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Proof of concept trials

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https://www.readbyqxmd.com/read/28923239/a-novel-formulation-of-inhaled-sodium-cromoglicate-pa101-in-idiopathic-pulmonary-fibrosis-and-chronic-cough-a-randomised-double-blind-proof-of-concept-phase-2-trial
#1
Surinder S Birring, Marlies S Wijsenbeek, Sanjay Agrawal, Jan W K van den Berg, Helen Stone, Toby M Maher, Ahmet Tutuncu, Alyn H Morice
BACKGROUND: Cough can be a debilitating symptom of idiopathic pulmonary fibrosis (IPF) and is difficult to treat. PA101 is a novel formulation of sodium cromoglicate delivered via a high-efficiency eFlow nebuliser that achieves significantly higher drug deposition in the lung compared with the existing formulations. We aimed to test the efficacy and safety of inhaled PA101 in patients with IPF and chronic cough and, to explore the antitussive mechanism of PA101, patients with chronic idiopathic cough (CIC) were also studied...
September 8, 2017: Lancet Respiratory Medicine
https://www.readbyqxmd.com/read/28901946/design-of-semg-assembly-to-detect-external-anal-sphincter-activity-a-proof-of-concept
#2
Arsam Shiraz, Brian Leaker, Charles Alexander Mosse, Eskinder Solomon, Michael Craggs, Andreas Demosthenous
Conditional trans-rectal stimulation of the pudendal nerve could provide a viable solution to treat hyperreflexive bladder in spinal cord injury. A set threshold of the amplitude estimate of the external anal sphincter surface electromyography (sEMG) may be used as the trigger signal. The efficacy of such a device should be tested in a large scale clinical trial. As such a probe should remain in situ for several hours while patients attend to their daily routine, the recording electrodes should be designed to be large enough to maintain good contact while observing design constraints...
September 13, 2017: Physiological Measurement
https://www.readbyqxmd.com/read/28901195/feedforward-coordinate-control-of-a-robotic-cell-injection-catheter
#3
Weyland Cheng, Peter K Law
Remote and robotically actuated catheters are the stepping-stones toward autonomous catheters, where complex intravascular procedures may be performed with minimal intervention from a physician. This article proposes a concept for the positional, feedforward control of a robotically actuated cell injection catheter used for the injection of myogenic or undifferentiated stem cells into the myocardial infarct boundary zones of the left ventricle. The prototype for the catheter system was built upon a needle-based catheter with a single degree of deflection, a 3-D printed handle combined with actuators, and the Arduino microcontroller platform...
August 2017: Cell Transplantation
https://www.readbyqxmd.com/read/28889819/advances-in-the-treatment-of-anorexia-nervosa-a-review-of-established-and-emerging-interventions
#4
T Brockmeyer, H-C Friederich, U Schmidt
BACKGROUND: Anorexia nervosa (AN) is a disabling, deadly and costly mental disorder. Until recently, treatment recommendations were based on expert opinion and limited evidence. The aim of this systematic review is to synthesise recent evidence on established and emerging AN treatments and to forecast trends for future developments. METHODS: We systematically review trials of established treatments and associated process outcome studies from the last 5 years, published since a previous review in this journal...
September 11, 2017: Psychological Medicine
https://www.readbyqxmd.com/read/28872549/89zr-trastuzumab-pet-ct-for-detection-of-human-epidermal-growth-factor-receptor-2-positive-metastases-in-patients-with-human-epidermal-growth-factor-receptor-2-negative-primary-breast-cancer
#5
Gary A Ulaner, David M Hyman, Serge K Lyashchenko, Jason S Lewis, Jorge A Carrasquillo
PURPOSE: The aim of this study was to determine if imaging with Zr-trastuzumab, a human epidermal growth factor receptor 2 (HER2)-targeting PET tracer, can detect HER2-positive metastases in patients with HER2-negative primary breast cancer. METHODS: As part of an institutional review board-approved, prospective clinical trial of Zr-trastuzumab PET/CT (ClinicalTrials.gov identifier NCT02286843), a second group of 11 patients with HER2-negative primary breast cancer and known metastatic disease were recruited...
September 1, 2017: Clinical Nuclear Medicine
https://www.readbyqxmd.com/read/28870120/virotherapy-research-in-germany-from-engineering-to-translation-a-review-as-contribution-to-the-combined-annual-meeting-of-the-german-and-european-societies-of-gene-and-cell-therapy-2017
#6
Guy Ungerechts, Christine E Engeland, Christian J Buchholz, Jürgen Eberle, Henry Fechner, Karsten Geletneky, Per Sonne Holm, Florian Kreppel, Florian Kühnel, Karl Sebastian Lang, Mathias F Leber, Antonio Marchini, Markus Moehler, Michael D Mühlebach, Jean Rommelaere, Christoph Springfeld, Ulrich M Lauer, Dirk M Nettelbeck
Virotherapy is a unique modality for treatment of cancer with oncolytic viruses (OVs) that selectively infect and lyse tumor cells, spread within tumors, and activate anti-tumor immunity. Different viruses are being developed as OVs preclinically and clinically, several of them engineered to encode therapeutic proteins for tumor-targeted gene therapy. Scientists and clinicians in Germany have made significant contributions to OV research and development, which are highlighted in this review article. Innovative strategies for "shielding", entry- or post-entry targeting, and "arming" of OVs have been established focusing on adeno-, measles, parvo-, and vaccinia virus platforms...
September 4, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28869295/the-clinical-potential-of-senolytic-drugs
#7
James L Kirkland, Tamara Tchkonia, Yi Zhu, Laura J Niedernhofer, Paul D Robbins
Senolytic drugs are agents that selectively induce apoptosis of senescent cells. These cells accumulate in many tissues with aging and at sites of pathology in multiple chronic diseases. In studies in animals, targeting senescent cells using genetic or pharmacological approaches delays, prevents, or alleviates multiple age-related phenotypes, chronic diseases, geriatric syndromes, and loss of physiological resilience. Among the chronic conditions successfully treated by depleting senescent cells in preclinical studies are frailty, cardiac dysfunction, vascular hyporeactivity and calcification, diabetes mellitus, liver steatosis, osteoporosis, vertebral disk degeneration, pulmonary fibrosis, and radiation-induced damage...
September 4, 2017: Journal of the American Geriatrics Society
https://www.readbyqxmd.com/read/28867605/high-throughput-and-sensitive-quantification-of-circulating-tumor-dna-by-microfluidic-based-multiplex-pcr-and-next-generation-sequencing
#8
Yinghui Guan, Oleg Mayba, Thomas Sandmann, Shan Lu, Younjeong Choi, Walter C Darbonne, Vincent Leveque, Lisa Ryner, Eric Humke, Nga Wan Rachel Tam, Sundari Sujathasarma, Anna Cheung, Richard Bourgon, Mark R Lackner, Yulei Wang
Circulating tumor DNA (ctDNA) has potential to serve as a biomarker for noninvasive monitoring of treatment response and disease progression. However, broad clinical applicability of ctDNA has been limited by the low sensitivity, throughput and patient coverage offered by existing ctDNA detection methods. Here we report the adaptation and characterization of the MMP-Seq technology for high-throughput and sensitive quantitation of ctDNA. A multiplex PCR preamplification (PreAmp) step was developed and incorporated into the MMP-seq workflow to enable low input ctDNA analysis with enhanced sensitivity...
August 31, 2017: Journal of Molecular Diagnostics: JMD
https://www.readbyqxmd.com/read/28866621/randomised-controlled-trial-to-assess-the-effect-of-a-just-in-time-training-on-procedural-performance-a-proof-of-concept-study-to-address-procedural-skill-decay
#9
Jeremy B Branzetti, Adeyinka A Adedipe, Matthew J Gittinger, Elizabeth D Rosenman, Sarah Brolliar, Anne K Chipman, James A Grand, Rosemarie Fernandez
BACKGROUND: A subset of high-risk procedures present significant safety threats due to their (1) infrequent occurrence, (2) execution under time constraints and (3) immediate necessity for patient survival. A Just-in-Time (JIT) intervention could provide real-time bedside guidance to improve high-risk procedural performance and address procedural deficits associated with skill decay. OBJECTIVE: To evaluate the impact of a novel JIT intervention on transvenous pacemaker (TVP) placement during a simulated patient event...
September 2, 2017: BMJ Quality & Safety
https://www.readbyqxmd.com/read/28862999/innovation-in-microbiome-based-strategies-for-promoting-metabolic-health
#10
Marina Romaní-Pérez, Ana Agusti, Yolanda Sanz
PURPOSE OF REVIEW: Update on the development of microbiome-based interventions and dietary supplements to combat obesity and related comorbidities, which are leading causes of global mortality. RECENT FINDINGS: The role of intestinal dysbiosis, partly resulting from unhealthy diets, in the development of obesity and metabolic disorders, is well documented by recent translational research. Human experimental trials with whole-faecal transplants are ongoing, and their results will be crucial as proof of concept that interventions intended to modulate the microbiome composition and function could be alternatives for the management of obesity and related comorbidities...
August 31, 2017: Current Opinion in Clinical Nutrition and Metabolic Care
https://www.readbyqxmd.com/read/28862464/use-of-cbct-imaging-open-source-modeling-software-and-desktop-stereolithography-3d-printing-to-duplicate-a-removable-dental-prosthesis-a-proof-of-concept
#11
Wei-Shao Lin, Bryan T Harris, Dean Morton
This article describes an alternative computer-aided design/computer-aided manufacturing (CAD/CAM) technique that uses cone-beam computed tomography (CBCT) imaging, open-source 3-dimensional (3D) modeling software, and a desktop stereolithography (SLA) 3D printer to duplicate an existing complete removable dental prosthesis (CRDP). Intended to offer a proof of concept, this proposed technique provides dental clinicians who have access to a CBCT imaging unit and a desktop SLA 3D printer an option to manufacture a duplicated prosthesis in-office...
September 2017: Compendium of Continuing Education in Dentistry
https://www.readbyqxmd.com/read/28859944/catheter-based-renal-denervation-in-patients-with-uncontrolled-hypertension-in-the-absence-of-antihypertensive-medications-spyral-htn-off-med-a-randomised-sham-controlled-proof-of-concept-trial
#12
Raymond R Townsend, Felix Mahfoud, David E Kandzari, Kazuomi Kario, Stuart Pocock, Michael A Weber, Sebastian Ewen, Konstantinos Tsioufis, Dimitrios Tousoulis, Andrew S P Sharp, Anthony F Watkinson, Roland E Schmieder, Axel Schmid, James W Choi, Cara East, Anthony Walton, Ingrid Hopper, Debbie L Cohen, Robert Wilensky, David P Lee, Adrian Ma, Chandan M Devireddy, Janice P Lea, Philipp C Lurz, Karl Fengler, Justin Davies, Neil Chapman, Sidney A Cohen, Vanessa DeBruin, Martin Fahy, Denise E Jones, Martin Rothman, Michael Böhm
BACKGROUND: Previous randomised renal denervation studies did not show consistent efficacy in reducing blood pressure. The objective of our study was to evaluate the effect of renal denervation on blood pressure in the absence of antihypertensive medications. METHODS: SPYRAL HTN-OFF MED was a multicentre, international, single-blind, randomised, sham-controlled, proof-of-concept trial. Patients were enrolled at 21 centres in the USA, Europe, Japan, and Australia...
August 25, 2017: Lancet
https://www.readbyqxmd.com/read/28853492/role-of-substance-p-and-its-receptor-neurokinin-1-in-chronic-prurigo-a-randomized-proof-of-concept-controlled-trial-with-topical-aprepitant
#13
Tatevik Ohanyan, Nicole Schoepke, Stefan Eirefelt, Gert Hoey, Witte Koopmann, Tomasz Hawro, Marcus Maurer, Martin Metz
Substance P (SP) and its receptor neurokinin 1 (NK1R) are thought to be involved in the pathogenesis of chronic prurigo. Here, we assessed SP serum levels, cutaneous NK1R expression, and the effects of topical aprepitant, an NK1R antagonist, in patients with chronic prurigo. SP and NK1R were increased, compared with controls, in the serum and in lesional vs. non-lesional skin of the patients, respectively. Aprepitant, in a randomized, placebo-controlled, split-sided, double-blind trial, reduced the intensity of pruritus as assessed by visual analogue scale by > 50% from baseline to day 28 (-35...
August 30, 2017: Acta Dermato-venereologica
https://www.readbyqxmd.com/read/28851316/a-non-controlled-single-arm-open-label-phase-ii-study-of-intravenous-and-intratumoral-administration-of-parvoryx-in-patients-with-metastatic-inoperable-pancreatic-cancer-parvoryx02-protocol
#14
Jacek Hajda, Monika Lehmann, Ottheinz Krebs, Meinhard Kieser, Karsten Geletneky, Dirk Jäger, Michael Dahm, Bernard Huber, Tilman Schöning, Oliver Sedlaczek, Albrecht Stenzinger, Niels Halama, Volker Daniel, Barbara Leuchs, Assia Angelova, Jean Rommelaere, Christine E Engeland, Christoph Springfeld, Guy Ungerechts
BACKGROUND: Metastatic pancreatic cancer has a dismal prognosis, with a mean six-month progression-free survival of approximately 50% and a median survival of about 11 months. Despite intensive research, only slight improvements of clinical outcome could be achieved over the last decades. Hence, new and innovative therapeutic strategies are urgently required. ParvOryx is a drug product containing native parvovirus H-1 (H-1PV). Since H-1PV was shown to exert pronounced anti-neoplastic effects in pre-clinical models of pancreatic cancer, the drug appears to be a promising candidate for treatment of this malignancy...
August 29, 2017: BMC Cancer
https://www.readbyqxmd.com/read/28851295/assessing-advances-in-regional-anesthesia-by-their-portrayals-in-meta-analyses-an-alternative-view-on-recent-progress
#15
Kamen V Vlassakov, Igor Kissin
BACKGROUND: The aim of this study was to delineate research reflecting advances in regional/local anesthesia where recent clinical progress was clearly defined by meta-analysis. METHODS: We conducted a search to identify all articles with meta-analyses of randomized clinical trials related to the field of regional/local anesthesia. From 279 titles, after multiple exclusions, 16 meta-analyses on important clinical practice developments with high potential for a positive conclusion on the effectiveness of the treatment were left for the assessment...
August 29, 2017: BMC Anesthesiology
https://www.readbyqxmd.com/read/28840255/-appropriate-treatment-of-acute-stroke-at-all-times-and-in-all-places-organizational-concepts-and-new-approaches
#16
REVIEW
J E Weber, H J Audebert
Stroke is one of the most common neurological diseases in acute care. The introduction of new organizational concepts in the rescue chain and in acute inpatient services can significantly reduce time to treatment and patients can receive specific therapeutic options that have been shown to improve acute stroke prognosis. This review provides an overview of organizational structures that lead to improved medical care and outlines the evidence-based therapeutic options. This is intended to give the reader a decision support on provision of specific treatment in acute ischemic stroke...
August 24, 2017: Der Internist
https://www.readbyqxmd.com/read/28837082/rna-interference-and-bmp-2-stimulation-allows-equine-chondrocytes-redifferentiation-in-3d-hypoxia-cell-culture-model-application-for-matrix-induced-autologous-chondrocyte-implantation
#17
Rodolphe Rakic, Bastien Bourdon, Magalie Hervieu, Thomas Branly, Florence Legendre, Nathalie Saulnier, Fabrice Audigié, Stéphane Maddens, Magali Demoor, Philippe Galera
As in humans, osteoarthritis (OA) causes considerable economic loss to the equine industry. New hopes for cartilage repair have emerged with the matrix-associated autologous chondrocyte implantation (MACI). Nevertheless, its limitation is due to the dedifferentiation occurring during the chondrocyte amplification phase, leading to the loss of its capacity to produce a hyaline extracellular matrix (ECM). To enhance the MACI therapy efficiency, we have developed a strategy for chondrocyte redifferentiation, and demonstrated its feasibility in the equine model...
August 24, 2017: International Journal of Molecular Sciences
https://www.readbyqxmd.com/read/28836869/renin-angiotensin-system-as-a-potential-target-for-new-therapeutic-approaches-in-parkinson-s-disease
#18
Santiago Perez-Lloret, Matilde Otero-Losada, Jorge E Toblli, Francisco Capani
Currently, available therapies for Parkinson's disease (PD) are symptomatic. Therefore, the search for neuroprotective drugs remains a top priority. Areas covered: In this review, the potential symptomatic or disease-modifying effect of drugs targeting the Renin-Angiotensin System (RAS) in PD will be explored. Expert opinion: The importance of nigrostriatal local RAS has only begun to be unraveled in the last decades. On one hand, there is a complex feedback cycle between RAS and dopamine (DA). On the other hand, RAS affects dopaminergic neurons vulnerability...
August 29, 2017: Expert Opinion on Investigational Drugs
https://www.readbyqxmd.com/read/28835536/single-cell-functional-and-chemosensitive-profiling-of-combinatorial-colorectal-therapy-in-zebrafish-xenografts
#19
Rita Fior, Vanda Póvoa, Raquel V Mendes, Tânia Carvalho, António Gomes, Nuno Figueiredo, Miguel Godinho Ferreira
Cancer is as unique as the person fighting it. With the exception of a few biomarker-driven therapies, patients go through rounds of trial-and-error approaches to find the best treatment. Using patient-derived cell lines, we show that zebrafish larvae xenotransplants constitute a fast and highly sensitive in vivo model for differential therapy response, with resolution to reveal intratumor functional cancer heterogeneity. We screened international colorectal cancer therapeutic guidelines and determined distinct functional tumor behaviors (proliferation, metastasis, and angiogenesis) and differential sensitivities to standard therapy...
August 23, 2017: Proceedings of the National Academy of Sciences of the United States of America
https://www.readbyqxmd.com/read/28835229/does-dapagliflozin-regress-left-ventricular-hypertrophy-in-patients-with-type-2-diabetes-a-prospective-double-blind-randomised-placebo-controlled-study
#20
Alexander J M Brown, Chim Lang, Rory McCrimmon, Allan Struthers
BACKGROUND: Patients with diabetes have a two to fourfold increased risk for development of and death from cardiovascular disease [CVD]. The current oral hypoglycaemic agents result in limited reduction in this cardiovascular risk. Sodium glucose linked co-transporter type 2 [SGLT2] inhibitors are a relatively new class of antidiabetic agent that have been shown to have potential cardiovascular benefits. In support of this, the EMPA-REG trial showed a striking 38% and 35% reduction in cardiovascular mortality and heart failure [HF] hospitalisation respectively...
August 23, 2017: BMC Cardiovascular Disorders
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