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Proof of concept trials

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https://www.readbyqxmd.com/read/29781072/ascites-index-a-novel-technique-to-evaluate-ascites-in-ovarian-hyperstimulation-syndrome-a-concept-proof-study
#1
Piotr Robert Szkodziak, Piotr Czuczwar, Wojciech Wrona, Tomasz Paszkowski, Filip Szkodziak, Sławomir Woźniak
OBJECTIVES: Controlled ovarian hyperstimulation is an important step in infertility treatment. In some cases, however, ovar-ian hyperstimulation syndrome (OHSS) can occur. In its severe forms, ascites is likely to develop, associated with dyspnea. The aim of this study was to explore the usefulness of Ascites Index (AsI), a new tool for quantitative determination of ascites in patients with OHSS, to obtain data for planning further trials. MATERIAL AND METHODS: Twelve patients with OHSS and ascites were included in the study...
2018: Ginekologia Polska
https://www.readbyqxmd.com/read/29772028/randomized-phase-i-trial-hiv-core-003-depletion-of-serum-amyloid-p-component-and-immunogenicity-of-dna-vaccination-against-hiv-1
#2
Nicola J Borthwick, Thirusha Lane, Nathifa Moyo, Alison Crook, Jung Min Shim, Ian Baines, Edmund G Wee, Philip N Hawkins, Julian D Gillmore, Tomáš Hanke, Mark B Pepys
BACKGROUND: The failure of DNA vaccination in humans, in contrast to its efficacy in some species, is unexplained. Observational and interventional experimental evidence suggests that DNA immunogenicity may be prevented by binding of human serum amyloid P component (SAP). SAP is the single normal DNA binding protein in human plasma. The drug (R)-1-[6-[(R)-2-carboxypyrrolidin-1-yl]-6-oxo-hexanoyl]pyrrolidine-2-carboxylic acid (CPHPC, miridesap), developed for treatment of systemic amyloidosis and Alzheimer's disease, depletes circulating SAP by 95-99%...
2018: PloS One
https://www.readbyqxmd.com/read/29764889/autologous-stem-cells-in-achilles-tendinopathy-ascat-protocol-for-a-phase-iia-single-centre-proof-of-concept-study
#3
Andrew J Goldberg, Razi Zaidi, Deirdre Brooking, Louise Kim, Michelle Korda, Lorenzo Masci, Ruth Green, Paul O'Donnell, Roger Smith
INTRODUCTION: Achilles tendinopathy (AT) is a cause of pain and disability affecting both athletes and sedentary individuals. More than 150 000 people in the UK every year suffer from AT.While there is much preclinical work on the use of stem cells in tendon pathology, there is a scarcity of clinical data looking at the use of mesenchymal stem cells to treat tendon disease and there does not appear to be any studies of the use of autologous cultured mesenchymal stem cells (MSCs) for AT...
May 14, 2018: BMJ Open
https://www.readbyqxmd.com/read/29759820/clustered-regularly-interspaced-short-palindromic-repeats-based-genome-surgery-for-the-treatment-of-autosomal-dominant-retinitis-pigmentosa
#4
Yi-Ting Tsai, Wen-Hsuan Wu, Ting-Ting Lee, Wei-Pu Wu, Christine L Xu, Karen S Park, Xuan Cui, Sally Justus, Chyuan-Sheng Lin, Ruben Jauregui, Pei-Yin Su, Stephen H Tsang
PURPOSE: To develop a universal gene therapy to overcome the genetic heterogeneity in retinitis pigmentosa (RP) resulting from mutations in rhodopsin (RHO). DESIGN: Experimental study for a combination gene therapy that uses both gene ablation and gene replacement. PARTICIPANTS: This study included 2 kinds of human RHO mutation knock-in mouse models: RhoP23H and RhoD190N . In total, 23 RhoP23H/P23H , 43 RhoP23H/+ , and 31 RhoD190N/+ mice were used for analysis...
May 5, 2018: Ophthalmology
https://www.readbyqxmd.com/read/29754952/early-time-restricted-feeding-improves-insulin-sensitivity-blood-pressure-and-oxidative-stress-even-without-weight-loss-in-men-with-prediabetes
#5
Elizabeth F Sutton, Robbie Beyl, Kate S Early, William T Cefalu, Eric Ravussin, Courtney M Peterson
Intermittent fasting (IF) improves cardiometabolic health; however, it is unknown whether these effects are due solely to weight loss. We conducted the first supervised controlled feeding trial to test whether IF has benefits independent of weight loss by feeding participants enough food to maintain their weight. Our proof-of-concept study also constitutes the first trial of early time-restricted feeding (eTRF), a form of IF that involves eating early in the day to be in alignment with circadian rhythms in metabolism...
May 8, 2018: Cell Metabolism
https://www.readbyqxmd.com/read/29750119/peer-mentoring-for-eating-disorders-evaluation-of-a-pilot-program
#6
Jennifer Beveridge, Andrea Phillipou, Kelly Edwards, Alice Hobday, Krissy Hilton, Cathy Wyett, Anna Saw, Georgia Graham, David Castle, Leah Brennan, Philippa Harrison, Rebecca de Gier, Narelle Warren, Freya Hanly, Benjamin Torrens-Witherow, J Richard Newton
Background: Eating disorders are serious psychiatric illnesses that are often associated with poor quality of life and low long-term recovery rates. Peer mentor programs have been found to improve psychiatric symptoms and quality of life in other mental illnesses, and a small number of studies have suggested that eating disorder patients may benefit from such programs. The aim of this study is to assess the efficacy of a peer mentor program for individuals with eating disorders in terms of improving symptomatology and quality of life...
2018: Pilot and Feasibility Studies
https://www.readbyqxmd.com/read/29746294/a-maintenance-three-day-per-week-schedule-with-the-single-tablet-regimen-efavirenz-emtricitabine-tenofovir-disoproxil-fumarate-is-effective-and-decreases-sub-clinical-toxicity-the-a-tri-week-pilot-trial
#7
Jhon Rojas, Jose L Blanco, Sonsoles Sanchez-Palomino, Maria A Marcos, Alberto C Guardo, Ana Gonzalez-Cordon, Montserrat Lonca, Amparo Tricas, Ana Rodriguez, Anabel Romero, Jose M Miro, Josep Mallolas, Jose M Gatell, Montserrat Plana, Esteban Martinez
BACKGROUND: Antiretroviral drugs contained in single tablet Atripla® have pharmacokinetic properties that could allow for longer than once-daily (OD) dosing. We hypothesized that simplifying Atripla® OD to 3-day per week would be feasible, able to maintain viral suppression and less toxic. METHODS: Virologically suppressed (≥2 years) HIV+ adults on Atripla® OD, CD4 >350/mm at inclusion, and no prior documented virological failure or evidence of resistance mutations to efavirenz, tenofovir, or emtricitabine were randomized to maintain their OD regimen or to reduce it to 3 days (Mondays, Wednesdays, and Fridays) a week (3W)...
May 9, 2018: AIDS
https://www.readbyqxmd.com/read/29742587/exploratory-application-of-neuropharmacometabolomics-in-severe-childhood-traumatic-brain-injury
#8
Fanuel T Hagos, Philip E Empey, Pengcheng Wang, Xiaochao Ma, Samuel M Poloyac, Hülya Bayır, Patrick M Kochanek, Michael J Bell, Robert S B Clark
OBJECTIVES: To employ metabolomics-based pathway and network analyses to evaluate the cerebrospinal fluid metabolome after severe traumatic brain injury in children and the capacity of combination therapy with probenecid and N-acetylcysteine to impact glutathione-related and other pathways and networks, relative to placebo treatment. DESIGN: Analysis of cerebrospinal fluid obtained from children enrolled in an Institutional Review Board-approved, randomized, placebo-controlled trial of a combination of probenecid and N-acetylcysteine after severe traumatic brain injury (Trial Registration NCT01322009)...
May 7, 2018: Critical Care Medicine
https://www.readbyqxmd.com/read/29741988/a-dynamic-compliance-cervix-phantom-robot-for-latent-labor-simulation
#9
Michelle Jennifer Luk, Derek Lobb, James Andrew Smith
Physical simulation systems are commonly used in training of midwifery and obstetrics students, but none of these systems offers a dynamic compliance aspect that would make them more truly representative of cervix ripening. In this study, we introduce a unique soft robot phantom that simulates the cervix softening during the latent labor phase of birth. This proof-of-concept robotic phantom can be dilated by 1 cm and effaced by 35% through the application of a Foley catheter-like loading mechanism. Furthermore, psychophysics trials demonstrate how untrained subjects can identify hard and soft states of the phantom with specificities of 91% and 87%, respectively...
May 9, 2018: Soft Robotics
https://www.readbyqxmd.com/read/29741608/jak-inhibitor-improves-type-i-interferon-induced-damage-proof-of-concept-in-dermatomyositis
#10
Leandro Ladislau, Xavier Suárez-Calvet, Ségolène Toquet, Océane Landon-Cardinal, Damien Amelin, Marine Depp, Mathieu P Rodero, Denisa Hathazi, Darragh Duffy, Vincent Bondet, Corinna Preusse, Boris Bienvenu, Flore Rozenberg, Andreas Roos, Claudia F Benjamim, Eduard Gallardo, Isabel Illa, Vincent Mouly, Werner Stenzel, Gillian Butler-Browne, Olivier Benveniste, Yves Allenbach
Dermatomyositis is an acquired auto-immune disease characterized by skin lesions and muscle-specific pathological features such as perifascicular muscle fibre atrophy and vasculopathy. Dermatomyositis patients display an upregulation of type I interferon-inducible genes in muscle fibres, endothelial cells, skin and peripheral blood. However, the effect of type I interferon on muscle tissue has not yet been determined. Our aim was to study the pathogenicity of type I interferon in vitro and to evaluate the efficacy of the type I interferon pathway blockade for therapeutic purposes...
May 8, 2018: Brain: a Journal of Neurology
https://www.readbyqxmd.com/read/29739754/dexpramipexole-as-an-oral-steroid-sparing-agent-in-hypereosinophilic-syndromes
#11
Sandhya R Panch, Michael E Bozik, Thomas Brown, Michelle Makiya, Calman Prussin, Donald G Archibald, Gregory T Hebrank, Mary Sullivan, Xiaoping Sun, Lauren Wetzler, JeanAnne Ware, Michael P Fay, Cynthia E Dunbar, Steven I Dworetzky, Paneez Khoury, Irina Maric, Amy D Klion
Hypereosinophilic syndromes (HES) are a heterogeneous group of disorders characterized by peripheral eosinophilia and eosinophil-related end organ damage. Whereas most patients respond to glucocorticoid (GC) therapy, high doses are often necessary and side effects are common. Dexpramipexole (KNS 760704), an orally bioavailable synthetic amino-benzothiazole, showed an excellent safety profile and was coincidentally noted to significantly decrease absolute eosinophil counts (AEC) in a phase 3 trial for amyotrophic lateral sclerosis...
May 8, 2018: Blood
https://www.readbyqxmd.com/read/29735994/therapeutic-activity-of-retroviral-replicating-vector-mediated-prodrug-activator-gene-therapy-for-pancreatic-cancer
#12
Kazuho Inoko, Kei Hiraoka, Akihito Inagaki, Mizuna Takahashi, Toshihiro Kushibiki, Koji Hontani, Hironobu Takano, Shoki Sato, Shintaro Takeuchi, Toru Nakamura, Takahiro Tsuchikawa, Toshiaki Shichinohe, Harry E Gruber, Douglas J Jolly, Noriyuki Kasahara, Satoshi Hirano
Toca 511, a retroviral replicating vector (RRV) encoding the yeast cytosine deaminase (yCD) prodrug activator gene, which mediates conversion of the prodrug 5-fluorocytosine (5-FC) to the anticancer drug 5-fluorouracil (5-FU), is currently being evaluated in Phase II/III clinical trials for glioma, and showing highly promising evidence of therapeutic activity. Here we evaluated RRV-mediated prodrug activator gene therapy as a new therapeutic approach for pancreatic ductal adenocarcinoma (PDAC). RRV spread rapidly and conferred significant cytotoxicity with prodrug in a panel of PDAC cells...
May 8, 2018: Cancer Gene Therapy
https://www.readbyqxmd.com/read/29735474/the-detroit-young-adult-asthma-project-proposal-for-a-multicomponent-technology-intervention-for-african-american-emerging-adults-with-asthma
#13
Karen MacDonell, Sylvie Naar, Wanda Gibson-Scipio, Jean-Marie Bruzzese, Bo Wang, Aaron Brody
BACKGROUND: Racial and ethnic minority youth have poorer asthma status than white youth, even after controlling for socioeconomic variables. Proper use of asthma controller medications is critical in reducing asthma mortality and morbidity. The clinical consequences of poor asthma management include increased illness complications, excessive functional morbidity, and fatal asthma attacks. There are significant limitations in research on interventions to improve asthma management in racial minority populations, particularly minority adolescents and young adults, although illness management tends to deteriorate after adolescence during emerging adulthood, the unique developmental period beyond adolescence but before adulthood...
May 7, 2018: JMIR Research Protocols
https://www.readbyqxmd.com/read/29724899/the-dot1l-inhibitor-pinometostat-reduces-h3k79-methylation-and-has-modest-clinical-activity-in-adult-acute-leukemia
#14
Eytan M Stein, Guillermo Garcia-Manero, David A Rizzieri, Raoul Tibes, Jesus G Berdeja, Michael R Savona, Mojca Jongen-Lavrenic, Jessica K Altman, Blythe Thomson, Stephen J Blakemore, Scott R Daigle, Nigel J Waters, A Benjamin Suttle, Alicia Clawson, Roy Pollock, Andrei Krivtsov, Scott A Armstrong, Jorge DiMartino, Eric Hedrick, Bob Löwenberg, Martin S Tallman
Pinometostat (EPZ-5676) is a first-in-class, small-molecule inhibitor of the histone methyltransferase DOT1L. In this phase 1 study, pinometostat was evaluated for safety and efficacy in adult patients with advanced acute leukemias, particularly those involving MLL rearrangements ( MLL-r ) resulting from 11q23 translocations. Fifty-one patients were enrolled into 6 dose escalation cohorts (n=26) and 2 expansion cohorts (n=25) at pinometostat doses of 54 and 90 mg/m2 /day by continuous intravenous infusion in 28-day cycles...
May 3, 2018: Blood
https://www.readbyqxmd.com/read/29722454/elephants-parkinson-s-disease-and-proof-of-concept-clinical-trials
#15
Jesse M Cedarbaum
No abstract text is available yet for this article.
May 3, 2018: Movement Disorders: Official Journal of the Movement Disorder Society
https://www.readbyqxmd.com/read/29719507/breathomics-for-assessing-the-effects-of-treatment-and-withdrawal-with-inhaled-beclomethasone-formoterol-in-patients-with-copd
#16
Paolo Montuschi, Giuseppe Santini, Nadia Mores, Alessia Vignoli, Francesco Macagno, Rugia Shoreh, Leonardo Tenori, Gina Zini, Leonello Fuso, Chiara Mondino, Corrado Di Natale, Arnaldo D'Amico, Claudio Luchinat, Peter J Barnes, Tim Higenbottam
Background: Prospective pharmacological studies on breathomics profiles in COPD patients have not been previously reported. We assessed the effects of treatment and withdrawal of an extrafine inhaled corticosteroid (ICS)-long-acting β2 -agonist (LABA) fixed dose combination (FDC) using a multidimensional classification model including breathomics. Methods: A pilot, proof-of-concept, pharmacological study was undertaken in 14 COPD patients on maintenance treatment with inhaled fluticasone propionate/salmeterol (500/50 μg b...
2018: Frontiers in Pharmacology
https://www.readbyqxmd.com/read/29714582/the-near-term-viability-and-benefits-of-elabels-for-patients-clinical-sites-and-sponsors
#17
Jodi Smith-Gick, Nicola Barnes, Rocco Barone, Jeff Bedford, Jason R James, Stacy Frankovitz Reisner, Michael Stephenson
BACKGROUND: Current clinical trial labels are designed primarily to meet regulatory requirements. These labels have low patient and site utility, few are opened, and they have limited space and small fonts. As our world transitions from paper to electronic, an opportunity exists to provide patients with information about their investigational clinical trial product in a way that is more easily accessible, meets Health Authority requirements, and provides valuable additional information for the patient and caregiver...
January 1, 2018: Therapeutic Innovation & Regulatory Science
https://www.readbyqxmd.com/read/29712630/efficacy-of-a-community-based-technology-enabled-physical-activity-counseling-program-for-people-with-knee-osteoarthritis-proof-of-concept-study
#18
Linda C Li, Eric C Sayre, Hui Xie, Ryan S Falck, John R Best, Teresa Liu-Ambrose, Navi Grewal, Alison M Hoens, Greg Noonan, Lynne M Feehan
BACKGROUND: Current practice guidelines emphasize the use of physical activity as the first-line treatment of knee osteoarthritis; however, up to 90% of people with osteoarthritis are inactive. OBJECTIVE: We aimed to assess the efficacy of a technology-enabled counseling intervention for improving physical activity in people with either a physician-confirmed diagnosis of knee osteoarthritis or having passed two validated criteria for early osteoarthritis. METHODS: We conducted a proof-of-concept randomized controlled trial...
April 30, 2018: Journal of Medical Internet Research
https://www.readbyqxmd.com/read/29706501/inducible-expression-of-gdnf-in-transplanted-ipsc-derived-neural-progenitor-cells
#19
Aslam Abbasi Akhtar, Genevieve Gowing, Naomi Kobritz, Steve E Savinoff, Leslie Garcia, David Saxon, Noell Cho, Gibum Kim, Colton M Tom, Hannah Park, George Lawless, Brandon C Shelley, Virginia B Mattis, Joshua J Breunig, Clive N Svendsen
Trophic factor delivery to the brain using stem cell-derived neural progenitors is a powerful way to bypass the blood-brain barrier. Protection of diseased neurons using this technology is a promising therapy for neurodegenerative diseases. Glial cell line-derived neurotrophic factor (GDNF) has provided benefits to Parkinsonian patients and is being used in a clinical trial for amyotrophic lateral sclerosis. However, chronic trophic factor delivery prohibits dose adjustment or cessation if side effects develop...
April 23, 2018: Stem Cell Reports
https://www.readbyqxmd.com/read/29703851/efficacy-and-safety-of-faecal-microbiota-transplantation-in-patients-with-psoriatic-arthritis-protocol-for-a-6-month-double-blind-randomised-placebo-controlled-trial
#20
Maja Skov Kragsnaes, Jens Kjeldsen, Hans Christian Horn, Heidi Lausten Munk, Finn Moeller Pedersen, Hanne Marie Holt, Jens Kristian Pedersen, Dorte Kinggaard Holm, Henning Glerup, Vibeke Andersen, Ulrich Fredberg, Karsten Kristiansen, Robin Christensen, Torkell Ellingsen
INTRODUCTION: An unbalanced intestinal microbiota may mediate activation of the inflammatory pathways seen in psoriatic arthritis (PsA). A randomised, placebo-controlled trial of faecal microbiota transplantation (FMT) infused into the small intestine of patients with PsA with active peripheral disease who are non-responsive to methotrexate (MTX) treatment will be conducted. The objective is to explore clinical aspects associated with FMT performed in patients with PsA. METHODS AND ANALYSIS: This trial is a randomised, two-centre stratified, double-blind (patient, care provider and outcome assessor), placebo-controlled, parallel-group study...
April 27, 2018: BMJ Open
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