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Enriched trial design

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https://www.readbyqxmd.com/read/29152847/a-case-study-in-identifying-targeted-patients-population-in-major-depressive-disorder-by-enhanced-enrichment-design
#1
Peter Zhang, Kevin Carroll, Mary Hobart, Carole Augustine, Gary Koch
Despite advances in clinical trial design, failure rates near 80% in phase 2 and 50% in phase 3 have recently been reported. The challenges to successful drug development are particularly acute in central nervous system trials such as for pain, schizophrenia, mania, and depression because high-placebo response rates lessen assay sensitivity, diminish estimated treatment effect sizes, and thereby decrease statistical power. This paper addresses the importance of rigorous patient selection in major depressive disorder trials through an enhanced enrichment paradigm...
November 19, 2017: Pharmaceutical Statistics
https://www.readbyqxmd.com/read/29152241/effects-of-matrix-on-plasma-levels-of-epa-and-dha-in-dogs
#2
Kay Goffin, Marc van Maris, Ronald J Corbee
EPA and DHA are often used in veterinary medicine due to their beneficial effects for several medical conditions such as osteoarthritis. EPA and DHA are administered to dogs through different matrices. The aim of the present study was to determine the effects on the plasma levels in dogs caused by various matrices for EPA and DHA administration. In this study, three different n-3 PUFA formulations were used: soft chew tablet (CCx); liquid fish oil (LFO); and enriched kibbles (EK). The formulations were administered single-dose and compared in a randomised, cross-over designed study with a 1-week wash-out period...
2017: Journal of Nutritional Science
https://www.readbyqxmd.com/read/29142972/a-drug-development-tool-for-trial-enrichment-in-patients-with-autosomal-dominant-polycystic-kidney-disease
#3
Ronald D Perrone, Mohamad-Samer Mouksassi, Klaus Romero, Frank S Czerwiec, Arlene B Chapman, Berenice Y Gitomer, Vicente E Torres, Dana C Miskulin, Steve Broadbent, Jean F Marier
Introduction: Total kidney volume (TKV) is a promising imaging biomarker for tracking and predicting the natural history of patients with autosomal dominant polycystic kidney disease. Methods: A drug development tool was developed by linking longitudinal TKV measurements to the probability of a 30% decline of estimated glomerular filtration rate (eGFR) or end-stage renal disease. Drug development tools were developed based on observational data collected over multiple decades for an eGFR decline and end-stage renal disease in 641 and 866 patients with autosomal dominant polycystic kidney disease, respectively...
May 2017: KI Reports
https://www.readbyqxmd.com/read/29110741/effect-of-fibre-additions-to-flatbread-flour-mixes-on-glucose-kinetics-a-randomised-controlled-trial
#4
Hanny M Boers, Theo H van Dijk, Harry Hiemstra, Anne-Roos Hoogenraad, David J Mela, Harry P F Peters, Roel J Vonk, Marion G Priebe
We previously found that guar gum (GG) and chickpea flour (CPF) added to flatbread wheat flour lowered postprandial blood glucose (PPG) and insulin responses dose dependently. However, rates of glucose influx cannot be determined from PPG, which integrates rates of influx, tissue disposal and hepatic glucose production. The objective was to quantify rates of glucose influx and related fluxes as contributors to changes in PPG with GG and CPF additions to wheat-based flatbreads. In a randomised cross-over design, twelve healthy males consumed each of three different 13C-enriched meals: control flatbreads (C), or C incorporating 15 % CPF with either 2 % (GG2) or 4 % (GG4) GG...
November 7, 2017: British Journal of Nutrition
https://www.readbyqxmd.com/read/29100397/correlating-programmed-death-ligand-1-pd-l1-expression-mismatch-repair-deficiency-and-outcomes-across-tumor-types-implications-for-immunotherapy
#5
Seung Tae Kim, Samuel J Klempner, Se Hoon Park, Joon Oh Park, Young Suk Park, Ho Yeong Lim, Won Ki Kang, Kyoung-Mee Kim, Jeeyun Lee
The identification of biomarkers associated with response to therapeutic agents is central to optimizing patient outcomes. Expression of the immune checkpoint proteins PD-1/L1, and DNA mismatch repair deficiency (dMMR) status may be predictive response biomarkers for immunotherapies, but their overlap requires further study. We prospectively conducted PD-L1 and MMR immunohistochemistry (IHC) on 430 consecutive patients with advanced gastrointestinal (GI) cancers, genitourinary (GU) cancers or rare cancers between June 2012 and March 2016...
September 29, 2017: Oncotarget
https://www.readbyqxmd.com/read/29094519/the-plan-of-enrichment-designs-for-dealing-with-high-placebo-response
#6
Xiangmin Zhang, Yeh-Fong Chen, Roy Tamura
To deal with high placebo response in clinical trials for psychiatric and other diseases, different enrichment designs, such as the sequential parallel design, two-way enriched design, and sequential enriched design, have been proposed and implemented recently. Depending on the historical trial information and the trial sponsors' resources, detailed design elements are needed for determining which design to adopt. To assist in making more suitable decisions, we perform evaluations for selecting required design elements in terms of power optimization and sample size planning...
November 1, 2017: Pharmaceutical Statistics
https://www.readbyqxmd.com/read/29082033/maintenance-efficacy-designs-in-psychiatry-randomized-discontinuation-trials-enriched-but-not-better
#7
REVIEW
S N Ghaemi, Harry P Selker
INTRODUCTION: Although classical randomized clinical trials (RCTs) are the gold standard for proof of drug efficacy, randomized discontinuation trials (RDTs), sometimes called "enriched" trials, are used increasingly, especially in psychiatric maintenance studies. METHODS: A narrative review of two decades of experience with RDTs. RESULTS: RDTs in psychiatric maintenance trials tend to use a dependent variable as a predictor: treatment response...
June 2017: Journal of clinical and translational science
https://www.readbyqxmd.com/read/29069041/effects-of-fiber-and-probiotics-on-diarrhea-associated-with-enteral-nutrition-in-gastric-cancer-patients-a-prospective-randomized-and-controlled-trial
#8
RANDOMIZED CONTROLLED TRIAL
Rui Zhao, Yong Wang, Yuqian Huang, Yaping Cui, Lin Xia, Zhiyong Rao, Yong Zhou, Xiaoting Wu
BACKGROUND AND OBJECTIVES: Diarrhea is a common complication of enteral nutrition (EN), which affects recovery and prolongs the length of hospital stay (LOHS). To investigate the effect of fiber and probiotics in reducing diarrhea associated with EN in postoperative patients with gastric cancer (GC), the authors designed this prospective randomized-controlled trial. METHODS AND STUDY DESIGN: This study included 120 patients with GC, and the patients were classified into 3 groups via random picking of envelopes: fiber-free nutrition formula (FF group, n = 40), fiber-enriched nutrition formula (FE group, n = 40), and fiber- and probiotic-enriched nutrition formula (FEP group, n = 40)...
October 2017: Medicine (Baltimore)
https://www.readbyqxmd.com/read/29064745/timing-of-the-interim-analysis-in-adaptive-enrichment-designs
#9
Laura Benner, Meinhard Kieser
With increasing interest in personalized medicine over the last years, study designs allowing to demonstrate efficacy in particular subgroups of the overall patient population become more important. Adaptive enrichment designs provide the possibility to both selecting the target population with the most promising treatment benefit and testing for efficacy within a single trial. Here, the target population is selected in a prespecified interim analysis. So far, it has not been very well investigated how timing of the interim analysis should be chosen...
October 24, 2017: Journal of Biopharmaceutical Statistics
https://www.readbyqxmd.com/read/29064567/learning-from-the-past-reflections-on-recently-completed-myasthenia-gravis-trials
#10
Michael Benatar, James F Howard, Richard Barohn, Gil I Wolfe, Gary Cutter
Recently competed clinical trials of therapeutics for myasthenia gravis have varied widely in design, but also perhaps in less explicit ways. We explore ways in which these design characteristics may have influenced recruitment and results, as well as the implications for forthcoming studies. Trial eligibility criteria may inadvertently select for incident versus prevalent cases or patients with relatively mild versus more severe disease. Trial enrichment with patients who have relatively mild disease may limit the sensitivity of the trial to detect a therapeutic effect...
October 24, 2017: Annals of the New York Academy of Sciences
https://www.readbyqxmd.com/read/29053142/resolution-of-inflammation-and-sepsis-survival-are-improved-by-dietary-%C3%AE-3-fatty-acids
#11
Andreas Körner, Martin Schlegel, Julia Theurer, Hannes Frohnmeyer, Michael Adolph, Marieke Heijink, Martin Giera, Peter Rosenberger, Valbona Mirakaj
Critical conditions such as sepsis following infection or traumatic injury disturb the complex state of homeostasis that may lead to uncontrolled inflammation resulting in organ failure, shock and death. They are associated with endogenous mediators that control the onset of acute inflammatory response, but the central problem remains the complete resolution of inflammation. Omega-3 enriched lipid emulsions (Ω-3(+) LEs) were used in experimental studies and clinical trials to establish homeostasis, yet with little understanding about their role on the resolution of inflammation and tissue regeneration...
October 20, 2017: Cell Death and Differentiation
https://www.readbyqxmd.com/read/29039265/hiv-prevention-trial-design-in-an-era-of-effective-pre-exposure-prophylaxis
#12
Amy Cutrell, Deborah Donnell, David T Dunn, David V Glidden, Anneke Grobler, Brett Hanscom, Britt S Stancil, R Daniel Meyer, Ronnie Wang, Robert L Cuffe
Pre-exposure prophylaxis (PrEP) has demonstrated remarkable effectiveness protecting at-risk individuals from HIV-1 infection. Despite this record of effectiveness, concerns persist about the diminished protective effect observed in women compared with men and the influence of adherence and risk behaviors on effectiveness in targeted subpopulations. Furthermore, the high prophylactic efficacy of the first PrEP agent, tenofovir disoproxil fumarate/emtricitabine (TDF/FTC), presents challenges for demonstrating the efficacy of new candidates...
October 17, 2017: HIV Clinical Trials
https://www.readbyqxmd.com/read/29018526/design-of-the-pf-ild-trial-a-double-blind-randomised-placebo-controlled-phase-iii-trial-of-nintedanib-in-patients-with-progressive-fibrosing-interstitial-lung-disease
#13
Kevin R Flaherty, Kevin K Brown, Athol U Wells, Emmanuelle Clerisme-Beaty, Harold R Collard, Vincent Cottin, Anand Devaraj, Yoshikazu Inoue, Florence Le Maulf, Luca Richeldi, Hendrik Schmidt, Simon Walsh, William Mezzanotte, Rozsa Schlenker-Herceg
600 patients aged ≥18 years will be randomised in a 1:1 ratio to nintedanib or placebo. Patients with diagnosis of IPF will be excluded. The study population will be enriched with two-thirds having a usual interstitial pneumonia-like pattern on HRCT. The primary endpoint is the annual rate of decline in forced vital capacity over 52 weeks. The main secondary endpoints are the absolute change from baseline in King's Brief Interstitial Lung Disease Questionnaire total score, time to first acute interstitial lung disease exacerbation or death and time to all-cause mortality over 52 weeks...
2017: BMJ Open Respiratory Research
https://www.readbyqxmd.com/read/29018432/maize-inoculation-with-azospirillum-brasilense-ab-v5-cells-enriched-with-exopolysaccharides-and-polyhydroxybutyrate-results-in-high-productivity-under-low-n-fertilizer-input
#14
André L M Oliveira, Odair J A P Santos, Paulo R F Marcelino, Karina M L Milani, Mónica Y A Zuluaga, Claudemir Zucareli, Leandro S A Gonçalves
Although Azospirillum strains used in commercial inoculant formulations presents diazotrophic activity, it has been reported that their ability to produce phytohormones plays a pivotal role in plant growth-promotion, leading to a general recommendation of its use in association with regular N-fertilizer doses. In addition, a high variability in the effectiveness of Azospirillum inoculants is still reported under field conditions, contributing to the adoption of the inoculation technology as an additional management practice rather than its use as an alternative practice to the use of chemical inputs in agriculture...
2017: Frontiers in Microbiology
https://www.readbyqxmd.com/read/28991832/adaptation-of-a-biomarker-based-sepsis-mortality-risk-stratification-tool-for-pediatric-acute-respiratory-distress-syndrome
#15
Nadir Yehya, Hector R Wong
OBJECTIVES: The original Pediatric Sepsis Biomarker Risk Model and revised (Pediatric Sepsis Biomarker Risk Model-II) biomarker-based risk prediction models have demonstrated utility for estimating baseline 28-day mortality risk in pediatric sepsis. Given the paucity of prediction tools in pediatric acute respiratory distress syndrome, and given the overlapping pathophysiology between sepsis and acute respiratory distress syndrome, we tested the utility of Pediatric Sepsis Biomarker Risk Model and Pediatric Sepsis Biomarker Risk Model-II for mortality prediction in a cohort of pediatric acute respiratory distress syndrome, with an a priori plan to revise the model if these existing models performed poorly...
October 6, 2017: Critical Care Medicine
https://www.readbyqxmd.com/read/28987076/potential-facilitators-and-barriers-to-awareness-of-n-of-1-trials-for-physicians-in-traditional-chinese-medicine
#16
Jiang Li, Dang Wei, Junqiang Niu, Min Yang, Long Ge, Xiaoqin Wang, Wenzhen Yuan, Jinhui Tian, Hongcai Shang, Jingbo Zhai, Kehu Yang, Min Dai
Context • N-of-1 trials are multiple crossover trials with randomized and blinded methods, conducted with a single participant to evaluate the effectiveness and safety of a therapy. They can be a helpful tool for enriching clinical research for traditional Chinese medicine (TCM), but the approach has gained little traction in TCM. Information is needed before supporters of the trials can pursue a change to that status. Objective • The study intended to obtain data needed to support TCM clinicians' adoption of N-of-1 trials...
October 2, 2017: Alternative Therapies in Health and Medicine
https://www.readbyqxmd.com/read/28974002/current-technical-approaches-for-the-early-detection-of-foodborne-pathogens-challenges-and-opportunities
#17
REVIEW
Il-Hoon Cho, Seockmo Ku
The development of novel and high-tech solutions for rapid, accurate, and non-laborious microbial detection methods is imperative to improve the global food supply. Such solutions have begun to address the need for microbial detection that is faster and more sensitive than existing methodologies (e.g., classic culture enrichment methods). Multiple reviews report the technical functions and structures of conventional microbial detection tools. These tools, used to detect pathogens in food and food homogenates, were designed via qualitative analysis methods...
September 30, 2017: International Journal of Molecular Sciences
https://www.readbyqxmd.com/read/28960913/willingness-to-pay-for-small-quantity-lipid-based-nutrient-supplements-for-women-and-children-evidence-from-ghana-and-malawi
#18
Katherine P Adams, Stephen A Vosti, Emmanuel Ayifah, Thokozani E Phiri, Seth Adu-Afarwuah, Kenneth Maleta, Ulla Ashorn, Mary Arimond, Kathryn G Dewey
Small-quantity lipid-based nutrient supplements (SQ-LNS) are designed to enrich maternal and child diets with the objective of preventing undernutrition during the first 1,000 days. Scaling up the delivery of supplements such as SQ-LNS hinges on understanding private demand and creatively leveraging policy-relevant factors that might influence demand. We used longitudinal stated willingness-to-pay (WTP) data from contingent valuation studies that were integrated into randomized controlled nutrition trials in Ghana and Malawi to estimate private valuation of SQ-LNS during pregnancy, postpartum, and early childhood...
September 28, 2017: Maternal & Child Nutrition
https://www.readbyqxmd.com/read/28948633/treatment-evaluation-for-a-data-driven-subgroup-in-adaptive-enrichment-designs-of-clinical-trials
#19
Zhiwei Zhang, Ruizhe Chen, Guoxing Soon, Hui Zhang
Adaptive enrichment designs (AEDs) of clinical trials allow investigators to restrict enrollment to a promising subgroup based on an interim analysis. Most of the existing AEDs deal with a small number of predefined subgroups, which are often unknown at the design stage. The newly developed Simon design offers a great deal of flexibility in subgroup selection (without requiring pre-defined subgroups) but does not provide a procedure for estimating and testing treatment efficacy for the selected subgroup. This article proposes a 2-stage AED which does not require predefined subgroups but requires a prespecified algorithm for choosing a subgroup on the basis of baseline covariate information...
September 26, 2017: Statistics in Medicine
https://www.readbyqxmd.com/read/28947466/randomised-controlled-trial-of-lactobacillus-rhamnosus-lgg-versus-placebo-in-children-presenting-to-the-emergency-department-with-acute-gastroenteritis-the-pecarn-probiotic-study-protocol
#20
David Schnadower, Phillip I Tarr, Casper T Charles, Marc H Gorelick, Michael J Dean, Karen J O'Connell, Prashant Mahajan, Thomas H Chun, Seema R Bhatt, Cindy G Roskind, Elizabeth C Powell, Alexander J Rogers, Cheryl Vance, Robert E Sapien, Feng Gao, Stephen B Freedman
INTRODUCTION: Acute gastroenteritis (AGE) is a common and burdensome condition that affects millions of children worldwide each year. Currently available strategies are limited to symptomatic management, treatment and prevention of dehydration and infection control; no disease-modifying interventions exist. Probiotics, defined as live microorganisms beneficial to the host, have shown promise in improving AGE outcomes, but existing studies have sufficient limitations such that the use of probiotics cannot currently be recommended with confidence...
September 24, 2017: BMJ Open
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