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adaptive clinical trials

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https://www.readbyqxmd.com/read/28821302/participation-of-adults-with-disorders-differences-of-sex-development-dsd-in-the-clinical-study-dsd-life-design-methodology-recruitment-data-quality-and-study-population
#1
Robert Röhle, Katharina Gehrmann, Maria Szarras-Czapnik, Hedi Claahsen-van der Grinten, Catherine Pienkowski, Claire Bouvattier, Peggy Cohen-Kettenis, Anna Nordenström, Ute Thyen, Birgit Köhler
BACKGROUND: dsd-LIFE is a comprehensive cross-sectional clinical outcome study of individuals with disorders/differences of sex development (DSD). This study focuses on various rare genetic conditions characterized by impaired gonadal or adrenal functionality. METHODS/DESIGN: The study aims to assess quality of life (QoL) as a measure of psychosocial adaptation, psychosexual and mental health aspects as major outcomes. Health status and functioning, medical and surgical therapies, participants' views on health care, psychological and social support, sociodemographic factors and their interrelations will be investigated as factors associated with the outcomes...
August 18, 2017: BMC Endocrine Disorders
https://www.readbyqxmd.com/read/28819075/-advances-in-neurological-therapeutics-for-friedreich-ataxia-and-machado-joseph-disease
#2
Ichiro Yabe, Hidenao Sasaki
We reviewed advances in therapeutics for both Friedreich ataxia and Machado-Joseph disease. Various clinical trials have been carried out, mainly for Friedreich ataxia; however, the therapeutic reports from these trials have not provided much evidence for success. Some interesting clinical trials have been reported, and further developments are expected. Regenerative therapy using umbilical cord mesenchymal stem cells and a therapeutic study investigating a new pathomechanism in animal and/or cell culture studies were reported...
August 2017: Brain and Nerve, Shinkei Kenkyū No Shinpo
https://www.readbyqxmd.com/read/28819024/trastuzumab-increases-her2-uptake-and-cross-presentation-by-dendritic-cells
#3
Victor A Gall, Anne V Philips, Na Qiao, Karen Clise-Dwyer, Alexander A Perakis, Mao Zhang, Guy Travis Clifton, Pariya Sukhumalchandra, Qing Ma, Sangeetha M Reddy, Dihua Yu, Jeffrey J Molldrem, George E Peoples, Gheath Alatrash, Elizabeth A Mittendorf
Early phase clinical trials evaluating CD8+ T cell-eliciting, HER2-derived peptide vaccines administered to HER2-positive breast cancer patients in the adjuvant setting suggest synergy between the vaccines and trastuzumab, the monoclonal antibody targeting the HER2 protein. Among 60 patients enrolled on clinical trials evaluating the E75+GM-CSF and GP2+GM-CSF vaccines, there have been no recurrences in patients vaccinated after receiving trastuzumab as part of standard therapy in the per treatment analyses conducted after a median follow-up of greater than 34 months...
August 17, 2017: Cancer Research
https://www.readbyqxmd.com/read/28818225/patient-centered-outcomes-of-a-dual-action-pneumatic-compression-device-in-comparison-to-compression-stockings-for-patients-with-chronic-venous-disease
#4
Fedor Lurie, Marc Schwartz
OBJECTIVE: The ACTitouch (AT) device (Tactile Medical, Minneapolis, Minn) represents a new generation of pneumatic compression devices by combining sustained compression with intermittent pneumatic compression. The sustained compression mode provides automatic pressure adjustment every 30 minutes, ensuring pressure consistency regardless of leg volume changes and environmental influences. Designed for mobile patients, this device has not been studied in comparison to standard compression stockings (CS)...
September 2017: Journal of Vascular Surgery. Venous and Lymphatic Disorders
https://www.readbyqxmd.com/read/28818060/rna-transfection-of-%C3%AE-%C3%AE-t-cells-with-a-chimeric-antigen-receptor-or-an-%C3%AE-%C3%AE-t-cell-receptor-a-safer-alternative-to-genetically-engineered-%C3%AE-%C3%AE-t-cells-for-the-immunotherapy-of-melanoma
#5
Dennis C Harrer, Bianca Simon, Shin-Ichiro Fujii, Kanako Shimizu, Ugur Uslu, Gerold Schuler, Kerstin F Gerer, Stefanie Hoyer, Jan Dörrie, Niels Schaft
BACKGROUND: Adoptive T-cell therapy relying on conventional T cells transduced with T-cell receptors (TCRs) or chimeric antigen receptors (CARs) has caused substantial tumor regression in several clinical trials. However, genetically engineered T cells have been associated with serious side-effects due to off-target toxicities and massive cytokine release. To obviate these concerns, we established a protocol adaptable to GMP to expand and transiently transfect γ/δ T cells with mRNA...
August 17, 2017: BMC Cancer
https://www.readbyqxmd.com/read/28815894/challenges-of-developing-and-conducting-clinical-trials-in-rare-disorders
#6
REVIEW
Lucas Kempf, Jonathan C Goldsmith, Robert Temple
Rare disease drug development is a rapidly expanding field. Clinical researchers in rare diseases face many challenges when conducting trials in small populations. Disease natural history is often poorly understood and the ability to detect clinically meaningful outcomes requires understanding of their rate of occurrence and variability, both of which contribute to difficulties in powering a study. Standard trial designs are not optimized to obtain adequate safety and efficacy data from small numbers of patients, so alternative designs (enrichment, crossover, adaptive, N-of 1) need to be considered...
August 16, 2017: American Journal of Medical Genetics. Part A
https://www.readbyqxmd.com/read/28815684/cognitive-psychophysiological-treatment-of-bodily-focused-repetitive-behaviors-in-adults-an-open-trial
#7
Kieron O'Connor, Marc Lavoie, Benoît Desaulniers, Jean-Sébastien Audet
BACKGROUND: Body-focused repetitive behaviors (BFRBs), such as hair pulling, skin picking, and nail biting, are repetitive, destructive, and nonfunctional habits that cause significant distress. Separate BFRBs form a cohesive group and could be assessed as part of the Tourette/tic spectrum or obsessive-compulsive spectrum of disorders. The treatment of choice is either antidepressant or behavioral treatment, both of which have shown effectiveness. The cognitive psychophysiological (CoPs) model focuses on the tension and emotional build up that triggers habits by addressing cognitive-behavioral, emotional and psychophysiological processes preceding onset rather than the habit itself...
August 16, 2017: Journal of Clinical Psychology
https://www.readbyqxmd.com/read/28815334/clinical-characteristics-of-patient-selection-and-imaging-predictors-of-outcome-in-solid-tumors-treated-with-checkpoint-inhibitors
#8
REVIEW
Sabrina Rossi, Luca Toschi, Angelo Castello, Fabio Grizzi, Luigi Mansi, Egesta Lopci
The rapidly evolving knowledge on tumor immunology and the continuous implementation of immunotherapy in cancer have recently led to the FDA and EMA approval of several checkpoint inhibitors as immunotherapic agents in clinical practice. Anti-CTLA-4, anti-PD-1, and anti-PDL-1 antibodies are becoming standard of care in advanced melanoma, as well as in relapsed or metastatic lung and kidney cancer, demonstrating higher and longer response compared to standard chemotherapy. These encouraging results have fostered the evaluation of these antibodies either alone or in combination with other therapies in several dozen clinical trials for the treatment of multiple tumor types...
August 16, 2017: European Journal of Nuclear Medicine and Molecular Imaging
https://www.readbyqxmd.com/read/28815218/capturing-the-systemic-immune-signature-of-a-norovirus-infection-an-n-of-1-case-study-within-a-clinical-trial
#9
Antony J Cutler, Joao Oliveira, Ricardo C Ferreira, Ben Challis, Neil M Walker, Sarah Caddy, Jia Lu, Helen E Stevens, Deborah J Smyth, Marcin L Pekalski, Jane Kennet, Kara M D Hunter, Ian Goodfellow, Linda S Wicker, John A Todd, Frank Waldron-Lynch
BACKGROUND:  The infection of a participant with norovirus during the adaptive study of interleukin-2 dose on regulatory T cells in type 1 diabetes (DILT1D) allowed a detailed insight into the cellular and cytokine immune responses to this prevalent gastrointestinal pathogen. METHODS:  Serial blood, serum and peripheral blood mononuclear cell (PBMC) samples were collected pre-, and post-development of the infection. To differentiate between the immune response to norovirus and to control for the administration of a single dose of aldesleukin (recombinant interleukin-2, rIL-2) alone, samples from five non-infected participants administered similar doses were analysed in parallel...
2017: Wellcome Open Research
https://www.readbyqxmd.com/read/28815145/evolving-research-data-sharing-networks-to-clinical-app-sharing-networks
#10
Kavishwar B Wagholikar, Rahul Jain, Eliel Oliveira, Joshua Mandel, Jeffery Klann, Ricardo Colas, Prasad Patil, Kuladip Yadav, Kenneth D Mandl, Thomas Carton, Shawn N Murphy
Research networks for data sharing are growing into a large platform for pragmatic clinical trials to generate quality evidence for shared medical decision-making. Institutions partnering in the networks have made large investments in developing the infrastructure for sharing data. We investigate whether institutions partnering on Patient-Centered Outcomes Research Institute's (PCORI) network can share clinical apps. At two different sites, we imported patient data in PCORI's clinical data model (CDM) format into i2b2 repositories, and adapted the SMART-on-FHIR cell to perform CDM-to-FHIR translation, serving demographics, laboratory results and diagnoses...
2017: AMIA Summits on Translational Science Proceedings
https://www.readbyqxmd.com/read/28814435/adaptive-global-innovative-learning-environment-for-glioblastoma-gbm-agile
#11
Brian M Alexander, Sujuan Ba, Mitchel S Berger, Donald A Berry, Webster K Cavenee, Susan M Chang, Timothy F Cloughesy, Tao Jiang, Mustafa Khasraw, Wenbin Li, Robert Mittman, George H Poste, Patrick Y Wen, W K Alfred Yung, Anna D Barker
Glioblastoma (GBM) is a deadly disease with few effective therapies. While much has been learned about the molecular characteristics of the disease, this knowledge has not been translated into clinical improvements for patients. At the same time, many new therapies are being developed. Many of these therapies have potential biomarkers to identify responders. The result is an enormous amount of testable clinical questions that must be answered efficiently. The GBM Adaptive Global Innovative Learning Environment (GBM AGILE) is a novel, multi-arm, platform trial designed to address these challenges...
August 16, 2017: Clinical Cancer Research: An Official Journal of the American Association for Cancer Research
https://www.readbyqxmd.com/read/28813982/validation-of-a-constrained-time-movement-task-for-use-in-rehabilitation-outcome-measures
#12
Daniel H Blustein, Jonathon W Sensinger
Current motor assessment tools can provide numerical indicators of performance but do not provide actionable information to target further improvement in rehabilitation interventions. Psychophysics-based outcome measures show promise to provide more useful information in the laboratory environment but have been limited in clinical implementation. Here we present a constrained-time task to assess paced and non-rhythmic movements. The task's output metrics include trial-by-trial adaptation rate and the just noticeable difference of a perturbation...
July 2017: IEEE ... International Conference on Rehabilitation Robotics: [proceedings]
https://www.readbyqxmd.com/read/28813943/conjunctive-rehabilitation-of-multiple-cognitive-domains-for-chronic-stroke-patients-in-virtual-reality
#13
Martina Maier, Nuria Leiva Banuelos, Belen Rubio Ballester, Esther Duarte, Paul F M J Verschure
Classically, cognitive deficits have been studied and treated in isolation from each other. A stroke patient is classified as being memory impaired, having executive dysfunction or showing attentional deficits after which a dedicated rehabilitation therapy is given. Studies seldom looked at the relationship between these different cognitive domains and syndromes, although, there is evidence that they might share common neuronal substrates and do not occur in isolation. Here, we propose a novel rehabilitation method in virtual reality to treat cognitive deficits in conjunction and report the preliminary results of an ongoing randomized controlled clinical trial...
July 2017: IEEE ... International Conference on Rehabilitation Robotics: [proceedings]
https://www.readbyqxmd.com/read/28813871/clinical-effectiveness-of-combined-virtual-reality-and-robot-assisted-fine-hand-motion-rehabilitation-in-subacute-stroke-patients
#14
Xianwei Huang, Fazel Naghdy, Golshah Naghdy, Haiping Du
Robot-assisted therapy is regarded as an effective and reliable method for the delivery of highly repetitive rehabilitation training in restoring motor skills after a stroke. This study focuses on the rehabilitation of fine hand motion skills due to their vital role in performing delicate activities of daily living (ADL) tasks. The proposed rehabilitation system combines an adaptive assist-as-needed (AAN) control algorithm and a Virtual Reality (VR) based rehabilitation gaming system (RGS). The developed system is described and its effectiveness is validated through clinical trials on a group of eight subacute stroke patients for a period of six weeks...
July 2017: IEEE ... International Conference on Rehabilitation Robotics: [proceedings]
https://www.readbyqxmd.com/read/28812250/miv-150-and-zinc-acetate-combination-provides-potent-and-broad-activity-against-hiv-1
#15
Olga Mizenina, Mayla Hsu, Ninochka Jean-Pierre, Meropi Aravantinou, Keith Levendosky, Gabriela Paglini, Thomas M Zydowsky, Melissa Robbiani, José A Fernández-Romero
We previously showed that the combination of the non-nucleoside reverse transcriptase inhibitor (NNRTI) MIV-150 with zinc acetate (ZA) formulated in a carrageenan (CG; MZC) gel provided macaques significant protection against vaginal simian-human immunodeficiency virus-RT (SHIV-RT) challenge, better than either MIV-150/CG or ZA/CG. The MZC gel was shown to be safe in a phase 1 clinical trial. Herein, we used in vitro approaches to study the antiviral properties of ZA and the MIV-150/ZA combination, compared to other NNRTIs...
August 15, 2017: Drug Delivery and Translational Research
https://www.readbyqxmd.com/read/28807656/opportunities-for-model-based-precision-dosing-in-the-treatment-of-sickle-cell-anemia
#16
Min Dong, Tomoyuki Mizuno, Alexander A Vinks
Hydroxyurea is the primary pharmacotherapy to prevent complications of sickle cell anemia (SCA). Accumulated clinical experience across multiple age ranges has suggested that the use of an individualized maximum tolerated dose (MTD) will achieve optimal benefit of hydroxyurea treatment. However, the current empirical and trial-and-error approach for dose escalation often results in a lengthy titration process and is not strictly implemented in many clinics. Opportunities exist for pharmacokinetics model-based precision dosing of hydroxyurea to quickly achieve individual MTD...
August 9, 2017: Blood Cells, Molecules & Diseases
https://www.readbyqxmd.com/read/28806908/changing-the-default-for-tobacco-cessation-treatment-in-an-inpatient-setting-study-protocol-of-a-randomized-controlled-trial
#17
Babalola Faseru, Edward F Ellerbeck, Delwyn Catley, Byron J Gajewski, Taneisha S Scheuermann, Theresa I Shireman, Laura M Mussulman, Niaman Nazir, Terry Bush, Kimber P Richter
BACKGROUND: Most health care providers do not treat tobacco dependence routinely. This may in part be due to the treatment "default." Current treatment guidelines recommend that providers (1) ask patients if they are willing to quit and (2) provide cessation-focused medications and counseling only to smokers who state that they are willing to quit. The default is that patients have to "opt in" to receive cessation assistance: providers ask smokers if they are willing to quit, and only offer medications and cessation support to those who say "yes...
August 14, 2017: Trials
https://www.readbyqxmd.com/read/28806427/fixed-dental-prostheses-and-single-tooth-crowns-based-on-ceria-stabilized-tetragonal-zirconia-alumina-nanocomposite-frameworks-outcome-after-2-years-in-a-clinical-trial
#18
Fabian Hüttig, Jan P Keitel, Andreas Prutscher, Sebastian Spintzyk, Andrea Klink
PURPOSE: This clinical trial tested bilayered restorations based on ceria-stabilized tetragonal zirconia/alumina frameworks veneered with feldspathic ceramic. MATERIALS AND METHODS: A total of 67 crowns and 40 fixed dental prostheses (FDPs) were luted in 57 patients with self-etching/self-adhesive composite resin cement. Dental status and integrity of restorations were evaluated at 2 weeks, 6 months, and then annually. RESULTS: A total of 66 crowns and 36 FDPs (88% posterior) survived for success rates of 93...
August 14, 2017: International Journal of Prosthodontics
https://www.readbyqxmd.com/read/28805255/mandibular-complete-denture-versus-single-implant-overdenture-a-systematic-review-of-patient-reported-outcomes
#19
REVIEW
Túlio Eduardo Nogueira, Danilo Rocha Dias, Cláudio Rodrigues Leles
The single-implant mandibular overdenture (SIMO) has been proposed as an alternative for edentulous patients who are poorly adapted to their dentures due to low retention and stability of the conventional mandibular complete denture (CD). However, there is a lack of evidence regarding the effectiveness of SIMO, which can be measured by examining patient perception of treatment effects. The aim of this systematic review was to assess the comparative results of CD and SIMO treatments by using patient-reported outcome measures...
August 14, 2017: Journal of Oral Rehabilitation
https://www.readbyqxmd.com/read/28804863/personalised-immunomodulating-treatments-for-graves-disease-fact-or-fiction
#20
Tristan Mirko Struja, Alexander Kutz, Stefan Fischli, Christian Meier, Beat Müller, Philipp Schütz
Although Graves' disease has been recognised for more than 100 years, its physiopathological mechanisms are incompletely understood. Treatment strategies today mainly focus on suppression of thyroid hormone production by use of antithyroid drugs or radio-iodine, but neglect the underlying immunological mechanisms. Although Graves' disease is often seen as a prototype for an autoimmune mechanism, it is more likely to be a heterogeneous syndrome showing characteristics of both autoimmunity and immunodeficiency...
August 14, 2017: Swiss Medical Weekly
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