Duchenne Muscular DystrophyValPedsQLDMDNCT03513367
The Validation Process for Confirmation of the French Version of the Pediatric Quality of Life Inventory :PedsQLTM.
An observational study of Duchenne Muscular Dystrophy, run by University Hospital, Toulouse at 1 site in France.
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. Read more about this study
- Who is it for?
- Ages 5 to 18 years, men onlyHealthy volunteers can take part
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- Duchenne Muscular Dystrophy of the PedsQL ™ 3.0 scale and The following data of motor function
- How many people
- 210 participants
- Where
- 1 siteFrance
- Study dates
- 19 Sep 2018to 19 Sep 2019
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 to 18 years
- Men only
- Healthy volunteers accepted
- Child, Adult
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)
Who may be able to join
- Boys aged 7 to 18, with genomic Duchenne muscular dystrophy whose parents (mother and / or father) or direct grandparents do not oppose.
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
France1 site, 1 recruiting
- Recruiting
University Hospital Toulouse
Toulouse, 31059
Claude Cances, MD, 05 34 55 87 28 ext. 33, Cances.c@chu-toulouse.fr
Isabelle Olivier, PhD, 05 61 77 70 51 ext. 33, olivier.i@chu-toulouse.fr
About this study
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.
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.
Read the 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
- Being tested
- Duchenne Muscular Dystrophy of the PedsQL ™ 3.0 scale, The following data of motor function
- Keywords
- Duchenne Muscular Dystrophy,, Pediatric Quality of Life Inventory, disability paradox
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Duchenne Muscular Dystrophy of the PedsQL ™ 3.0 scaleOther
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 functionOther
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).
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: Other: Duchenne Muscular Dystrophy of the PedsQL ™ 3.0 scale; Other: The following data of motor function | |
| 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 Receives: Other: Duchenne Muscular Dystrophy of the PedsQL ™ 3.0 scale |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 12 months
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
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 May 2018
- Study start19 Sep 2018
- Primary completion19 Sep 2019 (estimated)
- Study completion19 Sep 2019 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- More trials run by University Hospital, ToulouseThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Prospective
- Enrolment
- 210 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University Hospital, Toulouse
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Elisabeth Wallach, MD, Principal investigator, University Hospital, Toulouse
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03513367
- Sponsor's protocol ID
- RC31/18/0119
- Other IDs
- 2018-A00895-50
- Acronym
- ValPedsQLDMD
- Status last verified
- Apr 2019
- First submitted
- 19 Apr 2018
- Last updated
- 18 Apr 2019
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Apr 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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.
- ClinicalTrials.gov ID
- NCT03513367
- Acronym / sponsor ID
- ValPedsQLDMD / RC31/18/0119
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Duchenne Muscular Dystrophy,, Pediatric Quality of Life Inventory, disability paradox
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 210 participants (estimated)
- Lead sponsor
- University Hospital, ToulouseUniversity, hospital or other
- Study start
- 19 Sep 2018 (actual)
- Primary completion
- 19 Sep 2019 (estimated)
- Study completion
- 19 Sep 2019 (estimated)
- First posted
- 1 May 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Other
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Boys with Muscular Duchenne Dystrophy | – | Other: Duchenne Muscular Dystrophy of the PedsQL ™ 3.0 scale, Other: The following data of motor function105 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 |
| Parents of boys with Muscular Duchenne Dystrophy | – | Other: Duchenne Muscular Dystrophy of the PedsQL ™ 3.0 scale105 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 |
Who may qualify
- Ages
- 5 to 18 years
- Sex
- Male only
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
- 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)
Key inclusion criteria (1 listed)
- Boys aged 7 to 18, with genomic Duchenne muscular dystrophy whose parents (mother and / or father) or direct grandparents do not oppose.
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Duchenne Muscular Dystrophy of the PedsQL ™ 3.0 scaleScaling 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 | Boys with Muscular Duchenne Dystrophy, Parents of boys with Muscular Duchenne Dystrophy |
| The following data of motor functionIn 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). | Other | Boys with Muscular Duchenne Dystrophy |
Primary outcome
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
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Apr 2019.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Elisabeth Wallach, MD principal investigator
University Hospital, Toulouse
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Duchenne Muscular Dystrophy, run by University Hospital, Toulouse at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 18 Apr 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Claude Cances, MD (05 34 55 87 28 ext. 33, Cances.c@chu-toulouse.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evaluate the validity of the French version of the DMD module of the PedsQLTM 3.0 scale (time frame: 12 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Apr 2019.