The Lived Experience of Pediatric Gene Therapy Clinical Trial in Duchenne Muscular Dystrophy: Exploring Perceptions of Parents and Professionals Using Social Representation Method.

Abstract

In recent decades, medical and scientific advances have led to the development of new therapeutic approaches for Duchenne muscular dystrophy (DMD), including gene therapy (GT), which is currently being evaluated. Recruiting enough children in clinical trials remains a challenge, depending on parental decisions. Numerous studies have already been carried out to understand these decision-making factors. To date, no study in Europe has been conducted among the various stakeholders lived experience in a DMD GT trial. Our qualitative study explored participants' perceptions using a social representation method and compared them. We recruited 42 participants, divided into 2 groups comprising 21 parents and 21 professionals participating in GNT-014, a DMD natural history study. Each participant was interviewed on four questions about clinical trials, GT, and the facilitators and barriers of the clinical trial pathway. A prototypical and categorical analysis was carried out using "Pointe-au-Sel" software to analyze the data quantitatively. This method highlights which perceptions are shared within the same group and brings out the most important and most frequently evoked terms. We exported the data as a superimposed scatterplot of the representations of both groups for each question. We obtained a total of 453 evocations for the parents' group and a total of 611 evocations for the professionals' group. For clinical trial and GT, hope and scientific progress are common to the core of both groups but are not at the same level of representation. Parents evoked human contact as the main facilitator and what their child may undergo and become for barriers. For professionals, the facilitators and barriers are centered on the terms that can influence the proper conduct of the trial. These comparative results imply that the vision of the different stakeholders is not totally shared in trial participation. On the contrary, the term GT may also have an influence on professionals, including caregivers.