Transition to Adulthood in People With Muscular Dystrophy

Summary

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.

Conditions

• Duchenne / Becker Muscular Dystrophy

Interventions

DEVICE

VIRTUAL REALITY

The interventional part of the study is limited to 3 centers. It explores SOCIAL COGNITION throught a newly developed paradigm in Virtual Reality. In detail: an adapted version of the Virtual Reality Social Prediction Improvement and Rehabilitation Intensive Training (VR-SPIRIT) using a VR Head Mounted Display (HMD) will be implemented on a portable HMD, the Oculus Quest, equipped with two controllers with motion sensors that are used to track hand position. In a VR-SPIRIT session, participants are immersed in the playground scenario and engaged in a competition with four avatars. Each avatar has a pre-established probability to move toward one of the three described games. Different indices about a partecipant's performance are recorded automatically by the system during the session, such as the duration of each trial and the prediction score (percentage of trials in which the participant correctly predicted an avatar's intention).

Sponsors & Collaborators

• Università di Padova Italy

  collaborator UNKNOWN

• Fondazione Istituto Neurologico Nazionale Casimiro Mondino - IRCCS

  collaborator UNKNOWN

• Istituto Giannina Gaslini - IRCCS

  collaborator UNKNOWN

• Fondazione Serena Onlus - Centro Clinico NeMO Milano

  collaborator OTHER

• Centro Clinico NeMO Ancona

  collaborator UNKNOWN

• Fondazione Stella Maris - IRCCS

  collaborator UNKNOWN

• Università degli Studi di Milano Statale

  collaborator UNKNOWN

• Centro Clinico NeMO Brescia

  collaborator UNKNOWN

• Fondazione Istituto Neurologico Carlo Besta - IRCCS

  collaborator UNKNOWN

• Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

  collaborator OTHER

• I.R.C.C.S. Eugenio Medea - Associazione La Nostra Famiglia - Sede di Conegliano

  collaborator UNKNOWN

• Fondazione Policlinico Universitario Agostino Gemelli IRCCS

  collaborator OTHER

• Dipartimento di Neuroscienze Rita Levi Montalcini Torino

  collaborator UNKNOWN

• Università Vita-Salute San Raffaele

  collaborator OTHER

• Università di Pisa

  collaborator UNKNOWN

• Azienda Ospedaliera Universitaria Policlinico "G. Martino"

  collaborator OTHER

• Associazione La Nostra Famiglia Eugenio Medea - Brindisi

  collaborator UNKNOWN

• Scientific Institute, IRCCS E.Medea, Pasian di Prato

  collaborator UNKNOWN

• Istituto delle Scienze Neurologiche di Bologna - IRCCS

  collaborator UNKNOWN

• IRCCS Eugenio Medea

  lead OTHER

Study Design

Allocation

NA

Purpose

OTHER

Masking

NONE

Model

SINGLE_GROUP

Eligibility

Min Age

14 Years

Sex

MALE

Healthy Volunteers

No

Timeline & Regulatory

Start

2025-09-30

Primary Completion

2027-08-31

Completion

2027-08-31

Countries

• Italy

Study Locations