Transition to Adulthood in People With Muscular Dystrophy

The study will involve boys with Duchenne Muscular Dystrophy (DMD), from adolescence to adulthood living in Italy, and their families/ caregivers. The Investigators want to explore patients' mental health, social cognition and perception of body image as well as patients'and caregivers' ability to adapt to changes related to both the disease and various personal and social needs, through the use of short and simple questionnaires, neuropsychological tests and neurocognitive evaluation in ecological scenarios using Virtual Reality. The desire for autonomy and the awareness of both personal and social difficulties in achieving them put a strain on both patients and their families.Some studies conducted in the USA and in various European countries underline that the transition to adulthood of patients with muscular dystrophy is featured by a good ability to adapt to the more advanced stages of the disease in general; nevertheless, facing the worsening of health entails experiences of anxiety and depression.In this historical phase, clinical trials of gene therapy and gene modulating therapies are involving an increasing number of patients, mainly represented by children in the early phase of the disease. Hence, adolescents and young adults could develop feelings of exclusion from research efforts, losing confidence and hope in any future improvement of their condition, social participation and quality of life.This study could lead to identify strengths and weaknesses in social cognition and psychosocial adaptation; through the evaluation of characteristics of patients/caregivers or exogenous factors influencing higher levels of social adaptation, we ultimately aim to define strategies to improve not only physical (already defined by guidelines) but also psychological care, social cognition and social support in all stages of DMD, particularly in later ones.

NCT Number	NCT07101185
Lead Sponsor	IRCCS Eugenio Medea Collaborators: University of Padova, IRCCS National Neurological Institute "C. Mondino" Foundation, Istituto Giannina Gaslini - IRCCS, Fondazione Serena Onlus - Centro Clinico NeMO Milano, Centro Clinico NeMO Ancona, IRCCS Fondazione Stella Maris, Università degli Studi di Milano Statale, Centro Clinico NeMO Brescia, Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, I.R.C.C.S. Eugenio Medea - Associazione La Nostra Famiglia - Sede di Conegliano, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Dipartimento di Neuroscienze Rita Levi Montalcini Torino, Università Vita-Salute San Raffaele, Università di Pisa, Azienda Ospedaliera Universitaria Policlinico "G. Martino", Associazione La Nostra Famiglia Eugenio Medea - Brindisi, Scientific Institute, IRCCS E.Medea, Pasian di Prato, IRCCS Istituto delle Scienze Neurologiche di Bologna
Conditions	Duchenne / Becker Muscular Dystrophy
Enrollment	250 participants
Start Date	2025-09
Primary Completion	2027-08 (estimated)
Study Completion	2027-08 (estimated)
Updated on ClinicalTrials.gov	2025-08-03