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Clinical Trial Readiness for Spinal Muscular Atrophy: Experience of an International Educational-Training Initiative

Tizzano, Eduardo F; Christie-Brown, Vanessa; Baranello, Giovanni; Germanenko, Olga; Gray, Allyson; Krstic, Marija; Lilien, Charlotte; ... Scoto, Mariacristina; + view all (2022) Clinical Trial Readiness for Spinal Muscular Atrophy: Experience of an International Educational-Training Initiative. Journal of Neuromuscular Diseases , 9 (6) pp. 809-820. 10.3233/JND-221538. Green open access

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Abstract

Several successful clinical trials have been conducted in spinal muscular atrophy (SMA) over recent years which have led to the approval of splicing modifiers and gene transfer therapies. With an increasing number of other agents progressing through pre-clinical and clinical development, increasing worldwide clinical trial readiness is becoming essential.SMA Europe initiated a clinical trial readiness project, which included the development of a pilot face-to-face educational-training initiative for clinical specialists and physiotherapists involved in SMA, with an emphasis on the patient perspective. Participants were selected through two surveys and, ahead of the meeting, a mock protocol with specific questions was provided. The initiative involved a series of presentations, role-play and interactive exercises. We describe here our experience and evaluation of this educational-training initiative, emphasising scientific aspects, psychosocial implications and level of satisfaction.From a participant, patient and industry perspective, such training was considered successful and met the objective, which was to improve clinical trial readiness in emerging sites. Resource planning, ethical considerations and communication with patients were identified as three important topics for future training. This initiative highlights the need to develop a training programme to achieve clinical trial readiness across Europe and showcases a collaborative effort with different stakeholders, clinicians, patient advocacy groups and sponsors to address an important issue.

Type: Article
Title: Clinical Trial Readiness for Spinal Muscular Atrophy: Experience of an International Educational-Training Initiative
Location: Netherlands
Open access status: An open access version is available from UCL Discovery
DOI: 10.3233/JND-221538
Publisher version: http://dx.doi.org/10.3233/JND-221538
Language: English
Additional information: This version is the author accepted manuscript. For information on re-use, please refer to the publisher’s terms and conditions.
Keywords: Europe, Exercise, Humans, Muscular Atrophy, Spinal, Surveys and Questionnaires, clinical trial readiness, education and training, Spinal muscular atrophy, therapeutic advances
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Developmental Neurosciences Dept
URI: https://discovery.ucl.ac.uk/id/eprint/10164623
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