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Adaptive trial design

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https://www.readbyqxmd.com/read/28453376/control-of-the-unfolded-protein-response-in-health-and-disease
#1
Dimitrios Doultsinos, Tony Avril, Stéphanie Lhomond, Nicolas Dejeans, Philippe Guédat, Eric Chevet
The unfolded protein response (UPR) is an integrated, adaptive biochemical process that is inextricably linked with cell homeostasis and paramount to maintenance of normal physiological function. Prolonged accumulation of improperly folded proteins in the endoplasmic reticulum (ER) leads to stress. This is the driving stimulus behind the UPR. As such, prolonged ER stress can push the UPR past beneficial functions such as reduced protein production and increased folding and clearance to apoptotic signaling. The UPR is thus contributory to the commencement, maintenance, and exacerbation of a multitude of disease states, making it an attractive global target to tackle conditions sorely in need of novel therapeutic intervention...
April 1, 2017: SLAS Discovery
https://www.readbyqxmd.com/read/28448684/statistical-modeling-for-bayesian-extrapolation-of-adult-clinical-trial-information-in-pediatric-drug-evaluation
#2
Margaret Gamalo-Siebers, Jasmina Savic, Cynthia Basu, Xin Zhao, Mathangi Gopalakrishnan, Aijun Gao, Guochen Song, Simin Baygani, Laura Thompson, H Amy Xia, Karen Price, Ram Tiwari, Bradley P Carlin
Children represent a large underserved population of "therapeutic orphans," as an estimated 80% of children are treated off-label. However, pediatric drug development often faces substantial challenges, including economic, logistical, technical, and ethical barriers, among others. Among many efforts trying to remove these barriers, increased recent attention has been paid to extrapolation; that is, the leveraging of available data from adults or older age groups to draw conclusions for the pediatric population...
April 27, 2017: Pharmaceutical Statistics
https://www.readbyqxmd.com/read/28447664/efficient-design-of-clinical-trials-and-epidemiological-research-is-it-possible
#3
REVIEW
Michael S Lauer, David Gordon, Gina Wei, Gail Pearson
Randomized clinical trials and large-scale, cohort studies continue to have a critical role in generating evidence in cardiovascular medicine; however, the increasing concern is that ballooning costs threaten the clinical trial enterprise. In this Perspectives article, we discuss the changing landscape of clinical research, and clinical trials in particular, focusing on reasons for the increasing costs and inefficiencies. These reasons include excessively complex design, overly restrictive inclusion and exclusion criteria, burdensome regulations, excessive source-data verification, and concerns about the effect of clinical research conduct on workflow...
April 27, 2017: Nature Reviews. Cardiology
https://www.readbyqxmd.com/read/28446232/continuous-quality-improvement-interventions-to-improve-long-term-outcomes-of-antiretroviral-therapy-in-women-who-initiated-therapy-during-pregnancy-or-breastfeeding-in-the-democratic-republic-of-congo-design-of-an-open-label-parallel-group-randomized-trial
#4
Marcel Yotebieng, Frieda Behets, Bienvenu Kawende, Noro Lantoniaina Rosa Ravelomanana, Martine Tabala, Emile W Okitolonda
BACKGROUND: Despite the rapid adoption of the World Health Organization's 2013 guidelines, children continue to be infected with HIV perinatally because of sub-optimal adherence to the continuum of HIV care in maternal and child health (MCH) clinics. To achieve the UNAIDS goal of eliminating mother-to-child HIV transmission, multiple, adaptive interventions need to be implemented to improve adherence to the HIV continuum. METHODS: The aim of this open label, parallel, group randomized trial is to evaluate the effectiveness of Continuous Quality Improvement (CQI) interventions implemented at facility and health district levels to improve retention in care and virological suppression through 24 months postpartum among pregnant and breastfeeding women receiving ART in MCH clinics in Kinshasa, Democratic Republic of Congo...
April 26, 2017: BMC Health Services Research
https://www.readbyqxmd.com/read/28440861/findings-from-the-families-on-track-intervention-pilot-trial-for-children-with-fetal-alcohol-spectrum-disorders-and-their-families
#5
Christie L M Petrenko, Mary E Pandolfino, Luther K Robinson
BACKGROUND: Individuals with fetal alcohol spectrum disorders (FASD) are at high risk for costly, debilitating mental health problems and secondary conditions, such as school disruption, trouble with the law, and substance use. The study objective was to pilot a multi-component intervention designed to prevent secondary conditions in children with FASD and improve family adaptation. METHODS: Thirty children with FASD or prenatal alcohol exposure (PAE) (ages 4 to 8) and their primary caregivers were enrolled...
April 25, 2017: Alcoholism, Clinical and Experimental Research
https://www.readbyqxmd.com/read/28440024/immediate-and-delayed-loading-of-two-piece-reduced-diameter-implants-with-locator-analog-attachments-in-edentulous-mandibles-one-year-results-from-a-randomized-clinical-trial-examining-clinical-outcome-and-patient-expectation
#6
Nikolaos Nikitas Giannakopoulos, Kirsten Ariaans, Lydia Eberhard, Anna-Luisa Klotz, Keunyoung Oh, Stefanie Kappel
BACKGROUND: Prosthetic management of thin alveolar ridges in the edentulous mandibles of elderly patients, especially the time of loading, the number of implants needed, and patient expectations and perception, is a challenge in implant dentistry. PURPOSE: Survival of conventionally and immediately loaded 2-piece reduced-diameter implants in the interforaminal region of the edentulous mandible supporting locator-analog attachments was evaluated. Prosthetic complications and peri-implant hygiene were also studied, and patient expectation and subjective evaluation of the treatment were documented...
April 24, 2017: Clinical Implant Dentistry and related Research
https://www.readbyqxmd.com/read/28438707/contributions-of-the-parietal-cortex-to-increased-efficiency-of-planning-based-action-selection
#7
Jennifer Randerath, Kenneth Valyear, Benjamin A Philip, Scott H Frey
Response selection is foundational to adaptive behavior, and considerable attention has been devoted to investigating this behavior under conditions in which the mapping between stimuli and responses is fixed. Results from prior studies implicate the left supramarginal gyrus (SMg), premotor and prefrontal cortices, as well as the cerebellum in this essential function. Yet, many goal-directed motor behaviors have multiple solutions with flexible mappings between stimuli and responses whose solutions are believed to involve prospective planning...
April 21, 2017: Neuropsychologia
https://www.readbyqxmd.com/read/28435499/the-fast-and-the-furious-low-risk-chest-pain-and-the-rapid-rule-out-protocol
#8
REVIEW
Maite Anna Huis In 't Veld, Louise Cullen, Simon A Mahler, Barbra E Backus, Zachary D W Dezman, Amal Mattu
Accelerated diagnostic pathways (ADP) have been designed to identify low-risk chest pain patients in the emergency department. This review article discusses the Asia-Pacific Evaluation of Chest Pain Trial (ASPECT) score, the Accelerated Diagnostic Protocol for Chest Pain Trial (ADAPT) score, the Emergency Department Assessment of Chest Pain Score (EDACS), the HEARTScore and the HEART pathway. These ADPs have been validated in various studies and aid the emergency provider with identifying the low-risk chest pain patient who is appropriate for discharge home, while at the same time highlighting those patients who would benefit from further in-patient work up...
April 2017: Western Journal of Emergency Medicine
https://www.readbyqxmd.com/read/28434375/the-role-of-adaptive-trial-designs-in-drug-development
#9
François Curtin, Stephane Heritier
Clinical development of new drugs is a long and costly process. There is a need to find solutions which can improve and shorten this process. By introducing flexibility in to the design of clinical trials, adaptive design contributes to this improvement and allows to reach drug development decisions in a quicker way. Areas covered: We review the main methodological approaches to adaptive trial design, introducing key statistical concepts. For each phase of the clinical development, different uses and implementations of adaptive trial (AD) design are presented and examples of recent clinical trials are given...
April 24, 2017: Expert Review of Clinical Pharmacology
https://www.readbyqxmd.com/read/28430697/abusive-head-trauma-and-mortality-an-analysis-from-an-international-comparative-effectiveness-study-of-children-with-severe-traumatic-brain-injury
#10
Nikki Miller Ferguson, Ajit Sarnaik, Darryl Miles, Nadeem Shafi, Mark J Peters, Edward Truemper, Monica S Vavilala, Michael J Bell, Stephen R Wisniewski, James F Luther, Adam L Hartman, Patrick M Kochanek
OBJECTIVES: Small series have suggested that outcomes after abusive head trauma are less favorable than after other injury mechanisms. We sought to determine the impact of abusive head trauma on mortality and identify factors that differentiate children with abusive head trauma from those with traumatic brain injury from other mechanisms. DESIGN: First 200 subjects from the Approaches and Decisions in Acute Pediatric Traumatic Brain Injury Trial-a comparative effectiveness study using an observational, cohort study design...
April 20, 2017: Critical Care Medicine
https://www.readbyqxmd.com/read/28430547/enhancing-insights-into-pulmonary-vascular-disease-pvd-through-a-precision-medicine-approach-a-joint-nhlbi-cmref-workshop-report
#11
John H Newman, Stuart Rich, Steven H Abman, John H Alexander, John Barnard, Gerald J Beck, Raymond L Benza, Todd M Bull, Stephen Y Chan, Hyung J Chun, Declan Doogan, Jocelyn Dupuis, Serpil C Erzurum, Robert P Frantz, Mark Geraci, Hunter Gillies, Mark Gladwin, Michael P Gray, Anna R Hemnes, Roy S Herbst, Adrian F Hernandez, Nicholas S Hill, Evelyn M Horn, Kendall Hunter, Zhi-Cheng Jing, Roger Johns, Sanjay Kaul, Steven M Kawut, Tim Lahm, Jane A Leopold, Greg D Lewis, Stephen C Mathai, Vallerie V McLaughlin, Evangelos D Michelakis, Steven D Nathan, William Nichols, Grier Page, Marlene Rabinovitch, Jonathan Rich, Franz Rischard, Sharon Rounds, Sanjiv J Shah, Victor F Tapson, Naomi Lowy, Norman Stockbridge, Gail Weinmann, Lei Xiao
The Division of Lung Diseases of the National Heart, Lung and Blood Institute, (NHLBI) and the Cardiovascular Medical Education and Research Fund (CMREF), held a workshop to discuss to leverage of the anticipated scientific output from the recently launched "Redefining Pulmonary Hypertension (PH) through Pulmonary Vascular Disease Phenomics" (PVDOMICS) program. PVDOMICS is a protocol driven network to analyze PH patient populations to define novel pulmonary vascular disease (PVD) phenotypes. Basic, translational and clinical investigators, patient advocacy organizations, regulatory agencies, and pharmaceutical industry experts discussed the application of precision medicine to PVD clinical trials...
April 21, 2017: American Journal of Respiratory and Critical Care Medicine
https://www.readbyqxmd.com/read/28429241/pharmacological-approaches-to-the-management-of-secondary-progressive-multiple-sclerosis
#12
REVIEW
A Nandoskar, J Raffel, A S Scalfari, T Friede, R S Nicholas
It is well recognised that the majority of the impact of multiple sclerosis (MS), both personal and societal, arises in the progressive phase where disability accumulates inexorably. As such, progressive MS (PMS) has been the target of pharmacological therapies for many years. However, there are no current licensed treatments for PMS. This stands in marked contrast to relapsing remitting MS (RRMS) where trials have resulted in numerous licensed therapies. PMS has proven to be a more difficult challenge compared to RRMS and this review focuses on secondary progressive MS (SPMS), where relapses occur before the onset of gradual, irreversible disability, and not primary progressive MS where disability accumulation occurs without prior relapses...
April 20, 2017: Drugs
https://www.readbyqxmd.com/read/28424281/distinct-roles-of-vaccinia-virus-nf-kb-inhibitor-proteins-a52-b15-and-k7-in-the-immune-response
#13
Mauro Di Pilato, Ernesto Mejías-Pérez, Carlos Oscar S Sorzano, Mariano Esteban
Poxviruses use a complex strategy to escape immune control, by expressing immunomodulatory proteins that could limit their use as vaccine vectors. To test the role of poxvirus NF-κB pathway inhibitors A52, B15 and K7 in immunity, we deleted their genes in a NYVAC vaccinia virus strain that expresses HIV-1 clade C antigens. After infection of mice, ablation of A52R, B15R and K7R increased dendritic cell, natural killer cell and neutrophil migration as well as chemokine/cytokine expression. Revertant viruses for these genes confirmed their role in inhibiting the innate immune system...
April 19, 2017: Journal of Virology
https://www.readbyqxmd.com/read/28423744/design-and-validation-of-a-platform-to-evaluate-mhealth-apps
#14
Daniel Philpott, Aziz Guergachi, Karim Keshavjee
Emerging technologies show great potential in the field of patient care. One such technology is mobile heath applications (mhealth apps), which have exploded in number and variety in recent years, and offer great promise in the ability to collect and monitor patient health data. Despite their apparent success in proliferation and user adoption, these applications struggle to integrate into the primary care system and there is scant information regarding their efficacy to effect patient behavior and consequently health outcomes...
2017: Studies in Health Technology and Informatics
https://www.readbyqxmd.com/read/28412989/evaluation-of-methodological-aspects-of-digestibility-measurements-in-ponies-fed-different-haylage-to-concentrate-ratios
#15
F J W C Schaafstra, D A van Doorn, J T Schonewille, M M J van Riet, P Visser, M C Blok, W H Hendriks
Methodological aspects of digestibility measurements were studied in four Welsh pony geldings consuming haylage-based diets with increasing proportions of a pelleted concentrate according to a 4×4 Latin square design experiment. Ponies were fed four experimental, iso-energetic (net energy (NE) basis) diets (i.e. 22 MJ NE/day) with increasing proportions of a pelleted concentrate (C) in relation to haylage (H). The absolute amounts of diet dry matter fed per day were 4.48 kg of H (100H), 3.36 and 0.73 kg of H and C (75H25C), 2...
April 17, 2017: Animal: An International Journal of Animal Bioscience
https://www.readbyqxmd.com/read/28411508/interventions-to-support-people-with-dementia-and-their-caregivers-during-the-transition-from-home-care-to-nursing-home-care-a-systematic-review
#16
REVIEW
Christian Müller, Sindy Lautenschläger, Gabriele Meyer, Astrid Stephan
BACKGROUND: During the transition of people with dementia from home to nursing home family caregivers often feel burdened. OBJECTIVES: We aimed to 1) identify interventions which support people with dementia and their caregivers in the transition from home care to nursing home care, 2) synthesize the evidence for efficacy of these interventions, and 3) examine whether the identified interventions have been systematically developed, evaluated and implemented according to the Medical Research Council guidance on complex interventions...
April 3, 2017: International Journal of Nursing Studies
https://www.readbyqxmd.com/read/28408335/optimizing-delivery-of-a-behavioral-pain-intervention-in-cancer-patients-using-a-sequential-multiple-assignment-randomized-trial-smart
#17
Sarah A Kelleher, Caroline S Dorfman, Jen C Plumb Vilardaga, Catherine Majestic, Joseph Winger, Vicky Gandhi, Christine Nunez, Alyssa Van Denburg, Rebecca A Shelby, Shelby D Reed, Susan Murphy, Marie Davidian, Eric B Laber, Gretchen G Kimmick, Kelly W Westbrook, Amy P Abernethy, Tamara J Somers
BACKGROUND/AIMS: Pain is common in cancer patients and results in lower quality of life, depression, poor physical functioning, financial difficulty, and decreased survival time. Behavioral pain interventions are effective and nonpharmacologic. Traditional randomized controlled trials (RCT) test interventions of fixed time and dose, which poorly represent successive treatment decisions in clinical practice. We utilize a novel approach to conduct a RCT, the sequential multiple assignment randomized trial (SMART) design, to provide comparative evidence of: 1) response to differing initial doses of a pain coping skills training (PCST) intervention and 2) intervention dose sequences adjusted based on patient response...
April 11, 2017: Contemporary Clinical Trials
https://www.readbyqxmd.com/read/28405377/modulation-of-leg-joint-function-to-produce-emulated-acceleration-during-walking-and-running-in-humans
#18
Dominic James Farris, Brent J Raiteri
Understanding how humans adapt gait mechanics for a wide variety of locomotor tasks is important for inspiring the design of robotic, prosthetic and wearable assistive devices. We aimed to elicit the mechanical adjustments made to leg joint functions that are required to generate accelerative walking and running, using metrics with direct relevance to device design. Twelve healthy male participants completed constant speed (CS) walking and running and emulated acceleration (ACC) trials on an instrumented treadmill...
March 2017: Royal Society Open Science
https://www.readbyqxmd.com/read/28399988/youth-social-behaviour-and-network-therapy-y-sbnt-adaptation-of-a-family-and-social-network-intervention-for-young-people-who-misuse-alcohol-and-drugs-a-randomised-controlled-feasibility-trial
#19
Judith Watson, Paul Toner, Ed Day, Donna Back, Louca-Mai Brady, Caroline Fairhurst, Charlotte Renwick, Lorna Templeton, Shabana Akhtar, Charlie Lloyd, Jinshuo Li, Kim Cocks, Sangeeta Ambegaokar, Steve Parrott, Paul McArdle, Eilish Gilvarry, Alex Copello
BACKGROUND: Family interventions appear to be effective at treating young people's substance misuse. However, implementation of family approaches in UK services is low. This study aimed to demonstrate the feasibility of recruiting young people to an intervention based on an adaptation of adult social behaviour and network therapy. It also sought to involve young people with experience of using substance misuse services in the research process. OBJECTIVES: To demonstrate the feasibility of recruiting young people to family and social network therapy and to explore ways in which young people with experience of using substance misuse services could be involved in a study of this nature...
March 2017: Health Technology Assessment: HTA
https://www.readbyqxmd.com/read/28397083/cephalometric-and-dental-arch-changes-to-haas-type-rapid-maxillary-expander-anchored-to-deciduous-vs-permanent-molars-a-multicenter-randomized-controlled-trial
#20
Carmen Cerruto, Alessandro Ugolini, Luca Di Vece, Tiziana Doldo, Alberto Caprioglio, Armando Silvestrini-Biavati
OBJECTIVE: To assess radiographic changes and dental arch changes with Haas-type rapid maxillary expansion (H-RME) anchored to deciduous versus permanent molars in children with unilateral posterior crossbite. METHODS: In all, 70 patients with unilateral posterior crossbite were randomly allocated to group GrE (H-RME on second deciduous molars) or Gr6 (H-RME on first permanent molars) and compared between T0 (before treatment) and T1 (at the RME removal; i.e., 10 months after the end of the activation of the screw)...
April 10, 2017: Journal of Orofacial Orthopedics, Fortschritte der Kieferorthopädie
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