scholarly journals Timed walk as primary outcome measure of treatment response in clinical trials for HTLV-1-associated myelopathy: a feasibility study

2015 ◽  
Vol 1 (1) ◽  
Author(s):  
Fabiola Martin ◽  
Eisuke Inoue ◽  
Irene C. M. Cortese ◽  
Ramon de Almeida Kruschewsky ◽  
Adine Adonis ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Treatment Response ◽  
Feasibility Study ◽  
Primary Outcome ◽  
Outcome Measure ◽  
Primary Outcome Measure

scholarly journals Change in timed walk as primary outcome measure of treatment response in HAMLET-P: HAM/TSP MuLticentre Efficacy trial-Prednisolone

Retrovirology ◽  
2014 ◽  
Vol 11 (S1) ◽  
Author(s):  
Fabiola Martin ◽  
Eisuke Inoue ◽  
Maria Fernanda Rios Grassi ◽  
Ramon de Almeida Kruschewsky ◽  
Irene Cortese ◽  
...  
Keyword(s):  
Treatment Response ◽  
Primary Outcome ◽  
Outcome Measure ◽  
Primary Outcome Measure ◽  
Efficacy Trial

Statistical analysis, trial design and duration in Alzheimer's disease clinical trials: a review

2011 ◽  
Vol 24 (5) ◽  
pp. 689-697 ◽  
Author(s):  
P. A. Thompson ◽  
D. E. Wright ◽  
C. E. Counsell ◽  
J. Zajicek
Keyword(s):  
Alzheimer’S Disease ◽  
Alzheimer's Disease ◽  
Clinical Trials ◽  
Statistical Analysis ◽  
Statistical Methods ◽  
Outcome Measures ◽  
Primary Outcome ◽  
Outcome Measure ◽  
Primary Outcome Measure ◽  
Primary Analysis

ABSTRACTBackground: The social and economic burden of Alzheimer's disease (AD) and its increasing prevalence has led to much work on new treatment strategies and clinical trials. The search for surrogate markers of disease progression continues but traditional parallel group trial designs that use well-established, but often insensitive, clinical outcome measures predominate.Methods: We performed a systematic search across the Cochrane Library and PubMed abstracts published between January 2004 and August 2009. Information regarding the clinical trial methodology, outcome measures, intervention type and primary statistical analysis techniques was extracted and categorized, according to a standard protocol.Results: We identified 149 papers describing results from clinical trials in AD containing sufficient detail for our purposes. The largest proportion (38%) presented results of trials based on tests of cognition as the primary outcome measure. The primary analysis in most papers (85%) was a univariate significance test of a single primary outcome measure.Conclusions: The majority of trials reported a comparison of baseline and end-point assessment over relatively short patient follow-up periods, using univariate statistical methods to compare differences between intervention and control groups in the primary analysis. There is considerable scope to introduce newer statistical methods and trial designs in treatment evaluations in AD.

P07 Identifying the primary outcome measure and protocol components for a prospective feasibility study of corticosteroid regimens for CYP with JIA using consensus methods with young people, families and professionals

Rheumatology ◽  
2019 ◽  
Vol 58 (Supplement_4) ◽  
Author(s):  
Simon R. Stones ◽  
Heather Bagley ◽  
Michael W. Beresford ◽  
Ashley Jones ◽  
Flora McErlane ◽  
...  
Keyword(s):  
Young People ◽  
Outcome Measures ◽  
Feasibility Study ◽  
Primary Outcome ◽  
Outcome Measure ◽  
Conflicts Of Interest ◽  
Rapid Onset ◽  
Primary Outcome Measure ◽  
Feasibility Trial ◽  
Consensus Methods

Abstract Background Juvenile idiopathic arthritis (JIA) is an umbrella term for seven relapsing-remitting inflammatory conditions in children and young people (CYP). Early, intensive treatment can prevent long-term damage; however, established drugs exhibit a delayed response, prompting the need for rapid-onset treatment in the form of corticosteroids. Given a lack of consensus as to which corticosteroid induction regimen should be used for CYP with JIA, a feasibility trial of different regimens is needed. The aim was to achieve consensus among CYP, families, and healthcare professionals (HCPs) about the primary outcome measures and protocol components to include in a prospective feasibility study. Methods A modified Nominal Group Technique was used to achieve consensus on the most appropriate primary outcome measure to be included in a prospective feasibility study, in addition to other protocol components such as inclusion/exclusion criteria. Fifteen participants participated in the process, including a combination of CYP with JIA, families (n = 9) and HCPs (n = 6). Results In the first vote, participants agreed that Juvenile Arthritis Disease Activity Score (JADAS) and Physician Global Assessment Score were most meaningful. During sub-group discussions, the need for a composite score which captured the voice of CYP and families was emphasised. In the second vote, JADAS and the JIA Core Set were identified as the most important. Further discussions led to the results of the third vote, agreeing JADAS as the primary outcome measure of choice being measured at 6 weeks after commencement of treatment. The majority of HCPs, CYP and families voted for all JIA sub-types to be included in a prospective feasibility study, with some queries about the inclusion of systemic JIA given its unique presentation. Participants also identified the need for more frequent data collection time points to capture the rapid onset of corticosteroid action, while CYP and families opted for accessible mechanisms for participation, such as digital follow-up strategies. Conclusion It is feasible to include CYP, families and HCPs in synthesising complex concepts to agree by consensus the design components of clinical research. The primary outcome measure for inclusion in a prospective feasibility study of corticosteroid regimens in CYP with JIA was co-prioritised, with CYP and families taking a leading role in the ultimate selection of an appropriate outcome measure and other study protocol components. Using consensus methods with CYP, families and HCPs is a systematic and rigorous way in which to select outcome measures that are both meaningful and relevant to everyone involved in the care and treatment of CYP with JIA. Conflicts of Interest The authors declare no conflicts of interest.

Rationale for use of the Clinical Dementia Rating Sum of Boxes as a primary outcome measure for Alzheimer's disease clinical trials

2012 ◽  
Vol 9 (1S) ◽  
pp. S45-S55 ◽  
Author(s):  
Jesse M. Cedarbaum ◽  
Mark Jaros ◽  
Chito Hernandez ◽  
Nicola Coley ◽  
Sandrine Andrieu ◽  
...  
Keyword(s):  
Alzheimer’S Disease ◽  
Alzheimer's Disease ◽  
Clinical Trials ◽  
Primary Outcome ◽  
Outcome Measure ◽  
Primary Outcome Measure ◽  
Clinical Dementia Rating

The role of MRI as a surrogate outcome measure in multiple sclerosis

2002 ◽  
Vol 8 (1_suppl) ◽  
pp. 40-51 ◽  
Author(s):  
HF McFarland ◽  
F. Barkhof ◽  
J. Antel ◽  
DH Miller
Keyword(s):  
Multiple Sclerosis ◽  
Clinical Trials ◽  
Primary Outcome ◽  
Outcome Measure ◽  
Imaging Techniques ◽  
Primary Outcome Measure ◽  
New Therapies ◽  
Surrogate Outcome ◽  
Underlying Pathology ◽  
Surrogate Outcome Measure

The need for more specific and more sensitive outcome measures for use in testing new therapies in multiple sclerosis (MS) is generally accepted. This need has been accentuated by the realization that the ability to conduct large placebo-controlled trials will be limited in the future. From the first use of magnetic resonance imaging (MRI) to study MS, the ability of this imaging technique to identify areas of the central nervous system damage by the disease process in MS has been impressive. Thus, the possibility that MRI could serve as a surrogate outcome measure in clinical trials in MS has been attractive. The use of MRI as a surrogate outcome measure has been examined by an international group of investigators with expertise in clinical aspects of MS, the use of MRI in MS, and in experimental therapeutics. The group agreed that MRI does not represent a validated surrogate in any clinical form of MS. It was also agreed, however, that MRI does provide a reflection of the underlying pathology in the disease, but no single MRI measurement in isolation was seen as sufficient to monitor disease. The use for multiple imaging techniques, especially new, emerging techniques that may better reflect the underlying pathology, was seen as particularly important in monitoring studies of patients with either secondary or primary progressive MS. The choice of MRI techniques used to monitor new therapies needs to be consistent with the proposed mechanisms of the new therapy and phase of the disease. It was also noted, however, that additional validation is required for nonconventional imaging techniques. Finally, the participants noted that clinical trials using MRI as a primary outcome measure may fail to fully identify the effects of the therapy on clinical measures and that the risk and cost-benefit ratio of the treatment might be unresolved. Thus, before MRI is used as a primary outcome measure, new approaches to trial design must be given careful consideration. Multiple Sclerosis (2002)8, 40-51

Lesional magnetization transfer ratio: a feasible outcome for remyelinating treatment trials in multiple sclerosis

2010 ◽  
Vol 16 (6) ◽  
pp. 660-669 ◽  
Author(s):  
IJ van den Elskamp ◽  
DL Knol ◽  
H. Vrenken ◽  
G. Karas ◽  
A. Meijerman ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Clinical Trials ◽  
Primary Outcome ◽  
Outcome Measure ◽  
Magnetization Transfer ◽  
Primary Outcome Measure ◽  
Magnetization Transfer Ratio ◽  
Power Calculations ◽  
Transfer Ratio ◽  
Feasible Outcome

Magnetization transfer ratio (MTR) is a sensitive parameter to quantify the integrity of myelinated white matter in patients with multiple sclerosis. Lesional MTR decreases in the acute phase due to demyelination, and subsequently shows recovery depending on the degree of remyelination in the absence of axonal loss. Recovery of average lesion MTR therefore might prove a viable outcome measure to assess the effect of remyelinating agents. Our objective was to determine the required sample size for phase II multicentre clinical trials using the recovery of average lesion MTR as primary outcome measure. With 7-monthly MRI scans, the MTR evolution of 349 new enhancing lesions before and after enhancement was assessed in 32 MS patients from 5 centres. Multilevel models were fitted to the data yielding estimates for the variance components, which were applied in power calculations. Sample sizes were determined for placebo-controlled, multicentre trials using lesional MTR recovery post-enhancement as primary outcome measure. Average lesion MTR decreased slightly in the build-up to enhancement, decreased dramatically during enhancement and showed recovery in the period after cessation. The power calculations showed that for a power of 80%, approximately 136 patients per trial (mean number of 6 lesions per patient) are required to detect a 30% increase in lesional MTR post-enhancement compared with placebo, whereas 48 subjects are required to detect a 50% increase in lesional MTR compared with placebo. Recovery of lesion MTR is a feasible outcome measure for future multicentre clinical trials measuring the effect of remyelinating agents.

Comparison of Percentage of Syllables Stuttered With Parent-Reported Severity Ratings as a Primary Outcome Measure in Clinical Trials of Early Stuttering Treatment

2018 ◽  
Vol 61 (4) ◽  
pp. 811-819 ◽  
Author(s):  
Mark Onslow ◽  
Mark Jones ◽  
Sue O'Brian ◽  
Ann Packman ◽  
Ross Menzies ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Randomized Trials ◽  
Primary Outcome ◽  
Outcome Measure ◽  
Primary Outcome Measure ◽  
Analysis Of Covariance ◽  
Data Sets ◽  
Randomized Controlled ◽  
Absolute Agreement

Purpose This report investigates whether parent-reported stuttering severity ratings (SRs) provide similar estimates of effect size as percentage of syllables stuttered (%SS) for randomized trials of early stuttering treatment with preschool children. Method Data sets from 3 randomized controlled trials of an early stuttering intervention were selected for analyses. Analyses included median changes and 95% confidence intervals per treatment group, Bland–Altman plots, analysis of covariance, and Spearman rho correlations. Results Both SRs and %SS showed large effect sizes from pretreatment to follow-up, although correlations between the 2 measures were moderate at best. Absolute agreement between the 2 measures improved as percentage reduction of stuttering frequency and severity increased, probably due to innate measurement limitations for participants with low baseline severity. Analysis of covariance for the 3 trials showed consistent results. Conclusion There is no statistical reason to favor %SS over parent-reported stuttering SRs as primary outcomes for clinical trials of early stuttering treatment. However, there are logistical reasons to favor parent-reported stuttering SRs. We conclude that parent-reported rating of the child's typical stuttering severity for the week or month prior to each assessment is a justifiable alternative to %SS as a primary outcome measure in clinical trials of early stuttering treatment.

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