Pulse Brain · Growing Health Evidence Index
Peer-reviewed

The attitude of patients with progressive ataxias towards clinical trials

Gilbert Thomas‐Black, Andrada Dumitrascu, Héctor García‐Moreno, Julie Vallortigara, Julie Greenfield, Barry Hunt, Susan Walther, Mackenzie Wells, David R. Lynch, Hugh Montgomery, Paola Giunti

Orphanet Journal of Rare Diseases · 2022

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Summary

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 stu

Source type
Peer-reviewed study
DOI
10.1186/s13023-021-02091-x
Catalogue ID
BFmokjo2bz-0qp3yv
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