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 Digital Technology in Clinical Trials for Multiple Sclerosis: Systematic Review

2021 ◽  
Vol 10 (11) ◽  
pp. 2328
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 ◽  
Cognitive Rehabilitation ◽  
English Language ◽  
Self Assessment ◽  
Treatment Delivery ◽  
Electronic Recording

Clinical trials in multiple sclerosis (MS) have been including digital technology tools to overcome limitations in treatment delivery and disease monitoring. In March 2020, we conducted a systematic search on pubmed.gov and clinicaltrials.gov databases (with no restrictions) to identify all relevant published and unpublished clinical trials, in English language, including MS patients, in which digital technology was applied. We used “multiple sclerosis” and “clinical trial” as the main search words, and “app”, “digital”, “electronic”, “internet” and “mobile” as additional search words, separately. Digital technology is part of clinical trial interventions to deliver psychotherapy and motor rehabilitation, with exergames, e-training, and robot-assisted exercises. Digital technology has been used to standardise previously existing outcome measures, with automatic acquisitions, reduced inconsistencies, and improved detection of symptoms (e.g., electronic recording of motor performance). Other clinical trials have been using digital technology for monitoring symptoms that would be otherwise difficult to detect (e.g., fatigue, balance), for measuring treatment adherence and side effects, and for self-assessment purposes. Collection of outcome measures is progressively shifting from paper-based on site, to internet-based on site, and, in the future, to internet-based at home, with the detection of clinical and treatment features that would have remained otherwise invisible. Similarly, remote interventions provide new possibilities of motor and cognitive rehabilitation.

scholarly journals Problems in Clinical Trial Design in Multiple Sclerosis

2002 ◽  
Vol 4 (3) ◽  
pp. 125-138 ◽  
Author(s):  
Jeffrey I. Greenstein
Keyword(s):  
Multiple Sclerosis ◽  
Clinical Trials ◽  
Outcome Measures ◽  
Trial Design ◽  
Clinical Trial Design ◽  
Evidence Based ◽  
Controlled Clinical Trials ◽  
Cognitive Components ◽  
Basic Understanding ◽  
Clinical Measures

Historically, it has been difficult to demonstrate the effectiveness of treatments for multiple sclerosis (MS) because of the variability in the course of the disease, the lack of well-defined, reliable clinical measures, and the pervasiveness of poorly controlled clinical trials. Hence, to interpret the results of clinical trials in MS and make evidence-based decisions regarding treatment for their patients, neurologists should have a basic understanding of appropriate outcome measures and the necessary controls of a well-designed study. This paper reviews the controls required to test the efficacy of agents for the treatment of MS and offers examples of poorly controlled clinical trials to illustrate the problems in interpreting data without such controls. In addition, the outcome measures that should be used to assess the efficacy of treatments on the physical, inflammatory, and cognitive components of the disease are discussed. (Int J MS Care. 2002; 4: 125–131, 136–137)

scholarly journals Sharing data from MS clinical trials: Opportunities, challenges, and future directions

2015 ◽  
Vol 21 (11) ◽  
pp. 1365-1368 ◽  
Author(s):  
Timothy Coetzee
Keyword(s):  
Multiple Sclerosis ◽  
Clinical Trial ◽  
Clinical Trials ◽  
Clinical Trial Data ◽  
Therapeutic Agents ◽  
Research Community ◽  
Trial Data ◽  
Fundamental Question ◽  
Future Directions ◽  
Phase Ii And Iii

The treatment of people affected by multiple sclerosis, particularly the relapsing forms of the disease, has been transformed by the availability of various therapeutic agents. This landmark progress is due to an enormous foundation of clinical research and, particularly, numerous phase II and III clinical trials. Although the research community has many reasons to take pride in this progress, a fundamental question remains about whether opportunities for additional research are being lost due to inadequate clinical trial data sharing.

scholarly journals Clinical trial design for progressive MS trials

2017 ◽  
Vol 23 (12) ◽  
pp. 1642-1648 ◽  
Author(s):  
Matteo Pardini ◽  
Gary Cutter ◽  
Maria Pia Sormani
Keyword(s):  
Multiple Sclerosis ◽  
Clinical Trial ◽  
Clinical Trials ◽  
Trial Design ◽  
Clinical Trial Design ◽  
Progressive Multiple Sclerosis ◽  
Phase 2 ◽  
The Right ◽  
Selection Of Patients ◽  
Selection Of

The design of clinical trials is a key aspect to maximizing the possibility to detect a treatment effect. This fact is particularly challenging in progressive multiple sclerosis (PMS) studies due to the uncertainty about the right target and/or outcome in phase-2 studies. The aim of this review is to evaluate the current challenges facing the design of clinical trials for PMS. The selection of patients, the instrumental and clinical outcomes that can be used in PMS trials, and issues in their design will be covered in this report.

scholarly journals Methodology and Application of Adaptive and Sequential Approaches in Contemporary Clinical Trials

2012 ◽  
Vol 2012 ◽  
pp. 1-20 ◽  
Author(s):  
Zhengjia Chen ◽  
Yichuan Zhao ◽  
Ye Cui ◽  
Jeanne Kowalski
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Prospective Study ◽  
Trial Design ◽  
Clinical Trial Design ◽  
Modern Medicine ◽  
Future Directions ◽  
Sequential Methods ◽  
Significant Research ◽  
A Prospective Study

The clinical trial, a prospective study to evaluate the effect of interventions in humans under prespecified conditions, is a standard and integral part of modern medicine. Many adaptive and sequential approaches have been proposed for use in clinical trials to allow adaptations or modifications to aspects of a trial after its initiation without undermining the validity and integrity of the trial. The application of adaptive and sequential methods in clinical trials has significantly improved the flexibility, efficiency, therapeutic effect, and validity of trials. To further advance the performance of clinical trials and convey the progress of research on adaptive and sequential methods in clinical trial design, we review significant research that has explored novel adaptive and sequential approaches and their applications in Phase I, II, and III clinical trials and discuss future directions in this field of 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.

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