The Validation Process for Confirmation of the French Version of the Pediatric Quality of Life Inventory :PedsQLTM. (ValPedsQLDMD)

Toulouse University Hospital

Status: Unknown

Conditions: Duchenne Muscular Dystrophy

Treatments: Other: Duchenne Muscular Dystrophy of the PedsQL ™ 3.0 scale; Other: The following data of motor function

Study type: Observational

Funder types: Other

Identifiers

NCT03513367

RC31/18/0119

2018-A00895-50 (Other Identifier)

Details and patient eligibility

About

There isn't specific Health related quality of life measure for children with DMD in French. The aim of this study is to validate the French version of the Pediatric Quality of Life Inventory 3.0 Duchenne Muscular Dystrophy module with a multicentric study. The investigators will evaluate the following psychometric properties : convergent validity, internal validity, inter-rater reliability. The investigators would like to be able to use this scientific tool in future clinical trials.

Full description

The Duchenne Muscular Dystrophy, the commonest form of dystrophy, is an X-linked, recessive neuromuscular disease, in which there is an absence of the protein dystrophin. This chronic and progressive disease leads to an inevitable loss of autonomy (muscle weakness, respiratory and cardiac failure). With better multidisciplinary care, life expectancy has increased but also morbidity. From now one, the evaluation of the quality of life of children with DMD is necessary in therapeutic trials.

Given the specificities of the disease, it seems appropriate to have a specific scale. In the literature there isn't quality of life scale specific to Duchenne Muscular Dystrophy in French version. The only specific scale that exists is the specific module PedsQLTM DMD that was validated in English version in 2012. This scale is relevant for assessing the quality of life in clinical trials and in daily clinical practice given its psychometric properties (good internal consistency close to 0.8).The main hypothesis that we formulate is to validate the French translation of the pediatric module of Duchenne Muscular Dystrophy of the PedsQL ™ 3.0 scale.

Enrollment: 210 estimated patients

Sex: Male

Ages: 5 to 18 years old

Volunteers: Accepts Healthy Volunteers

Inclusion criteria

• Boys aged 7 to 18, with genomic Duchenne muscular dystrophy whose parents (mother and / or father) or direct grandparents do not oppose.

Exclusion criteria

• Inability for the child to understand the issues
• Absence of direct parents or grandparents
• Child receiving antidepressant treatment
• Non French speaking child
• Duchenne Muscular Dystrophy girls

Trial design

210 participants in 2 patient groups

Boys with Muscular Duchenne Dystrophy

Description:

105 boys with Muscular Duchenne Dystrophy (DMD) distributed as follows: 35 patients with Duchenne muscular dystrophy by age category, 8-12 years old and 13-18 years old. Children will complete the questionnaire of Duchenne Muscular Dystrophy of the PedsQL ™ 3.0 scale regardless of his parents

Treatment:

Other: Duchenne Muscular Dystrophy of the PedsQL ™ 3.0 scale

Other: The following data of motor function

Parents of boys with Muscular Duchenne Dystrophy

Description:

105 parents of boys with Muscular Duchenne Dystrophy For the 5-7 age group, only parents answer the questionnaire but medical data are collected : 35 by age category (5-7; 8-12; 13-18) Parents will complete the questionnaire of Duchenne Muscular Dystrophy of the PedsQL ™ 3.0 scale regardless of their children

Treatment:

Other: Duchenne Muscular Dystrophy of the PedsQL ™ 3.0 scale