Table 4 Primary and secondary objectives and endpoints
Objective | Endpoint |
|---|---|
Primary objective | |
Assess the effect of tezepelumab compared with placebo in reducing the prescribed OCS maintenance dose in adults with severe, uncontrolled asthma | Percentage reduction from baseline in daily OCS dose at week 48, defined as: 1. 90–100% reduction 2. 75–< 90% reduction 3. 50–< 75% reduction 4. 0–< 50% reduction 5. no change or any increase |
Key secondary objective | |
Assess the effect of tezepelumab on asthma exacerbations compared with placebo | AAER • Time to first asthma exacerbation • Rate of asthma exacerbations associated with ER visits, urgent care visits or hospitalization • Proportion of subjects who did not experience an asthma exacerbation |
Other secondary objectives | |
Assess the effect of tezepelumab on the prescribed OCS daily maintenance dose | Proportion of patients with 100% reduction from baseline in daily OCS dose at week 48 Proportion of patients with daily OCS dose ≤ 5 mg at week 48 Proportion of patients with ≥ 50% reduction from baseline in daily OCS dose at week 48 |
Assess the effect of tezepelumab on pulmonary function compared with placebo | Change from baseline in pre-BD FEV1 |
Assess the effect of tezepelumab on asthma symptoms and other asthma control metrics, compared with placebo | Change from baseline in: • weekly mean daily ASD score • weekly mean rescue medication use • weekly mean morning and evening PEF • weekly mean number of night-time awakenings • ACQ-6 score |
Assess the effect of tezepelumab on asthma-related and general health-related quality of life compared with placebo | Change from baseline in: • AQLQ(S)+12 total score • EQ-5D-5L score |
Assess the effect of tezepelumab on healthcare resource use and productivity loss owing to asthma | Asthma-specific resource use (e.g. unscheduled physician visits, use of other asthma medications) WPAI + CIQ scores |
Assess the effect of tezepelumab on biomarkers | Change from baseline in FeNO, peripheral blood eosinophil count |
Evaluate the pharmacokinetics and immunogenicity of tezepelumab | Pharmacokinetics: serum trough concentrations Immunogenicity: incidence of ADAs |