scholarly journals Hypothesis generating model-based wearable clinical trial

2016 ◽  
Author(s):  
Benjamin C Kirkup
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Human Subject ◽  
Clinical Trial Design ◽  
Shared Environment ◽  
Entire Cohort ◽  
Model Based ◽  
Physiological Impact ◽  
Physiological Sensors ◽  
Confirmatory Clinical Trials

Wearable physiological sensors have the projected capability to detect unknown and unreported health conditions. Development requires rounds of discovery-oriented human subject research and confirmatory clinical trials. However, each study is a significant investment and difficult to justify in isolation. This impasse requires bootstrapping spiral device development through hypothesis-generating, model-based clinical trials. An unconventional clinical trial design addresses environmental health and infectious disease, through the day-to-day observation of diverse people who occupy a shared environment. The design utilizes a flexible suite of developmental diagnostic devices to detect the physiological impact of exposures. Through advanced data analysis, the devices provide information about deviations from normal parameters for each human subject. The correlation of these anomalies across the entire cohort generates hypotheses about exposures that impact health. These hypotheses can be investigated further in targeted studies and lead to simultaneous refinement of the devices.

scholarly journals Hypothesis generating model-based wearable clinical trial

2016 ◽  
Author(s):  
Benjamin C Kirkup
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Human Subject ◽  
Clinical Trial Design ◽  
Shared Environment ◽  
Entire Cohort ◽  
Model Based ◽  
Physiological Impact ◽  
Physiological Sensors ◽  
Confirmatory Clinical Trials

Wearable physiological sensors have the projected capability to detect unknown and unreported health conditions. Development requires rounds of discovery-oriented human subject research and confirmatory clinical trials. However, each study is a significant investment and difficult to justify in isolation. This impasse requires bootstrapping spiral device development through hypothesis-generating, model-based clinical trials. An unconventional clinical trial design addresses environmental health and infectious disease, through the day-to-day observation of diverse people who occupy a shared environment. The design utilizes a flexible suite of developmental diagnostic devices to detect the physiological impact of exposures. Through advanced data analysis, the devices provide information about deviations from normal parameters for each human subject. The correlation of these anomalies across the entire cohort generates hypotheses about exposures that impact health. These hypotheses can be investigated further in targeted studies and lead to simultaneous refinement of the devices.

scholarly journals Recommendations on the Clinical Trial Programme for Diabetes Medicines

2021 ◽  
Vol 11 (2) ◽  
pp. 94-103
Author(s):  
I. A. Proskurina ◽  
E. A. Petraneva ◽  
D. V. Goryachev
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Treatment Effectiveness ◽  
Clinical Trial Design ◽  
Public Health Problem ◽  
The Body ◽  
Noncommunicable Diseases ◽  
The European Union ◽  
Antidiabetic Therapy ◽  
Confirmatory Clinical Trials

Diabetes is a serious public health problem and one of the major chronic noncommunicable diseases. A lengthy stepwise treatment, and the need for an individualised approach to antidiabetic therapy, pose serious challenges for medicine developers. For all new hypoglycaemic medicines, there has been a centralised authorisation procedure in the European Union (EU) since 2005, which ensures a unified approach to efficacy and safety assessment. The aim of the study was to analyse current requirements for planning clinical trials of hypoglycaemic medicines containing new active substances (except for insulin products). The recommendations for diagnosis and treatment of type 2 diabetes, prepared by the European Association for the Study of Diabetes (EASD) and the American Diabetes Association (ADA) in 2019, suggest a step-by-step approach to intensification of treatment to maintain glycaemic targets, which takes account of concomitant cardiovascular or other diseases, and clinical characteristics of patients. The analysis of EASD/ADA documents and scientific literature helped to develop recommendations on the basic principles of planning and conducting clinical trials at the final stages of hypoglycaemic medicine development. The paper describes new approaches to clinical trials, which allow for a more reliable assessment of the treatment effectiveness. The strategy for the assessment of therapeutic effect should be carefully planned, justified, and reflected in variables of interest, clinical trial design, and statistical analysis of the trial results. The main efficacy criterion in confirmatory clinical trials of hypoglycaemic medicines should be the demonstration of benefits in improving glycaemic control. The medicine’s effect on the body weight may be considered as a secondary endpoint. An essential requirement is confirmation of the medicines’ cardiovascular safety, while potential additional benefits are reduction or prevention of risks of cardiovascular disease development. The clinical trial protocol should provide definitions for intercurrent events and hypoglycaemia. A comprehensive safety study of a new hypoglycaemic medicine should involve identification of anticipated or known side effects characteristic of a particular pharmacological class. The provided recommendations may be helpful for medicine developers, and for experts who perform assessment of clinical trial programmes and regulatory submissions for hypoglycaemic medicines.           

Digital Technology in Clinical Trials for Multiple Sclerosis: a systematic review. (Preprint)

2020 ◽  
Author(s):  
Marcello De Angelis ◽  
Luigi Lavorgna ◽  
Antonio Carotenuto ◽  
Martina Petruzzo ◽  
Roberta Lanzillo ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Clinical Trial ◽  
Clinical Trials ◽  
Outcome Measures ◽  
Digital Technology ◽  
Clinical Trial Design ◽  
Motor Rehabilitation ◽  
Robot Assisted ◽  
Future Directions ◽  
Treatment Side Effects

BACKGROUND Clinical trials in multiple sclerosis (MS) have leveraged the use of digital technology to overcome limitations in treatment and disease monitoring. OBJECTIVE To review the use of digital technology in concluded and ongoing MS clinical trials. METHODS In March 2020, we searched for “multiple sclerosis” and “trial” on pubmed.gov and clinicaltrials.gov using “app”, “digital”, “electronic”, “internet” and “mobile” as additional search words, separately. Overall, we included thirty-five studies. RESULTS Digital technology is part of clinical trial interventions to deliver psychotherapy and motor rehabilitation, with exergames, e-training, and robot-assisted exercises. Also, digital technology has become increasingly used to standardise previously existing outcome measures, with automatic acquisitions, reduced inconsistencies, and improved detection of symptoms. Some trials have been developing new patient-centred outcome measures for the detection of symptoms and of treatment side effects and adherence. CONCLUSIONS We will discuss how digital technology has been changing MS clinical trial design, and possible future directions for MS and neurology research.

scholarly journals Conceptual Framework to Support Clinical Trial Optimization and End-to-End Enrollment Workflow

2019 ◽  
pp. 1-10 ◽  
Author(s):  
Neha M. Jain ◽  
Alison Culley ◽  
Teresa Knoop ◽  
Christine Micheel ◽  
Travis Osterman ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Knowledge Representation ◽  
Conceptual Framework ◽  
Trial Design ◽  
Clinical Trial Design ◽  
Prospective Clinical Trial ◽  
Future Trends ◽  
Current State ◽  
Evaluation Strategies

In this work, we present a conceptual framework to support clinical trial optimization and enrollment workflows and review the current state, limitations, and future trends in this space. This framework includes knowledge representation of clinical trials, clinical trial optimization, clinical trial design, enrollment workflows for prospective clinical trial matching, waitlist management, and, finally, evaluation strategies for assessing improvement.

scholarly journals Edaravone in Amyotrophic Lateral Sclerosis’Lessons from the Clinical Development Program and the Importance of a Strategic Clinical Trial Design

US Neurology ◽  
2018 ◽  
Vol 14 (1) ◽  
pp. 47 ◽  
Author(s):  
Said R Beydoun ◽  
Jeffrey Rosenfeld
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Amyotrophic Lateral Sclerosis ◽  
Trial Design ◽  
Development Program ◽  
Clinical Trial Design ◽  
Clinical Development ◽  
Disease Heterogeneity ◽  
Clinical Development Program ◽  
Lateral Sclerosis

Edaravone significantly slows progression of amyotrophic lateral sclerosis (ALS), and is the first therapy to receive approval by the Food and Drug Administration (FDA) for the disease in 22 years. Approval of edaravone has marked a new chapter in pharmaceutical development since the key trial included a novel strategic clinical design involving cohort enrichment. In addition, approval was based on clinical trials that had a relatively small patient number and were performed outside of the US. Edaravone was developed through a series of clinical trials in Japan where it was determined that a well-defined subgroup of patients was required to reveal a treatment effect within the study period. Amyotrophic lateral sclerosis is associated with wide-ranging disease heterogeneity (both within the spectrum of ALS phenotypes as well as in the rate of progression). The patient cohort enrichment strategy aimed to address this heterogeneity and should now be considered as a viable, and perhaps preferred, trial design for future studies. Future research incorporating relevant biomarkers may help to better elucidate edaravone’s mechanism of action, pharmacodynamics, and subsequently ALS phenotypes that may preferentially benefit from treatment. In this review, we discuss the edaravone clinical development program, outline the strategic clinical trial design, and highlight important lessons for future trials.

Clinical Trial Design and Statistics

2018 ◽  
Author(s):  
Julie Ann Sosa
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Data Analysis ◽  
Trial Design ◽  
New Technologies ◽  
Drug Application ◽  
Clinical Trial Design ◽  
Double Blind Study ◽  
Type I ◽  
End Points

A clinical trial is a planned experiment designed to prospectively measure the efficacy or effectiveness of an intervention by comparing outcomes in a group of subjects treated with the test intervention with those observed in one or more comparable group(s) of subjects receiving another intervention.  Historically, the gold standard for a clinical trial has been a prospective, randomized, double-blind study, but it is sometimes impractical or unethical to conduct such in clinical medicine and surgery. Conventional outcomes have traditionally been clinical end points; with the rise of new technologies, however, they are increasingly being supplemented and/or replaced by surrogate end points, such as serum biomarkers. Because patients are involved, safety considerations and ethical principles must be incorporated into all phases of clinical trial design, conduct, data analysis, and presentation. This review covers the history of clinical trials, clinical trial phases, ethical issues, implementing the study, basic biostatistics for data analysis, and other resources. Figures show drug development and clinical trial process, and type I and II error. Tables list Food and Drug Administration new drug application types, and types of missing data in clinical trials. This review contains 2 highly rendered figures, 2 tables, and 38 references

A novel, hybrid, single- and multi-site clinical trial design for CLN3 disease, an ultra-rare lysosomal storage disorder

2019 ◽  
Vol 16 (5) ◽  
pp. 555-560 ◽  
Author(s):  
Heather R Adams ◽  
Sara Defendorf ◽  
Amy Vierhile ◽  
Jonathan W Mink ◽  
Frederick J Marshall ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Rare Diseases ◽  
Trial Design ◽  
Neurodegenerative Disorder ◽  
Clinical Trial Design ◽  
Trial Participation ◽  
Local Participation ◽  
Cln3 Disease ◽  
Study Participants

Background Travel burden often substantially limits the ability of individuals to participate in clinical trials. Wide geographic dispersion of individuals with rare diseases poses an additional key challenge in the conduct of clinical trials for rare diseases. Novel technologies and methods can improve access to research by connecting participants in their homes and local communities to a distant research site. For clinical trials, however, understanding of factors important for transition from traditional multi-center trial models to local participation models is limited. We sought to test a novel, hybrid, single- and multi-site clinical trial design in the context of a trial for Juvenile Neuronal Ceroid Lipofuscinosis (CLN3 disease), a very rare pediatric neurodegenerative disorder. Methods We created a “hub and spoke” model for implementing a 22-week crossover clinical trial of mycophenolate compared with placebo, with two 8-week study arms. A single central site, the “hub,” conducted screening, consent, drug dispensing, and tolerability and efficacy assessments. Each participant identified a clinician to serve as a collaborating “spoke” site to perform local safety monitoring. Study participants traveled to the hub at the beginning and end of each study arm, and to their individual spoke site in the intervening weeks. Results A total of 18 spoke sites were established for 19 enrolled study participants. One potential participant was unable to identify a collaborating local site and was thus unable to participate. Study start-up required a median 6.7 months (interquartile range = 4.6–9.2 months). Only 33.3% (n = 6 of 18) of spoke site investigators had prior clinical trial experience, thus close collaboration with respect to study startup, training, and oversight was an important requirement. All but one participant completed all study visits; no study visits were missed due to travel requirements. Conclusions This study represents a step toward local trial participation for patients with rare diseases. Even in the context of close oversight, local participation models may be best suited for studies of compounds with well-understood side-effect profiles, for those with straightforward modes of administration, or for studies requiring extended follow-up periods.

scholarly journals Reporting guidelines for clinical trial reports for interventions involving artificial intelligence: the CONSORT-AI Extension

BMJ ◽  
2020 ◽  
pp. m3164 ◽  
Author(s):  
Xiaoxuan Liu ◽  
Samantha Cruz Rivera ◽  
David Moher ◽  
Melanie J Calvert ◽  
Alastair K Denniston
Keyword(s):  
Artificial Intelligence ◽  
Clinical Trial ◽  
Clinical Trials ◽  
Clinical Trial Design ◽  
Trial Protocols ◽  
General Readership ◽  
Peer Reviewers ◽  
Multi Stakeholder ◽  
Analysis Of Error

Abstract The CONSORT 2010 (Consolidated Standards of Reporting Trials) statement provides minimum guidelines for reporting randomised trials. Its widespread use has been instrumental in ensuring transparency when evaluating new interventions. More recently, there has been a growing recognition that interventions involving artificial intelligence (AI) need to undergo rigorous, prospective evaluation to demonstrate impact on health outcomes. The CONSORT-AI extension is a new reporting guideline for clinical trials evaluating interventions with an AI component. It was developed in parallel with its companion statement for clinical trial protocols: SPIRIT-AI. Both guidelines were developed through a staged consensus process, involving a literature review and expert consultation to generate 29 candidate items, which were assessed by an international multi-stakeholder group in a two-stage Delphi survey (103 stakeholders), agreed on in a two-day consensus meeting (31 stakeholders) and refined through a checklist pilot (34 participants). The CONSORT-AI extension includes 14 new items, which were considered sufficiently important for AI interventions, that they should be routinely reported in addition to the core CONSORT 2010 items. CONSORT-AI recommends that investigators provide clear descriptions of the AI intervention, including instructions and skills required for use, the setting in which the AI intervention is integrated, the handling of inputs and outputs of the AI intervention, the human-AI interaction and providing analysis of error cases. CONSORT-AI will help promote transparency and completeness in reporting clinical trials for AI interventions. It will assist editors and peer-reviewers, as well as the general readership, to understand, interpret and critically appraise the quality of clinical trial design and risk of bias in the reported outcomes.

Sign in / Sign up

Export Citation Format

Share Document