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

Article type: Meeting Report

Authors: Tizzano, Eduardo F.^a | Christie-Brown, Vanessa^b | Baranello, Giovanni^c | Germanenko, Olga^d | Gray, Allyson^e | Krstic, Marija^f | Lilien, Charlotte^g | Patel, Hinal^h | Servais, Laurentⁱ | Scoto, Mariacristina^{j; *}

Affiliations: [a] Department of Clinical and Molecular Genetics Hospital Vall d'Hebron, and Medicine Genetics Group, Vall d’Hebron Research Institute (VHIR), 08035 Barcelona, Spain | [b] Research Programme Manager, SMA Europe e.V., Freiburg, Germany | [c] The Dubowitz Neuromuscular Centre, NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College London, & Great Ormond Street Hospital NHS Foundation Trust, London, UK | [d] Patient Representative, Head of SMA Family Foundation Russia & Delegate of SMA Europe e. V., Freiburg, Germany | [e] Paediatric Research Nurse, Great Ormond Street Hospital NHS Trust, London, United Kingdom | [f] Patient Representative, SMA Serbia & Delegate of SMA Europe e.V., Freiburg, Germany | [g] MPT: MDUK Oxford Neuromuscular Centre, Oxford, United Kingdom | [h] The Dubowitz Neuromuscular Centre, NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College London, & Great Ormond Street Hospital NHS Foundation Trust, London, UK | [i] MDUK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford | [j] The Dubowitz Neuromuscular Centre, Great Ormond Street Hospital NHS Foundation Trust, London, UK & NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College London

Correspondence: [*] Correspondence to: Mariacristina Scoto, The Dubowitz Neuromuscular Centre, Great Ormond Street Hospital NHS Foundation Trust, London, UK & NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College London. E-mail: [email protected].

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.

Keywords: Spinal muscular atrophy, therapeutic advances, clinical trial readiness, education and training

DOI: 10.3233/JND-221538

Journal: Journal of Neuromuscular Diseases, vol. 9, no. 6, pp. 809-820, 2022