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Proof of concept trials

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https://www.readbyqxmd.com/read/28202358/a-prospective-multicenter-double-blind-randomized-trial-of-bladder-instillation-of-liposome-formulation-onabotulinumtoxina-for-interstitial-cystitis-bladder-pain-syndrome
#1
Yao-Chi Chuang, Hann-Chorng Kuo
BACKGROUND: Intravesical instillation of liposomal-formulated botulinum toxin A (lipotoxin) has shown therapeutic effects on treatment of refractory overactive bladder without needle injections. We hereby assessed lipotoxin for the treatment of refractory interstitial cystitis/bladder pain syndrome (IC/BPS). METHODS: This two-center, double-blind, randomized, placebo-controlled physician-initiated study enrolled patients with refractory IC/BPS. The patients were assigned to intravesical instillation of lipotoxin (onabotulinumtoxinA 200 U with 80mg sphingomyelin) (n=31), onabotulinumtoxinA 200 U in normal saline (n=28), or normal saline alone (n=31)...
February 12, 2017: Journal of Urology
https://www.readbyqxmd.com/read/28201851/the-design-of-clinical-trials-in-portal-hypertension
#2
Juan G Abraldes, Guadalupe Garcia-Tsao
Portal hypertension (PH) is the main consequence of cirrhosis and is responsible for the majority of its complications. Gastroesophageal varices and variceal hemorrhage are direct consequences of PH; therefore, most clinical trials in PH have been directed toward treating or preventing variceal hemorrhage. However, varices and variceal hemorrhage are not isolated events; they must be considered in the context of the presence (or absence) of other complications of cirrhosis/PH. Cirrhosis progresses across different stages, each with a different prognosis and pathophysiology and hence different therapeutic targets...
February 2017: Seminars in Liver Disease
https://www.readbyqxmd.com/read/28197884/therapies-of-early-advanced-and-late-onset-forms-of-axial-spondyloarthritis-and-the-need-for-treat-to-target-strategies
#3
REVIEW
Nurullah Akkoc, Gercek Can, Salvatore D'Angelo, Angela Padula, Ignazio Olivieri
PURPOSE OF REVIEW: This study aims to provide an update on current status of pharmacological therapies in early and advanced stages of axial spondyloarthritis (axSpA), as well as its late onset forms, and to discuss the need for treat to target strategies in this entity. RECENT FINDINGS: Efficacy of TNF inhibitors has been assessed in randomized controlled trials in axSpA, which included patients who had non-radiographic axSpA according to the ASAS classification criteria...
February 2017: Current Rheumatology Reports
https://www.readbyqxmd.com/read/28197000/impact-of-an-educational-module-in-antidepressant-naive-patients-prescribed-antidepressants-for-depression-pilot-proof-of-concept-randomized-controlled-trial
#4
Annie P John, Nagendra Madan Singh, Nagarajaiah, Chittaranjan Andrade
BACKGROUND: Patients are educated about their illness and its treatment at the time of diagnosis. However, little is known about how much of this education is retained and how it influences knowledge about, attitudes toward, and experiences with medication in antidepressant-naive patients with depression. METHODS: Antidepressant-naive outpatients with International Classification of Diseases-10 dysthymia or mild to moderate depression, who were advised antidepressant monotherapy, were randomized to control (n = 22) or intervention (n = 17) groups...
October 2016: Indian Journal of Psychiatry
https://www.readbyqxmd.com/read/28194410/acute-feasibility-of-neuromuscular-electrical-stimulation-in-severely-obese-patients-with-obstructive-sleep-apnea-syndrome-a-pilot-study
#5
RANDOMIZED CONTROLLED TRIAL
Isabelle Vivodtzev, Nicola A Maffiuletti, Anne-Laure Borel, Angélique Grangier, Bernard Wuyam, Renaud Tamisier, Jean-Louis Pépin
Objective. Obesity and obstructive sleep apnea (OSA) are closely interconnected conditions both leading to high cardiovascular risk. Inactivity is frequent and physical activity programs remain difficult in these patients. We investigated the acute feasibility of two neuromuscular electrical stimulation (NMES) modalities in extremely inactive obese patients with OSA. Design. A randomized cross-over study, with two experimental sessions (one per condition: multipath NMES versus conventional NMES). Setting. Outpatient research hospital...
2017: BioMed Research International
https://www.readbyqxmd.com/read/28193778/ag-221-a-first-in-class-therapy-targeting-acute-myeloid-leukemia-harboring-oncogenic-idh2-mutations
#6
Katharine Yen, Jeremy Travins, Fang Wang, Muriel D David, Erin Artin, Kim Straley, Anil Padyana, Stefan Gross, Byron DeLaBarre, Erica Tobin, Yue Chen, Raj Nagaraja, Sung Choe, Lei Jin, Zenon Konteatis, Giovanni Cianchetta, Jeffrey O Saunders, Francesco G Salituro, Cyril Quivoron, Paule Opolon, Olivia Bawa, Véronique Saada, Angelo Paci, Sophie Broutin, Olivier A Bernard, Stéphane de Botton, Benoît S Marteyn, Monika Pilichowska, YingXia Xu, Cheng Fang, Fan Jiang, Wentao Wei, Shengfang Jin, Lee Silverman, Wei Liu, Hua Yang, Lenny Dang, Marion Dorsch, Virginie Penard-Lacronique, Scott A Biller, Shin-San Michael Su
Somatic gain-of-function mutations in isocitrate dehydrogenase (IDH) 1 and 2 are found in multiple hematologic and solid tumors, leading to accumulation of the oncometabolite, (R)-2-hydroxyglutarate (2HG). 2HG competitively inhibits α-ketoglutarate-dependent dioxygenases, including histone demethylases and methylcytosine dioxygenases of the Tet family, causing epigenetic dysregulation and a block in cellular differentiation. In vitro studies have provided proof of concept for mutant IDH inhibition as a therapeutic approach...
February 13, 2017: Cancer Discovery
https://www.readbyqxmd.com/read/28193216/a-high-throughput-expression-screening-platform-to-optimize-the-production-of-antimicrobial-peptides
#7
Christine Schreiber, Hagen Müller, Oliver Birrenbach, Moritz Klein, Doreen Heerd, Tobias Weidner, Denise Salzig, Peter Czermak
BACKGROUND: Antimicrobial peptides (AMPs) are promising candidates for the development of novel antibiotics, but it is difficult to produce sufficient quantities for preclinical and clinical studies due to their toxicity towards microbial expression hosts. To avoid laborious trial-and-error testing for the identification of suitable expression constructs, we have developed a small-scale expression screening platform based on a combinatorial plasmid library. RESULTS: The combinatorial library is based on the Golden Gate cloning system...
February 13, 2017: Microbial Cell Factories
https://www.readbyqxmd.com/read/28192983/melted-paraffin-wax-as-an-innovative-liquid-and-solid-extractant-for-elemental-analysis-by-laser-induced-breakdown-spectroscopy
#8
Rodrigo Papai, Roseli Hiromi Sato, Lidiane Cristina Nunes, Francisco José Krug, Ivanise Gaubeur
This work proposes a new development in the use of melted paraffin wax as a new extractant in a procedure designed to aggregate the advantages of liquid phase extraction (extract homogeneity, fast, and efficient transfer, low cost and simplicity) to solid phase extraction. As proof of concept, copper(II) in aqueous samples was converted into a hydrophobic complex of copper(II) diethyldithiocarbamate and subsequently extracted into paraffin wax. Parameters which affect the complexation and extraction (pH, DDTC, and Triton X-100 concentration, vortex agitation time and complexation time) were optimized in a univariate way...
February 16, 2017: Analytical Chemistry
https://www.readbyqxmd.com/read/28191757/cartilage-regeneration-in-osteoarthritic-patients-by-a-composite-of-allogeneic-umbilical-cord-blood-derived-mesenchymal-stem-cells-and-hyaluronate-hydrogel-results-from-a-clinical-trial-for-safety-and-proof-of-concept-with-7-years-of-extended-follow-up
#9
Yong-Beom Park, Chul-Won Ha, Choong-Hee Lee, Young Cheol Yoon, Yong-Geun Park
Few methods are available to regenerate articular cartilage defects in patients with osteoarthritis. We aimed to assess the safety and efficacy of articular cartilage regeneration by a novel medicinal product composed of allogeneic human umbilical cord blood-derived mesenchymal stem cells (hUCB-MSCs). Patients with Kellgren-Lawrence grade 3 osteoarthritis and International Cartilage Repair Society (ICRS) grade 4 cartilage defects were enrolled in this clinical trial. The stem cell-based medicinal product (a composite of culture-expanded allogeneic hUCB-MSCs and hyaluronic acid hydrogel [Cartistem]) was applied to the lesion site...
February 2017: Stem Cells Translational Medicine
https://www.readbyqxmd.com/read/28191745/comparison-of-sampling-methods-to-recover-germinated-bacillus-anthracis-and-bacillus-thuringiensis-endospores-from-surface-coupons
#10
Tiffany M Mott, Jennifer L Shoe, Melissa Hunter, Amber M Woodson, Kristen A Fritts, Christopher P Klimko, Avery V Quirk, Susan L Welkos, Christopher K Cote
AIMS: In an attempt to devise decontamination methods that are both effective and minimally detrimental to the environment, we evaluated germination-induction as an enhancement to strategies for Bacillus anthracis spore decontamination. To determine an optimal method for the recovery of germinating spores from different matrices it was critical to ensure that the sampling procedures did not negatively impact the viability of the germinating spores possibly confounding the results and downstream analyses of field trial data...
February 12, 2017: Journal of Applied Microbiology
https://www.readbyqxmd.com/read/28191268/automated-electrotransformation-of-escherichia-coli-on-a-digital-microfluidic-platform-using-bioactivated-magnetic-beads
#11
J A Moore, M Nemat-Gorgani, A C Madison, M A Sandahl, S Punnamaraju, A E Eckhardt, M G Pollack, F Vigneault, G M Church, R B Fair, M A Horowitz, P B Griffin
This paper reports on the use of a digital microfluidic platform to perform multiplex automated genetic engineering (MAGE) cycles on droplets containing Escherichia coli cells. Bioactivated magnetic beads were employed for cell binding, washing, and media exchange in the preparation of electrocompetent cells in the electrowetting-on-dieletric (EWoD) platform. On-cartridge electroporation was used to deliver oligonucleotides into the cells. In addition to the optimization of a magnetic bead-based benchtop protocol for generating and transforming electrocompetent E...
January 2017: Biomicrofluidics
https://www.readbyqxmd.com/read/28186705/current-and-future-perspectives-on-alginate-encapsulated-pancreatic-islet
#12
Berit L Strand, Abba E Coron, Gudmund Skjak-Braek
Transplantation of pancreatic islets in immune protective capsules holds the promise as a functional cure for type 1 diabetes, also about 40 years after the first proof of principal study. The concept is simple in using semipermeable capsules that allow the ingress of oxygen and nutrients, but limit the access of the immune system. Encapsulated human islets have been evaluated in four small clinical trials where the procedure has been evaluated as safe, but lacking long-term efficacy. Host reactions toward the biomaterials used in the capsules may be one parameter limiting the long-term function of the graft in humans...
February 2, 2017: Stem Cells Translational Medicine
https://www.readbyqxmd.com/read/28177994/bilateral-posterior-tibial-nerve-stimulation-in-the-treatment-of-rectal-evacuation-disorder-a-preliminary-report
#13
Khaled M Madbouly, Khaled S Abbas, Emad Emanuel
BACKGROUND: Posterior tibial nerve stimulation influences both motor and sensory pathways, as well as the central nervous system. Stimulation of posterior tibial nerve roots (L4 to S3) could improve stool evacuation through S3 and/or S2 stimulation. OBJECTIVE: This study aimed to assess the efficiency of bilateral posterior tibial nerve stimulation in the treatment of rectal evacuation disorder without anatomic obstruction. DESIGN: This was a prospective case series studying the treatment of patients with obstructed defecation by posterior tibial nerve stimulation...
March 2017: Diseases of the Colon and Rectum
https://www.readbyqxmd.com/read/28176964/efficacy-and-safety-of-biological-and-targeted-synthetic-dmards-a-systematic-literature-review-informing-the-2016-update-of-the-asas-eular-recommendations-for-the-management-of-axial-spondyloarthritis
#14
Alexandre Sepriano, Andrea Regel, Désirée van der Heijde, Jürgen Braun, Xenofon Baraliakos, Robert Landewé, Filip Van den Bosch, Louise Falzon, Sofia Ramiro
OBJECTIVES: To update the evidence for the efficacy and safety of (b)biological and (ts)targeted-synthetic disease-modifying anti-rheumatic drugs (DMARDs) in patients with axial spondyloarthritis (axSpA) to inform the 2016 update of the Assessment of SpondyloArthritis international Society/European League Against Rheumatism (ASAS/EULAR) recommendations for the management of axSpA. METHODS: Systematic literature review (2009-2016) for randomised controlled trials (RCT), including long-term extensions, strategy trials and observational studies (the latter was only for safety assessment and a comparator was required)...
2017: RMD Open
https://www.readbyqxmd.com/read/28174390/-surgical-regeneration-therapy-using-myoblast-sheets-for-severe-heart-failure
#15
Yoshiki Sawa
Heart failure is a life-threatening disorder worldwide, and the current end-stage therapies for severe heart failure are replacement therapies such as ventricular-assist devices and heart transplantation. Although these therapies have been reported to be useful, there are many issues in terms of the durability, complications, limited donors, adverse effect of continuous administration of immunosuppressive agents, and high costs involved. Recently, regenerative therapy based on genetic, cellular, or tissue engineering techniques has gained attention as a new therapy to overcome the challenges encountered in transplantation medicine...
January 2017: Kyobu Geka. the Japanese Journal of Thoracic Surgery
https://www.readbyqxmd.com/read/28174194/safety-and-efficacy-of-remote-ischemic-preconditioning-in-patients-with-severe-carotid-artery-stenosis-prior-to-carotid-artery-stenting-a-proof-of-concept-randomized-controlled-trial
#16
Wenbo Zhao, Ran Meng, Chun Ma, Baojun Hou, Liqun Jiao, Fengshui Zhu, Weijuan Wu, Jingfei Shi, Yunxia Duan, Renling Zhang, Jing Zhang, Yongxin Sun, Hongqi Zhang, Feng Ling, Yuping Wang, Wuwei Feng, Yuchuan Ding, Bruce Ovbiagele, Xunming Ji
BACKGROUND: -Remote ischemic preconditioning (RIPC) can inhibit recurrent ischemic events effectively in patients with acute or chronic cerebral ischemia. However, it is still unclear that whether RIPC can impede ischemic injury after carotid artery stenting (CAS) in patients with severe carotid artery stenosis. METHODS: -Subjects with severe carotid artery stenosis were recruited in this randomized controlled study, and assigned to RIPC, sham and no intervention (control) groups...
February 7, 2017: Circulation
https://www.readbyqxmd.com/read/28174191/augmentation-of-muscle-blood-flow-by-ultrasound-cavitation-is-mediated-by-atp-and-purinergic-signaling
#17
J Todd Belcik, Brian P Davidson, Aris Xie, Melinda D Wu, Mrinal Yadava, Yue Qi, Sherry Liang, Chae Ryung Chon, Azzdine Y Ammi, Joshua Field, Leanne Harmann, William M Chilian, Joel Linden, Jonathan R Lindner
BACKGROUND: - Augmentation of tissue blood flow by therapeutic ultrasound is thought to rely on convective shear. Microbubble contrast agents that undergo ultrasound-mediated cavitation markedly amplify these effects. We hypothesized that purinergic signalling is responsible for shear-dependent increases in muscle perfusion during therapeutic cavitation. METHODS: -Unilateral exposure of the proximal hindlimb of mice (with or without ischemia produced by iliac ligation) to therapeutic ultrasound (1...
February 7, 2017: Circulation
https://www.readbyqxmd.com/read/28166459/a-bayesian-paradigm-for-decision-making-in-proof-of-concept-trials
#18
Erik Pulkstenis, Kaushik Patra, Jianliang Zhang
Decision making is central to every phase of drug development, and especially at the Proof of Concept stage where risk and evidence must be weighed carefully, often in the presence of significant uncertainty. The decision to proceed or not to large expensive Phase 3 trials has significant implications to both patients and sponsors alike. Recent experience has shown that Phase 3 failure rates remain high. We present a flexible Bayesian quantitative decision making paradigm that evaluates evidence relative to achieving a multilevel target product profile...
February 6, 2017: Journal of Biopharmaceutical Statistics
https://www.readbyqxmd.com/read/28164088/effective-implementation-of-novel-met-pharmacodynamic-assays-in-translational-studies
#19
REVIEW
Apurva K Srivastava, Tony Navas, William G Herrick, Melinda G Hollingshead, Donald P Bottaro, James H Doroshow, Ralph E Parchment
MET tyrosine kinase (TK) dysregulation is significantly implicated in many types of cancer. Despite over 20 years of drug development to target MET in cancers, a pure anti-MET therapeutic has not yet received market approval. The failure of two recently concluded phase III trials point to a major weakness in biomarker strategies to identify patients who will benefit most from MET therapies. The capability to interrogate oncogenic mutations in MET via circulating tumor DNA (ctDNA) provides an important advancement in identification and stratification of patients for MET therapy...
January 2017: Annals of Translational Medicine
https://www.readbyqxmd.com/read/28161513/the-burden-of-proof-the-current-state-of-atrial-fibrillation-prevention-and-treatment-trials
#20
Rosita Zakeri, David R Van Wagoner, Hugh Calkins, Tom Wong, Heather M Ross, E Kevin Heist, Timothy E Meyer, Peter R Kowey, Robert J Mentz, John G Cleland, Bertram Pitt, Faiez Zannad, Cecilia Linde
Atrial fibrillation (AF) is an age-related arrhythmia of enormous socio-economic significance. In recent years, our understanding of the basic mechanisms that initiate and perpetuate AF has evolved rapidly, catheter ablation of AF has progressed from concept to reality, and recent studies suggest lifestyle modification may help prevent AF recurrence. Emerging developments in genetics, imaging, and informatics also present new opportunities for personalised care. However considerable challenges remain. These include a paucity of studies examining AF prevention, modest efficacy of existing antiarrhythmic therapies, diverse ablation technologies and practice, and limited evidence to guide management in high-risk patients with multiple comorbidities...
February 1, 2017: Heart Rhythm: the Official Journal of the Heart Rhythm Society
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