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

NCT ID: NCT03513367

Last Updated: 2019-04-18

Basic Information

• Recruitment Status: UNKNOWN
• Total Enrollment: 210 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2018-09-19
• Study Completion Date: 2019-09-19

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.

Conditions

Duchenne Muscular Dystrophy

Study Design

• Observational Model Type: OTHER
• Study Time Perspective: PROSPECTIVE

Study Groups

Boys with Muscular Duchenne Dystrophy

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

Duchenne Muscular Dystrophy of the PedsQL ™ 3.0 scale

Intervention Type: OTHER

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

Intervention Type: OTHER

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).

Parents of boys with Muscular Duchenne Dystrophy

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

Duchenne Muscular Dystrophy of the PedsQL ™ 3.0 scale

Intervention Type: OTHER

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.

Interventions

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.

Intervention Type: OTHER

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).

Intervention Type: OTHER

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

• Minimum Eligible Age: 5 Years
• Maximum Eligible Age: 18 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: Yes

Sponsors

University Hospital, Toulouse

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Elisabeth Wallach, MD

Role: PRINCIPAL_INVESTIGATOR

University Hospital, Toulouse

Locations

University Hospital Toulouse

Toulouse, , France

Site Status: RECRUITING

Countries

France

Central Contacts

Claude Cances, MD

Role: CONTACT

Cances.c@chu-toulouse.fr

05 34 55 87 28 ext. 33

Isabelle Olivier, PhD

Role: CONTACT

olivier.i@chu-toulouse.fr

05 61 77 70 51 ext. 33

Facility Contacts

Claude Cances, MD

Role: primary

Cances.c@chu-toulouse.fr

05 34 55 87 28 ext. 33

Isabelle Olivier, PhD

Role: backup

olivier.i@chu-toulouse.fr

05 61 77 70 51 ext. 33

Other Identifiers

2018-A00895-50

Identifier Type: OTHER

Identifier Source: secondary_id

RC31/18/0119

Identifier Type: -

Identifier Source: org_study_id