Table 3 Endpoints of the SYGMA programme
SYGMA1 | SYGMA2 | |||
|---|---|---|---|---|
Comparator | Outcome measure | Comparator | Outcome measure | |
Primary endpoints | As-needed budesonide/formoterol vs as-needed terbutaline | • Evaluation of asthma control as measured by well-controlled asthma weeks | As-needed budesonide/formoterol vs budesonide bid plus as-needed terbutaline | • Annual severe asthma exacerbation rate |
Secondary efficacy endpoints | As-needed budesonide/formoterol vs budesonide bid plus as-needed terbutaline | • Evaluation of asthma control as measured by well-controlled asthma weeks | As-needed budesonide/formoterol vs budesonide bid plus as-needed terbutaline | • Time to first severe asthma exacerbation • Average change from baseline in pre-dose FEV1 • Time to study specific asthma-related discontinuation • Average change from baseline in number of inhalations of as-needed medication • Change from baseline in percent of as-needed free days • Percentage of controller use days • Average change from baseline in ACQ-5 score and responders based on MID • Average change from baseline in AQLQ score • Total ICS load and number of days with systemic corticosteroid treatment |
As-needed budesonide/formoterol vs as-needed terbutaline or budesonide bid plus as-needed terbutaline | • Time to first severe asthma exacerbation • Time to first moderate or severe asthma exacerbation • Average change from baseline in pre-dose FEV1 • Average change from baseline in morning and evening PEF • Average change from baseline in number of inhalations of as-needed medication • Average change from baseline in symptom score • Percentage of night-time awakenings due to asthma • Percentage of symptom-free days • Percentage of as-needed free days • Percentage of asthma control days • Percentage of controller use days • Time to asthma-related discontinuation • Poorly-controlled asthma weeks • Time to additional steroids for asthma • Average change from baseline in ACQ-5 score and responders based on MID • Average change from baseline in AQLQ score • Total ICS load and number of days with systemic corticosteroid treatment | |||
Safety endpoints | As-needed budesonide/formoterol vs as-needed terbutaline or budesonide bid plus as-needed terbutaline | • Adverse events (nature, incidence and severity) • Pulse, blood pressure and physical examination | As-needed budesonide/formoterol vs budesonide bid plus as-needed terbutaline | • Adverse events (nature, incidence and severity) • Pulse, blood pressure and physical examination |
Exploratory endpoints | N/A | • Coded transcriptions of patient interviews | As-needed budesonide/formoterol vs budesonide bid plus as-needed terbutaline | • EuroQol 5-dimensional 5-level questionnaire • Health Economics Questionnaire for resource utilisation |