scholarly journals Comparison of Alternative Primary Outcome Measures for Use in Lupus Nephritis Clinical Trials

2013 ◽  
Vol 65 (6) ◽  
pp. 1586-1591 ◽  
Author(s):  
David Wofsy ◽  
Jan L. Hillson ◽  
Betty Diamond
Keyword(s):  
Clinical Trials ◽  
Lupus Nephritis ◽  
Outcome Measures ◽  
Primary Outcome

scholarly journals IDENTIFYING BETTER OUTCOME MEASURES TO IMPROVE TREATMENT OF AGITATION IN DEMENTIA: A REPORT FROM THE EU/US/CTAD TASK FORCE

2018 ◽  
pp. 1-5
Author(s):  
M. Sano ◽  
M. Soto ◽  
M. Carrillo ◽  
J. Cummings ◽  
S. Hendrix ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Outcome Measures ◽  
Primary Outcome ◽  
Task Force ◽  
Neuropsychiatric Symptoms ◽  
Training And Education ◽  
The Past ◽  
Global Ratings ◽  
Power Studies ◽  
The Eu

For the second time in the past 3 years, the EU-US CTAD Task Force addressed challenges related to designing clinical trials for agitation in dementia, which is one of the most disruptive aspects of the condition for both patients and caregivers. Six recommendations emerged from the Task Force meeting: 1 – Operationalizing agitation criteria established by the IPA; 2 – Combining clinician- and caregiver-derived outcomes as primary outcome measures; 3 – Using global ratings to define clinically meaningful effects and power studies; 4 – Improving the accuracy of caregiver reports by better training and education of caregivers; 5 – Employing emerging technologies to collect near real-time behavioral data; and 6 – Utilizing innovative trial designs and increasing the use of biomarkers to maximize the productivity of clinical trials for neuropsychiatric symptoms.

Misclassification and Treatment Effect on Primary Outcome Measures in Clinical Trials of Severe Neurotrauma

2002 ◽  
Vol 19 (1) ◽  
pp. 17-22 ◽  
Author(s):  
Sung C. Choi ◽  
Guy L. Clifton ◽  
Anthony Marmarou ◽  
Emmy R. Miller
Keyword(s):  
Clinical Trials ◽  
Outcome Measures ◽  
Treatment Effect ◽  
Primary Outcome

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.

Agreement of Primary Outcomes in Chiropractic-Related Clinical Trials Registered in clinicaltrials.gov with Corresponding Publication.

2020 ◽  
Author(s):  
Robert Cote ◽  
Stephen Perle ◽  
Derek Martin
Keyword(s):  
Clinical Trials ◽  
Outcome Measures ◽  
Primary Outcome ◽  
A Priori ◽  
Feasibility Studies ◽  
Primary Outcome Measure ◽  
Publication Rate ◽  
Secondary Outcome ◽  
Study Protocols ◽  
Published Paper

Abstract Introduction:Previous analysis of registered clinical trials has found a disappointing number of study protocols result in publications which change what the registered a priori primary outcome measure is. Likewise, there is a disappointing rate of unpublished trials. Similar research has not been published on chiropractic-related studies. Primarily this investigation determined if reported primary outcomes in chiropractic-related clinical trials registered in clinicaltrials.gov match their published results. Secondarily, other outcome measures and publication status are assessed.Methodology:Clinicaltrials.gov was searched for chiropractic-related trials, using the search terms “chiropractic”, “chiropractor”, and having a completed status. Publication status was determined by searching PubMed (pubmed.gov), Index to Chiropractic Literature (chiroindex.org), and Google Scholar (scholar.google.com) through 29 May 2020. If the study was published, outcome measures were compared between the clinicaltrials.gov entry and the published paper to assess for consistency by two independent investigators. If there was disagreement between investigators, a third evaluated the data and decided if the published paper agreed with the clinicaltrials.gov entry.Results:Within clinicaltrials.gov 171 chiropractic-related protocols were identified. Twenty-five (25) had results posted and 102 were published. Twenty-nine of those entries produced multiple papers consisting of protocols, plot/feasibility studies, clinical trials, and poster presentations. Of the 102 studies published, 92 (90.2%) had agreement between their primary outcome and the listed entry on clinicaltrials.gov and 82 (80.4%) agreed with the secondary outcomes in the registered protocol. Entries on clinicaltrials.gov had a 59.6% (102/171) publication rate and a 14.6% (25/171) rate of displaying their results. Conclusion:A modest rate of agreement (90.2%) between clinicaltrails.gov entries and the 102 published papers (59.6% publication rate) were found. While chiropractic-related clinical trials are fewer in number compared to medical trials, chiropractic-related research has a substantially better rate of primary and secondary outcome concordance with registered protocols and a better publication rate. Investigators need to continue to upload results onto clinicaltrials.gov and seek publication regardless of the study findings. It is important to publish negative results so as not to introduce publication bias into systematic reviews and meta-analyses. Both positive and negative findings are important when evaluating treatments and determining the best care for patients.

Measuring Pain

1998 ◽  
Vol 16 (2) ◽  
pp. 83-87 ◽  
Author(s):  
Adrian White
Keyword(s):  
Clinical Trials ◽  
Clinical Practice ◽  
Outcome Measures ◽  
Primary Outcome ◽  
Crucial Importance ◽  
Beliefs And Attitudes

Accurate outcome measures are important both in clinical practice and in research. Measurement of pain should include intensity, relief-seeking behaviour and changes in function. Beliefs and attitudes to pain are of crucial importance and various instruments for assessing these are discussed. Primary outcome measures used in clinical trials must be valid, reliable, specific and sensitive.

scholarly journals Comprehensive Analysis of Clinical Trials Registration for Lupus Nephritis Therapy on ClinicalTrials.gov

2021 ◽  
Vol 8 ◽  
Author(s):  
Yanfang Gao ◽  
Yuhan Wang ◽  
Rongshan Li ◽  
Xiaoshuang Zhou
Keyword(s):  
Clinical Trials ◽  
Lupus Nephritis ◽  
Outcome Measures ◽  
Initial Treatment ◽  
Large Scale ◽  
Treatment Strategies ◽  
Maintenance Phase ◽  
Small Scale ◽  
Eligibility Criteria ◽  
Cell Therapies

Objective: Clinical trials are the most effective method for evaluating therapeutic strategies. The purpose of this study was to comprehensively assess the characteristics of trials on lupus nephritis (LN) and provide a reference for LN treatment and research.Methods: Registered therapeutic trials on drug interventions for LN were obtained from ClinicalTrials.gov up to December 3, 2020. The general characteristics, methodological characteristics, detailed characteristics, investigated drugs, eligibility criteria, and outcome measures of these trials were analyzed.Results: A total of 126 eligible trials were evaluated, and these trials mainly investigated the initial treatment of adult proliferative LN. Half of the trials enrolled <50 participants, and 70.7% of the trials lasted for 6–24 months. In total, 95.2% of trials adopted an interventional study design. Of intervention trials, 56.6% were in phase 2 or phase 3, 76.7% were randomized, 77.5% employed a parallel assignment, and 41.7% were masked. The eligibility criteria and outcome measures of the included trials varied and involved a variety of indicators. Chemical agents and biologics are the most widely studied immunotherapies, of which mycophenolate mofetil, tacrolimus, and rituximab are the most studied. In addition, some trials studied cell transplantation treatment.Conclusions: The majority of clinical trials for LN therapy registered on ClinicalTrials.gov investigated the initial treatment of adult proliferative LN, and most of these trials were randomized, parallel assigned, and insufficiently masked interventional trials with small scale, short duration, various eligibility criteria, and outcome measures. We hope that more large-scale, long-term multicenter, and high-quality RCT trials with standardized inclusion criteria/exclusion criteria and treatment effect evaluation systems will be conducted and that more energy and funding will be put into exploring biological products and stem cell therapies. In addition, trials for membranous LN, childhood-onset LN, and maintenance phase LN are needed to establish optimal treatment strategies.

scholarly journals Heterogeneity of primary outcome measures used in clinical trials of treatments for intermediate, posterior, and panuveitis

2015 ◽  
Vol 10 (1) ◽  
Author(s):  
Alastair K. Denniston ◽  
Gary N. Holland ◽  
Andrej Kidess ◽  
Robert B. Nussenblatt ◽  
Annabelle A. Okada ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Outcome Measures ◽  
Primary Outcome

scholarly journals Postpartum Haemorrhage in Anaemic Women: Assessing Outcome Measures for Clinical Trials

2021 ◽  
Author(s):  
Amy Charlotte Brenner ◽  
Ian Roberts ◽  
Eni Balogun ◽  
Folasade Adenike Bello ◽  
Rizwana Chaudhri ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Blood Loss ◽  
Clinical Diagnosis ◽  
Outcome Measures ◽  
Treatment Effect ◽  
Primary Outcome ◽  
Effect Estimate ◽  
Shock Index ◽  
Estimated Blood Loss

Abstract Background: Post-partum haemorrhage (PPH) is a leading cause of maternal mortality worldwide. Maternal anaemia greatly increases the risk of PPH and over a third of all pregnant women are anaemic. Because anaemia reduces the oxygen-carrying capacity of the blood, anaemic women cannot tolerate the same volume of blood loss as healthy women. Yet the same blood loss threshold is used to define PPH in all women. The lack of an established PPH definition in anaemic women means the most appropriate outcome measures for use in clinical trials are open to question. We used data from the WOMAN-2 trial to examine different definitions of PPH in anaemic women and consider their appropriateness as clinical trial outcome measures. Main body: The WOMAN-2 trial is assessing tranexamic acid (TXA) for PPH prevention in women with moderate or severe anaemia at baseline. To obtain an accurate, precise estimate of the treatment effect, outcome measures should be highly specific and reasonably sensitive. Some outcome misclassification is inevitable. Low sensitivity reduces precision, but low specificity biases the effect estimate towards the null. Outcomes should also be related to how patients feel, function, or survive. The primary outcome in the WOMAN-2 trial, a ‘clinical diagnosis of PPH’, is defined as estimated blood loss >500 ml or any blood loss within 24 hours sufficient to compromise haemodynamic stability. To explore the utility of several PPH outcome measures, we analysed blinded data from 4,521 participants. For each outcome, we assessed its: 1) frequency, 2) specificity for significant bleeding defined as shock index ≥1.0, and 3) association with fatigue (modified fatigue symptom inventory [MFSI]), physical endurance (six-minute walk test) and breathlessness. A clinical diagnosis of PPH was sufficiently frequent (7%), highly specific for clinical signs of early shock (95% specificity for shock index ≥1) and associated with worse maternal functioning after childbirth.Conclusion: Outcome measures in clinical trials of interventions for PPH prevention should facilitate valid and precise estimation of the treatment effect and be important to women. A clinical diagnosis of PPH appears to meet these criteria, making it an appropriate primary outcome for the WOMAN-2 trial.Clinical trial registration: ClinicalTrials.gov NCT03475342, registered on 23 March 2018; ISRCTN62396133, registered on 7 December 2017; Pan African Clinical Trial Registry PACTR201909735842379, registered on 18 September 2019.

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