Efficacy Analysis. 9.2.1 Analysis of Primary Endpoint The primary hypotheses are: *** The primary calculated endpoints for the trial are the percent weight loss at week 56 calculated as *** and the percentage of subjects achieving at least 5% weight loss at week 56. *** INDICATES MATERIAL THAT WAS OMITTED AND FOR WHICH CONFIDENTIAL TREATMENT WAS REQUESTED. ALL SUCH OMITTED MATERIAL WAS FILED SEPARATELY WITH THE SECURITIES AND EXCHANGE COMMISSION PURSUANT TO RULE 24b-2 PROMULGATED UNDER THE SECURITIES EXCHANGE ACT OF 1934, AS AMENDED. The intent to treat (ITT) population (Section 9.4) is the primary analysis population. For subjects who discontinue treatment prior to trial completion, every attempt will be made to have them return to the clinic at week 56 for a final weight assessment, regardless of when they discontinued treatment. For subjects who ***. Comparisons between treatments of percent weight loss will be assessed using a *** with factors of *** and ***and with ***. Comparisons between treatments of the percentage of subjects with at least 5% weight loss will be assessed by ***, with *** and *** and ***. A step-down multiple comparison procedure will be used to compare each dose group with ***. That is, comparison with *** will start at the ***. If the statistical test is significant for each of the co-primary end points at ***, then the testing will proceed to the *** also at ***. If the statistical comparison is not significant at the *** for the *** when compared with ***, then the statistical test will be stopped and the *** will not be tested. If both dose groups are significantly better than ***, then the *** and *** dose groups will be compared. The *** for the difference in the mean percent body weight reduction between treatment groups will be derived. The cumulative probability distribution as a function of percentage change in body weight for each treatment group will be plotted.
Efficacy Analysis. The primary analysis of efficacy will be based on the evaluable treated subjects, hence, only those subjects who received a complete or partial study treatment and completed a follow-up visit at 30-days post-treatment #2 will be included in the analysis using the Last Value Carried Forward method. Analyses of safety will include all subjects who received (complete or incomplete) study treatment. Efficacy will be the proportion of treated subjects determined to be improved by the reduction in the number of sweat glands from baseline to 30-days post-treatment #2.
Efficacy Analysis. The estimand for the primary efficacy analysis is defined as follows:
Efficacy Analysis. 4.4.1 Primary Efficacy Variable The primary end-point, that is, the proportion achieving “smoking reduction” at 24 weeks, will be analyzed with logistic regression modeling among eligible subjects using intent-to treat criteria and with participants who terminated the study prematurely for any reason considered to be failures.
Efficacy Analysis. Efficacy evaluation will be based on the full analysis set (see section 10.3) that includes all patients who received at least 1 dose of the study drug.
Efficacy Analysis. The key secondary endpoint, proportion of subjects achieving HiSCR50 at Week 12, will be analyzed using Xxxxxx’x exact test. Proportions and difference in proportions between treatment groups, and their associated 95% confidence intervals (CIs) will be reported. For subjects who discontinue study treatment prior to Week 12 due to any reason, their last observation will be used to impute response status. Xxxxxx’x exact test will also be used to analyze additional secondary endpoints which are dichotomized in nature, eg, proportion of subjects achieving HiSCR75 at Week 12, proportion of subjects with flare by Week 12, and other proportion endpoints. Change from baseline in NRS skin pain score, change from baseline in IHS4, and other continuous endpoints will be summarized using descriptive statistics. Change from baseline of these continuous endpoints will also be analyzed by a Mixed-effects model for repeated measures (MMRM). MMRM will include baseline as a covariate, treatment and visit as factors, and treatment-by-visit and baseline-by-visit interactions in the model.
