The attitude of patients with progressive ataxias towards clinical trials

Orphanet Journal of Rare Diseases

Barry Hunt¹  Julie Greenfield¹  Julie Vallortigara²  Andrada Dumitrascu²  Hector Garcia-Moreno³  Paola Giunti³  Gilbert Thomas-Black³  David R. Lynch⁴  Mackenzie Wells⁴  Susan Walther⁵  Hugh Montgomery⁶ 

[1] Ataxia UK, 12 Broadbent Close, N6 5JW, London, UK;Department of Clinical and Movement Neurosciences, The Ataxia Centre, UCL Queen Square Institute of Neurology, University College London, London, UK;Department of Clinical and Movement Neurosciences, The Ataxia Centre, UCL Queen Square Institute of Neurology, University College London, London, UK;National Hospital for Neurology and Neurosurgery, University College London Hospitals Foundation NHS Trust, London, UK;Departments of Pediatrics and Neurology, Children’s Hospital of Philadelphia, 19104, Philadelphia, PA, USA;Friedreich‘S Ataxia Research Alliance, 533 W Uwchlan Ave, 19335, Downingtown, PA, USA;Institute of Sport Exercise and Health, University College London, London, UK;

关键词: Clinical trials;    Patient attitude;    Ataxias;    Trial design;    Trial participation;    Trial investigations;    Time commitment;    Drug administration;   

DOI  :  10.1186/s13023-021-02091-x

来源: Springer

【 摘 要 】

BackgroundThe 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.MethodsWe 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.ResultsOf 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.ConclusionsKnowledge 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.

【 授权许可】

CC BY