scholarly journals 2200

2017 ◽  
Vol 1 (S1) ◽  
pp. 45-45
Author(s):  
Susan Lynn Murphy ◽  
Christy Byks-Jazayeri ◽  
Brenda Eakin ◽  
Jordan Hahn ◽  
Brandon Lynn ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Good Clinical Practice ◽  
Relevant Information ◽  
Behavioral Research ◽  
Preliminary Evaluation ◽  
Pass Rates ◽  
Job Aids ◽  
Additional Information ◽  
University Of Buffalo

OBJECTIVES/SPECIFIC AIMS: To conduct a preliminary evaluation of the Social and Behavioral Research Best Practices Course. METHODS/STUDY POPULATION: Learners are sampled from 5 institutions: University of Michigan, University of Rochester, University of Florida, Boston University, and University of Buffalo. Learners who take the course and consent to be in the study receive a web link to a survey immediately after course completion and at 2–3 months follow up. In addition to demographic information, learners will report their perceptions of usefulness and relevance of the course to their job, their satisfaction with the course and associated job aids, and at follow-up, if and how the course impacted their work. Additional information will be collected from the learning management systems which host the course at each institution. The data collected will include the number of participants who take the course, the number who complete, how many times the course was attempted, and pass rates. RESULTS/ANTICIPATED RESULTS: We anticipate that several hundred learners will take the course by the end of our project. Of learners who agree to participate in the survey, we anticipate that they will find the course useful and relevant to social and behavioral clinical trials and will be satisfied with the course. Information including suggestions about missing content, items or content that were not extremely clear, or any other comments will be collected to iterate and expand the course. DISCUSSION/SIGNIFICANCE OF IMPACT: This course was developed to fill a gap in training in good clinical practice for social and behavioral research. An evaluation of how the training provided in the course impacts the jobs of learners is needed both to ensure that the most relevant information is included in the course as well as to identify ways that the training may contribute to the quality and safety of social and behavioral clinical trials.

Valid Treatment Options for Osteoporosis and Osteopenia in HIV-Infected Persons

2008 ◽  
Vol 42 (5) ◽  
pp. 670-679 ◽  
Author(s):  
Patrick G Clay ◽  
Laura E Voss ◽  
Charlott Williams ◽  
Eric C Daume
Keyword(s):  
Clinical Trials ◽  
Randomized Clinical Trials ◽  
Calcium Supplementation ◽  
Data Extraction ◽  
Treatment Options ◽  
Relevant Information ◽  
Mineral Density ◽  
Safety And Efficacy ◽  
Additional Information ◽  
Study Selection

Objective: To review clinical data on bone ossification agents that may be considered for use in the treatment of osteoporosis and osteopenia in HIV-infected patients. Data Sources: A literature search was performed using MEDLINE (1950-January 2008), EMBASE, PubMed, and abstracts from major HIV conferences (February 2001-October 2007). These searches were limited to human data published in English and used the key words bisphosphonates, calcitonin, raloxifene. teriparatide, HAART, osteopenia, osteoporosis, and HIV/AIDS. Additional articles were retrieved from citations of selected references. Study Selection and Data Extraction: Relevant information on the pharmacology, pharmacokinetics, safety, and efficacy of available treatment with hormonal and nonhormonal agents was selected. Greater emphasis was placed on randomized clinical trials than on retrospective studies. Data Synthesis: Osteoporosis in HIV-infected persons is at least as prevalent as in postmenopausal women, yet this population is not listed in primary care guidelines as one that should be considered for screening. In addition to bisphosphonates, calcitonin, raloxifene, and teriparatide are used to treat bone disorders. Three clinical trials to date have evaluated the use of a bisphosphonate in HIV-infected persons. The trials showed a marked increase in bone mineral density in patients taking alendronate versus those in the control groups (with/without calcium, exercise, and/or vitamin D in 1 or both arms). Dosing restrictions complicate the use of these agents; diet, exercise, and calcium supplementation remain the foremost recommended strategies to prevent bone loss. The use of estrogen, testosterone, calcitonin, and teriparatide is less studied in HIV-positive patients, but may be considered in select cases. There are some investigational drugs and agents not available in the US; however, there are not enough data to support their use. Conclusions: Alendronate appears to be a promising treatment option for HIV-infected patients with osteoporosis and osteopenia. Further research is required to determine the safety and efficacy of other available drugs. Until additional information is provided, and with available knowledge on the metabolism profiles of antiretroviral and bone ossification agents, alendronate appears to be the preferred agent to use in this population.

scholarly journals An FDA Analysis of Inspected Entities After Receiving Official Action Indicated Letters for Good Clinical Practice Violations

2021 ◽  
Author(s):  
Miah Jung ◽  
Rachelle M. Swann ◽  
Michelle S. Anantha ◽  
Faranak Jamali
Keyword(s):  
Clinical Trials ◽  
Clinical Practice ◽  
Drug Evaluation ◽  
Good Clinical Practice ◽  
Cross Sectional Study ◽  
Voluntary Action ◽  
Cross Sectional ◽  
Depth Analysis ◽  
The Status

Abstract Background Limited research has been conducted to examine whether clinical investigators (CIs), sponsors (SPs), contract research organizations (CROs), and sponsor-investigators (SIs) continue conducting clinical trials following issuance of FDA Official Action Indicated (OAI) letters. FDA issues OAI letters for significant regulatory violations. The objective of this study was to evaluate the status of inspected entities who received OAI letters in the conduct of Center for Drug Evaluation and Research (CDER)-regulated clinical trials (CRCTs). Methods This cross-sectional study included an analysis of inspectional data from CDER’s Good Clinical Practice (GCP) inspections for OAI letters issued from October 1, 2010, to September 30, 2015, with an in-depth analysis of post-OAI status of inspected entities, including OAI follow-up inspections. Results Of the 2248 GCP letters issued during this period, 104 (4.6%) OAI letters were sent: 95 (4.2%) to CIs (91% of OAIs), 7 (0.3%) to SPs (7% of OAIs), and 2 (0.08%) to SIs (2% of OAIs). Majority of OAI letters were issued as a result of a for-cause inspection. Five CIs were excluded from analysis. No OAI letters were sent to CROs. Only 30% of CIs (27 out of 90) continued to conduct CRCTs. OAI follow-up inspections were completed for these CIs resulting in 16 No Action Indicated (NAI), 11 Voluntary Action Indicated (VAI), and no OAI letters. Majority (64%) of the VAI letters noted repeated but not significant violations. Conclusions Majority (70%) of CIs who received an OAI letter were no longer conducting CRCTs at the time of follow-up. Of the 27 CIs continuing CRCTs, 16 (59%) OAI follow-up inspections resulted in NAI classifications and 11 (41%) in VAI.

scholarly journals Best practices in social and behavioral research: report from the Enhancing Clinical Research Professional’s Training and Qualifications project

2017 ◽  
Vol 1 (1) ◽  
pp. 26-32 ◽  
Author(s):  
Susan L. Murphy ◽  
Christine Byks-Jazayeri ◽  
Nancy Calvin-Naylor ◽  
Vic Divecha ◽  
Elizabeth Anderson ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Best Practices ◽  
Clinical Research ◽  
Work Group ◽  
Good Clinical Practice ◽  
Behavioral Research ◽  
Research Report ◽  
Competency Based ◽  
Behavioral Trials ◽  
Training Need

IntroductionThis article discusses the process of defining competencies and development of a best practices training course for investigators and clinical research coordinators who conduct social and behavioral research.MethodsThe first project phase established recommendations for training in Good Clinical Practice (GCP) and was done in conjunction with representatives from 62 Clinical and Translational Science Award (CTSA) hubs. Diversity in behavioral clinical trials and differences in regulation of behavioral trials compared with clinical trials involving drugs, devices, or biologics necessitated a separate Social and Behavioral Work Group. This group worked with CTSA representatives to tailor competencies and fundamental GCP principles into best practices for social and behavioral research.ResultsAlthough concepts underlying GCP were deemed similar across all clinical trials, not all areas were equally applicable and the ways in which GCP would be enacted differ for behavioral trials. It was determined that suitable training in best practices for social and behavioral research was lacking.DiscussionBased on the training need, an e-learning course for best practices is available to all CTSA sites. Each institution is able to track outcomes for its employees to help achieve standardized competency-based best practices for social and behavioral investigators and staff.

The Use of Digital Clinical Trial Methods in the Evaluation of Digital Therapeutics: A Scoping Review (Preprint)

2019 ◽  
Author(s):  
Allison Hirsch ◽  
Mahip Grewal ◽  
Anthony James Martorell ◽  
Brian Michael Iacoviello
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Clinical Research ◽  
Scoping Review ◽  
Digital Technologies ◽  
Good Clinical Practice ◽  
The United States ◽  
Clinical Trial Research ◽  
Digital Methods ◽  
Clinical Trial Methods

BACKGROUND Digital Therapeutics (DTx) provide evidence based therapeutic health interventions that have been clinically validated to deliver therapeutic outcomes, such that the software is the treatment. Digital methodologies are increasingly adopted to conduct clinical trials due to advantages they provide including increases in efficiency and decreases in trial costs. Digital therapeutics are digital by design and can leverage the potential of digital and remote clinical trial methods. OBJECTIVE The principal purpose of this scoping review is to review the literature to determine whether digital technologies are being used in DTx clinical research, which type are being used and whether publications are noting any advantages to their use. As DTx development is an emerging field there are likely gaps in the knowledge base regarding DTx and clinical trials, and the purpose of this review is to illuminate those gaps. A secondary purpose is to consider questions which emerged during the review process including whether fully remote digital clinical research is appropriate for all health conditions and whether digital clinical trial methods are inline with the principles of Good Clinical Practice. METHODS 1,326 records were identified by searching research databases and 1,227 reviewed at the full-article level in order to determine if they were appropriate for inclusion. Confirmation of clinical trial status, use of digital clinical research methods and digital therapeutic status as well as inclusion and exclusion criteria were applied in order to determine relevant articles. Digital methods employed in DTx research were extracted from each article and these data were synthesized in order to determine which digital methods are currently used in clinical trial research. RESULTS After applying our criteria for scoping review inclusion, 11 articles were identified. All articles used at least one form of digital clinical research methodology enabling an element of remote research. The most commonly used digital methods are those related to recruitment, enrollment and the assessment of outcomes. A small number of articles reported using other methods such as online compensation (n = 3), or digital reminders for participants (n = 5). The majority of digital therapeutics clinical research using digital methods is conducted in the United States and increasing number of articles using digital methods are published each year. CONCLUSIONS Digital methods are used in clinical trial research evaluating DTx, though not frequently as evidenced by the low proportion of articles included in this review. Fully remote clinical trial research is not yet the standard, more frequently authors are using partially remote methods. Additionally, there is tremendous variability in the level of detail describing digital methods within the literature. As digital technologies continue to advance and the clinical research DTx literature matures, digital methods which facilitate remote research may be used more frequently.

scholarly journals Real-World Experience with Bezlotoxumab for Prevention of Recurrence of Clostridioides difficile Infection

2020 ◽  
Vol 10 (1) ◽  
pp. 2
Author(s):  
Rosa Escudero-Sánchez ◽  
María Ruíz-Ruizgómez ◽  
Jorge Fernández-Fradejas ◽  
Sergio García Fernández ◽  
María Olmedo Samperio ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Real World ◽  
Recurrence Rates ◽  
Factors Associated ◽  
Clostridioides Difficile ◽  
Multicentre Cohort Study ◽  
Clostridioides Difficile Infection ◽  
Prevention Of Recurrence ◽  
Multicentre Cohort

Bezlotoxumab is marketed for the prevention of recurrent Clostridioides difficile infection (rCDI). Its high cost could be determining its prescription to a different population than that represented in clinical trials. The objective of the study was to verify the effectiveness and safety of bezlotoxumab in preventing rCDI and to investigate factors related to bezlotoxumab failure in the real world. A retrospective, multicentre cohort study of patients treated with bezlotoxumab in Spain was conducted. We compared the characteristics of cohort patients with those of patients treated with bezlotoxumab in the pivotal MODIFY trials. We assessed recurrence rates 12 weeks after completion of treatment against C. difficile, and we analysed the factors associated with bezlotoxumab failure. Ninety-one patients were included in the study. The cohort presented with more risk factors for rCDI than the patients included in the MODIFY trials. Thirteen (14.2%) developed rCDI at 12 weeks of follow-up, and rCDI rates were numerically higher in patients with two or more previous episodes (25%) than in those who had fewer than two previous episodes of C. difficile infection (CDI) (10.4%); p = 0.09. There were no adverse effects attributable to bezlotoxumab. Despite being used in a more compromised population than that represented in clinical trials, we confirm the effectiveness of bezlotoxumab for the prevention of rCDI.

scholarly journals Teleconsultation and Teletreatment Protocol to Diagnose and Manage Patients with Benign Paroxysmal Positional Vertigo (BPPV) during the COVID-19 Pandemic

2021 ◽  
Vol 25 (01) ◽  
pp. e141-e149
Author(s):  
Renato Gonzaga Barreto ◽  
Darío Andrés Yacovino ◽  
Lázaro Juliano Teixeira ◽  
Mayanna Machado Freitas
Keyword(s):  
Disease Transmission ◽  
Benign Paroxysmal Positional Vertigo ◽  
Good Clinical Practice ◽  
Relevant Information ◽  
Remote Access ◽  
Vestibular Disorder ◽  
Face To Face ◽  
Positional Vertigo ◽  
Video And Audio ◽  
Paroxysmal Positional Vertigo

Abstract Introduction Telehealth consists in the application of technology to provide remote health service. This resource is considered safe and effective and has attracted an exponential interest in the context of the COVID pandemic. Expanded to dizzy patients, it would be able to provide diagnosis and treatment, minimizing the risk of disease transmission. Benign paroxysmal positional vertigo (BPPV) is the most common vestibular disorder. The diagnosis typically rests on the description of the symptoms along with the nystagmus observed at a well-established positional testing. Objectives The aim of the present study was to propose a teleconsultation and teletreatment protocol to manage patients with BPPV during the COVID-19 pandemic. Methods Specialists in the vestibular field met through remote access technologies to discuss the best strategy to manage BPPV patients by teleconsultation and teletreatment system. Additionally, several scientific sources were consulted. Technical issues, patient safety, and clinical assessment were independently analyzed. All relevant information was considered in order to design a clinical protocol to manage BPPV patients in the pandemic context. Results Teleconsultation for BPPV patients requires a double way (video and audio) digital system. An adapted informed consent to follow good clinical practice statements must be considered. The time, trigger and target eye bedside examination (TiTRaTe) protocol has proven to be a valuable first approach. The bow and lean test is the most rational screening maneuver for patients with suspected positional vertigo, followed by most specific maneuvers to diagnostic the sub-variants of BPPV. Conclusion Although with limited evidence, teleconsultation and teletreatment are both reasonable and feasible strategies for the management of patients with BPPV in adverse situations for face-to-face consultation.

scholarly journals Disability improvement as a clinically relevant outcome in clinical trials of relapsing forms of multiple sclerosis

2021 ◽  
pp. 135245852110002
Author(s):  
Bruce AC Cree ◽  
Jeffrey A Cohen ◽  
Anthony T Reder ◽  
Davorka Tomic ◽  
Diego Silva ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Clinical Trials ◽  
Therapeutic Benefit ◽  
Disease Modifying Therapies ◽  
Long Term Follow Up ◽  
Post Hoc ◽  
Switch Group ◽  
Relevant Outcome

Background: Disease-modifying therapies (DMTs) can reduce the risk of disability worsening in patients with relapsing forms of multiple sclerosis (RMS). High-efficacy DMTs can lead to confirmed or sustained disability improvement (CDI and SDI). Objective and Methods: Post hoc analyses of data from the TRANSFORMS, FREEDOMS, and FREEDOMS II trials and their extensions assessed the effects of fingolimod (0.5–1.25 mg/day) on stabilizing or improving disability over ⩽8 years in participants with RMS. CDI and SDI rates were compared between participants initially randomized to fingolimod, interferon (IFNβ-1a), or placebo. Results: At 8 years’ follow-up in TRANSFORMS, 35.1% (95% confidence interval [CI], 28.2%–43.1%) of assessed participants in the IFNβ-1a–fingolimod switch group and 41.9% (36.6%–47.6%) on continuous fingolimod experienced CDI; disability did not worsen in approximately 70%. Similar results were seen in the combined FREEDOMS population. Proportionally fewer TRANSFORMS participants achieved SDI in the IFNβ-1a–fingolimod switch group than on continuous fingolimod (5.4% [3.0%–9.5%] vs 14.2% [10.8%–18.4%], p = 0.01). Conclusion: CDI and SDI are outcomes of interest for clinical trials and for long-term follow-up of participants with RMS. Monitoring CDI and SDI in addition to disability worsening may facilitate understanding of the therapeutic benefit of RMS treatments.

Patients with shoulder pain referred to specialist care; treatment, predictors of pain and disability, emotional distress, main symptoms and sick-leave: a cohort study with a six-months follow-up

2020 ◽  
Vol 20 (4) ◽  
pp. 775-783
Author(s):  
Kaia B. Engebretsen ◽  
Jens Ivar Brox ◽  
Niels Gunnar Juel
Keyword(s):  
Clinical Trials ◽  
Cohort Study ◽  
Sick Leave ◽  
Shoulder Pain ◽  
Emotional Distress ◽  
Outpatient Clinic ◽  
Treatment Duration ◽  
Specialist Care ◽  
Yellow Flags

AbstractObjectivesRecommendations for referral of patients with shoulder pain from primary to specialist care are mainly clinical. Several patients are referred without meeting these criteria for referral, whereas some are referred for a second opinion although surgery is not recommended. The aims of this study were to describe a shoulder pain cohort in specialist healthcare according to demographic data, clinical, and psychological factors; evaluate changes in pain and disability, distress and main symptoms from baseline to six-month follow-up; and to assess predictors of pain and disability, changes in the main symptoms and sick-leave at six-months. Results were compared to previous randomised trials conducted at the same clinic in patients with subacromial shoulder pain.MethodsThis prospective study included 167 patients from an outpatient clinic in specialist healthcare with shoulder pain for more than 6 weeks. Clinical (pain duration, intensity, pain sites), sociodemographic (age, gender, educational level, work status) and psychological variables (emotional distress (HSCL-10), fear of pain, screening of “yellow flags”, health-related quality of life) were collected. Shoulder pain and disability (SPADI-score) were assessed and the patients were asked about their outcome expectation and to predict their status of their shoulder problem the next month. They underwent a clinical interview, a clinical assessment of shoulder function and orthopaedic tests for diagnostic purposes. After six months they received a questionnaire with main variables.ResultsOf the 167 patients (55% women), 50% had symptoms for more than 12 months and 37 (22%) were on sick-leave. Characteristics were in general comparable to patients previously included in clinical trials at the same department. The SPADI-score was 46 (23) points. Mean emotional distress was within the normal range (1.7 (SD 0.6)). More than 80% had received treatment before, mainly physiotherapy in addition to the GPs treatment. One hundred and thirty-seven patients (82%) were re-referred to physiotherapy, 74 (44%) in the outpatient clinic specialist healthcare, and 63 (38%) in primary care. One hundred and eighteen (71%) answered the follow-up questionnaire. Mean change in SPADI-score was 10.5 points (95% CI (6.5–14.5)), and 29% of the patients improved more than the smallest detectable difference (SDD). The percentage sick-listed was 19.5%, and mean change in main symptoms (−9 to +9) was 3.4 (SD 3.9). The subgroup of patients receiving physiotherapy in outpatient specialist care did not show any significant change in the main variables. The prediction models suggested that a lower level of education, more fear of pain and a high baseline SPADI-score, predicted a higher SPADI-score at follow-up. A high baseline HSCL-10 score was the only significant predictor for a high HSCL-10 score. At follow-up, less pain at rest predicted more change in main symptoms and more yellow flags (a higher score on the Örebro screening test) predicted sick-leave.ConclusionsWithin the limitations of a cohort study, patients with persistent shoulder pain referred to an outpatient specialist clinic had similar baseline characteristics but shorter treatment duration, inferior clinical results and predictors somewhat different compared with previous clinical trials conducted at the same clinic. The study raises some questions about the effectiveness of the routines in daily clinical practice, the selection of patients, the treatment duration and content.

scholarly journals Maximizing Acceleration and Change of Direction in Sport: A Case Series to Illustrate How the Force-Velocity Profile Provides Additional Information to That Derived from Linear Sprint Time

2021 ◽  
Vol 18 (11) ◽  
pp. 6140
Author(s):  
Andrés Baena-Raya ◽  
Manuel A. Rodríguez-Pérez ◽  
Pedro Jiménez-Reyes ◽  
Alberto Soriano-Maldonado
Keyword(s):  
Mechanical Properties ◽  
Case Series ◽  
Relevant Information ◽  
Maximal Power Output ◽  
Sprint Time ◽  
Change Of Direction ◽  
Training Interventions ◽  
Additional Information ◽  
Technical Report ◽  
Force Velocity

Sprint running and change of direction (COD) present similar mechanical demands, involving an acceleration phase in which athletes need to produce and apply substantial horizontal external force. Assessing the mechanical properties underpinning individual sprint acceleration might add relevant information about COD performance in addition to that obtained through sprint time alone. The present technical report uses a case series of three athletes with nearly identical 20 m sprint times but with different mechanical properties and COD performances. This makes it possible to illustrate, for the first time, a potential rationale for why the sprint force-velocity (FV) profile (i.e., theoretical maximal force (F0), velocity (V0), maximal power output (Pmax), ratio of effective horizontal component (RFpeak) and index of force application technique (DRF)) provides key information about COD performance (i.e., further to that derived from simple sprint time), which can be used to individualize training. This technical report provides practitioners with a justification to assess the FV profile in addition to sprint time when the aim is to enhance sprint acceleration and COD performance; practical interpretations and advice on how training interventions could be individualized based on the athletes’ differential sprint mechanical properties are also specified.

scholarly journals DNA Methylation at ATP11A cg11702988 Is a Biomarker of Lung Disease Severity in Cystic Fibrosis: A Longitudinal Study

Genes ◽  
2021 ◽  
Vol 12 (3) ◽  
pp. 441
Author(s):  
Fanny Pineau ◽  
Davide Caimmi ◽  
Sylvie Taviaux ◽  
Maurane Reveil ◽  
Laura Brosseau ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Dna Methylation ◽  
Clinical Trials ◽  
Lung Disease ◽  
Disease Severity ◽  
Genome Wide ◽  
A Genome ◽  
Lung Disease Severity ◽  
Epigenetic Biomarker

Cystic fibrosis (CF) is a chronic genetic disease that mainly affects the respiratory and gastrointestinal systems. No curative treatments are available, but the follow-up in specialized centers has greatly improved the patient life expectancy. Robust biomarkers are required to monitor the disease, guide treatments, stratify patients, and provide outcome measures in clinical trials. In the present study, we outline a strategy to select putative DNA methylation biomarkers of lung disease severity in cystic fibrosis patients. In the discovery step, we selected seven potential biomarkers using a genome-wide DNA methylation dataset that we generated in nasal epithelial samples from the MethylCF cohort. In the replication step, we assessed the same biomarkers using sputum cell samples from the MethylBiomark cohort. Of interest, DNA methylation at the cg11702988 site (ATP11A gene) positively correlated with lung function and BMI, and negatively correlated with lung disease severity, P. aeruginosa chronic infection, and the number of exacerbations. These results were replicated in prospective sputum samples collected at four time points within an 18-month period and longitudinally. To conclude, (i) we identified a DNA methylation biomarker that correlates with CF severity, (ii) we provided a method to easily assess this biomarker, and (iii) we carried out the first longitudinal analysis of DNA methylation in CF patients. This new epigenetic biomarker could be used to stratify CF patients in clinical trials.

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