Analysis Populations Sample Clauses

Analysis Populations. ‌ Three different analysis populations will be used for analysis of data from this study, as described below: • Randomized: This population will be comprised of all subjects who were initially randomized. This population will be used for summaries of subject disposition and baseline subject characteristics. • Intent-to-treat (ITT)/Safety: This population will be comprised of all subjects who were initially randomized and received at least one dose of study drug. This will be the primary population for all summaries of subject disposition and baseline characteristics, efficacy analyses, and safety analyses for purposes of regulatory submissions. • Modified Intent-to Treat (m-ITT): This population will be comprised of all randomized study subjects who receive study treatment and return for at least one post-randomization assessment of height and weight. This population will be used for the analysis of all efficacy variables for all other purposes, including but not limited to publications, presentations, and robustness of sensitivity of analyses.

Analysis Populations. The modified intent-to-treat population (mITT) is defined as all subjects who are randomized, receive study drug for at least 8 weeks, and have a baseline and at least 1 post-baseline MRI- PDFF assessment on or after Week 8. The mITT is the primary population for analysis of MRI- PDFF assessments and subjects will be analyzed according to the randomized treatment assignment. The intent-to-treat population (ITT) is defined as all subjects who are randomized and received at least 1 dose of study drug. The ITT will be used for analysis of secondary efficacy endpoints and supportive and sensitivity analyses of MRI-PDFF assessments. Subjects will be analyzed according to the randomized treatment assignment. Details will be provided in the Statistical Analysis Plan (SAP). The safety population is defined as all subjects who are randomized and received at least 1 dose of study drug and will be used for all analysis of safety. Subjects will be analyzed according to the treatment received. If all subjects were dosed according to randomized treatment assignment, then the safety population and ITT are identical.

Analysis Populations. The population valid for the safety analysis will be comprised of all patients who received treatment with IRE. Patients who received at least one treatment with IRE will be valid for safety analysis.

Analysis Populations. The following populations will be utilized for the analyses of data. Disposition of patients will use the All Patients Population. The safety analyses will be conducted on the Safety Population. The modified intent-to-treat (mITT) Population will be used for the primary efficacy analyses.

Analysis Populations. The PK Population will consist of all subjects who have received a dose of study drug, have at least 1 quantifiable plasma concentration, and for whom at least 1 PK parameter can be computed. A subject may be excluded from the PK summary statistics and statistical analysis if the subject has an AE of vomiting that occurs at or before 2 times median tmax. The impact of protocol deviations on the PK population will be evaluated on a case-by-case basis. The Safety Population will consist of all subjects who have received at least 1 dose of study drug. Subjects will be classified into groups based on actual treatment received.

Analysis Populations. The Safety Population will include all subjects who receive at least 1 dose of study drug. The PK Population will include subjects who receive study drug and have sufficient concentration data to facilitate the calculation of PK variables.

Analysis Populations. The following analysis populations will be used:

Analysis Populations. PART II Enrolled Population The Enrolled Population includes all screened subjects whose parent(s) provide informed consent, regardless of whether the subject is randomized to receive a study vaccine. This population will be used only to provide summary for subject disposition, starting with the informed consent. The enrolled population will not be used for analyses. Randomized Population The Randomized Population includes all eligible subjects who are randomized in the study, regardless of whether the subject received a study vaccination. This population will be used to provide summary for subject disposition as per randomization and vaccination status. Full Analysis Set Part I The Full Analysis Set (FAS) includes subjects in the enrolled population who were randomized, received study vaccination and have pre- and post-vaccination immunogenicity measurement(s) 4 weeks i.e. 28 days post vaccination. Part II The FAS includes subjects in the enrolled population who were randomized, received three doses of primary vaccination series of study vaccination and have pre- and post-vaccination immunogenicity measurement(s) 4 weeks i.e. 28 days from third dose of the primary vaccination series, available for subjects. Analysis for Part I and Part II will be according to the treatment group assigned at the time of randomization, regardless of whether subjects receive any study vaccine different from that to which they were randomized. The analysis based on this population will serve as supportive results for the immunogenicity objectives. Safety Population The Safety Population includes all subjects who were randomized and received at least one dose. Vaccine groups for safety analyses will be assigned according to the actual vaccine received at Dose 1. Per Protocol Population The Per Protocol Population (PP) includes subjects from FAS population who received all study vaccines as per the assigned vaccine group and have pre- and post-dose immunogenicity measurement(s) with no major protocol deviations that were determined to potentially interfere with immune response to the study vaccine. This population will serve as the primary analysis population for the immunogenicity objectives. The criteria for exclusion of subjects from the PP Population will be established prior to review and analysis of protocol deviations that occur in the study. Based on this, PP population will be finalized before database lock.

Analysis Populations. The statistical analysis of the HEROES PBC studies will follow a nested randomized trial emulation approach (▇▇▇▇▇▇ 2008, ▇▇▇▇▇▇ 2013) using a treatment decision design to identify index events (▇▇▇▇▇▇▇▇▇ 2015). The goal of the study design is to emulate a sequence of hypothetical randomized trials. In each hypothetical trial, patients meeting the inclusion and exclusion criteria who are making the decision whether to initiate OCA-based treatment are randomized to either continue their existing PBC treatment or switch to an OCA-based treatment. Each randomized patient is then followed until the earliest of the end of follow-up or the time of the study endpoint (death, liver transplant, or hospitalization for hepatic decompensation). Standard survival analysis methods (eg, a ▇▇▇▇▇▇-▇▇▇▇▇ estimator of the cumulative incidence function or a ▇▇▇ proportional hazards model to estimate the hazard ratio) will then be used to estimate the effect of OCA on the study endpoint. Due to the lack of randomization, non-randomized observational studies will instead be conducted, with the goal of mimicking the hypothetical randomized trial as closely as possible. All patients will be evaluated over time from the start of the study period to identify evidence of inadequate treatment response to UDCA. Each date of inadequate response identified in the data will be considered a treatment decision point, and that date will be considered an index date, with a corresponding record being created in the analytic data for that patient index. Each patient can contribute multiple index dates, and thus patient indices, corresponding to each date they have a treatment decision point, and therefore each patient may contribute person-time to multiple patient index records.