The Validation Process for Confirmation of the French Version of the Pediatric Quality of Life Inventory :PedsQLTM. (ValPedsQLDMD)
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| ClinicalTrials.gov Identifier: NCT03513367 |
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Recruitment Status : Unknown
Verified April 2019 by University Hospital, Toulouse.
Recruitment status was: Recruiting
First Posted : May 1, 2018
Last Update Posted : April 18, 2019
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| 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 |
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. |
| Actual Study Start Date : | September 19, 2018 |
| Estimated Primary Completion Date : | September 19, 2019 |
| Estimated Study Completion Date : | September 19, 2019 |
| Group/Cohort | Intervention/treatment |
|---|---|
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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). |
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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
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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. |
- 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
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| 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 |
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)
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
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
| 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 |
| France | |
| University Hospital Toulouse | 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 | |
| Principal Investigator: | Elisabeth Wallach, MD | University Hospital, Toulouse |
| Responsible Party: | University Hospital, Toulouse |
| ClinicalTrials.gov Identifier: | NCT03513367 |
| 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: | April 18, 2019 |
| Last Verified: | April 2019 |
| 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 |
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Duchenne Muscular Dystrophy, Pediatric Quality of Life Inventory disability paradox |
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Muscular Dystrophies Muscular Dystrophy, Duchenne Muscular Disorders, Atrophic Muscular Diseases Musculoskeletal Diseases |
Neuromuscular Diseases Nervous System Diseases Genetic Diseases, Inborn Genetic Diseases, X-Linked |