Evidence map›Paper›PMID 34983593›Full record

ArticleOrphanet journal of rare diseases2022

The attitude of patients with progressive ataxias towards clinical trials.

Gilbert Thomas-Black, Andrada Dumitrascu, Hector Garcia-Moreno, Julie Vallortigara, Julie Greenfield, Barry Hunt, Susan Walther, Mackenzie Wells, David R Lynch, Hugh Montgomery and 1 more

Open access · goldAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Orphanet journal of rare diseases, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
9citing papers in PubMed, 1 pooled it
2.7field-weighted citation impact, top 9% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

9 citing papers in PubMed, 1 synthesis or guideline pooled it, 28 citations in OpenAlex.

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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

11 authors at 6 institutions in 2 countries.

Gilbert Thomas-BlackDepartment of Clinical and Movement Neurosciences, The Ataxia Centre, UCL Queen Square Institute of Neurology, University College London, London, UK.
Andrada DumitrascuDepartment of Clinical and Movement Neurosciences, The Ataxia Centre, UCL Queen Square Institute of Neurology, University College London, London, UK.
Hector Garcia-MorenoDepartment of Clinical and Movement Neurosciences, The Ataxia Centre, UCL Queen Square Institute of Neurology, University College London, London, UK.
Julie VallortigaraDepartment of Clinical and Movement Neurosciences, The Ataxia Centre, UCL Queen Square Institute of Neurology, University College London, London, UK.
Julie GreenfieldAtaxia UK, 12 Broadbent Close, N6 5JW, London, UK.
Barry HuntAtaxia UK, 12 Broadbent Close, N6 5JW, London, UK.
Susan WaltherFriedreich'S Ataxia Research Alliance, 533 W Uwchlan Ave, Downingtown, PA, 19335, USA.
Mackenzie WellsDepartments of Pediatrics and Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, 19104, USA.
David R LynchDepartments of Pediatrics and Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, 19104, USA.
Hugh MontgomeryInstitute of Sport Exercise and Health, University College London, London, UK.
Paola GiuntiDepartment of Clinical and Movement Neurosciences, The Ataxia Centre, UCL Queen Square Institute of Neurology, University College London, London, UK. p.giunti@ucl.ac.uk.ORCID 0000-0003-3508-4788
University College London Hospitals NHS Foundation Trust · GBAtaxia UK · GBChildren's Hospital of Philadelphia · USNational Hospital for Neurology and Neurosurgery · GBEnglish Institute of Sport · GBFriedreich's Ataxia Research Alliance · US

Funding

Medical Research Council MR/N028767/1
6 · The paper itself

Abstract

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.

Indexed as

Cerebellar AtaxiaFriedreich AtaxiaSpinocerebellar DegenerationsClinical Trials as TopicHumansMotivationResearch DesignAtaxiasClinical trialsDrug administrationPatient attitudeTime commitmentTrial designTrial investigationsTrial participation

Identifiers

PMID34983593
PMCPMC8729009
OpenAlexW4221096694

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.