scholarly journals Psychometric validation and interpretation of the Nocturia Impact Diary in a clinical trial setting

2021 ◽  
Author(s):  
Stacie Hudgens ◽  
Amy Howerter ◽  
Ela Polek ◽  
Fredrik L. Andersson
Keyword(s):  
Clinical Trial ◽  
Convergent Validity ◽  
Psychometric Validation ◽  
Drug Candidate ◽  
Meaningful Improvement ◽  
Clinical Trial Setting ◽  
Exit Interviews ◽  
Patient Reported ◽  
The Impact ◽  
Trial Setting

Abstract Purpose Psychometric evaluation of the Nocturia Impact (NI) Diary was conducted to support its use as a trial endpoint. Methods As part of a randomized, controlled Phase 2 clinical trial investigating a novel drug candidate for nocturnal polyuria, adult nocturia patients completed the NI Diary and a voiding diary for three nights preceding their clinic visit at Baseline and Weeks 1, 4, 8, and 12 (end of treatment). Exit interviews were conducted to obtain patient impressions of the NI Diary. Results A total of N = 302 participants were included. Confirmatory factor analysis (CFA) indicated that the 11-item measure is unidimensional with values of CFI, TLI, and RMSEA meeting relevant thresholds. Good internal consistency (Cronbach’s α 0.941) and test–retest reliability (intra-class correlation coefficients 0.730–0.880). Convergent validity with two reference measures was demonstrated with strong correlations of 0.573–0.730 were shown. Significant differences (P = 0.0018, standardized effect size = 0.372) between groups defined by number of night-time voids supported known-groups validity. Exit interviews in 66 patients indicated all participants experienced improvement in at least 1 NI Diary item and that a 1-point improvement on the item response scale and 1-void reduction per night (associated with an average best cut point on ROC analysis of − 11.6) constituted meaningful improvement. Anchor and distribution-based analyses identified a meaningful change threshold of − 15 to − 18 points on the NI Diary. Conclusion The NI Diary is a reliable and valid patient-reported psychometric instrument which is fit-for-purpose to evaluate the impact of nocturia on patient quality of life in the clinical trial setting. Trial registration number and registration date NCT03201419; June 28, 2017.

scholarly journals Conducting Patient Interviews Within a Clinical Trial Setting

2018 ◽  
Author(s):  
Dana B. DiBenedetti ◽  
T. Michelle Brown ◽  
Carla Romano ◽  
Claire Ervin ◽  
Sandy Lewis ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Qualitative Interviews ◽  
Patient Reported Outcome ◽  
Treatment Needs ◽  
Clinical Trial Setting ◽  
Patient Interviews ◽  
Treatment Gaps ◽  
Patient Reported ◽  
Trial Participants ◽  
Trial Setting

Qualitative data centered on patients’ experiences and perspectives typically go uncollected in clinical trial settings. Yet patients’ treatment experiences offer complementary insights and context on topics such as disease management, treatment gaps, and previous treatments outside of those gathered in traditional patient-reported outcome questionnaires. Qualitative interviews can capture patients’ perceptions of treatment needs, more fully explore meaningful changes experienced as a result of treatment, and reveal outcomes that are most important to patients. Asking patients detailed questions can provide insight into the “why” of a patient’s expressed thought or feeling. The inclusion of patient interviews within clinical trials is a relatively new and evolving field of research. This article delineates the types of data that may be collected during interviews with clinical trial participants and outlines two approaches to conducting qualitative research in the clinical trial setting, with a focus on maximizing the value of the resulting data.

P4-045: Longitudinal ADC changes in Alzheimer's disease in a multi-center clinical trial setting

2011 ◽  
Vol 7 ◽  
pp. S713-S713
Author(s):  
Luc Bracoud ◽  
Angelika Caputo ◽  
Chahin Pachai ◽  
Boubakeur Belaroussi ◽  
Ana Graf ◽  
...  
Keyword(s):  
Alzheimer’S Disease ◽  
Alzheimer's Disease ◽  
Clinical Trial ◽  
Clinical Trial Setting ◽  
Trial Setting

Checkmate 577:Health-related quality of life (HRQoL) in a randomized, double-blind phase III study of nivolumab (NIVO) versus placebo (PBO) as adjuvant treatment in patients (pts) with resected esophageal or gastroesophageal junction cancer (EC/GEJC).

2021 ◽  
Vol 39 (3_suppl) ◽  
pp. 167-167
Author(s):  
Eric Van Cutsem ◽  
Prianka Singh ◽  
James M. Cleary ◽  
Ronan Joseph Kelly ◽  
Markus H. Moehler ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Visual Analogue Scale ◽  
Repeated Measures ◽  
Analogue Scale ◽  
Gastroesophageal Junction ◽  
Neoadjuvant Chemoradiotherapy ◽  
Phase Iii ◽  
Meaningful Improvement ◽  
Utility Index ◽  
Patient Reported

167 Background: NIVO is the first adjuvant therapy to provide a statistically significant and clinically meaningful improvement in disease-free survival (DFS) versus PBO in resected EC/GEJC following neoadjuvant chemoradiotherapy as demonstrated by CheckMate 577. NIVO was well tolerated with an acceptable safety profile. This analysis provides additional information on the exploratory HRQoL endpoints in this clinical trial. Methods: The effect of NIVO versus PBO on HRQoL, including general and disease-related symptoms, functioning, disease burden, and overall QoL, was assessed using FACT-E and EQ-5D-3L patient-reported outcome (PRO) questionnaires administered at baseline (BL), every 4 weeks during the 12-month treatment period, and at post-treatment follow-up visits (up to 2 years after last dose). Longitudinal change from BL in PRO scores over 12 months was assessed using descriptive statistics. Additionally, mixed model for repeated measures and time to deterioration analyses evaluated the difference between treatment with NIVO and PBO (data not shown). Results: 794 pts with EC/GEJC were randomized 2:1 to NIVO (n = 532) or PBO (n = 262). PRO completion rates were ≥ 95% at BL and ~ 90% at 12 months on treatment. Mean (SD) BL HRQoL scores were similar between treatment arms for the FACT-E total score (NIVO: 133.40 [20.97]; PBO: 134.03 [20.40]); esophageal cancer subscale (ECS; NIVO: 50.2 [9.3]; PBO: 50.1 [8.9]); EQ-5D Visual Analogue Scale (NIVO: 70.4 [22.3]; PBO: 69.1 [24.1]); and EQ-5D Utility Index (NIVO: 0.820 [0.179]; PBO: 0.831 [0.163]) based on the UK value set. Descriptive analyses showed a trend for increases from baseline at most time points through week 49 for both NIVO and PBO treatment groups for FACT-E total score, ECS, and EQ-5D Visual Analogue Scale and Utility Index. Conclusions: Preliminary results from CheckMate 577 demonstrated that pts on NIVO treatment showed trends of improvement in both esophageal-specific and general HRQoL. Similar trends were also observed in pts treated with PBO over 1 year. Pts treated with NIVO did not experience a reduction in HRQoL, further supporting clinical data to demonstrate treatment benefit and tolerability for adjuvant NIVO in pts with resected EC/GEJC. Clinical trial information: NCT02743494.

Data Security in Clinical Trials Using Blockchain Technology

2021 ◽  
pp. 250-268
Author(s):  
Marta de-Melo-Diogo ◽  
Jorge Tavares ◽  
Ângelo Nunes Luís
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Literature Review ◽  
Data Security ◽  
Critical Issue ◽  
The Other ◽  
Clinical Trial Setting ◽  
Blockchain Technology ◽  
Current Utilization ◽  
Trial Setting

Blockchain technology in a clinical trial setting is a valuable asset due to decentralization, immutability, transparency, and traceability features. For this chapter, a literature review was conducted to map the current utilization of blockchain systems in clinical trials, particularly data security managing systems and their characteristics, such as applicability, interests of use, limitations, and issues. The advantages of data security are producing a more transparent and tamper-proof clinical trial by providing accurate, validated data, therefore producing a more reliable and credible clinical trial. On the other hand, data integrity is a critical issue since data obtained from trials are not instantly made public to all participants. Work needs to be done to establish the significant implications in security data when applying blockchain technology in a real-world clinical trial setting and generalized conditions of use to establish its security.

Ibrutinib monotherapy outside of clinical trial setting in Waldenström macroglobulinaemia: practice patterns, toxicities and outcomes

2019 ◽  
Vol 188 (3) ◽  
pp. 394-403 ◽  
Author(s):  
Jithma P. Abeykoon ◽  
Saurabh Zanwar ◽  
Stephen M. Ansell ◽  
Morie A. Gertz ◽  
Shaji Kumar ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Practice Patterns ◽  
Clinical Trial Setting ◽  
Trial Setting

scholarly journals The impact of patient-reported outcome (PRO) data from clinical trials: a systematic review and critical analysis

2019 ◽  
Vol 17 (1) ◽  
Author(s):  
Samantha Cruz Rivera ◽  
Derek G. Kyte ◽  
Olalekan Lee Aiyegbusi ◽  
Anita L. Slade ◽  
Christel McMullan ◽  
...  
Keyword(s):  
Systematic Review ◽  
Clinical Trial ◽  
Clinical Trials ◽  
Case Studies ◽  
Real World ◽  
Clinical Trial Data ◽  
Research Impact ◽  
Trial Data ◽  
Patient Reported ◽  
The Impact

Abstract Background Patient-reported outcomes (PROs) are commonly collected in clinical trials and should provide impactful evidence on the effect of interventions on patient symptoms and quality of life. However, it is unclear how PRO impact is currently realised in practice. In addition, the different types of impact associated with PRO trial results, their barriers and facilitators, and appropriate impact metrics are not well defined. Therefore, our objectives were: i) to determine the range of potential impacts from PRO clinical trial data, ii) identify potential PRO impact metrics and iii) identify barriers/facilitators to maximising PRO impact; and iv) to examine real-world evidence of PRO trial data impact based on Research Excellence Framework (REF) impact case studies. Methods Two independent investigators searched MEDLINE, EMBASE, CINAHL+, HMIC databases from inception until December 2018. Articles were eligible if they discussed research impact in the context of PRO clinical trial data. In addition, the REF 2014 database was systematically searched. REF impact case studies were included if they incorporated PRO data in a clinical trial. Results Thirty-nine publications of eleven thousand four hundred eighty screened met the inclusion criteria. Nine types of PRO trial impact were identified; the most frequent of which centred around PRO data informing clinical decision-making. The included publications identified several barriers and facilitators around PRO trial design, conduct, analysis and report that can hinder or promote the impact of PRO trial data. Sixty-nine out of two hundred nine screened REF 2014 case studies were included. 12 (17%) REF case studies led to demonstrable impact including changes to international guidelines; national guidelines; influencing cost-effectiveness analysis; and influencing drug approvals. Conclusions PRO trial data may potentially lead to a range of benefits for patients and society, which can be measured through appropriate impact metrics. However, in practice there is relatively limited evidence demonstrating directly attributable and indirect real world PRO-related research impact. In part, this is due to the wider challenges of measuring the impact of research and PRO-specific issues around design, conduct, analysis and reporting. Adherence to guidelines and multi-stakeholder collaboration is essential to maximise the use of PRO trial data, facilitate impact and minimise research waste. Trial registration Systematic Review registration PROSPERO CRD42017067799.

scholarly journals Implementing a standard-of-care clinic for stroke prevention in children with sickle cell disease in Nigeria: a feasible strategy outside a clinical trial setting

2017 ◽  
Vol 1 (Suppl) ◽  
pp. 23-25
Author(s):  
Najibah A. Galadanci ◽  
Shehu U. Abdullahi ◽  
Leah D. Vance ◽  
Musa A. Tabari ◽  
Shehi Abubakar ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Stroke Prevention ◽  
Standard Of Care ◽  
Cell Disease ◽  
Care Clinic ◽  
Clinical Trial Setting ◽  
Trial Setting

scholarly journals Development of a new patient-reported outcome (PRO) measure on the Impact of Nighttime Urination (INTU) in patients with nocturia-Psychometric validation

2018 ◽  
Vol 37 (5) ◽  
pp. 1678-1685 ◽  
Author(s):  
Jason B. Bennett ◽  
Kristin Khalaf Gillard ◽  
Benjamin Banderas ◽  
Steven Abrams ◽  
Linda Cheng ◽  
...  
Keyword(s):  
Patient Reported Outcome ◽  
Psychometric Validation ◽  
Patient Reported ◽  
The Impact

FOLFIRINOX in locally advanced or metastatic pancreatic cancer.

2012 ◽  
Vol 30 (4_suppl) ◽  
pp. 313-313 ◽  
Author(s):  
Jason Edward Faris ◽  
Theodore S. Hong ◽  
Shaunagh McDermott ◽  
Alexander R Guimaraes ◽  
Dushyant Sahani ◽  
...  
Keyword(s):  
Pancreatic Cancer ◽  
Clinical Trial ◽  
Advanced Disease ◽  
Locally Advanced ◽  
Phase Iii ◽  
Metastatic Pancreatic Cancer ◽  
Clinical Trial Setting ◽  
Locally Advanced Disease ◽  
Grade 3 ◽  
Trial Setting

313 Background: The recently published Phase III trial of 5-FU, leucovorin, irinotecan, and oxaliplatin (FOLFIRINOX) demonstrated improved survival compared to gemcitabine in good performance status (PS) patients with metastatic pancreatic cancer (Conroy et al, NEJM 2011). Less is known about the efficacy and tolerability with FOLFIRINOX in the non-clinical trial setting. In this retrospective analysis, we report our institutional experience with FOLFIRINOX. Methods: 29 patients with locally advanced or metastatic pancreatic cancer treated with FOLFIRINOX between July 2010 and April 2011 were used for this analysis. Clinical characteristics, and gradeable toxicities were tabulated, and formal radiographic review performed to determine best overall response rates (ORR). Results: 17 patients received FOLFIRINOX for metastatic disease and 12 patients for locally advanced disease. The median age of patients was 60 (range 39-76). 22/29 patients were men. 18/29 patients had received no prior chemotherapy. There was one patient with PS 2; all others had PS 0 or 1. 8/29 patients had biliary stents. Overall, 11 partial responses (PR) were observed (ORR 38%); 10/11 partial responses were in chemo-naïve patients, who had an ORR of 56%. In the metastatic setting, there were 6 PR, for an ORR of 35%, and 7 patients with stable disease (SD). In the locally advanced setting, there were 5 PR (ORR 42%), and 7 patients with SD. Following treatment with FOLFIRINOX, one patient with locally advanced disease has subsequently undergone R0 resection. The median number of cycles performed was 8 in both the locally advanced and metastatic settings. 12/29 patients required an ED visit or hospitalization during treatment. Grade 3/4 neutropenia was observed in 10 patients; 7/10 had not received prophylactic growth factor treatment from the start of FOLFIRINOX. 4 patients developed febrile neutropenia, 4 patients developed grade 3/4 thrombocytopenia, and 1 patient developed grade 4 anemia. Conclusions: In a non-clinical trial setting, FOLFIRINOX demonstrated activity in both the metastatic and locally-advanced settings. FOLFIRINOX appears to be associated with manageable, but significant toxicities, with over 40% of patients requiring hospitalization.

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