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Estimands in epigenome-wide association studies
BackgroundIn DNA methylation analyses like epigenome-wide association studies, effects in differentially methylated CpG sites are assessed. Two kinds...
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Confirmatory prediction-driven RCTs in comparative effectiveness settings for cancer treatment
BackgroundMedical advances in the treatment of cancer have allowed the development of multiple approved treatments and prognostic and predictive...
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Strategies to manage auxiliary pain medications in chronic pain trials: a topical review
Chronic pain trials commonly allow auxiliary pain medications such as rescue and concomitant analgesics in addition to the randomized treatment....
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The UK BiLEVE and Mendelian randomisation: using multivariable instrumental variables to address “damned if you, damned if you don’t” adjustment problems
ObjectiveTo explore the use of multivariable instrumental variables to resolve the “damned if you do, damned if you don’t” adjustment problem created...
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General Overview of the Statistical Issues in Clinical Study Designs
This chapter introduces statistical issues often encountered in confirmatory clinical studies in a typical setting - randomized trials with parallel...
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Two-year effects of semaglutide in adults with overweight or obesity: the STEP 5 trial
The STEP 5 trial assessed the efficacy and safety of once-weekly subcutaneous semaglutide 2.4 mg versus placebo (both plus behavioral intervention)...
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Common Clinical Trial Designs
Clinical Trials are studies conducted for the purpose of assessing the effect of an intervention in a defined population, or to evaluate one or more...
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Do beta-blockers reduce negative intrusive thoughts and anxiety in cancer survivors? – An emulated trial
BackgroundHigh rates of negative intrusive thoughts have been reported among cancer patients. Prevalent users of beta-blocker therapy have reported...
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Deriving GWAS summary estimates for paternal smoking in UK biobank: a GWAS by subtraction
ObjectiveTo use genome-wide association study (GWAS) by subtraction, a method for deriving novel GWASs from existing summary statistics, to derive...
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The Importance of Assessing Drug Exposure and Medication Adherence in Evaluating Investigational Medications: Ensuring Validity and Reliability of Clinical Trial Results
The objective of this current opinion paper is to draw global attention to medication adherence, emphasizing its crucial role in drug trials....
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Variability and uncertainty: interpretation and usage of pharmacometric simulations and intervals
Variability and estimation uncertainty are important sources of variation in pharmacometric simulations. Different combinations of uncertainty and...
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Age-adjusted high-dose chemotherapy followed by autologous stem cell transplantation or conventional chemotherapy with R-MP as first-line treatment in elderly primary CNS lymphoma patients – the randomized phase III PRIMA-CNS trial
BackgroundOlder primary central nervous system lymphoma (PCNSL) patients have an inferior prognosis compared to younger patients because available...
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Semaglutide in HFpEF across obesity class and by body weight reduction: a prespecified analysis of the STEP-HFpEF trial
In the STEP-HFpEF trial, semaglutide improved symptoms, physical limitations and exercise function and reduced body weight in patients with obesity...
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The Randomised Controlled Trial at the Intersection of Research Ethics and Innovation
The randomised controlled trial (RCT) has been considered for a long time as the gold standard for evidence generation to support regulatory decision...
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Confidence bands in survival analysis
BackgroundProviding estimates of uncertainty for statistical quantities is important for statistical inference. When the statistical quantity of...
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Can incorporating genoty** data into efficacy estimators improve efficiency of early phase malaria vaccine trials?
BackgroundEarly phase malaria vaccine field trials typically measure malaria infection by PCR or thick blood smear microscopy performed on serially...
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Tirzepatide after intensive lifestyle intervention in adults with overweight or obesity: the SURMOUNT-3 phase 3 trial
The effects of tirzepatide, a glucose-dependent insulinotropic polypeptide and glucagon-like peptide-1 receptor agonist, on weight reduction after...
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MDMA-assisted therapy for severe PTSD: a randomized, double-blind, placebo-controlled phase 3 study
Post-traumatic stress disorder (PTSD) presents a major public health problem for which currently available treatments are modestly effective. We...
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Accurate estimation of SNP-heritability from biobank-scale data irrespective of genetic architecture
SNP-heritability is a fundamental quantity in the study of complex traits. Recent studies have shown that existing methods to estimate genome-wide...
