scholarly journals The Attitude of Patients with Progressive Ataxias Towards Clinical Trials

2021 ◽  
Author(s):  
Gilbert Thomas-Black ◽  
Andrada Dumitrascu ◽  
Hector Garcia-Moreno ◽  
Julie Vallortigara ◽  
Julie Greenfield ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Drug Administration ◽  
Online Survey ◽  
Drug Repurposing ◽  
Friedreich’S Ataxia ◽  
Friedreich's Ataxia ◽  
Therapeutic Interventions ◽  
Human Experimentation ◽  
Trial Participation ◽  
Routes Of Drug Administration

Abstract Background: The development of new therapies may rely on the conduct of human experimentation as well as later clinical trials of therapeutic interventions. Ethical considerations seek to protect the patient from risk but few have sought to ascertain the attitude to such risk of patients with progressive debilitating or terminal conditions, for which no mitigating or curative therapies exist. Such understanding is also important if recruitment is to be maximized. We therefore sought to define the motivations for and barriers to trial participation amongst patients with progressive ataxias, as well as their condition-specific trial preferences. Methods: We conducted an online survey consisting of 29 questions covering four key domains (demographics, personal motivation, drug therapy and study design) relating to the design of clinical trials. Two major ataxia charities, Ataxia UK and the Friedreich’s Ataxia Research Alliance (FARA) sent the survey to their members. Responses were analysed by disease and by ambulatory status.Results: Of 342 respondents, 204 reported a diagnosis of Friedreich’s ataxia (FRDA), 55 inherited cerebellar ataxia (CA) and 70 idiopathic CA. The most important symptoms to be addressed by a trial were considered to be balance problems and ambulation, although these were superseded by speech problems in wheelchair users. Common motivations for participation were potential benefits to self and others. Reasons for non-participation included concerns about side effects, and the burden and cost of travel. Financial reimbursement for expenses was reported to be likely to increase trial engagement, Phase two trials were the most popular to participate in, and the use of a placebo arm was seen as a disincentive. Across all disease subgroups, drug repurposing trials proved popular and just under 70% of participants would be prepared to undergo intrathecal drug administration.Conclusions: Knowledge of motivations for and barriers to trial participation as well as the acceptability of investigations, time commitments and routes of drug administration should inform better, more patient focused trial design. This in turn may improve recruitment and retention of participants to future trials.

scholarly journals The attitude of patients with progressive ataxias towards clinical trials

2022 ◽  
Vol 17 (1) ◽  
Author(s):  
Gilbert Thomas-Black ◽  
Andrada Dumitrascu ◽  
Hector Garcia-Moreno ◽  
Julie Vallortigara ◽  
Julie Greenfield ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Drug Administration ◽  
Online Survey ◽  
Drug Repurposing ◽  
Friedreich’S Ataxia ◽  
Friedreich's Ataxia ◽  
Therapeutic Interventions ◽  
Human Experimentation ◽  
Trial Participation ◽  
Routes Of Drug Administration

Abstract Background The development of new therapies may rely on the conduct of human experimentation as well as later clinical trials of therapeutic interventions. Ethical considerations seek to protect the patient from risk but few have sought to ascertain the attitude to such risk of patients with progressive debilitating or terminal conditions, for which no mitigating or curative therapies exist. Such understanding is also important if recruitment is to be maximized. We therefore sought to define the motivations for and barriers to trial participation amongst patients with progressive ataxias, as well as their condition-specific trial preferences. Methods We conducted an online survey consisting of 29 questions covering four key domains (demographics, personal motivation, drug therapy and study design) relating to the design of clinical trials. Two major ataxia charities, Ataxia UK and the Friedreich’s Ataxia Research Alliance (FARA) sent the survey to their members. Responses were analysed by disease and by ambulatory status. Results Of 342 respondents, 204 reported a diagnosis of Friedreich’s ataxia (FRDA), 55 inherited cerebellar ataxia (CA) and 70 idiopathic CA. The most important symptoms to be addressed by a trial were considered to be balance problems and ambulation, although these were superseded by speech problems in wheelchair users. Common motivations for participation were potential benefits to self and others. Reasons for non-participation included concerns about side effects, and the burden and cost of travel. Financial reimbursement for expenses was reported to be likely to increase trial engagement, Phase two trials were the most popular to participate in, and the use of a placebo arm was seen as a disincentive. Across all disease subgroups, drug repurposing trials proved popular and just under 70% of participants would be prepared to undergo intrathecal drug administration. Conclusions Knowledge of motivations for and barriers to trial participation as well as the acceptability of investigations, time commitments and routes of drug administration should inform better, more patient focused trial design. This in turn may improve recruitment and retention of participants to future trials.

P003 INFLAMMATORY BOWEL DISEASE PATIENTS’ PERSPECTIVES OF CLINICAL TRIALS

2020 ◽  
Vol 26 (Supplement_1) ◽  
pp. S53-S53
Author(s):  
David Rubin ◽  
Laurent Peyrin-Biroulet ◽  
Walter Reinisch ◽  
Swati Tole ◽  
Laura Sullivan ◽  
...  
Keyword(s):  
Health Care ◽  
Clinical Trial ◽  
Clinical Trials ◽  
Health Care Providers ◽  
Online Survey ◽  
The United States ◽  
Trial Participation ◽  
Pharmaceutical Manufacturer ◽  
Care Providers ◽  
Inflammatory Bowel

Abstract Background Despite recent progress in treatment for inflammatory bowel diseases (IBD), there is a need for therapies with long-term efficacy and improved safety. Clinical trials in IBD face challenges with patient recruitment because of study designs, competitive or overlapping trials, and a limited number of eligible patients. We sought to better understand patients’ motivations, awareness of, and experience with IBD clinical trials. Methods We conducted an international survey of adult patients with IBD consisting of 2 components. The quantitative component, a 15-minute online survey, was completed by all patients. A qualitative component, a 30-minute telephone interview, was completed by a subset of patients from the United States (US). All percentages indicate results from the online survey. Results 226 patients (mean age, 41.9 y) completed the online survey. Survey respondents included patients with ulcerative colitis (52%) and Crohn’s disease (48%) from the US (n=100, 21 of whom underwent a phone interview), Brazil (n=26), Canada (n=25), France (n=25), Germany (n=25), and Spain (n=25). Ninety-six percent of respondents reported at least a basic understanding of clinical trials, and 34 (15%) were current or past clinical trial participants. Patients reported learning about trials through 1 or more sources (could select as many as applied): health care providers (42%), pharmaceutical manufacturer websites (31%), social media (30%), online support groups (28%), and foundations (18%-23%). In the survey, patients rated conversations with health care providers most helpful, but patients who were interviewed revealed that most physicians often do not initiate conversations about clinical trials, and patients typically do not ask. Primary motivators for trial participation (rated from “does not encourage me at all” to “encourages me very much”) included altruistic goals of advancing medicine (67%), potentially mitigating risks of uncontrolled IBD such as colon cancer (59%), and access to treatment options that could improve quality of life (59%) or would otherwise be unaffordable (52%). Major barriers to participation (rated from “does not discourage me at all” to “discourages me very much”) included invasive screening and monitoring (35%), concern over receiving placebo (35%), or suboptimal treatment (33%), and concerns about posttrial access to study medication (27%). The majority (68%) reported that being in a clinical trial means being a “guinea pig” for an experimental treatment. Conclusion Opportunities to improve patients’ clinical trial experience in IBD include better communication with health care providers and improved patient education about clinical trial design and ethics. Ultimately, a better understanding of the patient perspective will be important for more informed patients and potentially higher recruitment and enrollment.

Differentially Regulated Cell-Free MicroRNAs in the Plasma of Friedreich's Ataxia Patients and Their Association with Disease Pathology

2017 ◽  
Vol 49 (01) ◽  
pp. 035-043 ◽  
Author(s):  
Subrahamanyam Dantham ◽  
Achal Srivastava ◽  
Sheffali Gulati ◽  
Moganty Rajeswari
Keyword(s):  
Microarray Platform ◽  
Friedreich’S Ataxia ◽  
Friedreich's Ataxia ◽  
Therapeutic Interventions ◽  
Healthy Controls ◽  
Disease Pathogenesis ◽  
Multisystem Disease ◽  
Mirna Microarray ◽  
Thermo Fisher Scientific ◽  
Fisher Scientific

AbstractFriedreich's ataxia (FRDA) is a multisystem disease affecting the predominately nervous system, followed by muscle, heart, and pancreas. Current research focused on therapeutic interventions aimed at molecular amelioration, but there are no reliable noninvasive signatures available to understand disease pathogenesis. The present study investigates the alterations of plasma cell-free microRNAs (miRNAs) in FRDA patients and attempts to find the significance in relevance with the pathogenesis. Total RNA from the plasma of patients and healthy controls were subjected to miRNA microarray analysis using Agilent Technologies microarray platform. Differentially regulated miRNAs were validated by SYBR-green real-time polymerase chain reaction (Thermo Fisher Scientific). The study identified 20 deregulated miRNAs (false discovery rate < 0.01, fold change ≥ 2.0 ≤) in comparison with healthy controls; out of which 17 miRNAs were upregulated, and 3 miRNAs were downregulated. Target and pathway analysis of these miRNAs have shown association with neurodegenerative and other clinical features in FRDA. Further validation (n = 21) identified a set of significant (p < 0.05) deregulated miRNAs; hsa-miR-15a-5p, hsa-miR-26a-5p, hsa-miR-29a-3p, hsa-miR-223–3p, hsa-24–3p, and hsa-miR-21–5p in comparison with healthy controls. These miRNAs were reported to influence various pathological features associated with FRDA. The present study is expected to aid in the understanding of disease pathogenesis.

scholarly journals Antioxidant Therapies and Oxidative Stress in Friedreich’s Ataxia: The Right Path or Just a Diversion?

Antioxidants ◽  
2020 ◽  
Vol 9 (8) ◽  
pp. 664 ◽  
Author(s):  
Laura R. Rodríguez ◽  
Tamara Lapeña ◽  
Pablo Calap-Quintana ◽  
María Dolores Moltó ◽  
Pilar Gonzalez-Cabo ◽  
...  
Keyword(s):  
Oxidative Stress ◽  
Clinical Trials ◽  
Autosomal Recessive ◽  
Friedreich’S Ataxia ◽  
Friedreich's Ataxia ◽  
Antioxidant Defenses ◽  
Stress Markers ◽  
The One ◽  
The Right ◽  
And Oxidative Stress

Friedreich’s ataxia is the commonest autosomal recessive ataxia among population of European descent. Despite the huge advances performed in the last decades, a cure still remains elusive. One of the most studied hallmarks of the disease is the increased production of oxidative stress markers in patients and models. This feature has been the motivation to develop treatments that aim to counteract such boost of free radicals and to enhance the production of antioxidant defenses. In this work, we present and critically review those “antioxidant” drugs that went beyond the disease’s models and were approved for its application in clinical trials. The evaluation of these trials highlights some crucial aspects of the FRDA research. On the one hand, the analysis contributes to elucidate whether oxidative stress plays a central role or whether it is only an epiphenomenon. On the other hand, it comments on some limitations in the current trials that complicate the analysis and interpretation of their outcome. We also include some suggestions that will be interesting to implement in future studies and clinical trials.

scholarly journals Results from a Theory-Guided Survey to Support Breast Cancer Trial Participation: Barriers, Enablers, and What to Do about them

2021 ◽  
Vol 28 (3) ◽  
pp. 2014-2028
Author(s):  
Jamie C. Brehaut ◽  
Kelly Carroll ◽  
Jenn Gordon ◽  
Justin Presseau ◽  
Dawn P. Richards ◽  
...  
Keyword(s):  
Breast Cancer ◽  
Clinical Trials ◽  
Online Survey ◽  
Theoretical Domains Framework ◽  
Previous Experience ◽  
Trial Participation ◽  
Detailed Knowledge ◽  
Cancer Trial ◽  
Comprehensive List ◽  
Barriers And Enablers

Background: Ensuring adequate, informed, and timely participation in clinical trials is a multifactorial problem. We have previously developed a systematic, tailorable survey development approach that is informed by theory, can identify barriers and enablers to participation, and can suggest recruitment strategies to address these issues. In this study, we surveyed subscribers to the Canadian Breast Cancer Network (CBCN) in order to identify a comprehensive list of theory-informed barriers and enablers relevant to participation in a hypothetical breast cancer trial. Methods: We developed and conducted an online survey of breast cancer patients informed by the Theoretical Domains Framework and designed to determine previous experience with clinical trials, knowledge about clinical trials, and importance of a comprehensive list of barriers and enablers to trial participation. Participants were contacted by email or through social media. Results: From 2451 subscribers of the CBCN, we received 244 responses and 210 completed surveys (244/2451 or 9.9% participation, 210/244 or 86.1% completion). A total of 38% of respondents indicated experience in trial participation, but 83% indicated confidence in their knowledge about clinical trials. Those who had previously participated in clinical trials were more confident in their knowledge (χ2= 6.77, p = 0.009) and answered more knowledge questions (t = −3.90 p = 0.000). Endorsed barriers and enablers to participation included 39 factors across 12 of 14 domains relevant to behaviour change. Our approach identifies barriers that might be meaningfully addressed by careful knowledge provision (‘If I would learn more about my condition’; ‘If I find the trial documents hard to understand’), those that may require other theory-informed approaches to address (‘my feelings about the quality of my drug plan’; ‘my worry over unknown side effects’), and those that may require tailored approaches depending on participant differences such as previous experience in trials (‘If there were patient-friendly decision-making tools to help you make your participation decision’). Discussion: This work demonstrates that a comprehensive, theory-guided survey of barriers and enablers to participation in breast cancer clinical trials is feasible, can lead to detailed knowledge about the issues related to participation in specific trials, and most importantly, can lead to insights about evidence-based ways to better support patient participation.

scholarly journals Primary care physicians’ attitudes and beliefs about cancer clinical trials

2017 ◽  
Vol 14 (5) ◽  
pp. 518-525 ◽  
Author(s):  
Carma L Bylund ◽  
Elisa S Weiss ◽  
Margo Michaels ◽  
Shilpa Patel ◽  
Thomas A D’Agostino ◽  
...  
Keyword(s):  
Primary Care ◽  
Clinical Trials ◽  
Primary Care Physicians ◽  
Online Survey ◽  
Minority Groups ◽  
Clinical Care ◽  
Trial Participation ◽  
Cancer Clinical Trials ◽  
Attitudes And Beliefs ◽  
Positive Attitudes

Background/Aims: Cancer clinical trials give patients access to state-of-the-art treatments and facilitate the translation of findings into mainstream clinical care. However, patients from racial and ethnic minority groups remain underrepresented in clinical trials. Primary care physicians are a trusted source of information for patients, yet their role in decision-making about cancer treatment and referrals to trial participation has received little attention. The aim of this study was to determine physicians’ knowledge, attitudes, and beliefs about cancer clinical trials, their experience with trials, and their interest in appropriate training about trials. Methods: A total of 613 physicians in the New York City area primarily serving patients from ethnic and racial minority groups were invited via email to participate in a 20-min online survey. Physicians were asked about their patient population, trial knowledge and attitudes, interest in training, and personal demographics. Using calculated scale variables, we used descriptive statistical analyses to better understand physicians’ knowledge, attitudes, and beliefs about trials. Results: A total of 127 physicians completed the survey. Overall, they had low knowledge about and little experience with trials. However, they generally had positive attitudes toward trials, with 41.4% indicating a strong interest in learning more about their role in trials, and 35.7% indicating that they might be interested. Results suggest that Black and Latino physicians and those with more positive attitudes and beliefs were more likely to be interested in future training opportunities. Conclusion: Primary care physicians may be an important group to target in trying to improve cancer clinical trial participation among minority patients. Future work should explore methods of educational intervention for such interested providers.

Friedreich's Ataxia: From Disease Mechanisms to Therapeutic Interventions

2006 ◽  
Vol 8 (3-4) ◽  
pp. 438-443 ◽  
Author(s):  
Raffaele Lodi ◽  
Caterina Tonon ◽  
Vittorio Calabrese ◽  
Anthony H.V. Schapira
Keyword(s):  
Friedreich’S Ataxia ◽  
Friedreich's Ataxia ◽  
Therapeutic Interventions ◽  
Disease Mechanisms

Cancer clinical trials education: Insights from patient experiences and preferences.

2017 ◽  
Vol 35 (15_suppl) ◽  
pp. e21554-e21554
Author(s):  
Claire Saxton
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Cancer Patients ◽  
Online Survey ◽  
Patient Experiences ◽  
Trial Participation ◽  
Trial Participant ◽  
Healthcare Team ◽  
Cancer Support ◽  
Clinical Trial Information

e21554 Background: Clinical trials offer cancer patients access to innovative treatments, quality care, and hope for a better future. Yet, participation rates across adult cancers remain low. Education is a target intervention. The following research examines patient experiences, beliefs, and preferences related to cancer clinical trial participation. Methods: In 2015-16, the Cancer Support Community conducted an online survey with the goal of developing an educational program that would increase clinical trial awareness and participation. Survey responses from 506 cancer patients were collected. Most respondents were female (85.5%) and Caucasian (84.8%). The average age was 58.7 years old. Nearly half (47.2%) were diagnosed with breast cancer. Of the respondents, 48.3% were currently receiving treatment. Results: Most (81.4%) had heard of clinical trials prior to diagnosis. While 36.3% had participated in trials, 52.5% never considered it. Results found that 56.7% had discussed clinical trials with their healthcare team during treatment, but only 20.2% felt that their goals and concerns were meaningfully addressed. Additionally, 93.1% reported that more time with their doctors to discuss clinical trials would be “somewhat” or “very” helpful. Websites with clinical trial information were shared with just 34.4% of respondents. Survey results indicated that 91.9% felt that a website including clinical trial information would be “somewhat” or “very” helpful. Specifically, 60.2% of respondents reported that a website with patient narratives would be “very” helpful. While, 60.9% felt that tools to help locate clinical trials would be “very” helpful. Most had not been offered or couldn’t independently locate videos (80.7%) or illustrations/images (73.8%) explaining clinical trials. Of the respondents, 94.0% reported that speaking directly with a clinical trial participant would be helpful. Yet, this was only offered to 10.3% of patients. Conclusions: Findings were used to develop content for educational products and programs delivered by CSC for those potentially eligible for clinical trials. Survey data highlight the importance of providing information relevant to eligible participants across multiple formats.

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