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Proof of concept trials

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https://www.readbyqxmd.com/read/28330821/beyond-precision-surgery-molecularly-motivated-precision-care-for-gastric-cancer
#1
REVIEW
Y Y Choi, J-H Cheong
Gastric cancer is one of the leading causes of cancer-related deaths worldwide. Despite the high disease prevalence, gastric cancer research has not gained much attention. Recently, genome-scale technology has made it possible to explore the characteristics of gastric cancer at the molecular level. Accordingly, gastric cancer can be classified into molecular subtypes that convey more detailed information of tumor than histopathological characteristics, and these subtypes are associated with clinical outcomes...
March 1, 2017: European Journal of Surgical Oncology
https://www.readbyqxmd.com/read/28328012/free-fatty-acid-receptor-1-ffar1-as-an-emerging-therapeutic-target-for-type-2-diabetes-mellitus-recent-progress-and-prevailing-challenges
#2
REVIEW
Zheng Li, Xue Xu, Wenlong Huang, Hai Qian
The free fatty acid receptor 1 (FFAR1/GPR40) amplifies glucose-dependent insulin secretion; therefore, it has attracted widespread attention as a promising antidiabetic target. Current clinical proof of concept also indicates that FFAR1 agonists achieve the initially therapeutic endpoint for the treatment of type 2 diabetes mellitus (T2DM) without the hypoglycemic risk. Thus, many pharmaceutical companies and academic institutes are competing to develop FFAR1 agonists. However, several candidates have been discontinued in clinical trials, often without reporting the underlying reasons...
March 22, 2017: Medicinal Research Reviews
https://www.readbyqxmd.com/read/28326351/effects-of-low-dose-ibuprofen-supplementation-and-resistance-training-on-bone-and-muscle-in-postmenopausal-women-a-randomized-controlled-trial
#3
Whitney R D Duff, Saija A Kontulainen, Darren G Candow, Julianne J Gordon, Riley S Mason, Regina Taylor-Gjevre, Bindu Nair, Michael Szafron, Adam D G Baxter-Jones, Gordon A Zello, Philip D Chilibeck
PURPOSE: To compare the effects of nine months of exercise training and ibuprofen supplementation (given immeditately after exercise sessions) on bone and muscle in postmenopausal women. METHODS: In a double-blind randomized trial, participants (females: n = 90, mean age 64.8, SD 4.3 years) were assigned (computer generated, double blind) to receive supervised resistance training or stretching 3 days/week, and ibuprofen (400 mg, post-exercise) or placebo (i...
December 2016: Bone Reports
https://www.readbyqxmd.com/read/28326010/impact-of-resveratrol-on-glucose-control-hippocampal-structure-and-connectivity-and-memory-performance-in-patients-with-mild-cognitive-impairment
#4
Theresa Köbe, A Veronica Witte, Ariane Schnelle, Valentina A Tesky, Johannes Pantel, Jan-Philipp Schuchardt, Andreas Hahn, Jens Bohlken, Ulrike Grittner, Agnes Flöel
In healthy older adults, resveratrol supplementation has been shown to improve long-term glucose control, resting-state functional connectivity (RSFC) of the hippocampus, and memory function. Here, we aimed to investigate if these beneficial effects extend to individuals at high-risk for dementia, i.e., patients with mild cognitive impairment (MCI). In a randomized, double-blind interventional study, 40 well-characterized patients with MCI (21 females; 50-80 years) completed 26 weeks of resveratrol (200 mg/d; n = 18) or placebo (1,015 mg/d olive oil; n = 22) intake...
2017: Frontiers in Neuroscience
https://www.readbyqxmd.com/read/28325150/managing-expectations-in-the-transition-to-proof-of-concept-studies
#5
Thomas Kieber-Emmons, Issam Makhoul, Angela Pennisi, Eric R Siegel, Peter D Emanuel, Bejotaloh Monzavi-Karbassi, Zenon Steplewski, J Thaddeus Beck, Laura F Hutchins
BACKGROUND: As we are moving away from the traditional chemotherapy era to targeted therapy, the validity of old assessment paradigms associated with therapeutics are being raised in the context of immunotherapy. The old paradigm required elaborate assessment of toxicity with no expectation of efficacy in early phase trials. Safety data from Phase 1 and 2 studies with many immunotherapeutics show that they display limited toxicities and draw attention to the need to demonstrate efficacy in the early evaluation of new agents...
March 21, 2017: Reviews on Recent Clinical Trials
https://www.readbyqxmd.com/read/28319444/retro-orbital-venous-sinus-delivery-of-raav9-mediates-high-level-transduction-of-brain-and-retina-compared-with-temporal-vein-delivery-in-neonatal-mouse-pups
#6
Alisha M Gruntman, Lin Su, Terence R Flotte
In order to pursue a clinical gene therapy for a human neurologic disease, it is often necessary to perform proof-of-concept trials in mouse models of that disease. In order to demonstrate a potential clinical efficacy, one must be able to select an appropriate vector and route of delivery for the appropriate age group in the disease model. Since many diseases require correction early in life, investigators often need to deliver recombinant adeno-associated viral (rAAV) vectors to neonatal mice. Herein, general central nervous system expression patterns of nuclear GFP following delivery of rAAV by three different routes are reported...
March 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28318816/use-of-electromyography-to-optimize-lokomat-%C3%A2-settings-for-subject-specific-gait-rehabilitation-in-post-stroke-hemiparetic-patients-a-proof-of-concept-study
#7
Yosra Cherni, Mickael Begon, Hicham Chababe, Florent Moissenet
OBJECTIVES: While generic protocols exist for gait rehabilitation using robotic orthotics such as the Lokomat(®), several settings - guidance, body-weight support (BWS) and velocity - may be adjusted to individualize patient training. However, no systematic approach has yet emerged. Our objective was to assess the feasibility and effects of a systematic approach based on electromyography to determine subject-specific settings with application to the strengthening of the gluteus maximus muscle in post-stroke hemiparetic patients...
March 16, 2017: Neurophysiologie Clinique, Clinical Neurophysiology
https://www.readbyqxmd.com/read/28318420/bayesian-penalized-log-likelihood-ratio-approach-for-dose-response-clinical-trial-studies
#8
Yuanyuan Tang, Chunyan Cai, Liangrui Sun, Jianghua He
In literature, there are a few unified approaches to test proof of concept and estimate a target dose, including the multiple comparison procedure using modeling approach, and the permutation approach proposed by Klingenberg. We discuss and compare the operating characteristics of these unified approaches and further develop an alternative approach in a Bayesian framework based on the posterior distribution of a penalized log-likelihood ratio test statistic. Our Bayesian approach is much more flexible to handle linear or nonlinear dose-response relationships and is more efficient than the permutation approach...
February 13, 2017: Journal of Biopharmaceutical Statistics
https://www.readbyqxmd.com/read/28314707/biometrics-and-policing-a-protocol-for-multichannel-sensor-data-collection-and-exploratory-analysis-of-contextualized-psychophysiological-response-during-law-enforcement-operations
#9
Robert D Furberg, Travis Taniguchi, Brian Aagaard, Alexa M Ortiz, Meghan Hegarty-Craver, Kristin H Gilchrist, Ty A Ridenour
BACKGROUND: Stress experienced by law enforcement officers is often extreme and is in many ways unique among professions. Although past research on officer stress is informative, it is limited, and most studies measure stress using self-report questionnaires or observational studies that have limited generalizability. We know of no research studies that have attempted to track direct physiological stress responses in high fidelity, especially within an operational police setting. The outcome of this project will have an impact on both practitioners and policing researchers...
March 17, 2017: JMIR Research Protocols
https://www.readbyqxmd.com/read/28314411/small-molecule-inhibition-of-interleukin-1-receptor-associated-kinase-4-irak4
#10
N E Genung, K M Guckian
In recent years, interleukin-1 receptor-associated kinase 4, IRAK4, has become an attractive target for many medicinal chemistry programmes. Target inhibition is of potential therapeutic value in areas including autoimmune disorders, cancer, inflammatory diseases, and possibly neurodegenerative diseases. Results from high-throughput screening efforts have led, in conjunction with structure-based drug design, to the identification of highly potent and selective small molecule IRAK4 inhibitors from many diverse chemical series...
2017: Progress in Medicinal Chemistry
https://www.readbyqxmd.com/read/28296167/what-can-intracoronary-pressure-measurements-tell-us-about-flow-reserve-pressure-bounded-coronary-flow-reserve-and-example-application-to-the-randomized-defer-trial
#11
Frederik M Zimmermann, Nico H J Pijls, Bernard De Bruyne, G Jan-Willem Bech, Pepijn van Schaardenburgh, Richard L Kirkeeide, K Lance Gould, Nils P Johnson
OBJECTIVE: We propose a novel technique called pressure-bounded coronary flow reserve (pb-CFR) and demonstrate its application to the randomized DEFER trial. BACKGROUND: Intracoronary flow reserve assessment remains underutilized relative to pressure measurements partly due to less robust tools. METHODS: While rest and hyperemic intracoronary pressure measurements cannot quantify CFR exactly, they do provide upper and lower bounds. We validated pb-CFR invasively against traditional CFR, then applied it to high fractional flow reserve (FFR ≥ 0...
March 15, 2017: Catheterization and Cardiovascular Interventions
https://www.readbyqxmd.com/read/28294453/a-translational-approach-for-nmda-receptor-profiling-as-a-vulnerability-biomarker-for-depression-and-schizophrenia
#12
Handan Gunduz-Bruce, Joshua Kenney, Suravi Changlani, Aldo Peixoto, Ralitza Gueorguieva, Cheryl Leone, Nina Stachenfeld
Altered N-methyl-D-aspartate (NMDA) receptor activity and glutamate signaling may underlie the pathogenesis of both schizophrenia and depression in subgroups of patients. In schizophrenia, pharmacologic modeling, postmortem and imaging data suggest reduced NMDA signaling. In contrast, recent clinical trials demonstrating the efficacy of the NMDA antagonist ketamine in severely depressed patients suggest increased NMDA receptor signaling. We conducted a proof of concept study to assess whether there is any in vivo evidence for an inverse association in depression and schizophrenia with respect to the NMDA receptor function...
March 13, 2017: Experimental Physiology
https://www.readbyqxmd.com/read/28289776/-research-funding-for-rare-diseases-in-germany
#13
Frank Wissing, Leena Bruckner-Tuderman
There is high need for more research in the field of rare diseases. Not only must the causes and mechanisms of the numerous and often heterogeneous diseases be delineated, but criteria must also be defined for optimal stratification of patients for individualized therapies. In this context, research and innovative diagnostics are linked together more closely than in other fields of medicine. The early stages of disease-oriented research can be performed in individual institutions but, due to low numbers of patients, late translation and transfer into clinics requires multicentric and international collaboration...
March 13, 2017: Bundesgesundheitsblatt, Gesundheitsforschung, Gesundheitsschutz
https://www.readbyqxmd.com/read/28287872/development-of-a-modular-automated-system-for-maintenance-and-differentiation-of-adherent-human-pluripotent-stem-cells
#14
Duncan E Crombie, Maciej Daniszewski, Helena H Liang, Tejal Kulkarni, Fan Li, Grace E Lidgerwood, Alison Conquest, Damian Hernández, Sandy S Hung, Katherine P Gill, Elisabeth De Smit, Lisa S Kearns, Linda Clarke, Valentin M Sluch, Xitiz Chamling, Donald J Zack, Raymond C B Wong, Alex W Hewitt, Alice Pébay
Patient-specific induced pluripotent stem cells (iPSCs) have tremendous potential for development of regenerative medicine, disease modeling, and drug discovery. However, the processes of reprogramming, maintenance, and differentiation are labor intensive and subject to intertechnician variability. To address these issues, we established and optimized protocols to allow for the automated maintenance of reprogrammed somatic cells into iPSCs to enable the large-scale culture and passaging of human pluripotent stem cells (PSCs) using a customized TECAN Freedom EVO...
March 1, 2017: SLAS Discov
https://www.readbyqxmd.com/read/28286668/a-protocol-for-a-cluster-randomised-feasibility-study-of-an-adolescent-incentive-intervention-to-increase-uptake-of-hpv-vaccination-among-girls
#15
Alice S Forster, Victoria Cornelius, Lauren Rockliffe, Laura A V Marlow, Helen Bedford, Jo Waller
BACKGROUND: Uptake of the human papillomavirus (HPV) vaccine in the UK is good, but there are pockets of the community who remain unprotected. Immunisation teams usually require written parental consent for a girl to receive the vaccine. Evidence suggests that uptake of the vaccine might be improved by promoting consent form return (if returned, forms are likely to grant consent). Incentivising girls to return consent forms is a promising approach to promoting consent form return. Before testing the efficacy of an incentive intervention in a randomised controlled trial (RCT), we must first establish whether the RCT is feasible...
2017: Pilot and Feasibility Studies
https://www.readbyqxmd.com/read/28285644/effects-of-transdermal-testosterone-gel-or-an-aromatase-inhibitor-on-serum-concentration-and-pulsatility-of-growth-hormone-in-older-men-with-age-related-low-testosterone
#16
Jenny Pena Dias, Johannes D Veldhuis, Olga Carlson, Michelle Shardell, Chee W Chia, Denise Melvin, Josephine M Egan, Shehzad Basaria
Growth hormone is the major regulator of growth and body composition. Pulsatile GH secretion declines exponentially with age. Testosterone replacement is being increasingly offered to older men with age-related low testosterone. Testosterone administration has been shown to stimulate GH secretion. However, little is known about the effect of testosterone aromatization to estradiol on GH pulsatility and its impact on IGF-1 in older men. OBJECTIVE: This randomized controlled proof-of-concept trial investigated the relative effects of testosterone and estradiol on GH pulsatility and IGF-1 in older men with low testosterone...
April 2017: Metabolism: Clinical and Experimental
https://www.readbyqxmd.com/read/28284655/improving-resident-performance-in-oculoplastic-surgery-a-new-curriculum-using-surgical-wet-laboratory-videos
#17
Kapil Mishra, Mariam Mathai, Robert C Della Rocca, Harsha S Reddy
OBJECTIVE: To develop a new oculoplastic curriculum that incorporates learning theory of skill acquisition. To develop and evaluate the effectiveness of instructional videos for an oculoplastic surgical wet laboratory. DESIGN: Proof of concept, randomized controlled trial. SETTING: New York Eye and Ear Infirmary of Mount Sinai-tertiary care academic institution. PARTICIPANTS AND METHODS: In total, 16 ophthalmology residents were randomly assigned to 1 of 2 groups and given either video and text or text instructions alone for the following 2 procedures: blepharoplasty and eyelid laceration repair...
March 8, 2017: Journal of Surgical Education
https://www.readbyqxmd.com/read/28274108/small-intestinal-bacterial-overgrowth-and-irritable-bowel-syndrome-a-bridge-between-functional-organic-dichotomy
#18
REVIEW
Uday C Ghoshal, Ratnakar Shukla, Ujjala Ghoshal
The pathogenesis of irritable bowel syndrome (IBS), once thought to be largely psychogenic in origin, is now understood to be multifactorial. One of the reasons for this paradigm shift is the realization that gut dysbiosis, including small intestinal bacterial overgrowth (SIBO), causes IBS symptoms. Between 4% and 78% of patients with IBS and 1% and 40% of controls have SIBO; such wide variations in prevalence might result from population differences, IBS diagnostic criteria, and, most importantly, methods to diagnose SIBO...
March 15, 2017: Gut and Liver
https://www.readbyqxmd.com/read/28269635/wearable-embroidered-muscle-activity-sensing-device-for-the-human-upper-leg
#19
R B Ribas Manero, A Shafti, B Michael, J Grewal, J Ll Ribas Fernandez, K Althoefer, M J Howard
Within the last decade, running has become one of the most popular physical activities in the world. Although the benefits of running are numerous, there is a risk of Running Related Injuries (RRIs) of the lower extremities. Electromyography (EMG) techniques have previously been used to study causes of RRIs, but the complexity of this technology limits its use to a laboratory setting. As running is primarily an outdoors activity, this lack of technology acts as a barrier to the study of RRIs in natural environments...
August 2016: Conference Proceedings: Annual International Conference of the IEEE Engineering in Medicine and Biology Society
https://www.readbyqxmd.com/read/28266963/safety-and-efficacy-of-a-topical-sodium-channel-inhibitor-tv-45070-in-patients-with-postherpetic-neuralgia-phn-a-randomized-controlled-proof-of-concept-crossover-study-with-a-subgroup-analysis-of-the-nav1-7-r1150w-genotype
#20
Nicola Price, Rostam Namdari, Judith Neville, Katie J W Proctor, Samer Kaber, Jeffery Vest, Michael Fetell, Richard Malamut, Robin P Sherrington, Simon N Pimstone, Yigal P Goldberg
OBJECTIVE: The objective was to evaluate the safety and efficacy of TV-45070 ointment, as a treatment for postherpetic neuralgia, and to explore the response in patients with the Nav1.7 R1150W gain-of-function polymorphism. MATERIALS AND METHODS: This was a randomized, placebo-controlled, 2-period, 2-treatment crossover trial. Patients with postherpetic neuralgia with moderate or greater pain received TV-45070 and placebo ointments, each applied twice daily for 3 weeks...
April 2017: Clinical Journal of Pain
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