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Enriched trial design

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https://www.readbyqxmd.com/read/28795844/sample-size-adjustment-designs-with-time-to-event-outcomes-a-caution
#1
Boris Freidlin, Edward L Korn
BACKGROUND: Sample size adjustment designs, which allow increasing the study sample size based on interim analysis of outcome data from a randomized clinical trial, have been increasingly promoted in the biostatistical literature. Although it is recognized that group sequential designs can be at least as efficient as sample size adjustment designs, many authors argue that a key advantage of these designs is their flexibility; interim sample size adjustment decisions can incorporate information and business interests external to the trial...
August 1, 2017: Clinical Trials: Journal of the Society for Clinical Trials
https://www.readbyqxmd.com/read/28795420/inference-for-multimarker-adaptive-enrichment-trials
#2
Richard Simon, Noah Simon
Identification of treatment selection biomarkers has become very important in cancer drug development. Adaptive enrichment designs have been developed for situations where a unique treatment selection biomarker is not apparent based on the mechanism of action of the drug. With such designs, the eligibility rules may be adaptively modified at interim analysis times to exclude patients who are unlikely to benefit from the test treatment.We consider a recently proposed, particularly flexible approach that permits development of model-based multifeature predictive classifiers as well as optimized cut-points for continuous biomarkers...
August 10, 2017: Statistics in Medicine
https://www.readbyqxmd.com/read/28793995/transcriptome-profiling-from-adipose-tissue-during-a-low-calorie-diet-reveals-predictors-of-weight-and-glycemic-outcomes-in-obese-nondiabetic-subjects
#3
Claudia Armenise, Gregory Lefebvre, Jérôme Carayol, Sophie Bonnel, Jennifer Bolton, Alessandro Di Cara, Nele Gheldof, Patrick Descombes, Dominique Langin, Wim Hm Saris, Arne Astrup, Jörg Hager, Nathalie Viguerie, Armand Valsesia
Background: A low-calorie diet (LCD) reduces fat mass excess, improves insulin sensitivity, and alters adipose tissue (AT) gene expression, yet the relation with clinical outcomes remains unclear.Objective: We evaluated AT transcriptome alterations during an LCD and the association with weight and glycemic outcomes both at LCD termination and 6 mo after the LCD.Design: Using RNA sequencing (RNAseq), we analyzed transcriptome changes in AT from 191 obese, nondiabetic patients within a multicenter, controlled dietary intervention...
August 9, 2017: American Journal of Clinical Nutrition
https://www.readbyqxmd.com/read/28783881/point-estimation-in-adaptive-enrichment-designs
#4
Kevin Kunzmann, Laura Benner, Meinhard Kieser
Adaptive enrichment designs are an attractive option for clinical trials that aim at demonstrating efficacy of therapies, which may show different benefit for the full patient population and a prespecified subgroup. In these designs, based on interim data, either the subgroup or the full population is selected for further exploration. When selection is based on efficacy data, this introduces bias to the commonly used maximum likelihood estimator. For the situation of two-stage designs with a single prespecified subgroup, we present six alternative estimators and investigate their performance in a simulation study...
August 7, 2017: Statistics in Medicine
https://www.readbyqxmd.com/read/28771664/outcome-dependent-sampling-with-interval-censored-failure-time-data
#5
Qingning Zhou, Jianwen Cai, Haibo Zhou
Epidemiologic studies and disease prevention trials often seek to relate an exposure variable to a failure time that suffers from interval-censoring. When the failure rate is low and the time intervals are wide, a large cohort is often required so as to yield reliable precision on the exposure-failure-time relationship. However, large cohort studies with simple random sampling could be prohibitive for investigators with a limited budget, especially when the exposure variables are expensive to obtain. Alternative cost-effective sampling designs and inference procedures are therefore desirable...
August 3, 2017: Biometrics
https://www.readbyqxmd.com/read/28761069/evidence-for-treatment-by-biomarker-interaction-for-fda-approved-oncology-drugs-with-required-pharmacogenomic-biomarker-testing
#6
Alexandre Vivot, Isabelle Boutron, Geoffroy Béraud-Chaulet, Jean-David Zeitoun, Philippe Ravaud, Raphaël Porcher
For oncology drugs that were approved by the US Food and Drug Administration (FDA) and required pharmacogenomic biomarker testing, we describe 1) the use of enrichment (biomarker-positive patients) and a randomized controlled design by pre-approval trials and 2) the treatment-by-biomarker interaction. From the 137 drugs included in the FDA table, we selected the 22 oncology drugs with required genetic testing in their labels. These drugs corresponded to 35 approvals supported by 80 clinical studies included in the FDA medical officer reviews of efficacy...
July 31, 2017: Scientific Reports
https://www.readbyqxmd.com/read/28756942/identifying-incipient-dementia-individuals-using-machine-learning-and-amyloid-imaging
#7
Sulantha Mathotaarachchi, Tharick A Pascoal, Monica Shin, Andrea L Benedet, Min Su Kang, Thomas Beaudry, Vladimir S Fonov, Serge Gauthier, Pedro Rosa-Neto
Identifying individuals destined to develop Alzheimer's dementia within time frames acceptable for clinical trials constitutes an important challenge to design studies to test emerging disease-modifying therapies. Although amyloid-β protein is the core pathologic feature of Alzheimer's disease, biomarkers of neuronal degeneration are the only ones believed to provide satisfactory predictions of clinical progression within short time frames. Here, we propose a machine learning-based probabilistic method designed to assess the progression to dementia within 24 months, based on the regional information from a single amyloid positron emission tomography scan...
July 11, 2017: Neurobiology of Aging
https://www.readbyqxmd.com/read/28681133/innovative-clinical-trial-designs-for-precision-medicine-in-heart-failure-with-preserved-ejection-fraction
#8
Sanjiv J Shah
A major challenge in the care of patients with heart failure and preserved ejection fraction (HFpEF) is the lack of proven therapies due to disappointing results from randomized controlled trials (RCTs). The heterogeneity of the HFpEF syndrome and the use of conventional RCT designs are possible reasons underlying the failure of these trials. There are several factors-including the widespread adoption of electronic health records, decreasing costs of obtaining high-dimensional data, and the availability of a wide variety of potential therapeutics-that have evolved to enable more innovative clinical trial designs in HFpEF...
June 2017: Journal of Cardiovascular Translational Research
https://www.readbyqxmd.com/read/28673539/trial-designs-and-results-supporting-treatment-de-escalation-and-escalation
#9
Meredith M Regan, William T Barry
Trials for escalation and de-escalation of treatment aim to improve patient care, but from different sides of the same coin with respect to disease control and burdens of treatment. De-escalation of therapy is inherently a non-inferiority question. A design with random assignment to standard of care versus de-escalated therapy is typically implemented but ordinarily will require a large sample size. Some research questions of treatment de-escalation might be asked in select patient populations using single-arm designs...
June 30, 2017: Breast: Official Journal of the European Society of Mastology
https://www.readbyqxmd.com/read/28664851/clinical-trials-in-acute-respiratory-distress-syndrome-challenges-and-opportunities
#10
REVIEW
Michael A Matthay, Daniel F McAuley, Lorraine B Ware
This year is the 50th anniversary of the first description of acute respiratory distress syndrome (ARDS). Since then, much has been learned about the pathogenesis of lung injury in ARDS, with an emphasis on the mechanisms of injury to the lung endothelium and the alveolar epithelium. In terms of treatment, major progress has been made in reducing mortality from ARDS with lung-protective ventilation, using a tidal volume of 6 mL per kg of predicted bodyweight and a plateau airway pressure of less than 30 cm H2O...
June 2017: Lancet Respiratory Medicine
https://www.readbyqxmd.com/read/28637773/follow-up-of-a-randomized-trial-on-postdischarge-nutrition-in-preterm-born-children-at-age-8-y
#11
Charlotte A Ruys, Monique van de Lagemaat, Martijn Jj Finken, Harrie N Lafeber
Background: Early nutritional interventions may modulate health risks in preterm-born infants. Previously, we showed that preterm-born infants fed an isocaloric protein- and mineral-enriched postdischarge formula (PDF) from term age to 6-mo corrected age (CA) gained more lean mass than did those fed term formula (TF). Long-term follow-up of randomized nutritional trials is important to test the hypothesis that short-term positive effects on health are sustainable.Objective: The aim of this follow-up study was to compare body size, body composition, and metabolic health at age 8 y in preterm-born children who were randomly assigned to receive either PDF or TF from term age until 6-mo CA...
June 21, 2017: American Journal of Clinical Nutrition
https://www.readbyqxmd.com/read/28636752/comparison-of-effect-sizes-between-enriched-and-non-enriched-trials-of-analgesics-for-chronic-musculoskeletal-pain-a-systematic-review
#12
Tie P Yamato, Chris G Maher, Bruno T Saragiotto, Christina Abdel Shaheed, Anne M Moseley, Chung-Wei Christine Lin, Bart Koes, Andrew J McLachlan
OBJECTIVE: To investigate the use of an enriched study design on the estimates of treatment effect in analgesic trials for chronic musculoskeletal pain METHODS: Database searches were conducted from 2004 to 2014. We included randomised placebo-controlled trials evaluating pain medications for chronic musculoskeletal pain. Methodological quality was assessed using the PEDro scale. The estimates of treatment effect on pain and adverse events were compared between enriched and non-enriched designs...
June 21, 2017: British Journal of Clinical Pharmacology
https://www.readbyqxmd.com/read/28636403/applying-precision-medicine-to-trial-design-using-physiology-extracorporeal-co2-removal-for-ards
#13
Ewan C Goligher, Marcelo B P Amato, Arthur S Slutsky
In clinical trials of therapies for ARDS, the average treatment effect in the study population may be attenuated because individual patient responses vary widely. This inflates sample size requirements and increases the cost and difficulty of conducting successful clinical trials. One solution is to enrich the study population with patients most likely to benefit based on predicted patient response to treatment (predictive enrichment). In this perspective, we apply the precision medicine paradigm to the emerging use of extracorporeal CO2 removal (ECCO2R) for ultra-protective ventilation in ARDS...
June 21, 2017: American Journal of Respiratory and Critical Care Medicine
https://www.readbyqxmd.com/read/28631582/effect-of-green-tea-extract-supplementation-on-glycogen-replenishment-in-exercised-human-skeletal-muscle
#14
Tsen-Wei Tsai, Chia-Chen Chang, Su-Fen Liao, Yi-Hung Liao, Chien-Wen Hou, Jung-Piao Tsao, I-Shiung Cheng
The purpose of this study was to investigate the effects of 8-week green tea extract (GTE) supplementation on promoting postexercise muscle glycogen resynthesis and systemic energy substrate utilisation in young college students. A total of eight healthy male participants (age: 22·0 (se 1·0) years, BMI: 24·2 (se 0·7) kg/m2, VO2max: 43·2 (se 2·4) ml/kg per min) participated in this study. GTE (500 mg/d for 8 weeks) was compared with placebo in participants in a double-blind/placebo-controlled and crossover study design with an 8-week washout period...
May 2017: British Journal of Nutrition
https://www.readbyqxmd.com/read/28626811/the-enrich-study-to-evaluate-the-effectiveness-of-a-combination-intervention-package-to-improve-isoniazid-preventive-therapy-initiation-adherence-and-completion-among-people-living-with-hiv-in-ethiopia-rationale-and-design-of-a-mixed-methods-cluster-randomized
#15
Andrea A Howard, Yael Hirsch-Moverman, Suzue Saito, Tsigereda Gadisa, Amrita Daftary, Zenebe Melaku
BACKGROUND: Isoniazid preventive therapy (IPT) prevents tuberculosis among HIV-positive individuals, however implementation is suboptimal. Implementation science studies are needed to identify interventions to address this evidence-to-program gap. OBJECTIVE: The ENRICH Study is a mixed methods cluster randomized trial aimed at evaluating the effectiveness and acceptability of a combination intervention package (CIP) to improve IPT implementation in Ethiopia. DESIGN: Ten health centers were randomized to receive the CIP or standard of care...
June 2017: Contemporary Clinical Trials Communications
https://www.readbyqxmd.com/read/28593470/effect-of-selenium-from-selenium-enriched-kale-sprout-versus-other-selenium-sources-on-productivity-and-selenium-concentrations-in-egg-and-tissue-of-laying-hens
#16
Anut Chantiratikul, Orawan Chinrasri, Piyanete Chantiratikul
A 6-week trial was conducted to compare the effect of selenium (Se) from hydroponically produced Se-enriched kale sprout (HPSeKS), sodium selenite (SS), and Se-enriched yeast (SeY) in laying hens. A total of 144 40-week-old hens were randomly divided into four groups, according to a completely randomized design. Each group consisted of four replicates with nine hens per replicate. The dietary treatments were T1 (basal diet) and T2, T3, and T4 (basal diets supplemented with 0.30 mg Se/kg from SS, SeY, and HPSeKS, respectively)...
June 7, 2017: Biological Trace Element Research
https://www.readbyqxmd.com/read/28581200/natural-history-of-second-biochemical-failure-following-salvage-radiation-therapy-for-prostate-cancer-a-multi-institution-study
#17
Vasu Tumati, William C Jackson, Ahmed E Abugharib, Ganesh Raj, Claus Roehrborn, Yair Lotan, Kevin Courtney, Aditya Bagrodia, Jeffrey C Gahan, Zachary S Zumsteg, Michael R Folkert, Aaron M Laine, Raquibul Hannan, Daniel E Spratt, Neil B Desai
OBJECTIVES: To describe the natural history of prostate cancer in men who experience a second biochemical recurrence (BCR) after salvage radiotherapy (SRT) following prostatectomy. SUBJECTS/PATIENTS AND METHODS: Following SRT at two institutions from 1986-2013, 286 patients developed second BCR, defined as two rises in PSA of ≥0.2 ng/mL above nadir. Event rates for distant metastasis (DM) or freedom from DM (FFDM), castration-resistant prostate cancer (CRPC), prostate cancer-specific survival (PCSS), and overall survival (OS) were estimated using the Kaplan-Meier method...
June 5, 2017: BJU International
https://www.readbyqxmd.com/read/28570268/network-analysis-of-the-genomic-basis-of-the-placebo-effect
#18
Rui-Sheng Wang, Kathryn T Hall, Franco Giulianini, Dani Passow, Ted J Kaptchuk, Joseph Loscalzo
The placebo effect is a phenomenon in which patients who are given an inactive treatment (e.g., inert pill) show a perceived or actual improvement in a medical condition. Placebo effects in clinical trials have been investigated for many years especially because placebo treatments often serve as the control arm of randomized clinical trial designs. Recent observations suggest that placebo effects may be modified by genetics. This observation has given rise to the term "placebome," which refers to a group of genome-related mediators that affect an individual's response to placebo treatments...
June 2, 2017: JCI Insight
https://www.readbyqxmd.com/read/28569123/diffusion-weighted-imaging-or-computerized-tomography-perfusion-assessment-with-clinical-mismatch-in-the-triage-of-wake-up-and-late-presenting-strokes-undergoing-neurointervention-with-trevo-dawn-trial-methods
#19
Tudor G Jovin, Jeffrey L Saver, Marc Ribo, Vitor Pereira, Anthony Furlan, Alain Bonafe, Blaise Baxter, Rishi Gupta, Demetrius Lopes, Olav Jansen, Wade Smith, Daryl Gress, Steven Hetts, Roger J Lewis, Ryan Shields, Scott M Berry, Todd L Graves, Tim Malisch, Ansaar Rai, Kevin N Sheth, David S Liebeskind, Raul G Nogueira
Rationale Efficacy of mechanical thrombectomy for acute stroke due to large vessel occlusion initiated beyond 6 h of time last seen well has not been demonstrated in randomized trials. Aim To establish whether subjects considered to have substantial areas of salvageable brain based on age-adjusted clinical core mismatch who can undergo endovascular treatment within 6-24 h from time last seen well (TLSW) have better outcomes at three months compared to subjects treated with standard medical therapy alone...
August 2017: International Journal of Stroke: Official Journal of the International Stroke Society
https://www.readbyqxmd.com/read/28536178/adaptive-enrichment-designs-for-stroke-clinical-trials
#20
REVIEW
Michael Rosenblum, Daniel F Hanley
No abstract text is available yet for this article.
July 2017: Stroke; a Journal of Cerebral Circulation
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