Transition to Adulthood in People With Muscular Dystrophy

Trial statusNot yet recruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexMale
Age14+
SponsorIRCCS Eugenio Medea

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Defined diagnosis of DMD

Age > 14 years

Willingness and ability to comply to study procedures

Comprehension of the study and Signature of the informed consent

Disqualifiers

Clinical history of cerebral palsy, severe intellectual deficit, severe behavioral disturbances

absence of compliance to the studyof the patient and the caregivers.

Trial design

Design model

Single group

Treatments tested in this trial

  • VIRTUAL REALITY

    Device

    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).

Treatment groups

250 Participants
are divided into 1 treatment group
Group A: one armExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

psychosocial adjustment, mental health and coping strategies

To assess psychosocial adjustment, mental health and coping strategies through standardized measures in a large population of teenagers and adults with DMD and their caregivers

Time frame
baseline visit
2

social cognition

To test social cognition mechanisms with standardized neuropsychological evaluation and newly-developed cognitive neuroscience paradigms that investigate emotional and social processing in ecological settings using Virtual reality in a subsample of partecipants

Time frame
baseline visit
3

body image

To explore body image perception and assess its impact on psychological well-being exploring the role of cognitive and motor impairment as possible mediators

Time frame
baseline visit
4

Correlation with medical conditions

To investigate the interaction patterns between medical conditions and test on social cognition, psychosocial adjustment, mental health and coping strategies, to identify strengths to be supported and weaknesses to be reduced or counterbalanced.

Time frame
baseline visit

Secondary outcomes

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

IRCCS Eugenio Medea

Lead sponsor

University of Padova

Collaborator

IRCCS National Neurological Institute "C. Mondino" Foundation

Collaborator

Istituto Giannina Gaslini - IRCCS

Collaborator

Fondazione Serena Onlus - Centro Clinico NeMO Milano

Collaborator

Centro Clinico NeMO Ancona

Collaborator

IRCCS Fondazione Stella Maris

Collaborator

Università degli Studi di Milano Statale

Collaborator

Centro Clinico NeMO Brescia

Collaborator

Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta

Collaborator

Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

Collaborator

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

Collaborator

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Collaborator

Dipartimento di Neuroscienze Rita Levi Montalcini Torino

Collaborator

Università Vita-Salute San Raffaele

Collaborator

Università di Pisa

Collaborator

Azienda Ospedaliera Universitaria Policlinico "G. Martino"

Collaborator

Associazione La Nostra Famiglia Eugenio Medea - Brindisi

Collaborator

Scientific Institute, IRCCS E.Medea, Pasian di Prato

Collaborator

IRCCS Istituto delle Scienze Neurologiche di Bologna

Collaborator

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