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

Duchenne Muscular Dystrophy Clinical Trial

— ValPedsQLDMD  

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.

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03513367
• Other study ID #: RC31/18/0119
• Secondary ID: 2018-A00895-50
• Status: Recruiting
• Start date: September 19, 2018
• Est. completion date: September 19, 2019

Study information

• Verified date: April 2019
• Source: University Hospital, Toulouse
• Contact: Claude Cances, MD
• Phone: 05 34 55 87 28
• Email: Cances.c[@]chu-toulouse.fr
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 210
• Est. completion date: September 19, 2019
• Est. primary completion date: September 19, 2019
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: Male
• Age group: 5 Years to 18 Years

Eligibility

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

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Other:
• Duchenne Muscular Dystrophy of the PedsQL ™ 3.0 scale
Scaling in multidisciplinary consultations in the form of a self-administered questionnaire with the help of a third party (psychologist). The child and his / her parent complete the questionnaire independently. The result of the questionnaire will then be scored. To validate the French translation of the pediatric module of Duchenne Muscular Dystrophy of the PedsQL ™ 3.0 scale. The validation process is confirmatory, the scale being widely used in English. The scale will measure the quality of life of the child using two independent assessments : children and their parents.
• The following data of motor function
In parallel, the following data are collected on the day of the consultation: assessment of motor function (MFM, use of a wheelchair, age of loss of walking); assessment of respiratory function (EFR, FVC, respiratory assistance, type of respiratory aid); evaluation of cardiac function (FE); assessment of nutritional status (weight, height, BMI, nutritional support by gastrostomy), school status; ongoing drug treatments (corticosteroids, IEC).

Locations

Country	Name	City	State
France	University Hospital Toulouse	Toulouse

Sponsors (1)

Lead Sponsor	Collaborator
University Hospital, Toulouse

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Evaluate the validity of the French version of the DMD module of the PedsQLTM 3.0 scale	The validation process is confirmatory, the scale being widely used in English 201/5000 The internal consistency of the 4 dimensions of the PedsQL ™ DMD module will be evaluated by measuring the Cronbach Alpha. In terms of data availability to children (activity report).; the validation of the DMD module will focus on the validity of constructs, internal structure validity, discriminant validity and reliability	12 months
Primary	Evaluate the reliability of the French version of the DMD module of the PedsQLTM 3.0 scale	PedsQLTM is a model for measuring quality of life in children with acute or chronic pathology. Pathology-specific PedsQL ™ provides a better assessment of the quality of life of this population	12 months