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This method uses the "expected" (imputed) behavior of placebo, based on prior trials.
For biomarker analyses (primarily muscle biopsy) the sample size calculation is based on prior trials detecting changes in muscle protein synthesis and associated signalling following exercise, nutritional and pharmacological interventions (increase in signalling >20% with SD in signalling of 2%).
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As expected, based on prior randomized trials, the distribution of posterior mean ORRs for trials testing monoclonal antibodies was shifted toward higher ORRs relative to the distribution for trials not including monoclonal antibodies (54% vs 46%, p-value of 0.0042 based on a Kolmogorov-Smirnov test).
Based on prior clinical trials indicating that γ-aminobutyric acid (GABA -based anticonvulsant medications reduce druGABA -basedn cocanticonvulsant study participants, we tested the effects of valproate treatmedications-induced cocaine creduce.
Based on prior local trials with early dementia patients, the recruitment of 201 participants is required (67 per arm), assuming an attrition rate of about 35%% [ 33].
An attrition rate of approximately 25% is expected, based on prior community trials of Tai-chi interventions in elderly patients, and exercise programs for patients with severe mental illnesses [ 33].
We assumed a difference of 1.5 days between both groups with a standard deviation (SD) of 1.5, based on prior preventive clinical trial studies.
One of the other safety concerns closely monitored on this trial was based on prior observations in the early phase I trials [ 19, 20] of significant peripheral neuropathy and neurologic AEs.
The sample size estimation was based on prior data from a similar clinical trial showing mean (SD) in MADRS score (from baseline to end of the study (week 24)) was decreased by around 10.0 ± 4.3 and increased by around 0.9 ± 2.6 (in respectively the NAC and the placebo group (Berk et al. 2008).
The designer of a clinical trial needs to make many assumptions about real-life practice based on prior knowledge.
Expensive trials can only be justified if there is a reasonable probability of success based on prior data.
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