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Proof of concept clinical trial

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https://www.readbyqxmd.com/read/28088167/randomized-double-blind-placebo-controlled-clinical-trial-to-assess-the-safety-and-effectiveness-of-a-novel-dual-action-oral-topical-formulation-against-upper-respiratory-infections
#1
Pranab K Mukherjee, Frank Esper, Ken Buchheit, Karen Arters, Ina Adkins, Mahmoud A Ghannoum, Robert A Salata
BACKGROUND: Current prevention options for upper respiratory infections (URIs) are not optimal. We conducted a randomized, double-blinded, placebo-controlled pilot clinical trial to evaluate the safety and efficacy of ARMS-I™ (currently marketed as Halo™) in the prevention of URIs. METHODS: ARMS-I is patented novel formulation for the prevention and treatment of influenza, comprising a broad-spectrum antimicrobial agent (cetylpyridinium chloride, CPC) and components (glycerin and xanthan gum) that form a barrier on the host mucosa, thus preventing viral contact and invasion...
January 14, 2017: BMC Infectious Diseases
https://www.readbyqxmd.com/read/28078750/metabolic-adaptation-of-colonic-microbiota-to-galactooligosaccharides-a-proof-of-concept-study
#2
M Mego, C Manichanh, A Accarino, D Campos, M Pozuelo, E Varela, J Vulevic, G Tzortzis, G Gibson, F Guarner, F Azpiroz
BACKGROUND: Prebiotics have been shown to reduce abdominal symptoms in patients with functional gut disorders, despite that they are fermented by colonic bacteria and may induce gas-related symptoms. AIM: To investigate changes in the metabolic activity of gut microbiota induced by a recognised prebiotic. METHODS: Healthy subjects (n = 20) were given a prebiotic (2.8 g/day HOST-G904, HOST Therabiomics, Jersey, Channel Islands) for 3 weeks...
January 12, 2017: Alimentary Pharmacology & Therapeutics
https://www.readbyqxmd.com/read/28078079/looking-forward-to-new-targeted-treatments-for-chronic-spontaneous-urticaria
#3
REVIEW
Emek Kocatürk, Marcus Maurer, Martin Metz, Clive Grattan
The introduction of omalizumab to the management of chronic spontaneous urticaria (CSU) has markedly improved the therapeutic possibilities for both, patients and physicians dealing with this disabling disease. But there is still a hard core of patients who do not tolerate or benefit from existing therapies and who require effective treatment. Novel approaches include the use of currently available drugs off-licence, investigational drugs currently undergoing clinical trials and exploring the potential for therapies directed at pathophysiological targets in CSU...
2017: Clinical and Translational Allergy
https://www.readbyqxmd.com/read/28075482/human-induced-pluripotent-stem-hips-cells-from-urine-samples-a-non-integrative-and-feeder-free-reprogramming-strategy
#4
Clara Steichen, Karim Si-Tayeb, Fanny Wulkan, Thayane Crestani, Graça Rosas, Rafael Dariolli, Alexandre C Pereira, Jose E Krieger
Human induced pluripotent stem (hiPS) cell technology has already revolutionized some aspects of fundamental and applied research such as study of disease mechanisms and pharmacology screening. The first clinical trial using hiPS cell-derived cells began in Japan, only 10 years after the publication of the proof-of concept article. In this exciting context, strategies to generate hiPS cells have evolved quickly, tending towards non-invasive protocols to sample somatic cells combined with "safer" reprogramming strategies...
January 11, 2017: Current Protocols in Human Genetics
https://www.readbyqxmd.com/read/28062705/novel-p21-activated-kinase-4-pak4-allosteric-modulators-overcome-drug-resistance-and-stemness-in-pancreatic-ductal-adenocarcinoma
#5
Amro Aboukameel, Irfana Muqbil, William Senapedis, Erkan Baloglu, Yosef Landesman, Sharon Shacham, Michael Kauffman, Philip A Philip, Ramzi M Mohammad, Asfar S Azmi
The p21-activated kinase 4 (PAK4) is a key downstream effector of the Rho family GTPases and is found to be overexpressed in pancreatic ductal adenocarcinoma (PDAC) cells but not in normal human pancreatic ductal epithelia (HPDE). Gene copy number amplification studies in PDAC patient cohorts confirmed PAK4 amplification making it an attractive therapeutic target in PDAC. We investigated the antitumor activity of novel PAK4 allosteric modulators (PAM) on a panel of PDAC cell lines and chemotherapy-resistant flow-sorted PDAC cancer stem cells (CSC)...
January 2017: Molecular Cancer Therapeutics
https://www.readbyqxmd.com/read/28060186/acupuncture-for-pain-in-chronic-pancreatitis-a-single-blinded-randomized-crossover-trial
#6
Jacob Juel, Stefano Liguori, Aldo Liguori, Jakob L Poulsen, Massimiliano Valeriani, Carina Graversen, Søren S Olesen, Asbjørn M Drewes
OBJECTIVES: Many patients with painful chronic pancreatitis (CP) have insufficient effect of treatment, and the prevalence of adverse effects is high. Consequently, alternatives to conventional management are needed. We aimed to study the effect of acupuncture in painful CP. METHODS: This was a prospective, single-blinded, randomized crossover trial. Fifteen patients with CP were assigned to a session of acupuncture followed by sham stimulation or vice versa. Patients rated clinical pain scores daily on a 0 to 10 visual analogue scale (VAS) and completed the Patient Global Impression of Change...
February 2017: Pancreas
https://www.readbyqxmd.com/read/28046370/th-cd-202-09-free-breathing-proton-mri-functional-lung-avoidance-maps-to-guide-radiation-therapy
#7
D Capaldi, K Sheikh, D Hoover, B Yaremko, D Palma, G Parraga
PURPOSE: Pulmonary functional MRI using inhaled gas contrast agents was previously investigated as a way to identify well-functioning lung in patients with NSCLC who are clinical candidates for radiotherapy. Hyperpolarized noble-gas ((3) He and (129) Xe) MRI has also been optimized to measure functional lung information, but for a number of reasons, the clinical translation of this approach to guide radiotherapy planning has been limited. As an alternative, free-breathing pulmonary 1H MRI using clinically available MRI systems and pulse sequences provides a non-contrast-enhanced method to generate both ventilation and perfusion maps...
June 2016: Medical Physics
https://www.readbyqxmd.com/read/28041817/combined-diabetes-renal-multifactorial-intervention-in-patients-with-advanced-diabetic-nephropathy-proof-of-concept
#8
Leon Fogelfeld, Peter Hart, Jadwiga Miernik, Jocelyn Ko, Donna Calvin, Bettina Tahsin, Anwar Adhami, Rajeev Mehrotra, Louis Fogg
AIMS: To evaluate efficacy of a multifactorial-multidisciplinary approach in delaying CKD 3-4 progression to ESRD. METHODS: Two-year proof-of-concept stratified randomized control trial conducted in an outpatient clinic of a large public hospital system. This intervention, led by a team of endocrinologists, nephrologists, nurse practitioners, and registered dietitians, integrated intensive diabetes-renal care with behavioral/dietary and pharmacological interventions...
December 8, 2016: Journal of Diabetes and its Complications
https://www.readbyqxmd.com/read/28032901/structure-of-proof-of-concept-studies-that-precede-a-nonalcoholic-steatohepatitis-development-program
#9
R N Williams, C Filozof, B J Goldstein, K Cusi
Surrogate endpoints for clinical proof of concept (POC) trials in nonalcoholic steatohepatitis (NASH) are based upon expert pathological review of liver biopsies. During early development, these long-term POC studies (≥48 weeks) add cost and time to the "Go/No Go" decision process. However, it is possible to conduct short-term noninvasive POC studies utilizing biomarkers and magnetic resonance imaging. Here, we discuss the use of shorter noninvasive POC studies relative to biopsy-driven studies for drug development in NASH...
November 7, 2016: Clinical Pharmacology and Therapeutics
https://www.readbyqxmd.com/read/28032481/safety-tolerability-and-antihypertensive-effect-of-ser100-an-opiate-receptor-like-1-orl-1-partial-agonist-in-patients-with-isolated-systolic-hypertension
#10
Ilkka Kantola, Mika Scheinin, Trygve Gulbrandsen, Nils Meland, Knut T Smerud
The purpose of the present trial was to evaluate safety, tolerability, and effect on systolic blood pressure (SBP) of SER100 in a small group of patients with isolated systolic hypertension (ISH) in treatment with at least 1 antihypertensive drug. Eligible patients were randomized to either SER100 (10 mg) or placebo in a crossover design, and 2 doses were given subcutaneously (SC), 8 hours apart, on 2 consecutive days. On all treatment days patients were monitored with an ambulatory blood pressure measurement device for 12 daytime hours...
December 29, 2016: Clinical Pharmacology in Drug Development
https://www.readbyqxmd.com/read/28026860/pioglitazone-together-with-imatinib-in-chronic-myeloid-leukemia-a-proof-of-concept-study
#11
Philippe Rousselot, Stéphane Prost, Joelle Guilhot, Lydia Roy, Gabriel Etienne, Laurence Legros, Aude Charbonnier, Valérie Coiteux, Pascale Cony-Makhoul, Francoise Huguet, Emilie Cayssials, Jean-Michel Cayuela, Francis Relouzat, Marc Delord, Heriberto Bruzzoni-Giovanelli, Laure Morisset, François-Xavier Mahon, François Guilhot, Philippe Leboulch
BACKGROUND: We recently reported that peroxisome proliferator-activated receptor γ agonists target chronic myeloid leukemia (CML) quiescent stem cells in vitro by decreasing transcription of STAT5. Here in the ACTIM phase 2 clinical trial, we asked whether pioglitazone add-on therapy to imatinib would impact CML residual disease, as assessed by BCR-ABL1 transcript quantification. METHODS: CML patients were eligible if treated with imatinib for at least 2 years at a stable daily dose, having yielded major molecular response (MMR) but not having achieved molecular response 4...
December 27, 2016: Cancer
https://www.readbyqxmd.com/read/28010129/pharmaceutical-development-of-an-amorphous-solid-dispersion-formulation-of-elacridar-hydrochloride-for-proof-of-concept-clinical-studies
#12
E Sawicki, J H M Schellens, J H Beijnen, B Nuijen
OBJECTIVE: A novel tablet formulation containing an amorphous solid dispersion (ASD) of elacridar hydrochloride was developed with the purpose to resolve the drug's low solubility in water and to conduct proof-of-concept clinical studies. SIGNIFICANCE: Elacridar is highly demanded for proof-of-concept clinical trials that study the drug's suitability to boost brain penetration and bioavailability of numerous anticancer agents. Previously, clinical trials with elacridar were performed with a tablet containing elacridar hydrochloride...
December 23, 2016: Drug Development and Industrial Pharmacy
https://www.readbyqxmd.com/read/28009725/cognitive-decline-and-dementia-in-down-syndrome
#13
Rosalyn Hithersay, Sarah Hamburg, Bernice Knight, André Strydom
PURPOSE OF REVIEW: Alzheimer's disease is most likely universal in older individuals with Down syndrome, due to having three copies of the amyloid precursor protein gene, resulting in amyloid-beta plaque deposition. Down syndrome is an important population in which to consider clinical trials of treatments to prevent or delay the development of dementia. However, assessment of subtler cognitive changes is challenging due to the presence of intellectual disability. RECENT FINDINGS: Recent research confirmed that older adults with Down syndrome often present with cognitive decline: more than 80% may experience dementia by age 65 years...
December 21, 2016: Current Opinion in Psychiatry
https://www.readbyqxmd.com/read/27998961/do-all-patients-with-advanced-her2-positive-breast-cancer-need-upfront-chemo-when-receiving-trastuzumab-randomized-phase-iii-trial-sakk-22-99
#14
O Pagani, D Klingbiel, T Ruhstaller, F Nolè, S Eppenberger, C Oehlschlegel, J Bernhard, P Brauchli, D Hess, C Mamot, E Munzone, B Pestalozzi, M Rabaglio, S Aebi, K Ribi, C Rochlitz, K Rothgiesser, B Thürlimann, R von Moos, K Zaman, A Goldhirsch
BackgroundHER2-targeted therapy plus chemotherapy is standard treatment in advanced HER2+ breast cancer. Trastuzumab alone followed by addition of chemotherapy at disease progression versus upfront combination therapy has not been elucidated.Patients and methodsOne-hundred-seventy-five patients with measurable/evaluable HER2+ advanced disease without previous HER2-directed therapy were randomized to trastuzumab alone followed, at disease progression, by the combination with chemotherapy (Arm A) or upfront trastuzumab plus chemotherapy (Arm B)...
December 19, 2016: Annals of Oncology: Official Journal of the European Society for Medical Oncology
https://www.readbyqxmd.com/read/27997307/caervest-%C3%A2-a-novel-endothermic-hypothermic-device-for-core-temperature-cooling-safety-and-efficacy-testing
#15
Ashley G B Willmott, Alex Bliss, William H Simpson, Steve M Tocker, Rowland Cottingham, Neil S Maxwell
INTRODUCTION: Cooling of the body is used to treat hyperthermic individuals with heatstroke or to depress core temperature below normal for neuroprotection. A novel chemically activated, unpowered cooling device, CAERvest(®), was investigated for safety and efficacy, with the feedback used to inform device development. METHODS: Eight healthy male participants (body mass 79.9±1.9kg and body fat percentage 16.1±3.8%) visited the laboratory (20°C, 40% RH) on four occasions...
December 20, 2016: International Journal of Occupational Safety and Ergonomics: JOSE
https://www.readbyqxmd.com/read/27980779/distinct-immunological-activation-profiles-of-dslim%C3%A2-and-promune%C3%A2-depend-on-their-different-structural-context
#16
Kerstin Kapp, Jacqueline Schneider, Lisa Schneider, Nadine Gollinge, Stefanie Jänsch, Matthias Schroff, Burghardt Wittig, Christiane Kleuss
INTRODUCTION: DNA-based TLR9 agonists are potent activators of the immune system. ProMune® and dSLIM® belong to different families of TLR9 agonists and both have been established as cancer immunotherapeutics in clinical proof-of-concept studies. Unfortunately, ProMune® failed in pivotal oncological trials. dSLIM®, the active ingredient of Lefitolimod (MGN1703), successfully finished a double-blinded, placebo-controlled phase II study in patients with advanced colorectal cancer, exhibiting improved progression-free survival and durable disease control...
December 2016: Immunity, Inflammation and Disease
https://www.readbyqxmd.com/read/27979834/ciclosporin-to-protect-renal-function-in-cardiac-surgery-ciprics-a-study-protocol-for-a-double-blind-randomised-placebo-controlled-proof-of-concept-study
#17
Per Ederoth, Edgars Grins, Alain Dardashti, Björn Brondén, Carsten Metzsch, André Erdling, Shahab Nozohoor, Arash Mokhtari, Magnus J Hansson, Eskil Elmér, Lars Algotsson, Stefan Jovinge, Henrik Bjursten
INTRODUCTION: Acute kidney injury (AKI) after cardiac surgery is common and results in increased morbidity and mortality. One possible mechanism for AKI is ischaemia-reperfusion injury caused by the extracorporeal circulation (ECC), resulting in an opening of the mitochondrial permeability transition pore (mPTP) in the kidneys, which can lead to cell injury or cell death. Ciclosporin may block the opening of mPTP if administered before the ischaemia-reperfusion injury. We hypothesised that ciclosporin given before the start of ECC in cardiac surgery can decrease the degree of AKI...
December 15, 2016: BMJ Open
https://www.readbyqxmd.com/read/27965880/lifestyle-change-in-the-cancer-setting-using-the-teachable-moment-protocol-for-a-proof-of-concept-pilot-in-a-urology-service
#18
Alyssa Sara Lee, Gozde Ozakinci, Steve Leung, Gerry Humphris, Hannah Dale, Neil Hamlet
BACKGROUND: Previous research has shown diagnosis or screening for cancer may be a 'teachable moment' for prevention through lifestyle change. Previous trials have been successful but have been delivered via national programmes targeting patients being screened for colorectal cancer. This manuscript reports the protocol for a proof-of-concept study to assess the feasibility and acceptability of a lifestyle change service targeting men suspected or diagnosed with cancer of the prostate in a secondary care cancer service within the UK...
2016: Pilot and Feasibility Studies
https://www.readbyqxmd.com/read/27965597/transcranial-direct-current-stimulation-effects-on-athletes-cognitive-performance-an-exploratory-proof-of-concept-trial
#19
Davimar M M Borducchi, July Silveira Gomes, Henrique Akiba, Quirino Cordeiro, José Henrique M Borducchi, Lívia Stocco Sanches Valentin, Gabrielle M Borducchi, Álvaro Machado Dias
Among the 2016 Olympic and Paralympic Games' unforgettable moments, one could not overlook performances by Phelps and Bolt, which challenge old premises about the maximum extension of individual supremacism in ultracompetitive modalities and the doping scandals. Different media channels resonated these two trends, with an unseen rise on discussions about traits and practices that may set ultrahigh performance athletes apart from the more ordinary ones. Yet, some key issues remain undebated. This paper aims to add to this debate, with a proof of concept trial, which investigates whether transcranial direct current stimulation (tDCS) may serve as an aid for professional athletes...
2016: Frontiers in Psychiatry
https://www.readbyqxmd.com/read/27956060/plasmodium-vivax-controlled-human-malaria-infection-progress-and-prospects
#20
REVIEW
Ruth O Payne, Paul M Griffin, James S McCarthy, Simon J Draper
Modern controlled human malaria infection (CHMI) clinical trials have almost entirely focussed on Plasmodium falciparum, providing a highly informative means to investigate host-pathogen interactions as well as assess potential new prophylactic and therapeutic interventions. However, in recent years, there has been renewed interest in Plasmodium vivax, with CHMI models developed by groups in Colombia, the USA, and Australia. This review summarizes the published experiences, and examines the advantages and disadvantages of the different models that initiate infection either by mosquito bite or using a blood-stage inoculum...
December 9, 2016: Trends in Parasitology
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