Clinical trial NCT03513367: The Validation Process for Confirmation of the French Version of the Pediatric Quality of Life Inventory :PedsQLTM.
Official title: The Validation Process for Confirmation of the French Version of the Pediatric Quality of Life Inventory (PedsQLTM) 3.0 Duchenne Muscular Dystrophy Module.
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Duchenne Muscular Dystrophy
Keywords: Duchenne Muscular Dystrophy,
Pediatric Quality of Life Inventory
disability paradox
Lead sponsor: University Hospital, Toulouse
Sponsor class: OTHER
First posted: 2018-05-01
Enrollment: 210
Sex: MALE
Minimum age (years): 5
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: yes
Observational model: OTHER
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: 105 boys with DMD distributed as follows: 35 patients with Duchenne muscular dystrophy by age category, 8-12 years old and 13-18 years old.

For the 5-7 age group, only parents answer the questionnaire but medical data are collected.

105 parents of boys with DMD, broken down as follows: 35 by age group (5-7, 8-12, 13-18)

Brief summary: 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.

Detailed 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.

Eligibility criteria: 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

Primary outcomes:
measure: Evaluate the validity of the French version of the DMD module of the PedsQLTM 3.0 scale
timeFrame: 12 months
measure: Evaluate the reliability of the French version of the DMD module of the PedsQLTM 3.0 scale
timeFrame: 12 months
Secondary outcomes:
Other outcomes:
Locations:
city: Toulouse
country: France
facility: University Hospital Toulouse
Study officials:
affiliation: University Hospital, Toulouse
name: Elisabeth Wallach, MD
role: PRINCIPAL_INVESTIGATOR
References: