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Enriched trial design

W Kievit, F A C Berden, J P H Drenth
Pharmaceutical companies are under increasing scrutiny because of their strategy for gaining market access and reimbursement authorisation for novel drugs. The tool most often used is that of a randomised controlled trial (RCT) in a highly selected population that has a high chance of responding on the treatment but a low chance of developing side effects. This population differs to a large extent from real-life patients, who have diverging characteristics that can influence effectiveness and safety; these include co-morbidity, age and disease severity...
2016: Nederlands Tijdschrift Voor Geneeskunde
Sheena Derry, Cathy Stannard, Peter Cole, Philip J Wiffen, Roger Knaggs, Dominic Aldington, R Andrew Moore
BACKGROUND: Opioid drugs, including fentanyl, are commonly used to treat neuropathic pain, and are considered effective by some professionals. Most reviews have examined all opioids together. This review sought evidence specifically for fentanyl, at any dose, and by any route of administration. Other opioids are considered in separate reviews. OBJECTIVES: To assess the analgesic efficacy of fentanyl for chronic neuropathic pain in adults, and the adverse events associated with its use in clinical trials...
October 11, 2016: Cochrane Database of Systematic Reviews
Sheena Derry, Malene Cording, Philip J Wiffen, Simon Law, Tudor Phillips, R Andrew Moore
BACKGROUND: This review updates part of an earlier Cochrane review on 'Pregabalin for acute and chronic pain in adults' (Moore 2009), and considers only fibromyalgia pain.Antiepileptic drugs have been used in pain management since the 1960s. Pregabalin is an antiepileptic drug also used in management of chronic pain conditions, including fibromyalgia. Pain response with pregabalin is associated with major benefits for other symptoms, and improved quality of life and function in people with chronic painful conditions...
September 29, 2016: Cochrane Database of Systematic Reviews
Eliza G Miller, Caryl A Nowson, David W Dunstan, Deborah A Kerr, Vicky Solah, David Menzies, Robin M Daly
BACKGROUND: Recruitment of participants into long-term community-based lifestyle intervention trials, particularly adults with a chronic disease, is often slow and challenging. Currently there is limited data on successful recruitment strategies suitable for older adults with type 2 diabetes into community-based exercise and nutrition programs, and no information on cost estimates associated with such recruitment. The aim of this report is to describe the recruitment strategies used and the success of each approach in recruiting older adults with type 2 diabetes into a 6-month community-based exercise and nutritional supplementation randomised controlled trial (RCT)...
September 26, 2016: Trials
Kim-Tiu Teng, Lin Faun Chang, Shireene Ratna Vethakkan, Kalanithi Nesaretnam, Tom A B Sanders
BACKGROUND & AIMS: Modification of the amount and type of dietary fat has diverse effects on cardiovascular risk. METHODS: We recruited 54 abdominally obese subjects to participate in a prospective cross-over design, single-blind trial comparing isocaloric 2000 kcal MUFA or carbohydrate-enriched diet with SFA-enriched diet (control). The control diet consisted of 15E% protein, 53E% carbohydrate and 32E% fat (12E% SFA, 13E% MUFA). A total of ∼7E% of MUFA or refined carbohydrate was exchanged with SFA in the MUFA-rich and carbohydrate-rich diets respectively for 6-weeks...
September 8, 2016: Clinical Nutrition: Official Journal of the European Society of Parenteral and Enteral Nutrition
Yong Zang, Ying Yuan
In the early phase development of molecularly targeted agents (MTAs), a commonly encountered situation is that the MTA is expected to be more effective for a certain biomarker subgroup, say marker-positive patients, but there is no adequate evidence to show that the MTA does not work for the other subgroup, that is, marker-negative patients. After establishing that marker-positive patients benefit from the treatment, it is often of great clinical interest to determine whether the treatment benefit extends to marker-negative patients...
September 19, 2016: Statistics in Medicine
Akkarach Bumrungpert, Patcharanee Pavadhgul, Ruchaneekorn W Kalpravidh
Camellia oil is commonly used as an adjuvant in medicine. It is rich in monounsaturated fatty acids, vitamin E, and phytochemicals. The objective of this study was to examine effects of camellia oil consumption on oxidative stress, low-density lipoprotein-cholesterol (LDL-C) oxidation, and inflammatory markers in hypercholesterolemic subjects. The study design was a randomized, single-blind controlled trial. Women with hypercholesterolemia (n = 50) were randomly divided into two groups. The treatment group (n = 25) was provided camellia oil-enriched diets and the control group (n = 25) was provided diets cooked with soybean oil three meals (45 mL oil) a day for 8 weeks...
September 2016: Journal of Medicinal Food
Nikola Sprigg, Rebecca O'Connor, Lisa Woodhouse, Kailash Krishnan, Timothy J England, Louise A Connell, Marion F Walker, Philip M Bath
BACKGROUND: Granulocyte-colony stimulating factor (G-CSF) mobilises endogenous haematopoietic stem cells and enhances recovery in experimental stroke. Recovery may also be dependent on an enriched environment and physical activity. G-CSF may have the potential to enhance recovery when used in combination with physiotherapy, in patients with disability late after stroke. METHODS: A pilot 2 x 2 factorial randomised (1:1) placebo-controlled trial of G-CSF (double-blind), and/or a 6 week course of physiotherapy, in 60 participants with disability (mRS >1), at least 3 months after stroke...
2016: PloS One
Ying Liu, Yuanjia Wang, Donglin Zeng
Sequential multiple assignment randomization trial (SMART) is a powerful design to study Dynamic Treatment Regimes (DTRs) and allows causal comparisons of DTRs. To handle practical challenges of SMART, we propose a SMART with Enrichment (SMARTER) design, which performs stage-wise enrichment for SMART. SMARTER can improve design efficiency, shorten the recruitment period, and partially reduce trial duration to make SMART more practical with limited time and resource. Specifically, at each subsequent stage of a SMART, we enrich the study sample with new patients who have received previous stages' treatments in a naturalistic fashion without randomization, and only randomize them among the current stage treatment options...
September 6, 2016: Biometrics
Dimitrios Draganidis, Leonidas G Karagounis, Ioannis Athanailidis, Athanasios Chatzinikolaou, Athanasios Z Jamurtas, Ioannis G Fatouros
Inflammaging is the chronic low-grade inflammatory state present in the elderly, characterized by increased systemic concentrations of proinflammatory cytokines. It has been shown that inflammaging increases the risk of pathologic conditions and age-related diseases, and that it also has been associated with increased skeletal muscle wasting, strength loss, and functional impairments. Experimental evidence suggests that the increased concentrations of proinflammatory cytokines and primary tumor necrosis factor α observed in chronic inflammation lead to protein degradation through proteasome activation and reduced skeletal muscle protein synthesis (MPS) via protein kinase B/Akt downregulation...
October 2016: Journal of Nutrition
Marisa Dolled-Filhart, Charlotte Roach, Grant Toland, Dave Stanforth, Malinka Jansson, Gregory M Lubiniecki, Gary Ponto, Kenneth Emancipator
Context .- Programmed death ligand-1 (PD-L1) expression by tumors may enable them to avoid immunosurveillance. Objective .- To develop a PD-L1 immunohistochemical assay using the 22C3 anti-PD-L1 murine monoclonal antibody on the Dako platform as a possible companion diagnostic for pembrolizumab in patients with non-small cell lung cancer. Design .- Tumor samples from 146 patients with non-small cell lung cancer treated with pembrolizumab in KEYNOTE-001 and for whom response data were available were scored according to their staining intensity by a single pathologist using 4 methods: percentage of tumor cells staining at any intensity (PS1), moderate/strong intensity (PS2), strong intensity (PS3), and H-score (PS1 + PS2 + PS3)...
August 23, 2016: Archives of Pathology & Laboratory Medicine
Dean M Thompson, Deborah A Hall, Dawn-Marie Walker, Derek J Hoare
BACKGROUND: Tinnitus is associated with depression and anxiety disorders, severely and adversely affecting the quality of life and functional health status for some people. With the dearth of clinical psychologists embedded in audiology services and the cessation of training for hearing therapists in the UK, it is left to audiologists to meet the psychological needs of many patients with tinnitus. However, there is no universally standardized training or manualized intervention specifically for audiologists across the whole UK public healthcare system and similar systems elsewhere across the world...
August 18, 2016: Ear and Hearing
Fugui Yin, Hai Yu, Dion Lepp, Xuejiang Shi, Xiaojian Yang, Jielun Hu, Steve Leeson, Chengbo Yang, Shaoping Nie, Yongqing Hou, Joshua Gong
BACKGROUND & AIMS: Butyrate has been shown to potently regulate energy expenditure and lipid metabolism in animals, yet the underlying mechanisms remain to be fully understood. The aim of this study was to investigate the molecular mechanisms of butyrate (in the form of butyrate glycerides, BG)-induced lipid metabolism at the level of gene expression in the jejunum and liver of broilers. METHODOLOGY/PRINCIPAL FINDINGS: Two animal experiments were included in this study...
2016: PloS One
María V Irazabal, Kaleab Z Abebe, Kyongtae Ty Bae, Ronald D Perrone, Arlene B Chapman, Robert W Schrier, Alan S Yu, William E Braun, Theodore I Steinman, Peter C Harris, Michael F Flessner, Vicente E Torres
BACKGROUND: Patients with mild autosomal dominant polycystic kidney disease (ADPKD) are less likely to be informative in randomized clinical trials (RCTs). We previously developed an imaging classification of ADPKD (typical diffuse cyst distribution Class 1A-E and atypical cyst distribution Class 2) for prognostic enrichment design in RCTs. We investigated whether using this classification would have increased the power to detect a beneficial treatment effect of rigorous blood pressure (BP) control on HALT-PKD participants with early disease (Study A)...
August 2, 2016: Nephrology, Dialysis, Transplantation
D García-Villamisar, J Dattilo, C Muela
BACKGROUND: The purpose of this research was to examine effects of a therapeutic recreation (TR) program designed to increase executive function (EF), social skills, adaptive behaviours and well-being of adults with autism spectrum disorder (ASD) and intellectual disability (ID). METHOD: A preliminary pre-test, post-test randomized control group experimental design was used to measure effects of a 40-week TR program designed to increase EF (TR-EF). The TR-EF used instructional electronically based games delivered during 200 1-h sessions (5/week)...
July 28, 2016: Journal of Intellectual Disability Research: JIDR
Konstantina Poulianiti, Christina Karatzaferi, Andreas D Flouris, Ioannis G Fatouros, Yiannis Koutedakis, Athanasios Z Jamurtas
CONTEXT: It has been indicated that acute active and passive tobacco cigarette smoking may cause changes on redox status balance that may result in significant pathologies. However, no study has evaluated the effects of active and passive e-cigarette smoking on redox status of consumers. OBJECTIVE: To examine the acute effects of active and passive e-cigarette and tobacco cigarette smoking on selected redox status markers. METHODS: Using a randomized single-blind crossover design, 30 participants (15 smokers and 15 nonsmokers) were exposed to three different experimental conditions...
July 2016: Toxicology Mechanisms and Methods
Josephine Ho, Raylene A Reimer, Manpreet Doulla, Carol Huang
BACKGROUND: The gut microbiome is increasingly recognized as a contributor to disease states. Patients with type 1 diabetes (DM1) have distinct gut microbiota in comparison to non-diabetic individuals, and it has been linked to changes in intestinal permeability, inflammation and insulin resistance. Prebiotics are non-digestible carbohydrates that alter gut microbiota and could potentially improve glycemic control in children with DM1. This pilot study aims to determine the feasibility of a 12-week dietary intervention with prebiotics in children with DM1...
2016: Trials
J E Grilley-Olson, P L Bedard, A Fasolo, M Cornfeld, L Cartee, A R Abdul Razak, L-A Stayner, Y Wu, R Greenwood, R Singh, C B Lee, J Bendell, H A Burris, G Del Conte, C Sessa, J R Infante
Introduction This Phase Ib trial investigated the safety, tolerability, and recommended phase 2 dose for the pan-PI3K/mTOR inhibitor, GSK2126458 (GSK458), and trametinib combination when administered to patients with advanced solid tumors. Patients and Methods Patients with advanced solid tumors received escalating doses of GSK458 (once or twice daily, and continuous or intermittent) and trametinib following a zone-based 3 + 3 design to determine the maximum tolerated dose (MTD). Assessments included monitoring for adverse events and response, and evaluating pharmacokinetic (PK) measures...
July 23, 2016: Investigational New Drugs
Tony Shu Kam Mok, Sarayut Lucien Geater, Wu-Chou Su, Eng-Huat Tan, James Chi-Hsin Yang, Gee-Chen Chang, May Han, Philip Komarnitsky, Francis Payumo, Jennifer E Garrus, Sandra Close, Keunchil Park
INTRODUCTION: A randomized phase 2 study was designed to compare the combination of ficlatuzumab (AV-299), a humanized hepatocyte growth factor-neutralizing monoclonal antibody, plus gefitinib versus gefitinib monotherapy in a pulmonary adenocarcinoma population clinically enriched for EFGR tyrosine kinase inhibitor-sensitizing mutations. METHODS: A total of 188 patients were randomized 1:1 to receive either gefitinib or ficlatuzumab plus gefitinib treatment. Patients who demonstrated disease control in the single-agent gefitinib arm were allowed to cross over to ficlatuzumab plus gefitinib treatment upon disease progression...
October 2016: Journal of Thoracic Oncology
Janne Grønli, Ida Kristiansen Byrkjedal, Bjørn Bjorvatn, Øystein Nødtvedt, Børge Hamre, Ståle Pallesen
OBJECTIVE: To objectively and subjectively compare whether reading a story for 30 min from an iPad or from a book in bed prior to sleep will differentially affect sleep. METHODS: Sixteen students (12 females, mean age 25.1 ± 2.9 years) underwent ambulatory (sleeping in their own beds at home) polysomnographic (PSG) recordings in a counterbalanced crossover design consisting of three PSG nights (one adaptation night, two test nights) and two different reading materials: read from an iPad or from a book...
May 2016: Sleep Medicine
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