Duchenne Muscular DystrophyCouDuMyoNCT06861270
Estimation of Non-Reimbursable Costs for Patients With Duchenne Muscular Dystrophy in France
An observational study of Duchenne Muscular Dystrophy, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial summary
Duchenne muscular dystrophy (DMD) is a chronic neuromuscular disorder affecting approximately 150 to 200 newborns annually, predominantly males, and is characterized by progressive muscle atrophy and weakness due to a complete absence of dystrophin. Read more about this study
- Who is it for?
- Ages 1 year to 99 years
- Conditions the study is for
- Duchenne Muscular Dystrophy
- How many people
- 28 participants
- Where
- 1 siteFrance
- Study dates
- 28 Mar 2024to 31 Jul 2025
- 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 1 year to 99 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: This study will focus on patients with Duchenne muscular dystrophy, whatever their age, and included in the BNDMR. This database brings together data on all patients treated in the rare disease expert centres approved by the DGOS, and shows a gross total of 2,028 patients with DMD on 1st March 2022.
Who may be able to join
- Patients of any age with DMD diagnosed more than 6 months ago;
- Patient included in the BNDMR, not objecting to the collection of his or her data (see BNDMR 'Agreement to be contacted for a protocol') and alive at the time of his or her request to participate in the study.
Who cannot join
- Patient taking part in an interventional clinical study with changes to usual management (see BNDMR 'Patient taking part in a protocol');
- Patient and/or main carer objecting to the use of their data;
- Patient and/or main carer whose understanding of the French language does not allow them to complete the questionnaire.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Estimation of Non-Reimbursable Direct Medical Costs, Direct Non-Medical Costs, and Indirect Costs for Patients With Duchenne Muscular Dystrophy in France
Duchenne muscular dystrophy (DMD) is a chronic neuromuscular disorder affecting approximately 150 to 200 newborns annually, predominantly males, and is characterized by progressive muscle atrophy and weakness due to a complete absence of dystrophin. DMD presents a severe phenotype, with life expectancy typically extending into the third decade. Data on healthcare utilization and associated costs for DMD patients, particularly within the French healthcare context, remain limited. The EPARDYS study aims to examine the care pathways of these patients using data from the Banque Nationale des Données Maladies Rares (BNDMR) and the Système National des Données de Santé (SNDS), enabling a detailed characterization of patient demographics, healthcare utilization patterns, and associated costs.
However, only direct costs eligible for reimbursement under the Affection Longue Durée (ALD 100%) scheme can be evaluated. Additional resources are consumed by DMD patients, particularly for medical care that falls outside the scope of reimbursement. Furthermore, the high level of dependency in these patients may lead to the need for home adaptations or other expenditures outside the healthcare domain but linked to the disease. Informal caregiving also plays a significant role.
These additional family-incurred costs must be considered. The CouDuMyo study will aim to estimate all relevant cost components from a societal perspective, considering both minors and adults, whether they attend school or work. The medico-social aspect of care, along with the collection of data on aids and benefits received, will offer insights into the nature of the support available to families in France. The knowledge gained regarding non-reimbursable, disease-related costs according to patient characteristics will provide a comprehensive view of the financial burden borne by patients and their caregivers. This assessment is particularly relevant in the context of emerging therapies, which may reduce these associated costs. The main objective will be to describe the resource utilization associated with DMD care that is not eligible for reimbursement, including direct medical costs not covered, medico-social expenses, direct non-medical costs, and indirect costs.
- Conditions
- Duchenne Muscular Dystrophy
- Medical subject headings
- Muscular Dystrophy, Duchenne
What is being measured
The primary outcome is the main question the study is designed to answer.
Average annualised cost per patient/family.
The consumption of resources related to the care of patients with Duchenne muscular dystrophy (DMD) that are not eligible for reimbursement. This includes direct medical costs not covered by insurance, such as certain treatments, therapies, or medical devices that patients may require. Additionally, medico-social costs will be assessed, including services that support daily living and social integration, such as specialized transportation, caregiving, and educational support. Direct non-medical costs will also be evaluated, particularly those related to home modifications or equipment necessary for adapting the living environment to the patient's needs. Finally, the study will analyze indirect costs, which encompass the loss of productivity both for the patient and their caregivers, as well as the value of informal care provided by family members or other non-professional caregivers.
Measured: 1 year
Secondary outcomes (3)
Different types of costs identified for managing the disease
All relevant expenses associated with the care of Duchenne muscular dystrophy (DMD) patients. The analysis will cover various categories of costs, including direct medical costs (both reimbursable and non-reimbursable), medico-social costs (such as home care or special education), direct non-medical costs (such as home adaptations or specialized equipment), and indirect costs (such as loss of productivity and informal caregiving)
Measured: from disease identification to questionnaire's completion, up to 98 years
Costs based on the severity of the disease
How costs vary depending on the stage of disease progression. As DMD is a degenerative condition, the severity of the disease evolves over time, requiring different levels of care and resources at each stage. This analysis will examine how costs increase or change as the patient's functional abilities decline
Measured: from disease identification to questionnaire's completion, up to 98 years
Determinants of the identified costs
The key factors that drive the costs of managing DMD. These determinants may include patient characteristics (such as age, disease duration, and comorbidities), socioeconomic factors (such as income, access to care, and geographic location), and the type of support or assistance available (such as public benefits, informal caregiving, and specialized services).
Measured: from disease identification to questionnaire's completion, up to 98 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Mar 2025
- Study start28 Mar 2024
- Primary completion31 Mar 2024
- Study completion31 Jul 2025
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 Assistance Publique - Hôpitaux de ParisThe 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
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 28 participants (actual)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Isabelle Durand-Zaleski, Professor, Study director, Assistance Publique - Hôpitaux de Paris
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06861270
- Sponsor's protocol ID
- APHP240695
- Acronym
- CouDuMyo
- Status last verified
- Jul 2024
- First submitted
- 25 Sep 2024
- Last updated
- 28 May 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 28 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Estimation of Non-Reimbursable Direct Medical Costs, Direct Non-Medical Costs, and Indirect Costs for Patients With Duchenne Muscular Dystrophy in France
- ClinicalTrials.gov ID
- NCT06861270
- Acronym / sponsor ID
- CouDuMyo / APHP240695
- Conditions
- Duchenne Muscular Dystrophy
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 28 participants (actual)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Study start
- 28 Mar 2024 (actual)
- Primary completion
- 31 Mar 2024 (actual)
- Study completion
- 31 Jul 2025 (actual)
- First posted
- 6 Mar 2025 (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.
Duchenne muscular dystrophy (DMD) is a chronic neuromuscular disorder affecting approximately 150 to 200 newborns annually, predominantly males, and is characterized by progressive muscle atrophy and weakness due to a complete absence of dystrophin. DMD presents a severe phenotype, with life expectancy typically extending into the third decade. Data on healthcare utilization and associated costs for DMD patients, particularly within the French healthcare context, remain limited. The EPARDYS study aims to examine the care pathways of these patients using data from the Banque Nationale des Données Maladies Rares (BNDMR) and the Système National des Données de Santé (SNDS), enabling a detailed characterization of patient demographics, healthcare utilization patterns, and associated costs.
However, only direct costs eligible for reimbursement under the Affection Longue Durée (ALD 100%) scheme can be evaluated. Additional resources are consumed by DMD patients, particularly for medical care that falls outside the scope of reimbursement. Furthermore, the high level of dependency in these patients may lead to the need for home adaptations or other expenditures outside the healthcare domain but linked to the disease. Informal caregiving also plays a significant role.
These additional family-incurred costs must be considered. The CouDuMyo study will aim to estimate all relevant cost components from a societal perspective, considering both minors and adults, whether they attend school or work. The medico-social aspect of care, along with the collection of data on aids and benefits received, will offer insights into the nature of the support available to families in France. The knowledge gained regarding non-reimbursable, disease-related costs according to patient characteristics will provide a comprehensive view of the financial burden borne by patients and their caregivers. This assessment is particularly relevant in the context of emerging therapies, which may reduce these associated costs. The main objective will be to describe the resource utilization associated with DMD care that is not eligible for reimbursement, including direct medical costs not covered, medico-social expenses, direct non-medical costs, and indirect costs.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
Who may qualify
- Ages
- 1 year to 99 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- This study will focus on patients with Duchenne muscular dystrophy, whatever their age, and included in the BNDMR. This database brings together data on all patients treated in the rare disease expert centres approved by the DGOS, and shows a gross total of 2,028 patients with DMD on 1st March 2022.
Key inclusion criteria (2 listed)
- Patients of any age with DMD diagnosed more than 6 months ago;
- Patient included in the BNDMR, not objecting to the collection of his or her data (see BNDMR 'Agreement to be contacted for a protocol') and alive at the time of his or her request to participate in the study.
Key exclusion criteria (3 listed)
- Patient taking part in an interventional clinical study with changes to usual management (see BNDMR 'Patient taking part in a protocol');
- Patient and/or main carer objecting to the use of their data;
- Patient and/or main carer whose understanding of the French language does not allow them to complete the questionnaire.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Average annualised cost per patient/family.
Time frame: 1 year
The consumption of resources related to the care of patients with Duchenne muscular dystrophy (DMD) that are not eligible for reimbursement. This includes direct medical costs not covered by insurance, such as certain treatments, therapies, or medical devices that patients may require. Additionally, medico-social costs will be assessed, including services that support daily living and social integration, such as specialized transportation, caregiving, and educational support. Direct non-medical costs will also be evaluated, particularly those related to home modifications or equipment necessary for adapting the living environment to the patient's needs. Finally, the study will analyze indirect costs, which encompass the loss of productivity both for the patient and their caregivers, as well as the value of informal care provided by family members or other non-professional caregivers.
3 secondary outcomes
Different types of costs identified for managing the disease
Time frame: from disease identification to questionnaire's completion, up to 98 years
Costs based on the severity of the disease
Time frame: from disease identification to questionnaire's completion, up to 98 years
Determinants of the identified costs
Time frame: from disease identification to questionnaire's completion, up to 98 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 May 2026.
- Individual participant dataSharing planned: Undecided
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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.
Isabelle Durand-Zaleski, Professor study director
Assistance Publique - Hôpitaux de Paris
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 Assistance Publique - Hôpitaux de Paris 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 2 inclusion and 3 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: finished, as of the sponsor's last update on 28 May 2026. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Average annualised cost per patient/family. (time frame: 1 year). 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 28 May 2026.