scholarly journals Possible clinical outcome measures for clinical trials in patients with multiple sclerosis

2010 ◽  
Vol 3 (4) ◽  
pp. 229-239 ◽  
Author(s):  
Myla D. Goldman ◽  
Robert W. Motl ◽  
Richard A. Rudick
Keyword(s):  
Multiple Sclerosis ◽  
Clinical Trials ◽  
Clinical Outcome ◽  
Outcome Measures

The Multiple Sclerosis Functional Composite: a new clinical outcome measure for multiple sclerosis trials

2002 ◽  
Vol 8 (5) ◽  
pp. 359-365 ◽  
Author(s):  
R A Rudick ◽  
G Cutter ◽  
S Reingold
Keyword(s):  
Multiple Sclerosis ◽  
Clinical Trials ◽  
Cognitive Function ◽  
Clinical Outcome ◽  
Outcome Measures ◽  
Standard Therapy ◽  
Outcome Measure ◽  
Task Force ◽  
Multiple Sclerosis Functional Composite ◽  
Clinical Outcome Measure

With the advent and widespread use of partially effective disease modifying drug therapies for multiple sclerosis (MS), future clinical trials will undoubtedly test experimental interventions against standard therapy, or will test combinations of drugs against standard therapy. In either case, incremental progress in slowing disability progression in future MS clinical trials will require much larger sample sizes, more sensitive outcome measures, or a combination of the two. Because improved clinical outcome methods would likely accelerate progress in MS therapeutics, the National Multiple Sclerosis Society (NMSS) convened an international task force in 1994 to recommend improved clinical outcome measures. As the result of a two-year process of discussion and data analysis, the task force recommended the Multiple Sclerosis Functional Composite (MSFC) as a new clinical outcome measure for future MS trials. MSFC consists of timed tests of walking, arm function, and cognitive function, expressed as a single score along a continuous scale. The task force recommended that MSFC be included in future MS trials, and recommended a series of validation studies. Subsequent studies have provided evidence that MSFC correlates moderately with Expanded Disability Status Scale (EDSS), and that correlation is driven by strong correlations with the ambulatory function component; arm function and cognitive function correlate at lower levels with EDSS. The MSFC correlates better than EDSS with magnetic resonance imaging (MRI) variables, including brain atrophy, and shows significant correlation with patient-reported disease-related quality of life (QOL). MSFC and short-term change in MSFC correlate with future clinical and MRI status, and the strength of the correlations compares favorably with well-known cardiovascular risk factors. The studies in aggregate indicate that MSFC and MSFC change are clinically meaningful, and that MSFC has substantial advantages over alternative clinical outcome measures for MS clinical trials.

scholarly journals Clinical outcome measures for progressive MS trials

2017 ◽  
Vol 23 (12) ◽  
pp. 1627-1635 ◽  
Author(s):  
Daniel Ontaneda ◽  
Jeffrey A Cohen ◽  
Maria Pia Amato
Keyword(s):  
Multiple Sclerosis ◽  
Clinical Trials ◽  
Clinical Outcome ◽  
Outcome Measures ◽  
Treatment Options ◽  
Unmet Need ◽  
Screening Tests ◽  
Future Research ◽  
Neuroprotective Effects ◽  
Patient Reported

Treatment options for progressive multiple sclerosis remain the main unmet need of the field. As the understanding of multiple sclerosis (MS) pathogenesis improves, new pathways and molecules will be tested for potential reparative, remyelinating, or neuroprotective effects. The clinical outcomes used will determine successful demonstration of beneficial treatment effects to regulatory agencies, clinicians, and persons with MS. This review focuses on clinical outcome measures including the Expanded Disability Status Scale, Multiple Sclerosis Functional Composite, and novel composite measures of disability. The paper also covers cognitive outcomes and screening tests for use in clinical trials. The growing importance of patient-reported outcomes and their suitability for clinical trials is also presented. The review aims to create consensus in regard to these topics and suggestions for future research.

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 Development of a Retinopathy of Prematurity Activity Scale and Clinical Outcome Measures for Use in Clinical Trials

2019 ◽  
Vol 137 (3) ◽  
pp. 305 ◽  
Author(s):  
Lois E. H. Smith ◽  
Ann Hellström ◽  
Andreas Stahl ◽  
Alistair Fielder ◽  
Wiley Chambers ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Clinical Outcome ◽  
Outcome Measures ◽  
Retinopathy Of Prematurity ◽  
Activity Scale

Disease progression in multiple sclerosis: combining physicians’ and patients’ perspectives?

2010 ◽  
Vol 17 (2) ◽  
pp. 234-240 ◽  
Author(s):  
JJ Kragt ◽  
JM Nielsen ◽  
FAH van der Linden ◽  
CH Polman ◽  
BMJ Uitdehaag
Keyword(s):  
Multiple Sclerosis ◽  
Clinical Outcome ◽  
Disease Progression ◽  
Outcome Measures ◽  
Added Value ◽  
Impact Scale ◽  
Disability Status ◽  
The Impact ◽  
Nine Hole Peg Test ◽  
Combine Outcome

Background: To assess disease progression in multiple sclerosis (MS) several outcome measures are available. The interrelation of changes on different scales has not been studied extensively and the concept of combining scales has only recently been introduced in MS. Objective: To explore combining different clinical outcome measures in the evaluation of disease progression in MS. Methods: In 553 patients we studied the presence of relevant changes according to standard definitions on the Expanded Disability Status Scale (EDSS), Nine-Hole Peg Test (9HPT), Timed 25-Foot Walk (T25FW) and the Multiple Sclerosis Impact Scale (MSIS-29). We examined ‘exclusive worsening’ (worsening on one measure while not worsening on any other measure) and ‘opposing changes’ (worsening on one measure while improving on another measure). Finally, we investigated the impact of combining assessments. Results: Based on the EDSS alone, 140 patients progressed. However, almost twice as many (275) showed worsening on any of the clinical outcome measures. Exclusive worsening was observed in 37 patients on the EDSS, 13 on the 9HPT, 39 on the T25FW and 44 on the MSIS physical. Of all worsened patients 76 (28%) showed opposing changes, a phenomenon predominantly observed when combining physician-based and patient-derived outcome measures. Conclusion: When assessing disease progression in MS, sensitivity to change can be increased by combining different outcome measures. The added value is especially present when combining measures from different perspectives. However, further research is needed to evaluate the optimal way to combine outcome measures before implementing this strategy in clinical studies.

scholarly journals The added value of cognition-targeted exercise versus symptom-targeted exercise for multiple sclerosis fatigue: A randomized controlled pilot trial

PLoS ONE ◽  
2021 ◽  
Vol 16 (11) ◽  
pp. e0258752
Author(s):  
Azza Alketbi ◽  
Salah Basit ◽  
Nouran Hamza ◽  
Lori M. Walton ◽  
Ibrahim M. Moustafa
Keyword(s):  
Multiple Sclerosis ◽  
Clinical Outcome ◽  
Outcome Measures ◽  
Recruitment Rate ◽  
Control Group ◽  
Entire Study ◽  
Randomized Controlled ◽  
Significant Difference ◽  
Intervention Protocol

Background Fatigue is considered one of the most common symptoms of multiple sclerosis (MS) and lacks a current standardized treatment. Therefore, the aim of this study was to examine the feasibility and effectiveness of a cognition-targeted exercise versus symptom-targeted exercise for MS fatigue. Methods In this Pilot, parallel-group, randomized controlled trial, sixty participants with multiple sclerosis, were randomly assigned to either a Cognition-Targeted Exercise (CTE) (N = 30, mean age 41) or a Symptom-Targeted Exercise (STE) (N = 30, mean age 42). The participants in the experimental group received eight, 50-minute sessions of weekly Cognitive Behavior Therapy (CBT) in addition to a CTE Program; whereas, participants in the control group received eight, 50-minute sessions of weekly CBT in addition to the standardized physiotherapy program (STE Program). Feasibility was assessed through recruitment rate, participant retention, adherence and safety, in addition to clinical outcome measures, including: (1) Modified Fatigue Impact Scale (MFIS), (2) Work and Social Adjustment Scale (WSAS), (3) Hospital Anxiety and Depression Scale (HADS), and Perceived Stress Scale (PSS). All outcome measures were assessed at baseline (pretreatment), following completion of the eight visit intervention protocol, and at 3-months follow-up. Results The recruitment rate was 60% and 93% of participants completed the entire study. The recruited participants complied with 98% of the required visits. No adverse events were recorded. A Generalized Estimation Equation Model revealed a significant difference over time as an interaction term during the post and follow up visit for all clinical outcome measures (p < .001). Conclusion The addition of CTE to CBT exhibited positive and more lasting influence on MS fatigue outcomes compared to Symptom-Targeted Exercise (STE). Feasibility and efficacy data from this pilot study provide support for a full-scale RCT of CTE as an integral component of Multiple Sclerosis fatigue management.

Current Clinical Trials in Dry AMD and the Definition of Appropriate Clinical Outcome Measures

2011 ◽  
Vol 26 (3) ◽  
pp. 167-180 ◽  
Author(s):  
Zohar Yehoshua ◽  
Philip J. Rosenfeld ◽  
Thomas A. Albini
Keyword(s):  
Clinical Trials ◽  
Clinical Outcome ◽  
Outcome Measures ◽  
Dry Amd ◽  
Definition Of

Clinical Outcome Measures and Rating Scales in Multiple Sclerosis Trials

1997 ◽  
Vol 72 (11) ◽  
pp. 1070-1079 ◽  
Author(s):  
Dean M. Wingerchuk ◽  
John H. Noseworthy ◽  
Brian G. Weinshenker
Keyword(s):  
Multiple Sclerosis ◽  
Clinical Outcome ◽  
Outcome Measures ◽  
Rating Scales

Outcome Measures in Multiple Sclerosis

2016 ◽  
Author(s):  
Robert M. Herndon
Keyword(s):  
Quality Of Life ◽  
Multiple Sclerosis ◽  
Clinical Trials ◽  
Disease Activity ◽  
Outcome Measures

This chapter discusses the challenges involved in measuring disease activity and progression in multiple sclerosis and how different aspects of the disease are measured in clinical trials. Metrics have been developed to assess multiple aspects of the disease., These include, in addition to more global instruments, measures of specific functions such as, cognition, mobility, quality of life, fatigue, and so on. These scales are discussed along with their strengths and weaknesses.

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