The evaluation of heart muscle changes in muscular dystrophies applying cardiac magnetic resonance: follow-up study
| ISRCTN | ISRCTN16491505 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN16491505 |
| Secondary identifying numbers | MD-CMR- Follow-up |
- Submission date
- 26/08/2017
- Registration date
- 29/11/2017
- Last edited
- 10/11/2021
- Recruitment status
- No longer recruiting
- Overall study status
- Completed
- Condition category
- Musculoskeletal Diseases
Plain English Summary
Background and study aims
Facioscapulohumeral muscular dystrophy (FSHD) and Myotonic dystrophy Type 2 (PROMM) are a genetic disease that causes weakness in the skeletal muscles. Patients may suffer from heart problems like dangerous heart rhythm disturbances, shortness of breath also sudden death have been reported. It is suspected that an unknown injury of the heart can be a reason. Over the last couple of year’s cardiac magnetic resonance imaging (MRI) (a type of scan that uses strong magnetic fields and radio waves to create an image of the heart) has become the best method for looking at scars and other changes in the heart muscle (fat, inflammation). The aim of this study is to identify heart muscle changes within of 2-4 years in patients with DM1/FSHD and its relation to heart rhythm disturbances.
Who can participate?
Participants who have a FSHD Type 1 and PROMM
What does the study involve?
All participants get a detailed work-up by a cardiologist (heart doctor) including a cardiac MRI due to muscle dystrophy. The participant receives a cardiac MRI protocol and innovative techniques. This prolongs the MRI scan by only approximately ten minutes. There are no additional applications of contrast-media or medication.
What are the possible benefits and risks of participating?
Participants may benefit from the detection of early myocardial changes applying MRI may help to stratify further therapy and has a potential impact on prognosis and heart muscle remodeling. The clinical results will also be available for each patient. There are no direct risks with participating.
Where is the study run from?
This study is a work group Cardiovascular Magnetic Resonance, Experimental and Clinical Research Center, a joint cooperation between the Charité University Medicine Berlin (Germany) and the Max-Delbrueck Center for Molecular Medicine (Germany), and HELIOS Klinikum Berlin Buch, Department of Cardiology and Nephrology, Berlin (Germany).
When is the study starting and how long is it expected to run for?
November 2016 to January 2019
Who is funding the study?
Charité University Medicine Berlin (Germany)
Who is the main contact?
Professor Jeanette Schulz-Menger
Contact information
Scientific
Charité University Medicine Berlin Campus Buch
Working Group Kardiale MRT Lindenberger Weg 80
Berlin
13125
Germany
 ORCID ID |
0000-0003-3100-1092 |
|---|
Study information
| Study design | Prospective diagnostic follow-up trial |
|---|---|
| Primary study design | Observational |
| Secondary study design | Longitudinal study |
| Study setting(s) | Hospital |
| Study type | Diagnostic |
| Participant information sheet | Not available in web format, please use the contact details to request a patient information sheet |
| Scientific title | Cardiac involvement in muscular dystrophies assessment by cardiovascular magnetic resonance: follow up study |
| Study hypothesis | The aim of this prospective diagnostic follow up trial is to evaluate the efficacy of a comprehensive cardiovascular magnetic resonance (CMR) protocol to predict myocardial tissue injury and heart rhythm abnormalities in facioscapulohumeral muscular dystrophy type 1 (FSHD1) patients and myotonic dystrophy Type 2 (DM2, PROMM) patients. |
| Ethics approval(s) | Charité University Medicine Berlin Campus Mitte, 10/04/2017, ref: EA1/042/17 |
| Condition | Patients with a genetically confirmed diagnosis FSHD Type 1 and PROMM who took part in a previous studies |
| Intervention | Participants receive a detailed work up (including an ECG, Holter and Echocardiogram) by a cardiologist. The participants undergo a CMR. They receive the same CMR protocol as during the previous study. CMR is applied at a 1.5 T Scanner (MAGNETOM AvantoFit®, Siemens Healthcare, Erlangen, Germany) using a 32 channel surface coil. Cine imaging is performed applying state of the art steady state precession sequences to determine the global cardiac performance. For myocardial tissue differentiation, parametric T1 and T2 mapping, fat/water separated imaging and focal fibrosis imaging (Late Gadolinium Enhancement, LGE) are performed. There are no additional treatments. |
| Intervention type | Procedure/Surgery |
| Primary outcome measure | Increase of myocardial tissue injuries is measured using magnetic resonance imaging visually looking for presence and extent of lesions like fat and scar as well as diffuse myocardial changes at yearly follow up. |
| Secondary outcome measures | Heart rhythm disturbances is measured using ECG and ECG monitoring at yearly follow up |
| Overall study start date | 01/11/2016 |
| Overall study end date | 01/01/2019 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Adult |
| Sex | Both |
| Target number of participants | 84 |
| Total final enrolment | 83 |
| Participant inclusion criteria | FSHD and PROMM patients who took part in the previous study PROMM 2013, FSHD 2015/2016 |
| Participant exclusion criteria | 1. Known vascular, cardiac diseases (e.g. coronary artery disease, significant valvular disease, myocarditis) 2. Malign diseases or known contraindications for CMR 3. Gadolinium-based contrast-media |
| Recruitment start date | 10/04/2017 |
| Recruitment end date | 01/08/2018 |
Locations
Countries of recruitment
- Germany
Study participating centres
Berlin
13125
Germany
Berlin
13125
Germany
Berlin
-
Germany
Sponsor information
University/education
Working Group Kardiale
MRT Lindenberger Weg 80
Berlin
13125
Germany
| Website | http://www.cmr-berlin.org |
|---|---|
"ROR" |
https://ror.org/001w7jn25 |
Funders
Funder type
University/education
Private sector organisation / For-profit companies (industry)
- Alternative name(s)
- Medical School - Charité - University Medicine Berlin
- Location
- Germany
Results and Publications
| Intention to publish date | 31/12/2018 |
|---|---|
| Individual participant data (IPD) Intention to share | No |
| IPD sharing plan summary | Not expected to be made available |
| Publication and dissemination plan | Results of this study shall be published in a high-ranking peer-reviewed journal. |
| IPD sharing plan | The datasets generated during and/or analysed during the current study are not expected to be made available due to reasons of data protection laws in Germany. However, upon request methodology and dataset structure can be shared. |
Study outputs
| Output type | Details | Date created | Date added | Peer reviewed? | Patient-facing? |
|---|---|---|---|---|---|
| Protocol article | protocol | 01/07/2016 | Yes | No | |
| Results article | 08/11/2021 | 10/11/2021 | Yes | No |
Editorial Notes
10/11/2021: The following changes were made to the trial record:
1. Publication reference added.
2. The total final enrolment was added.
