Duchenne / Becker Muscular DystrophyNCT02470962
Cardiac Involvement in Patients With Duchenne/Becker Muscular Dystrophy
An observational study of Duchenne / Becker Muscular Dystrophy, run by University Children's Hospital, Zurich at 1 site in Switzerland.
Trial summary
This study evaluates the function of the heart in young patients with muscular dystrophy type Duchenne or Becker. Participants have their hearts examined at regular intervals by ultrasound (echocardiography) and cardiac magnetic resonance imaging. Read more about this study
- Who is it for?
- Ages 8 to 18 years, men onlyHealthy volunteers can take part
- Conditions the study is for
- Duchenne / Becker Muscular Dystrophy
- What is being tested?
- Observation
- How many people
- 59 participants
- Where
- 1 siteSwitzerland
- Study dates
- 1 Jun 2015to 22 Mar 2022
- 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 8 to 18 years
- Men only
- Healthy volunteers accepted
- Child, Adult
Study population: * Boys aged 8 to 18 years with DMD/BMD confirmed genetically or by muscle biopsy * Control group for CMR: children without heart disease
Who may be able to join
- Boys aged 8 to 18 years with DMD/BMD confirmed genetically or by muscle biopsy
- Informed consent
Who cannot join
- Other clinically significant concomitant disease states (e.g., renal failure)
- Inability to follow the procedures of the study, e.g. due to language problems, psychological disorders, dementia, etc. of the participant or his/her parents or legal caregivers,
- Inability to lie still for the duration of the imaging procedures (approximately 45 minutes each for echocardiography and CMR)
- MR-incompatible implanted or accidentally incorporated metal device or claustrophobia that prohibits use of magnetic resonance imaging
Trial locations and contacts
1 site in 1 country.
About this study
This study evaluates the function of the heart in young patients with muscular dystrophy type Duchenne or Becker. Participants have their hearts examined at regular intervals by ultrasound (echocardiography) and cardiac magnetic resonance imaging.
Read the detailed description
Muscular dystrophy leads to progressive loss of function in all muscles during childhood and adolescence, including the heart. The usual method to evaluate the heart is echocardiography, emphasizing few parameters. Cardiac magnetic resonance imaging is not as widely available as echocardiography, but early changes can be detected before they become visible on echocardiography. In this study, the investigators compare the methods of measuring heart function in order to find the best measurements for follow up and to see how fast the degenerative changes occur in the hearts of patients with muscular dystrophy.
- Conditions
- Duchenne / Becker Muscular Dystrophy
- Being tested
- Observation
- 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.
ObservationOther
Observation by serial echocardiography with extended techniques and cardiac magnetic resonance imaging
| Study group | Role | What they receive |
|---|---|---|
| Patients with muscular dystrophy | Boys aged 8 to 18 years with muscular dystrophy of the Duchenne / Becker type | |
| Children without heart disease | Children without heart disease, aged 8-18 years, as CMR comparison group Receives: Other: Observation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Left ventricular ejection fraction
Measured: 3 years per patient
Secondary outcomes (2)
Quantification of fibrosis by LGE/T1 mapping
Measured: 3 years per patient
NT-proBNP
Measured: 3 years per patient
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jun 2015 (estimated)
- Study start1 Jun 2015
- Primary completion22 Mar 2022
- Study completion22 Mar 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne / Becker 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
- Case only
- Time perspective
- Prospective
- Enrolment
- 59 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Children's Hospital, Zurich
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Barbara EU Burkhardt, MD, Principal investigator, Children's Hospital Zürich, Switzerland
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02470962
- Sponsor's protocol ID
- DMD-Herz
- Status last verified
- Mar 2025
- First submitted
- 10 Jun 2015
- Last updated
- 20 Mar 2025
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Cardiac Involvement in Patients With Duchenne/Becker Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT02470962
- Acronym / sponsor ID
- DMD-Herz
- Conditions
- Duchenne / Becker Muscular Dystrophy
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 59 participants (actual)
- Lead sponsor
- University Children's Hospital, ZurichUniversity, hospital or other
- Study start
- 1 Jun 2015 (actual)
- Primary completion
- 22 Mar 2022 (actual)
- Study completion
- 22 Mar 2022 (actual)
- First posted
- 12 Jun 2015 (estimated)
- 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.
This study evaluates the function of the heart in young patients with muscular dystrophy type Duchenne or Becker. Participants have their hearts examined at regular intervals by ultrasound (echocardiography) and cardiac magnetic resonance imaging.
Detailed description
Muscular dystrophy leads to progressive loss of function in all muscles during childhood and adolescence, including the heart. The usual method to evaluate the heart is echocardiography, emphasizing few parameters. Cardiac magnetic resonance imaging is not as widely available as echocardiography, but early changes can be detected before they become visible on echocardiography. In this study, the investigators compare the methods of measuring heart function in order to find the best measurements for follow up and to see how fast the degenerative changes occur in the hearts of patients with muscular dystrophy.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Patients with muscular dystrophy | – | –Boys aged 8 to 18 years with muscular dystrophy of the Duchenne / Becker type |
| Children without heart disease | – | Other: ObservationChildren without heart disease, aged 8-18 years, as CMR comparison group |
Who may qualify
- Ages
- 8 to 18 years
- Sex
- Male only
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
- Study population
- * Boys aged 8 to 18 years with DMD/BMD confirmed genetically or by muscle biopsy * Control group for CMR: children without heart disease
Key inclusion criteria (2 listed)
- Boys aged 8 to 18 years with DMD/BMD confirmed genetically or by muscle biopsy
- Informed consent
Key exclusion criteria (4 listed)
- Other clinically significant concomitant disease states (e.g., renal failure)
- Inability to follow the procedures of the study, e.g. due to language problems, psychological disorders, dementia, etc. of the participant or his/her parents or legal caregivers,
- Inability to lie still for the duration of the imaging procedures (approximately 45 minutes each for echocardiography and CMR)
- MR-incompatible implanted or accidentally incorporated metal device or claustrophobia that prohibits use of magnetic resonance imaging
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 |
|---|---|---|
| ObservationObservation by serial echocardiography with extended techniques and cardiac magnetic resonance imaging | Other | Children without heart disease |
Primary outcome
Left ventricular ejection fraction
Time frame: 3 years per patient
2 secondary outcomes
Quantification of fibrosis by LGE/T1 mapping
Time frame: 3 years per patient
NT-proBNP
Time frame: 3 years per patient
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 20 Mar 2025.
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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.
Barbara EU Burkhardt, MD principal investigator
Children's Hospital Zürich, Switzerland
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 / Becker Muscular Dystrophy, run by University Children's Hospital, Zurich at 1 site in Switzerland.
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 4 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 20 Mar 2025. 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 Switzerland. 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?
Left ventricular ejection fraction (time frame: 3 years per patient). 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 20 Mar 2025.