Additional exploratory endpoints included components of the macrovascular and microvascular composite endpoints, the composite endpoint of failure requiring hospitalisation or CV ‘heart death’, and all-cause mortality. Exploratory efficacy parameters included HbA1c, FPG (fasting plasma glucose), body weight, SBP (systolic blood pressure), and DBP (diastolic blood pressure). A clinical event committee was established for the central adjudication of potential cardiovascular end points. Comment: The number of efficacy endpoints is noted. Neuropathy was not assessed. There was no physiological assessment of heart function. Randomisation and blinding methods Randomisation as centrally via a computer generated random sequence and interactive voice and web response system. It was stratified for HbA1c, BMI, eGFR and geographical area. Analysis populations Table 1. Analysis populations in the EMPA-REG OUTCOME study Population Abbreviation Definition Screened set SCR All patients screened for the trial with informed consent give and who completed at least 1 screening procedure at Visit 1 Randomised set RS All patients from the screened set who were randomised to study medication Treated set TS All randomised patients who received at least 1 dose of study medication. This population was the basis of primary analysis On treatment set OS Patients who received treatment for at least 30 days (cumulative). Events were considered that occurred not later than 30 days after last intake of the study medication or until the end of the entire trial Full analysis set FAS All patients randomised, treated with at least 1 dose of medication and with a baseline HbA1c value Treated set follow up TS-FU All patients in the TS for whom a follow up visit was performed between 28 and 50 days after last intake of study medication Per Protocol set PPS All patients who were treated with at least 1 dose of study medication and did not have important protocol violations Sample size The trial continued until an adjudicated outcome event (a component of the 3-point MACE) had occurred in at least 691 patients. The criteria used to determine this sample size included:
Additional exploratory endpoints included components of the macrovascular and microvascular composite endpoints, the composite endpoint of ‘heart failure requiring hospitalisation or CV death’, and all-cause mortality. Exploratory efficacy parameters included HbA1c, FPG (fasting plasma glucose), body weight, SBP (systolic blood pressure), and DBP (diastolic blood pressure). A clinical event committee was established for the central adjudication of potential cardiovascular end points. Comment: The number of efficacy endpoints is noted. Neuropathy was not assessed. There was no physiological assessment of heart function. Randomisation and blinding methods Randomisation as centrally via a computer generated random sequence and interactive voice and web response system. It was stratified for HbA1c, BMI, eGFR and geographical area. Analysis populations Table 1. Analysis populations in the EMPA-REG OUTCOME study Population Abbreviation Definition Screened set SCR All patients screened for the trial with informed consent give and who completed at least 1 screening procedure at Visit 1 Randomised set RS All patients from the screened set who were randomised to study medication Treated set TS All randomised patients who received at least 1 dose of study medication. This population was the basis of primary analysis On treatment set OS Patients who received treatment for at least 30 days (cumulative). Events were considered that occurred not later than 30 days after last intake of the study medication or until the end of the entire trial Full analysis set FAS All patients randomised, treated with at least 1 dose of medication and with a baseline HbA1c value Treated set follow up TS-FU All patients in the TS for whom a follow up visit was performed between 28 and 50 days after last intake of study medication Per Protocol set PPS All patients who were treated with at least 1 dose of study medication and did not have important protocol violations Sample size The trial continued until an adjudicated outcome event (a component of the 3-point MACE) had occurred in at least 691 patients. The criteria used to determine this sample size included:
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