keyword
MENU ▼
Read by QxMD icon Read
search

Proof of concept clinical trial

keyword
https://www.readbyqxmd.com/read/28901946/design-of-semg-assembly-to-detect-external-anal-sphincter-activity-a-proof-of-concept
#1
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/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
#2
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
#3
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
#4
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
#5
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/28862999/innovation-in-microbiome-based-strategies-for-promoting-metabolic-health
#6
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
#7
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/28853492/role-of-substance-p-and-its-receptor-neurokinin-1-in-chronic-prurigo-a-randomized-proof-of-concept-controlled-trial-with-topical-aprepitant
#8
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
#9
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
#10
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/28837082/rna-interference-and-bmp-2-stimulation-allows-equine-chondrocytes-redifferentiation-in-3d-hypoxia-cell-culture-model-application-for-matrix-induced-autologous-chondrocyte-implantation
#11
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
#12
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/28835229/does-dapagliflozin-regress-left-ventricular-hypertrophy-in-patients-with-type-2-diabetes-a-prospective-double-blind-randomised-placebo-controlled-study
#13
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
https://www.readbyqxmd.com/read/28830912/mapping-genetic-vulnerabilities-reveals-btk-as-a-novel-therapeutic-target-in-oesophageal-cancer
#14
Irene Yushing Chong, Lauren Aronson, Hanna Bryant, Aditi Gulati, James Campbell, Richard Elliott, Stephen Pettitt, Paul Wilkerson, Maryou B Lambros, Jorge S Reis-Filho, Anisha Ramessur, Michael Davidson, Ian Chau, David Cunningham, Alan Ashworth, Christopher J Lord
OBJECTIVE: Oesophageal cancer is the seventh most common cause of cancer-related death worldwide. Disease relapse is frequent and treatment options are limited. DESIGN: To identify new biomarker-defined therapeutic approaches for patients with oesophageal cancer, we integrated the genomic profiles of 17 oesophageal tumour-derived cell lines with drug sensitivity data from small molecule inhibitor profiling, identifying drug sensitivity effects associated with cancer driver gene alterations...
August 22, 2017: Gut
https://www.readbyqxmd.com/read/28822719/thalidomide-is-associated-with-increased-t-cell-activation-and-inflammation-in-antiretroviral-naive-hiv-infected-individuals-in-a-randomised-clinical-trial-of-efficacy-and-safety
#15
Tânia R C Vergara, Sadia Samer, Joanna R Santos-Oliveira, Leila B Giron, Muhammad Shoaib Arif, Maria Luciana Silva-Freitas, Lia A Cherman, Mauro S Treitsman, Alberto Chebabo, Maria Cecilia A Sucupira, Alda M Da-Cruz, Ricardo Sobhie Diaz
TRIAL DESIGN: Open-label, randomised, controlled, pilot proof-of-concept clinical trial. METHODS: Participants: Antiretroviral naïve adult males with CD4 count ≥350cells/mm(3). INTERVENTIONS: Patients were randomised to receive thalidomide 200mg QD for 3weeks (Thalidomide group) or not (Control group) and followed for 48weeks. OBJECTIVE: We hypothesized that short-term Thalidomide use would reduce HIV related inflammation and HIV replication among antiretroviral naïve HIV infected individuals...
August 12, 2017: EBioMedicine
https://www.readbyqxmd.com/read/28819076/-molecularly-targeted-therapy-of-spinocerebellar-ataxia-type-1-by-hmgb1
#16
Kyota Fujita, Hitoshi Okazawa
Spinocerebellar ataxia type 1 (SCA1) is an untreatable neurodegenerative disease. We reported a decrease in HMGB1 levels in the nucleus of cerebellar neurons in mouse SCA 1. The decrease in this DNA architectural protein leads to the impairment of DNA repair and transcription, the two essential nuclear functions, and eventually causes neurodegeneration. We have designed a gene therapy using AAV-HMGB1 and tested it using the mouse model. Based on the results of these proof of concept (POC) studies, we are now preparing GMP-level AAV vector and designing human clinical trials...
August 2017: Brain and Nerve, Shinkei Kenkyū No Shinpo
https://www.readbyqxmd.com/read/28818518/seladelpar-mbx-8025-a-selective-ppar-%C3%AE-agonist-in-patients-with-primary-biliary-cholangitis-with-an-inadequate-response-to-ursodeoxycholic-acid-a-double-blind-randomised-placebo-controlled-phase-2-proof-of-concept-study
#17
David Jones, Pol F Boudes, Mark G Swain, Christopher L Bowlus, Michael R Galambos, Bruce R Bacon, Yvonne Doerffel, Norman Gitlin, Stuart C Gordon, Joseph A Odin, David Sheridan, Markus-Alexander Wörns, Virginia Clark, Linsey Corless, Heinz Hartmann, Mark E Jonas, Andreas E Kremer, George F Mells, Peter Buggisch, Bradley L Freilich, Cynthia Levy, John M Vierling, David E Bernstein, Marek Hartleb, Ewa Janczewska, Fedja Rochling, Hemant Shah, Mitchell L Shiffman, John H Smith, Yun-Jung Choi, Alexandra Steinberg, Monika Varga, Harinder Chera, Robert Martin, Charles A McWherter, Gideon M Hirschfield
BACKGROUND: Many patients with primary biliary cholangitis have an inadequate response to first-line therapy with ursodeoxycholic acid. Seladelpar is a potent, selective agonist for the peroxisome proliferator-activated receptor-delta (PPAR-δ), which is implicated in bile acid homoeostasis. This first-in-class study evaluated the anti-cholestatic effects and safety of seladelpar in patients with an inadequate response to ursodeoxycholic acid. METHODS: The study was a 12-week, double-blind, placebo-controlled, phase 2 trial of patients with alkaline phosphatase of at least 1·67 times the upper limit of normal (ULN) despite treatment with ursodeoxycholic acid...
August 14, 2017: Lancet. Gastroenterology & Hepatology
https://www.readbyqxmd.com/read/28801373/metformin-and-melatonin-in-adrenocortical-carcinoma-morphoproteomics-and-biomedical-analytics-provide-proof-of-concept-in-a-case-study
#18
Robert E Brown, Jamie Buryanek, Mary F McGuire
Metformin has been proposed as a novel anti-cancer drug for adrenocortical carcinoma (ACC) based upon Poli's recent preclinical studies that 1. "in vitro" metformin modulates the ACC cell model H295R and 2. "in vivo" metformin inhibits tumor growth in a xenograft model as confirmed by a significant reduction of Ki67 [1]. Here we report on our prior clinical case study that provides proof of concept for Poli's studies. We were requested to perform morphoproteomic analysis to further define the biology of, and raise targeted therapeutic options, for a case of post-treatment and chemoresistant ACC metastatic to the liver and the lung...
August 2017: Annals of Clinical and Laboratory Science
https://www.readbyqxmd.com/read/28798986/a-phase-ii-multicentre-open-label-proof-of-concept-study-of-tasquinimod-in-hepatocellular-ovarian-renal-cell-and-gastric-cancers
#19
Bernard Escudier, Sandrine Faivre, Eric Van Cutsem, Nathalie Germann, Jean-Christophe Pouget, Ruth Plummer, Ignace Vergote, Fiona Thistlethwaite, Georg A Bjarnason, Robert Jones, Helen Mackay, Julien Edeline, Laetitia Fartoux, Hal Hirte, Amit Oza
BACKGROUND: Tasquinimod is a small molecule with immunomodulatory, anti-angiogenic, and anti-metastatic properties that targets the tumor microenvironment. This study aimed to obtain a clinical proof of concept that tasquinimod was active and tolerable in patients with advanced solid tumors. PATIENTS AND METHODS: This early stopping design, open-label, proof-of-concept clinical trial evaluated the clinical activity of tasquinimod in four independent cohorts of patients with advanced hepatocellular (n = 53), ovarian (n = 55), renal cell (n = 38), and gastric (n = 21) cancers...
August 10, 2017: Targeted Oncology
https://www.readbyqxmd.com/read/28797473/biomarker-research-to-improve-clinical-outcomes-of-peritoneal-dialysis-consensus-of-the-european-training-and-research-in-peritoneal-dialysis-eutripd-network
#20
REVIEW
Christoph Aufricht, Robert Beelen, Matthias Eberl, Michel Fischbach, Donald Fraser, Achim Jörres, Klaus Kratochwill, Manuel LópezCabrera, Peter Rutherford, Claus-Peter Schmitt, Nicholas Topley, Janusz Witowski
Peritoneal dialysis (PD) therapy substantially requires biomarkers as tools to identify patients who are at the highest risk for PD-related complications and to guide personalized interventions that may improve clinical outcome in the individual patient. In this consensus article, members of the European Training and Research in Peritoneal Dialysis Network (EuTRiPD) review the current status of biomarker research in PD and suggest a selection of biomarkers that can be relevant to the care of PD patients and that are directly accessible in PD effluents...
August 8, 2017: Kidney International
keyword
keyword
85926
1
2
Fetch more papers »
Fetching more papers... Fetching...
Read by QxMD. Sign in or create an account to discover new knowledge that matter to you.
Remove bar
Read by QxMD icon Read
×

Search Tips

Use Boolean operators: AND/OR

diabetic AND foot
diabetes OR diabetic

Exclude a word using the 'minus' sign

Virchow -triad

Use Parentheses

water AND (cup OR glass)

Add an asterisk (*) at end of a word to include word stems

Neuro* will search for Neurology, Neuroscientist, Neurological, and so on

Use quotes to search for an exact phrase

"primary prevention of cancer"
(heart or cardiac or cardio*) AND arrest -"American Heart Association"