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adaptive clinical trials

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https://www.readbyqxmd.com/read/28346927/microbiota-in-functional-gastrointestinal-disorders-in-infancy-implications-for-management
#1
Thomas R Abrahamsson, Richard Y Wu, Philip M Sherman
The complex and diverse intestinal microbiome is recognized as important in promoting human health. An altered gut microflora, referred to as dysbiosis, is increasingly recognized as having an etiologic role in a variety of conditions, including functional gastrointestinal disorders: colic in infants and irritable bowel syndrome in older children. Probiotics are defined as live microorganisms that, if ingested in sufficient amounts, restore microbial homeostasis and have a benefit on health. Randomized controlled trials indicate that probiotics can be effective in a variety of intestinal conditions, including colic and irritable bowel syndrome...
2017: Nestlé Nutrition Institute Workshop Series
https://www.readbyqxmd.com/read/28346823/design-and-monitoring-of-multi-arm-multi-stage-clinical-trials
#2
Pranab Ghosh, Lingyun Liu, P Senchaudhuri, Ping Gao, Cyrus Mehta
Two-arm group sequential designs have been widely used for over 40 years, especially for studies with mortality endpoints. The natural generalization of such designs to trials with multiple treatment arms and a common control (MAMS designs) has, however, been implemented rarely. While the statistical methodology for this extension is clear, the main limitation has been an efficient way to perform the computations. Past efforts were hampered by algorithms that were computationally explosive. With the increasing interest in adaptive designs, platform designs, and other innovative designs that involve multiple comparisons over multiple stages, the importance of MAMS designs is growing rapidly...
March 27, 2017: Biometrics
https://www.readbyqxmd.com/read/28345263/nmr-solution-structure-of-the-red-subdomain-of-the-sleeping-beauty-transposase
#3
Tatiana A Konnova, Christopher M Singer, Irina V Nesmelova
DNA transposons can be employed for stable gene transfer in vertebrates. The Sleeping Beauty (SB) DNA transposon has been recently adapted for human application and is being evaluated in clinical trials, however its molecular mechanism is not clear. SB transposition is catalyzed by the transposase enzyme, which is a multi-domain protein containing the catalytic and the DNA-binding domains. The DNA-binding domain of SB transposase contains two structurally independent subdomains, PAI and RED. Recently, the structures of the catalytic domain and the PAI subdomain have been determined, however no structural information on the RED subdomain and its interactions with DNA has been available...
March 27, 2017: Protein Science: a Publication of the Protein Society
https://www.readbyqxmd.com/read/28344643/goliah-gaming-open-library-for-intervention-in-autism-at-home-a-6-month-single-blind-matched-controlled-exploratory-study
#4
Anne-Lise Jouen, Antonio Narzisi, Jean Xavier, Elodie Tilmont, Nicolas Bodeau, Valentina Bono, Nabila Ketem-Premel, Salvatore Anzalone, Koushik Maharatna, Mohamed Chetouani, Filippo Muratori, David Cohen
BACKGROUND: To meet the required hours of intensive intervention for treating children with autism spectrum disorder (ASD), we developed an automated serious gaming platform (11 games) to deliver intervention at home (GOLIAH) by mapping the imitation and joint attention (JA) subset of age-adapted stimuli from the Early Start Denver Model (ESDM) intervention. Here, we report the results of a 6-month matched controlled exploratory study. METHODS: From two specialized clinics, we included 14 children (age range 5-8 years) with ASD and 10 controls matched for gender, age, sites, and treatment as usual (TAU)...
2017: Child and Adolescent Psychiatry and Mental Health
https://www.readbyqxmd.com/read/28342990/telephone-adapted-mindfulness-based-stress-reduction-tmbsr-for-patients-awaiting-kidney-transplantation
#5
Cynthia R Gross, Maryanne Reilly-Spong, Taehwan Park, Ruizhi Zhao, Olga V Gurvich, Hassan N Ibrahim
BACKGROUND: Patients with progressive kidney disease experience increasing physiologic and psychosocial stressors and declining health-related quality of life (HRQOL). METHODS: We conducted a randomized, active-controlled, open-label trial to test whether a Mindfulness-based Stress Reduction (MBSR) program delivered in a novel workshop-teleconference format would reduce symptoms and improve HRQOL in patients awaiting kidney transplantation. Sixty-three transplant candidates were randomized to one of two arms: i) telephone-adapted MBSR (tMBSR, an 8-week program of meditation and yoga); or ii) a telephone-based support group (tSupport)...
March 22, 2017: Contemporary Clinical Trials
https://www.readbyqxmd.com/read/28342083/classification-of-advanced-stages-of-parkinson-s-disease-translation-into-stratified-treatments
#6
REVIEW
Rejko Krüger, Jochen Klucken, Daniel Weiss, Lars Tönges, Pierre Kolber, Stefan Unterecker, Michael Lorrain, Horst Baas, Thomas Müller, Peter Riederer
Advanced stages of Parkinson's disease (advPD) still impose a challenge in terms of classification and related stage-adapted treatment recommendations. Previous concepts that define advPD by certain milestones of motor disability apparently fall short in addressing the increasingly recognized complexity of motor and non-motor symptoms and do not allow to account for the clinical heterogeneity that require more personalized approaches. Therefore, deep phenotyping approaches are required to characterize the broad-scaled, continuous and multidimensional spectrum of disease-related motor and non-motor symptoms and their progression under real-life conditions...
March 24, 2017: Journal of Neural Transmission
https://www.readbyqxmd.com/read/28341761/cardiac-safety-of-dual-anti-her2-therapy-in-the-neoadjuvant-setting-for-treatment-of-her2-positive-breast-cancer
#7
Anthony F Yu, Jasmeet C Singh, Rui Wang, Jennifer E Liu, Anne Eaton, Kevin C Oeffinger, Richard M Steingart, Clifford A Hudis, Chau T Dang
BACKGROUND: Trastuzumab and pertuzumab are approved for the neoadjuvant treatment of human epidermal growth receptor 2 (HER2)-positive breast cancer, but cardiac safety data is limited. We report the cardiac safety of dose-dense doxorubicin and cyclophosphamide (AC) followed by paclitaxel, trastuzumab, and pertuzumab (THP) in the neoadjuvant setting followed by adjuvant trastuzumab-based therapy. METHODS: Fifty-seven patients treated with neoadjuvant dose-dense AC-THP followed by adjuvant trastuzumab-based therapy between September 1, 2013, and March 1, 2015, were identified...
March 24, 2017: Oncologist
https://www.readbyqxmd.com/read/28339723/leveraging-molecular-datasets-for-biomarker-based-clinical-trial-design-in-glioblastoma
#8
Shyam K Tanguturi, Lorenzo Trippa, Shakti H Ramkissoon, Kristine Pelton, David Knoff, David Sandak, Neal I Lindeman, Azra H Ligon, Rameen Beroukhim, Giovanni Parmigiani, Patrick Y Wen, Keith L Ligon, Brian M Alexander
Background.: Biomarkers can improve clinical trial efficiency, but designing and interpreting biomarker-driven trials require knowledge of relationships among biomarkers, clinical covariates, and endpoints. We investigated these relationships across genomic subgroups of glioblastoma (GBM) within our institution (DF/BWCC), validated results in The Cancer Genome Atlas (TCGA), and demonstrated potential impacts on clinical trial design and interpretation. Methods.: We identified genotyped patients at DF/BWCC, and clinical associations across 4 common GBM genomic biomarker groups were compared along with overall survival (OS), progression-free survival (PFS), and survival post-progression (SPP)...
February 20, 2017: Neuro-oncology
https://www.readbyqxmd.com/read/28339555/an-item-bank-for-abuse-of-prescription-pain-medication-from-the-patient-reported-outcomes-measurement-information-system-promis%C3%A2
#9
Paul A Pilkonis, Lan Yu, Nathan E Dodds, Kelly L Johnston, Suzanne M Lawrence, Thomas F Hilton, Dennis C Daley, Ashwin A Patkar, Dennis McCarty
Objective.:  There is a need to monitor patients receiving prescription opioids to detect possible signs of abuse. To address this need, we developed and calibrated an item bank for severity of abuse of prescription pain medication as part of the Patient-Reported Outcomes Measurement Information System (PROMIS ® ). Methods.:  Comprehensive literature searches yielded an initial bank of 5,310 items relevant to substance use and abuse, including abuse of prescription pain medication, from over 80 unique instruments...
October 8, 2016: Pain Medicine: the Official Journal of the American Academy of Pain Medicine
https://www.readbyqxmd.com/read/28339416/improving-vocational-outcomes-in-first-episode-psychosis-by-addressing-cognitive-impairments-using-cognitive-adaptation-training
#10
Kelly A Allott, Eoin Killackey, Pamela Sun, Warrick J Brewer, Dawn I Velligan
BACKGROUND: Cognitive Adaptation Training (CAT) uses compensatory strategies and environmental supports to support cognitive impairments and improve functioning. CAT may be useful for addressing vocational recovery in first-episode psychosis (FEP) because cognitive impairments are common and vocational recovery is a key goal of young people with FEP. OBJECTIVE: To describe clinical observations and practice experience when delivering CAT with FEP clients and explore potential benefits via objective outcome measures for improving vocational outcomes...
March 23, 2017: Work: a Journal of Prevention, Assessment, and Rehabilitation
https://www.readbyqxmd.com/read/28338763/mesenchymal-stromal-cells-and-autoimmunity
#11
Vito Pistoia, Lizzia Raffaghello
Mesenchymal stromal cells (MSCs) are committed progenitors of mesodermal origin that are found virtually in every organ and exhibit multilineage differentiation into osteocytes, adipocytes and chondrocytes. MSCs also mediate a wide spectrum of immunoregulatory activities that usually dampen innate and adaptive immune responses. These features have attracted interest in the perspective of developing novel cell therapies for autoimmune disease. However, depending on the microenvironmental conditions, MSCs may show a plastic behavior and switch to an immunostimulatory phenotype...
February 18, 2017: International Immunology
https://www.readbyqxmd.com/read/28337139/a-mixed-methods-trial-of-broad-band-noise-and-nature-sounds-for-tinnitus-therapy-group-and-individual-responses-modeled-under-the-adaptation-level-theory-of-tinnitus
#12
Mithila Durai, Grant D Searchfield
Objectives: A randomized cross-over trial in 18 participants tested the hypothesis that nature sounds, with unpredictable temporal characteristics and high valence would yield greater improvement in tinnitus than constant, emotionally neutral broadband noise. Study Design: The primary outcome measure was the Tinnitus Functional Index (TFI). Secondary measures were: loudness and annoyance ratings, loudness level matches, minimum masking levels, positive and negative emotionality, attention reaction and discrimination time, anxiety, depression and stress...
2017: Frontiers in Aging Neuroscience
https://www.readbyqxmd.com/read/28335798/treatment-of-mucocutaneous-manifestations-in-beh%C3%A3-et-s-disease-with-anakinra-a-pilot-open-label-study
#13
Peter C Grayson, Yusuf Yazici, Melissa Merideth, H Nida Sen, Michael Davis, Elaine Novakovich, Elizabeth Joyal, Raphaela Goldbach-Mansky, Cailin H Sibley
BACKGROUND: The effect of IL-1 blocking therapy on mucocutaneous manifestations of Behçet's disease is incompletely understood. METHODS: Six patients with Behçet's disease and ongoing oral/genital ulcers for ≥1 month were enrolled into an adaptive, two-phase clinical trial and included in the analysis. Study duration was 6 months with extension up to 16 months. All were treated non-blinded with anakinra 100 mg subcutaneous daily with the option to escalate the dose to 200 mg in partial responders after 1 month and 300 mg after 6 months...
March 24, 2017: Arthritis Research & Therapy
https://www.readbyqxmd.com/read/28335643/prospects-and-progress-of-atezolizumab-in-non-small-cell-lung-cancer
#14
Johan Vansteenkiste, Els Wauters, Keunchil Park, Achim Rittmeyer, Alan Sandler, Alexander Spira
Immunotherapy has recently come to the forefront of oncology treatment as a potential means of combating cancer by restoring the body's adaptive cancer-immunity cycle. Atezolizumab is a monoclonal antibody agent that specifically targets programmed death ligand 1 (PD-L1), a key molecule in the cancer-immunity pathway, to block binding to its receptors PD-1 and B7.1. Areas covered: This review covers the role of atezolizumab in the treatment of non-small-cell lung cancer (NSCLC). Several studies have reported promising efficacy in this indication...
March 24, 2017: Expert Opinion on Biological Therapy
https://www.readbyqxmd.com/read/28335618/aav-an-overview-of-unanswered-questions
#15
Kenneth I Berns, Nicholas Muzyczka
Today AAV is one of the most promising and successful gene therapy vectors. AAV vectors have been successful in the treatment of several monogenic diseases in early clinical trials1. Although work in the past has focused primarily on gene replacement, many investigators are now adapting the vector system to new clinical modalities including RNAi and gene modifying strategies such as Crisper/cas91. Moreover, Glybera2 has been licensed for clinical use in the European Union for treatment of a lysosomal storage disease...
March 23, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28334851/the-modulatory-effect-of-adaptive-deep-brain-stimulation-on-beta-bursts-in-parkinson-s-disease
#16
Gerd Tinkhauser, Alek Pogosyan, Simon Little, Martijn Beudel, Damian M Herz, Huiling Tan, Peter Brown
Adaptive deep brain stimulation uses feedback about the state of neural circuits to control stimulation rather than delivering fixed stimulation all the time, as currently performed. In patients with Parkinson's disease, elevations in beta activity (13-35 Hz) in the subthalamic nucleus have been demonstrated to correlate with clinical impairment and have provided the basis for feedback control in trials of adaptive deep brain stimulation. These pilot studies have suggested that adaptive deep brain stimulation may potentially be more effective, efficient and selective than conventional deep brain stimulation, implying mechanistic differences between the two approaches...
February 13, 2017: Brain: a Journal of Neurology
https://www.readbyqxmd.com/read/28334399/association-of-hiv-status-with-local-immune-response-to-anal-squamous-cell-carcinoma-implications-for-immunotherapy
#17
Elizabeth L Yanik, Genevieve J Kaunitz, Tricia R Cottrell, Farah Succaria, Tracee L McMiller, Maria L Ascierto, Jessica Esandrio, Haiying Xu, Aleksandra Ogurtsova, Toby Cornish, Evan J Lipson, Suzanne L Topalian, Eric A Engels, Janis M Taube
Importance: The programmed cell death protein 1 (PD-1) and programmed death-ligand 1 (PD-L1) pathway play an important immunosuppressive role in cancer and chronic viral infection, and have been effectively targeted in cancer therapy. Anal squamous cell carcinoma (SCC) is associated with both human papillomavirus and HIV infection. To date, patients with HIV have been excluded from most trials of immune checkpoint blocking agents, such as anti-PD-1 and anti-PD-L1, because it was assumed that their antitumor immunity was compromised compared with immunocompetent patients...
March 23, 2017: JAMA Oncology
https://www.readbyqxmd.com/read/28332352/development-of-inhibitors-targeting-hypoxia-inducible-factor-1-and-2-for-cancer-therapy
#18
REVIEW
Tianchi Yu, Bo Tang, Xueying Sun
Hypoxia is frequently observed in solid tumors and also one of the major obstacles for effective cancer therapies. Cancer cells take advantage of their ability to adapt hypoxia to initiate a special transcriptional program that renders them more aggressive biological behaviors. Hypoxia-inducible factors (HIFs) are the key factors that control hypoxia-inducible pathways by regulating the expression of a vast array of genes involved in cancer progression and treatment resistance. HIFs, mainly HIF-1 and -2, have become potential targets for developing novel cancer therapeutics...
May 2017: Yonsei Medical Journal
https://www.readbyqxmd.com/read/28330497/atopic-dermatitis-anti-ige-paediatric-trial-adapt-the-role-of-anti-ige-in-severe-paediatric-eczema-study-protocol-for-a-randomised-controlled-trial
#19
Susan Chan, Victoria Cornelius, Tao Chen, Suzana Radulovic, Mandy Wan, Rahi Jahan, Gideon Lack
BACKGROUND: The evidence for systemic treatments for severe childhood eczema is limited and largely based on extrapolation of data from adult studies. Current therapies are often immunosuppressant and may be associated with both short- and long-term side effects. There is increasing in vitro and murine-model evidence for the role of IgE in the immunopathogenesis of atopic eczema. The aim of the study is to assess whether anti-IgE treatment (omalizumab) improves eczema, compared to placebo...
March 22, 2017: Trials
https://www.readbyqxmd.com/read/28328699/a-mindfulness-program-adapted-for-adolescents-with-chronic-pain-feasibility-acceptability-and-initial-outcomes
#20
Danielle A Ruskin, Michelle M Gagnon, Sara A Kohut, Jennifer N Stinson, Kathryn S Walker
OBJECTIVES: Pediatric chronic pain is a major health issue which can lead to significant interference in daily functioning. Mindfulness-based interventions (MBI's), which emphasize acceptance rather than control of pain, have gained increasing attention as a viable treatment option among adults with chronic pain. The effectiveness of MBIs for chronic pain in pediatric populations remains largely unknown. This prospective pre-post interventional study was conducted to examine the feasibility, acceptability, and initial effectiveness of an 8-week group MBI for adolescents (MBI-A) with chronic pain...
March 21, 2017: Clinical Journal of Pain
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