (AEs)
-treatment emergent serious adverse events
-treatment emergent AEs leading to discontinuation |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
52 weeks (end Treatment Period 1)
160 weeks (end Treatment Period 2) |
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E.5.2 | Secondary end point(s) |
-Rate of patients with at least 1 treatment emergent AE by primary system organ class
-Rate of treatment emergent patient deaths for asthma exacerbations
-Rate of treatment emergent patient hospitalizations for an asthma exacerbation |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
52 weeks (end Treatment Period 1)
160 weeks (end Treatment Period 2) |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | No |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | Yes |
E.8.1.7.1 | Other trial design description |
While Part 1 is double-blind, Part 2 is single-blind |
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E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 3 |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 5 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 165 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Argentina |
Australia |
Austria |
Belgium |
Brazil |
Bulgaria |
Canada |
China |
Colombia |
Czech Republic |
Estonia |
Finland |
France |
Germany |
Greece |
Guatemala |
Hungary |
India |
Israel |
Japan |
Latvia |
Lebanon |
Lithuania |
Malaysia |
Mexico |
Netherlands |
Peru |
Philippines |
Poland |
Romania |
Russian Federation |
Saudi Arabia |
Serbia |
Singapore |
Slovakia |
Spain |
Switzerland |
Taiwan |
Turkey |
United Kingdom |
United States |
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E.8.7 | Trial has a data monitoring committee | Yes |
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
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 5 |
E.8.9.1 | In the Member State concerned months | 3 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 5 |
E.8.9.2 | In all countries concerned by the trial months | 4 |
E.8.9.2 | In all countries concerned by the trial days | 0 |