Carrier of Duchenne Muscular Dystrophy Clinical Trial
| Verified date | October 2016 |
| Source | Hospital Rudolfstiftung |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | Austria: Ethikkommission |
| Study type | Observational |
Carriers of Duchenne muscular dystrophy (DMD) often have no severe symptoms of the scelet muscles, but they may develop a poor heart function due to the involvement of the heart muscle. Ultrasound of the heart is recommended, but it can detect a cardiac affection only after the heart has already become weaker. Cardiac magnetic resonance tomography can detect myocardial fibrosis already at preserved heart function and may facilitate early therapy. In our study, diagnosed carriers of DMD will undergo examinations of the heart by blood tests, EKG, heart ultrasound and magnetic resonance at enrollment and after one year in order to assess the extent of cardiac affection and its association with the clinical development.
| Status | Completed |
| Enrollment | 20 |
| Est. completion date | September 2015 |
| Est. primary completion date | April 2014 |
| Accepts healthy volunteers | No |
| Gender | Female |
| Age group | 18 Years and older |
| Eligibility |
Inclusion Criteria: genetic and/or histological identification as a carrier of DMD age above 18 years able and willing to conform to the requirements of the study provided written informed consent exclusion of pregnancy in women of childbearing potential Exclusion Criteria: Claustrophobia Excessive obesity to an extent where CMR cannot be performed Chronic renal failure with a GFR <30 ml/min/1,73m² Implanted pacemakers/defibrillators Severe arrhythmia Inability to cooperate during the CMR Known intolerance to gadolinium Positive pregnancy test Unable or unwilling to conform to the study protocol |
Observational Model: Cohort, Time Perspective: Prospective
| Country | Name | City | State |
|---|---|---|---|
| Austria | 2nd Medical Dept., Rudolfstiftung | Vienna |
| Lead Sponsor | Collaborator |
|---|---|
| Hospital Rudolfstiftung | Oesterreichische Muskelforschung |
Austria,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | T1 mapping | T1 mapping by MOLLI sequences will be performed and compared between study entry and one year follow-up | 1 year | No |
| Secondary | left ventricular function | left ventricular function on MRI will be compared between study entry and one year follow-up | 1 year | No |