Determination of Sample Size Clause Samples
The Determination of Sample Size clause establishes how the number of samples to be tested, inspected, or analyzed will be set within an agreement or protocol. Typically, this clause outlines the criteria, statistical methods, or standards that will be used to decide the appropriate sample size, such as referencing industry guidelines or specifying a calculation based on population size. Its core function is to ensure that both parties have a clear, agreed-upon method for determining sample quantities, thereby reducing disputes and ensuring that testing or inspection is both sufficient and efficient.
Determination of Sample Size. The planned total number of randomized patients is approximately 300 and will be randomized 1:1:1 to TNX-1900 30 IU QAM, placebo QPM: TNX-1900 30 IU BID: or placebo BID. The sample size estimate was derived using a two-sided t-test with 90% power and 5% two-sided significance level to test the primary efficacy statistical superiority hypothesis of TNX-1900 30 IU BID over placebo. Based on a post-hoc analysis of a chronic migraine subgroup from a prior study, it is anticipated that a difference in mean change from Baseline to study end (last 28 days of double-blind) in migraine headache days between TNX-1900 30 IU BID and placebo BID would be approximately 1.84 with a common standard deviation of ± 3.95. As such the required sample size per group is approximately 100. Accounting for the multiplicity adjustment by applying an alpha of 2.5% to the first test yields power of approximately 85% with the above assumptions.
Determination of Sample Size. The sample size of this study is based on the results obtained with a randomized, double blind, study with Racecadotril in the treatment of hospitalized children aged 3-60 months suffering from acute watery diarrhea (Bio-Projet Study nr. 45, ▇▇▇▇▇▇▇ ▇▇▇▇▇ et al. 1998). A total of 135 subjects were analyzed in this study. Data of the study showed following recovery rates over time: Duration of treatment Percentage of recovered patients Placebo Racecadotril Sample size per group required for a power of 80% using log-rank test (+10% drop out rate) after 1 day (24 hours) 18.3% 46.6% 41 (46) After 2 days (50 hours) 35.8% 70.1% 36 (40) After 3 days (72 hours) 59.4% 84.2% 55 (62) After 4 days (96 hours) 76.3% 90.1% 119 (132) After 5 days (120 hours) 80.6% 94.7% 90 (100) The mean duration of diarrhea was 64 (±4.6) hours in the placebo group (median 64 hours) and 40 (±4.1) hours in the Racecadotril group (median 28 hours), Therefore an appropriate approach to investigate a difference between recovery rates of the two treatment groups would be after a treatment duration of at least 3 days (i.e 72 hours).When the sample size in each group is 55 (with a total number of recoveries of at least 26), the two-sided log-rank test for equality of survival curves will have 80% power to detect the difference between 84.2% rate of recovery in the Racecadotril group and the 59.4% rate of recovery in the placebo group after a treatment duration of 72 hours. Adding a 10% drop-out rate the sample size per groups would be 62 subjects i.e. 124 subjects in total. A minimum number of 20 subjects should be available in each age group (3 – < 24 months, 2 years to 11 years, 12 to < 18 years). A subgroup analysis per age group (3 – < 24 months, 2 years to 11 years, 12 to < 18 years) will be performed.
Determination of Sample Size. To reliably detect (p<0.05, statistical power >80%) a more than two-fold increase in the odds of achieving smoking reduction at 24 weeks among the active versus placebo groups, and assuming a smoking reduction rate of 15% in the placebo group versus 25% in the active snus group (corresponding to an odds ratio of 1.9), the target sample size can be estimated at 250 per group, that is, a total size of 500 study participants. In previous randomized studies of NRT versus placebo, NRT has resulted in an increased proportion of participants achieving short- to medium term smoking cessation with odds ratios in the order of 1.5-3. Epidemiological, cross-sectional studies from Sweden have indicated that snus might be more effective as an aid in smoking cessation than NRT. Against this background the mentioned target sample size should provide reasonable statistical power for current purposes.
Determination of Sample Size. There is no formal sample size calculation. The sample size is based on qualitative considerations and is sufficient to provide safety evaluation of eteplirsen in the studied population.
Determination of Sample Size. The sample size is based on a fixed-sequence strategy which tests for a treatment difference in the primary endpoint (percent change at Week 12 in liver fat from baseline as determined by MRI-PDFF) between each randomized TVB-2640 dose group (25 and 50 mg) versus placebo. The fixed-sequence strategy will start with the highest dose group comparison. If statistically significant, then testing will proceed to the low dose group comparison. Each TVB-2640 dose group will be compared to the pooled placebo group using an F-test test from an analysis of covariance (ANCOVA) model with fixed effects for the stratification factor (diabetes presence/absence) and treatment group (i.e., TVB-2640 dose groups and pooled placebo) and with the baseline MRI-PDFF value as a covariate. Since the primary efficacy endpoint may not be normally distributed, the non-parametric Wilcoxon rank-sum test is conservatively used, instead of the F-test, for power calculations. The sample size is based on a conservative assumption that the primary analysis is going to be performed in the US population only, resulting in 30 evaluable subjects in the placebo group, and 30 evaluable subjects in each of the randomized TVB-2640 dose levels. Based on ▇▇▇▇▇ (2016), it is conservatively assumed that the primary endpoint has a standard deviation of 30. Power calculations assume the primary endpoint is lognormally distributed with a standard deviation of 30, there are at least 30 evaluable subjects in each treatment group, and the two-sided Wilcoxon rank sum test will be used to test each pairwise treatment difference at the 0.05 Type I error level. Under the fixed-sequence strategy which maintains an overall 0.05 Type I error rate, the study has at least 80% overall power to detect both treatment differences (i.e., both high dose [50 mg] versus placebo and low dose [25 mg] versus placebo), if each mean treatment difference is at least 24. If the study does not proceed to the high-dose level, (i.e., there are 30 evaluable subjects treated with 25 mg TVB-2640 and only 15 with placebo), then the study has at least 77% power to detect a mean treatment difference of at least 24. The study will have more power if the subjects from China are included in the primary analysis and the same assumptions of treatment difference and variability hold. Power calculations for the key secondary endpoint (percentage of subjects with at least a 30% reduction in the primary endpoint) are based on pairwise comparisons w...
Determination of Sample Size. This is a double-blind study. Each cohort will be independently analyzed. A total of approximately 26 subjects will be randomized in each cohort. The first 12 subjects will be randomized to the KPL-716 and the placebo arms in a 3:1 randomization ratio. The rest of the 14 subjects will be randomized in a 1:1 randomization ratio. There will be approximately 16 subjects and 10 subjects randomized to the KPL-716 arm and the placebo arm respectively. Based on a two-sample t-test for the primary efficacy endpoint, change from baseline in weekly average of WI-NRS at Week 8, assumed mean changes of 4 for the KPL-716 arm and
Determination of Sample Size. Using the sample size in the 2 populations (Section 6.2), the Sponsor computed power to detect treatment effects (relative hazards) of varying sizes, estimated using ▇▇▇ proportional hazards regression. Computations are based on the formulae of ▇▇▇▇▇▇▇▇▇▇ 1983. To account for confounding control due to the application of IP weights, the Sponsor inflated the variance from the ▇▇▇▇▇▇▇▇▇▇ formula using factors derived by Shook-Sa 2020. It was conservatively assumed just one control observation per patient. In the Komodo Health data, a preliminary investigation found that approximately 395 patients who meet entry criteria will initiate OCA and that 5916 patients will contribute at least one record to the control group (UDCA nonresponders). In the Global PBC registry, the Sponsor expects to have 344 patients meeting entry criteria who initiate OCA, and 2200 who contribute at least one observation to the control group. Power in both cohorts was computed for relative hazards ranging from 0.5 to 0.9 with an alpha level of
Determination of Sample Size. A total of up to 24 subjects with renal impairment (up to 8 subjects with severe impairment, and, if necessary, up to 8 subjects with moderate impairment, and/or up to 8 subjects with mild impairment, per eGFR using the CKD-EPI equation) and approximately 8 to 24 matched-control healthy subjects with normal renal function will be enrolled in the study with the goal of having at least 6 subjects from each renal impairment group enrolled in the study and at least 6 subjects with normal renal function complete the study. The sample size chosen for this study was based upon precedent set by other PK studies of similar nature and was not based on power calculations to detect statistically significant differences among groups. This number is considered a sufficient sample size to evaluate the PK of LOXO-305 under various degrees of renal function.
Determination of Sample Size. It is expected that the responder rate for this study is approximately 80% based on the results of a pivotal study of a similar investigational product Juvéderm Volbella XC. The margin of error (associated with 95% CI) for estimating an 80% expected responder rate is 10%, which is desirable to attain by having a sample size of 60 subjects for the study. Patients’ dropout rate is expected to be negligibly small at Day 30 post-treatment when the primary efficacy assessment is collected, thus 60 subjects will be recruited into the study.
Determination of Sample Size. This is a double-blind study. Each cohort will be independently analyzed. A total of approximately 26 subjects will be randomized in each cohort. The first 12 subjects will be randomized to the KPL-716 and the placebo arms in a 3:1 randomization ratio. The rest of the 14 subjects will be randomized in a 1:1 randomization ratio. There will be approximately 16 subjects and 10 subjects randomized to the KPL-716 arm and the placebo arm respectively. Based on a two- sample t-test for the primary efficacy endpoint, change from baseline in weekly average of WI- NRS at Week 8, assumed mean changes of 4 for the KPL-716 arm and 1.5 for the placebo arm, a total sample size of 26 subjects per cohort will provide about 80% power to detect a 2.5-point mean difference with a standard deviation of 2.8, given a two-sided alpha of 0.2. Given the exploratory nature of this pilot study, the precision attained with the above cohort samples size is acceptable for early signal of efficacy assessments.