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Enriched trial design

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https://www.readbyqxmd.com/read/28935438/lipids-in-the-intensive-care-unit-recommendations-from-the-espen-expert-group
#1
REVIEW
Philip C Calder, Michael Adolph, Nicolaas E Deutz, Teodoro Grau, Jacqueline K Innes, Stanislaw Klek, Shaul Lev, Konstantin Mayer, Adina T Michael-Titus, Lorenzo Pradelli, Mark Puder, Hester Vlaardingerbroek, Pierre Singer
This article summarizes the presentations given at an ESPEN Workshop on "Lipids in the ICU" held in Tel Aviv, Israel in November 2014 and subsequent discussions and updates. Lipids are an important component of enteral and parenteral nutrition support and provide essential fatty acids, a concentrated source of calories and building blocks for cell membranes. Whilst linoleic acid-rich vegetable oil-based enteral and parenteral nutrition is still widely used, newer lipid components such as medium-chain triglycerides and olive oil are safe and well tolerated...
September 7, 2017: Clinical Nutrition: Official Journal of the European Society of Parenteral and Enteral Nutrition
https://www.readbyqxmd.com/read/28933670/on-enrichment-strategies-for-biomarker-stratified-clinical-trials
#2
Xiaofei Wang, Jingzhu Zhou, Ting Wang, Stephen L George
In the era of precision medicine, drugs are increasingly developed to target subgroups of patients with certain biomarkers. In large all-comer trials using a biomarker stratified design (BSD), the cost of treating and following patients for clinical outcomes may be prohibitive. With a fixed number of randomized patients, the efficiency of testing certain treatments parameters, including the treatment effect among biomarker positive patients and the interaction between treatment and biomarker, can be improved by increasing the proportion of biomarker positives on study, especially when the prevalence rate of biomarker positives is low in the underlying patient population...
September 21, 2017: Journal of Biopharmaceutical Statistics
https://www.readbyqxmd.com/read/28922068/influence-of-flaxseed-lignan-supplementation-to-older-adults-on-biochemical-and-functional-outcome-measures-of-inflammation
#3
Yunyun Di, Jennifer Jones, Kerry Mansell, Susan Whiting, Sharyle Fowler, Lilian Thorpe, Jennifer Billinsky, Navita Viveky, Pui Chi Cheng, Ahmed Almousa, Thomas Hadjistavropoulos, Jane Alcorn
Evidence from the literature suggests that dietary flaxseed lignans have the ability to modulate inflammation, which is recognized as the underlying basis of multiple chronic human diseases in older adults. Our objective was to determine the effects of oral lignan supplementation on biochemical and functional indicators of inflammation as well as safety and tolerability in older healthy adults. We designed a randomized, double-blind, placebo-controlled clinical trial in older healthy adults (60-80 years) to assess flaxseed lignan-enriched complex (∼38% secoisolariciresinol diglucoside [SDG]; 600 mg SDG dose) oral supplementation effects on biochemical and functional indicators of inflammation and safety and tolerability in older healthy adults after 6 months of once-daily oral administration...
September 18, 2017: Journal of the American College of Nutrition
https://www.readbyqxmd.com/read/28906031/design-optimization-for-clinical-trials-in-early-stage-manifest-huntington-s-disease
#4
Chris Frost, Amy Mulick, Rachael I Scahill, Gail Owen, Elizabeth Aylward, Blair R Leavitt, Alexandra Durr, Raymund A C Roos, Beth Borowsky, Julie C Stout, Ralf Reilmann, Douglas R Langbehn, Sarah J Tabrizi, Cristina Sampaio
OBJECTIVES: The purpose of this study was to inform the design of randomized clinical trials in early-stage manifest Huntington's disease through analysis of longitudinal data from TRACK-Huntington's Disease (TRACK-HD), a multicenter observational study. METHODS: We compute sample sizes required for trials with candidate clinical, functional, and imaging outcomes, whose aims are to reduce rates of change. The calculations use a 2-stage approach: first using linear mixed models to estimate mean rates of change and components of variability from TRACK-HD data and second using these to predict sample sizes for a range of trial designs...
September 14, 2017: Movement Disorders: Official Journal of the Movement Disorder Society
https://www.readbyqxmd.com/read/28903955/in-the-elderly-meat-protein-assimilation-from-rare-meat-is-lower-than-that-from-meat-that-is-well-done
#5
Caroline Buffière, Claire Gaudichon, Noureddine Hafnaoui, Carole Migné, Valérie Scislowsky, Nadezda Khodorova, Laurent Mosoni, Adeline Blot, Yves Boirie, Dominique Dardevet, Véronique Santé-Lhoutellier, Didier Rémond
Background: Meat cooking conditions in in vitro and in vivo models have been shown to influence the rate of protein digestion, which is known to affect postprandial protein metabolism in the elderly.Objectives: The present study was conducted to demonstrate the effect of cooking conditions on meat protein assimilation in the elderly. We used a single-meal protocol to assess the meat protein absorption rate and estimate postprandial meat protein utilization in elderly subjects.Design: The study recruited 10 elderly volunteers aged 70-82 y...
September 13, 2017: American Journal of Clinical Nutrition
https://www.readbyqxmd.com/read/28899446/affective-and-emotional-dysregulation-as-pre-dementia-risk-markers-exploring-the-mild-behavioral-impairment-symptoms-of-depression-anxiety-irritability-and-euphoria
#6
Zahinoor Ismail, Jennifer Gatchel, Daniel R Bateman, Ricardo Barcelos-Ferreira, Marc Chantillon, Judith Jaeger, Nancy J Donovan, Moyra E Mortby
BACKGROUND: Affective and emotional symptoms such as depression, anxiety, euphoria, and irritability are common neuropsychiatric symptoms (NPS) in pre-dementia and cognitively normal older adults. They comprise a domain of Mild Behavioral Impairment (MBI), which describes their emergence in later life as an at-risk state for cognitive decline and dementia, and as a potential manifestation of prodromal dementia. This selective scoping review explores the epidemiology and neurobiological links between affective and emotional symptoms, and incident cognitive decline, focusing on recent literature in this expanding field of research...
September 13, 2017: International Psychogeriatrics
https://www.readbyqxmd.com/read/28895274/methodological-recommendations-for-cognition-trials-in-bipolar-disorder-by-the-international-society-for-bipolar-disorders-targeting-cognition-task-force
#7
REVIEW
K W Miskowiak, K E Burdick, A Martinez-Aran, C M Bonnin, C R Bowie, A F Carvalho, P Gallagher, B Lafer, C López-Jaramillo, T Sumiyoshi, R S McIntyre, A Schaffer, R J Porter, I J Torres, L N Yatham, A H Young, L V Kessing, E Vieta
OBJECTIVES: To aid the development of treatment for cognitive impairment in bipolar disorder, the International Society for Bipolar Disorders (ISBD) convened a task force to create a consensus-based guidance paper for the methodology and design of cognition trials in bipolar disorder. METHODS: The task force was launched in September 2016, consisting of 18 international experts from nine countries. A series of methodological issues were identified based on literature review and expert opinion...
September 12, 2017: Bipolar Disorders
https://www.readbyqxmd.com/read/28893090/an-in-depth-review-of-chemical-angiogenesis-inhibitors-for-treating-hepatocellular-carcinoma
#8
Jean-Luc Raoul, Marine Gilabert, Xavier Adhoute, Julien Edeline
Hepatocellular carcinoma (HCC) is a frequent and severe complication of cirrhosis. Most HCC patients initially present with or progress to advanced stage disease and require systemic treatment. As hypervascularization is a major characteristic of HCC, antiangiogenic drugs have been tested. Areas covered: In this review, we summarize data on the use of drugs targeting the angiogenesis. Despite many trials, in 2017 only 3 drugs, all antiangiogenic, have demonstrated efficacy in first (sorafenib, lenvatinib) or second line (regorafenib) treatment of advanced HCC...
October 2017: Expert Opinion on Pharmacotherapy
https://www.readbyqxmd.com/read/28887317/biomarker-stratified-phase-iii-clinical-trials-enhancement-with-a-subgroup-focused-sequential-design
#9
Shigeyuki Matsui, John Crowley
Among various design approaches to phase III clinical trials with a predictive biomarker, the marker-stratified all-comers design is advantageous because it allows for establishing the utility of both treatment and biomarker, but it is often criticized for requiring large sample sizes, since the design includes both marker-positive and marker-negative patients. In this paper, we propose a simple but flexible subgroup-focused design for marker-stratified trials that allows both sequential assessment across marker-defined subgroups and adaptive subgroup selection, while retaining an assessment using the entire patient cohort at the final analysis stage, possibly using established marker-based multiple testing procedures...
September 8, 2017: Clinical Cancer Research: An Official Journal of the American Association for Cancer Research
https://www.readbyqxmd.com/read/28877896/intake-of-potassium-and-magnesium-enriched-salt-improves-functional-outcome-after-stroke-a-randomized-multicenter-double-blind-controlled-trial
#10
Wen-Harn Pan, Ying-Ho Lai, Wen-Ting Yeh, Jiunn-Rong Chen, Jiann-Shing Jeng, Chyi-Huey Bai, Ruey-Tay Lin, Tsong-Hai Lee, Ku-Chou Chang, Huey-Juan Lin, Chin-Fu Hsiao, Chang-Ming Chern, Li-Ming Lien, Chung-Hsiang Liu, Wei-Hung Chen, Anna Chang
Background: Stroke is one of the leading causes of mortality and neurologic deficits. Management measures to improve neurologic outcomes are in great need. Our previous intervention trial in elderly subjects successfully used salt as a carrier for potassium, demonstrating a 41% reduction in cardiovascular mortality by switching to potassium-enriched salt. Dietary magnesium has been associated with lowered diabetes and/or stroke risk in humans and with neuroprotection in animals.Objective: Because a large proportion of Taiwanese individuals are in marginal deficiency states for potassium and for magnesium and salt is a good carrier for minerals, it is justifiable to study whether further enriching salt with magnesium at an amount near the Dietary Reference Intake (DRI) amount may provide additional benefit for stroke recovery...
September 6, 2017: American Journal of Clinical Nutrition
https://www.readbyqxmd.com/read/28856711/rationale-and-design-of-the-canari-study-a-case-control-study-investigating-the-association-between-prostate-cancer-and-5-alpha-reductase-inhibitors-for-symptomatic-benign-prostate-hypertrophy-by-linking-sniiram-and-pathology-laboratories-in-a-specific-region
#11
Lucie-Marie Scailteux, Frédéric Balusson, Sébastien Vincendeau, Nathalie Rioux-Leclercq, Emmanuel Nowak
Benign prostate hypertrophy (BPH) could be associated with low urinary symptoms requiring medical treatment: 5 alpha-reductase inhibitors (5-ARI) or ɑ-blockers. Two clinical trials investigating 5-ARI use in prostate cancer (PCa) primary prevention highlighted a potential safety signal with an increased risk of high grade PCa. Later observational studies failed to show similar results but have some limits. The present paper focuses on describing the protocol of the CANARI study and its feasibility, as regards the matching process of two pseudo-anonymous databases...
August 30, 2017: Fundamental & Clinical Pharmacology
https://www.readbyqxmd.com/read/28842510/impact-of-therapy-on-genomics-and-transcriptomics-in-high-risk-prostate-cancer-treated-with-neoadjuvant-docetaxel-and-androgen-deprivation-therapy
#12
Himisha Beltran, Alexander W Wyatt, Edmund Chedgy, Adam Donoghue, Matti Annala, Evan Warner, Kevin Beja, Michael Sigouros, Fan Mo, Ladan Fazli, Colin C Collins, James A Eastham, Michael J Morris, Mary-Ellen Taplin, Andrea Sboner, Susan Halabi, Martin E Gleave
BACKGROUND: The combination of docetaxel chemotherapy and androgen deprivation therapy (ADT) has become a standard treatment for patients with metastatic prostate cancer. The recently accrued Phase III CALGB 90203 trial was designed to investigate the clinical effectiveness of this treatment approach earlier in the disease. Specimens from this trial offer a unique opportunity to interrogate the acute molecular response to docetaxel and ADT and identify predictive biomarkers. METHODS: We evaluated baseline clinical data, needle biopsies and radical prostatectomy (RP) specimens from 52 (of 788) patients enrolled on CALGB 90203 at one high volume center...
August 25, 2017: Clinical Cancer Research: An Official Journal of the American Association for Cancer Research
https://www.readbyqxmd.com/read/28830428/the-effect-of-oligofructose-enriched-inulin-supplementation-on-gut-microbiota-nutritional-status-and-gastrointestinal-symptoms-in-paediatric-coeliac-disease-patients-on-a-gluten-free-diet-study-protocol-for-a-pilot-randomized-controlled-trial
#13
Urszula Krupa-Kozak, Natalia Drabińska, Elżbieta Jarocka-Cyrta
BACKGROUND: A lifelong gluten-free diet (GFD) is regarded as the only proven and accepted therapy for coeliac disease (CD). However, even patients who strictly follow a GFD often suffer from intestinal symptoms and malabsorption. Selective modulation of intestinal microbiota with prebiotics could remedy various symptoms associated with CD. The use of prebiotics in the treatment of intestinal diseases remains insufficiently investigated. To our knowledge, this study makes the first attempt to evaluate the effect of prebiotic supplementation on gastrointestinal symptoms and nutritional status of children with CD...
August 22, 2017: Nutrition Journal
https://www.readbyqxmd.com/read/28815894/challenges-of-developing-and-conducting-clinical-trials-in-rare-disorders
#14
REVIEW
Lucas Kempf, Jonathan C Goldsmith, Robert Temple
Rare disease drug development is a rapidly expanding field. Clinical researchers in rare diseases face many challenges when conducting trials in small populations. Disease natural history is often poorly understood and the ability to detect clinically meaningful outcomes requires understanding of their rate of occurrence and variability, both of which contribute to difficulties in powering a study. Standard trial designs are not optimized to obtain adequate safety and efficacy data from small numbers of patients, so alternative designs (enrichment, crossover, adaptive, N-of 1) need to be considered...
August 16, 2017: American Journal of Medical Genetics. Part A
https://www.readbyqxmd.com/read/28814393/soya-maize-and-sorghum-based-ready-to-use-therapeutic-food-with-amino-acid-is-as-efficacious-as-the-standard-milk-and-peanut-paste-based-formulation-for-the-treatment-of-severe-acute-malnutrition-in-children-a-noninferiority-individually-randomized-controlled
#15
Paluku Bahwere, Peter Akomo, Mwawi Mwale, Hitoshi Murakami, Chrissy Banda, Sylvester Kathumba, Chimwemwe Banda, Solomon Jere, Kate Sadler, Steve Collins
Background: Development of more cost-effective ready-to-use therapeutic food (RUTF) is a global public health priority. To date, previous lower-cost recipes have been less effective than the standard peanut and milk (PM)-based RUTF, particularly in children aged <24 mo.Objective: We aimed to compare the efficacy of the PM-RUTF to a milk-free soya, maize, and sorghum (FSMS)-RUTF enriched with crystalline amino acids without cow milk powder and a milk, soya, maize, and sorghum (MSMS)-RUTF containing 9.3% skim cow milk powder...
August 16, 2017: American Journal of Clinical Nutrition
https://www.readbyqxmd.com/read/28795844/sample-size-adjustment-designs-with-time-to-event-outcomes-a-caution
#16
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
#17
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
#18
RANDOMIZED CONTROLLED TRIAL
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...
September 2017: American Journal of Clinical Nutrition
https://www.readbyqxmd.com/read/28783881/point-estimation-in-adaptive-enrichment-designs
#19
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
#20
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
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