Duchenne Muscular DystrophyDMD-HSNCT03443115
Duchenne Muscular Dystrophy Heart Study
An observational study of Duchenne Muscular Dystrophy and Duchenne Muscular Dystrophy-Associated Dilated Cardiomyopathy, run by Hôpital Cochin at 1 site in France.
Trial summary
Retrospective cohort study including patients with genetically proven Duchenne muscular dystrophy, diagnosed from January 1993 to March 2020. Inclusion of the data relative to genetic diagnosis, clinical characteristics at baseline, cardiac and respiratory workup, medical treatments (ACE inhibitors, steroids)… Read more about this study
- Who is it for?
- Any age, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy, Duchenne Muscular Dystrophy-Associated Dilated Cardiomyopathy
- How many people
- 700 participants
- Where
- 1 siteFrance
- Study dates
- 27 Jun 2017to 15 Mar 2020
- 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 Any age
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Study population: Consecutive patients diagnosed with Duchenne muscular dystrophy and referred to reference centers for neuromuscular diseases for the management of their condiction.
Who may be able to join
- Genetically-proven Duchenne Muscular Dystrophy (DMD mutation)
- Covering by social security
Who cannot join
- Patient refusal to participate to the study
Trial locations and contacts
1 site in 1 country.
France1 site, 1 recruiting
- Recruiting
Cochin Reference Center for Neuromuscular Diseases
Paris, 75014
Karim Wahbi, MD, PhD, +33158411653, karim.wahbi@aphp.fr
About this study
Official title: Multicenter Cohort Study on Duchenne Muscular Dystrophy Cardiomyopathy
Retrospective cohort study including patients with genetically proven Duchenne muscular dystrophy, diagnosed from January 1993 to March 2020.
Inclusion of the data relative to genetic diagnosis, clinical characteristics at baseline, cardiac and respiratory workup, medical treatments (ACE inhibitors, steroids), surgical procedures, and occurrence during follow-up of cardiac, respiratory and fatal events.
Objectives are to describe long-term natural history of the disease, vital prognosis, genotype-phenotype correlations, effect of treatments.
- Conditions
- Duchenne Muscular Dystrophy, Duchenne Muscular Dystrophy-Associated Dilated Cardiomyopathy
- Medical subject headings
- Muscular Dystrophy, Duchenne
What is being measured
The primary outcome is the main question the study is designed to answer.
All-cause mortality
Measured: Follow-up completed in March 2020
Secondary outcomes (3)
Hospitalization for heart failure
Measured: Follow-up completed in March 2020
Dilated cardiomyopathy
Measured: Follow-up completed in March 2020
Hospitalization for acute respiratory failure
Measured: Follow-up completed in March 2020
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Feb 2018
- Study start27 Jun 2017
- Primary completion16 Feb 2018
- Study completion15 Mar 2020 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- 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
- 700 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hôpital Cochin
- Sponsor type
- University, hospital or other
- Collaborators
- Association Monégasque contre les Myopathies
- Responsible party
- Principal investigator, Karim Wahbi, Hôpital Cochin
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03443115
- Sponsor's protocol ID
- DMD-HS
- Acronym
- DMD-HS
- Status last verified
- Feb 2018
- First submitted
- 16 Feb 2018
- Last updated
- 22 Feb 2018
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Porcher R, Desguerre I, Amthor H, Chabrol B, Audic F, Rivier F, Isapof A, Tiffreau V, Campana-Salort E, Leturcq F, Tuffery-Giraud S, Ben Yaou R, Annane D, Amedro P, Barnerias C, Becane HM, Behin A, Bonnet D, Bassez G, Cossee M, de La Villeon G, Delcourte C, Fayssoil A, Fontaine B, Godart F, Guillaumont S, Jaillette E, Laforet P, Leonard-Louis S, Lofaso F, Mayer M, Morales RJ, Meune C, Orlikowski D, Ovaert C, Prigent H, Saadi M, Sochala M, Tard C, Vaksmann G, Walther-Louvier U, Eymard B, Stojkovic T, Ravaud P, Duboc D, Wahbi K. Association between prophylactic angiotensin-converting enzyme inhibitors and overall survival in Duchenne muscular dystrophy-analysis of registry data. Eur Heart J. 2021 May 21;42(20):1976-1984. doi: 10.1093/eurheartj/ehab054.
PubMed 33748842
Study at a glance
From the registry record, last updated by the sponsor on 22 Feb 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multicenter Cohort Study on Duchenne Muscular Dystrophy Cardiomyopathy
- ClinicalTrials.gov ID
- NCT03443115
- Acronym / sponsor ID
- DMD-HS / DMD-HS
- Conditions
- Duchenne Muscular Dystrophy, Duchenne Muscular Dystrophy-Associated Dilated Cardiomyopathy
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 700 participants (estimated)
- Lead sponsor
- Hôpital CochinUniversity, hospital or other
- Collaborators
- Association Monégasque contre les Myopathies
- Study start
- 27 Jun 2017 (actual)
- Primary completion
- 16 Feb 2018 (actual)
- Study completion
- 15 Mar 2020 (estimated)
- First posted
- 22 Feb 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.
Retrospective cohort study including patients with genetically proven Duchenne muscular dystrophy, diagnosed from January 1993 to March 2020.
Inclusion of the data relative to genetic diagnosis, clinical characteristics at baseline, cardiac and respiratory workup, medical treatments (ACE inhibitors, steroids), surgical procedures, and occurrence during follow-up of cardiac, respiratory and fatal events.
Objectives are to describe long-term natural history of the disease, vital prognosis, genotype-phenotype correlations, effect of treatments.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
Who may qualify
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Consecutive patients diagnosed with Duchenne muscular dystrophy and referred to reference centers for neuromuscular diseases for the management of their condiction.
Key inclusion criteria (2 listed)
- Genetically-proven Duchenne Muscular Dystrophy (DMD mutation)
- Covering by social security
Key exclusion criteria (1 listed)
- Patient refusal to participate to the study
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
All-cause mortality
Time frame: Follow-up completed in March 2020
3 secondary outcomes
Hospitalization for heart failure
Time frame: Follow-up completed in March 2020
Dilated cardiomyopathy
Time frame: Follow-up completed in March 2020
Hospitalization for acute respiratory failure
Time frame: Follow-up completed in March 2020
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Feb 2018.
- Derived from this study: PubMed 33748842Porcher R, Desguerre I, Amthor H, Chabrol B, Audic F, Rivier F, Isapof A, Tiffreau V, Campana-Salort E, Leturcq F, Tuffery-Giraud S, Ben Yaou R, Annane D, Amedro P, Barnerias C, Becane HM, Behin A, Bonnet D, Bassez G, Cossee M, de La Villeon G, Delcourte C, Fayssoil A, Fontaine B, Godart F, Guillaumont S, Jaillette E, Laforet P, Leonard-Louis S, Lofaso F, Mayer M, Morales RJ, Meune C, Orlikowski D, Ovaert C, Prigent H, Saadi M, Sochala M, Tard C, Vaksmann G, Walther-Louvier U, Eymard B, Stojkovic T, Ravaud P, Duboc D, Wahbi K. Association between prophylactic angiotensin-converting enzyme inhibitors and overall survival in Duchenne muscular dystrophy-analysis of registry data. Eur Heart J. 2021 May 21;42(20):1976-1984. doi: 10.1093/eurheartj/ehab054.
- Individual participant dataSharing planned: No
Related trials for Duchenne Muscular Dystrophy
Enrolling by invitationObservational study
An Observational Study Comparing Delandistrogene Moxeparvovec (ELEVIDYS) With Standard of Care in Participants With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
RecruitingObservational study
Does Chronic Pain Change Over Time in Children With Duchenne Muscular Dystrophy?
Duchenne Muscular Dystrophy (DMD), Chronic Pain
RecruitingObservational study
Bone Health in Adults With Duchenne Muscular Dystrophy: A Survey on Osteoporosis, Fractures and Treatment.
Duchenne Muscular Dystrophy (DMD)
RecruitingObservational study
Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders
Invasive PreNatal Diagnosis in a Context of Family History of Single-gene Disorders, Including, Sickle Cell Disease
RecruitingPhase 1/2
PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD)
Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMD
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.
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 and Duchenne Muscular Dystrophy-Associated Dilated Cardiomyopathy, run by Hôpital Cochin 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 1 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 22 Feb 2018. 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 Karim Wahbi, MD, PhD (+33158411653, karim.wahbi@aphp.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
All-cause mortality (time frame: Follow-up completed in March 2020). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03443115, which also lists 1 linked publication.
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 22 Feb 2018.