ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 39 of 95 for:    Recruiting, Not yet recruiting, Available Studies | "Muscular Dystrophies"

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03513367
Recruitment Status : Not yet recruiting
First Posted : May 1, 2018
Last Update Posted : May 11, 2018
Sponsor:
Information provided by (Responsible Party):
University Hospital, Toulouse

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.

Condition or disease Intervention/treatment
Duchenne Muscular Dystrophy Other: Duchenne Muscular Dystrophy of the PedsQL ™ 3.0 scale Other: The following data of motor function

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.


Study Type : Observational
Estimated Enrollment : 210 participants
Observational Model: Other
Time Perspective: Prospective
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.
Estimated Study Start Date : May 2018
Estimated Primary Completion Date : May 2019
Estimated Study Completion Date : May 2019


Group/Cohort Intervention/treatment
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

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.


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).

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
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.





Primary Outcome Measures :
  1. 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


  2. 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



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   5 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   Yes
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)

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03513367


Contacts
Contact: Claude Cances, MD 05 34 55 87 28 ext 33 Cances.c@chu-toulouse.fr
Contact: Isabelle Olivier, PhD 05 61 77 70 51 ext 33 olivier.i@chu-toulouse.fr

Locations
France
University Hospital Toulouse Not yet recruiting
Toulouse, France, 31059
Contact: Claude Cances, MD    05 34 55 87 28 ext 33    Cances.c@chu-toulouse.fr   
Contact: Isabelle Olivier, PhD    05 61 77 70 51 ext 33    olivier.i@chu-toulouse.fr   
Principal Investigator: Elisabeth Wallach, MD         
Sponsors and Collaborators
University Hospital, Toulouse
Investigators
Principal Investigator: Elisabeth Wallach, MD University Hospital, Toulouse

Responsible Party: University Hospital, Toulouse
ClinicalTrials.gov Identifier: NCT03513367     History of Changes
Other Study ID Numbers: RC31/18/0119
2018-A00895-50 ( Other Identifier: ID-RCB )
First Posted: May 1, 2018    Key Record Dates
Last Update Posted: May 11, 2018
Last Verified: May 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by University Hospital, Toulouse:
Duchenne Muscular Dystrophy,
Pediatric Quality of Life Inventory
disability paradox

Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked