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

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

scholarly journals Longitudinal Assessment of Strength, Functional Capacity, Oropharyngeal Function, and Quality of Life in Oculopharyngeal Muscular Dystrophy

Neurology ◽  
2021 ◽  
pp. 10.1212/WNL.0000000000012640
Author(s):  
Rosemarie H.M.J.M. Kroon ◽  
Johanna G. Kalf ◽  
Bert J.M. de Swart ◽  
Barbara M. van der Sluijs ◽  
Jeffrey C. Glennon ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Clinical Trials ◽  
Muscular Dystrophy ◽  
Muscle Strength ◽  
Clinical Outcome ◽  
Disease Progression ◽  
Outcome Measures ◽  
Deltoid Muscle ◽  
Oculopharyngeal Muscular Dystrophy

ObjectiveOculopharyngeal muscular dystrophy (OPMD) is a late-onset, progressive muscle disease. Disease progression is known to be slow, but details on the natural history remain unknown. We aimed to examine the natural history of OPMD in a large nationwide cohort to determine clinical outcome measures that capture disease progression and can be used in future clinical trials.MethodsPatients, invited by their treating physicians or from the national neuromuscular database, and invited family members, were examined twice, 20 months apart, using fixed dynamometry, MRC grading, maximum bite force and isometric tongue strength, Motor Function Measure (MFM), 10-step stair test, maximum swallowing-, chewing-, and speech-tasks and quality of life assessments.ResultsDisease progression was captured by 8 out of 18 measures over 20 months in forty-three genetically confirmed OPMD patients. The largest deterioration was seen in deltoid muscle strength (-27% (range -17 – -37%)), followed by the quadriceps (-14% (range -6 – -23%)), iliopsoas (-12.2%), tongue (-9.9%) and MRC sum-score (-2.5%). The 10-step stair test (-12.5%), MFM part D1 (-7.1%), and maximum repetition rate of /pa/ (-5.3%) showed a significant decrease as well (all p<0.05). Domain ‘Physical functioning’ of the SF-36 Health Survey significantly deteriorated (p=0.044). No relationship was found between disease progression and genotype or disease duration (p>0.05).ConclusionsDespite the slow disease progression of OPMD, this study showed that several outcome measures detected progression within 20 months. The deltoid muscle strength, measured by fixed dynamometry, showed the greatest decline. This longitudinal data provides clinical outcome measures that can be used as biomarkers in future clinical trials.

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.

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