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

study id #: NCT03513367

condition: Duchenne Muscular Dystrophy

status: recruiting

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.

intervention:
Duchenne Muscular Dystrophy of the PedsQL ™ 3.0 scale,
The following data of motor function

mechanism of action: No pharmaceutical intervention

results: https://clinicaltrials.gov/ct2/show/results/NCT03513367

last updated: May 06, 2019

start date: September 19, 2018

estimated completion: September 19, 2019

size / enrollment: 210

study 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.

primary outcomes:

• Evaluate the validity of the French version of the DMD module of the PedsQLTM 3.0 scale [Time Frame: 12 months]
  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
  
• Evaluate the reliability of the French version of the DMD module of the PedsQLTM 3.0 scale [Time Frame: 12 months]
  
• 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

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

sponsor: University Hospital, Toulouse

contacts: Claude Cances, MD, 05 34 55 87 28, Cances.c@chu-toulouse.fr;
Isabelle Olivier, PhD, 05 61 77 70 51, olivier.i@chu-toulouse.fr

investigators: Elisabeth Wallach, MD

locations: France