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Metoprolol Safety and Efficacy in the Prevention of Cardiomyopathy in Patients with Duchenne Muscular Dystrophy (DMD)
EU CTIS ID: 2024-515057-19-00
What this study is testing
The primary objective of this study is to evaluate whether metoprolol succinate in addition to standard of care cardiac treatment introduced before the onset of echocardiography detectable left ventricular dysfunction, can slow the rate of progression left ventricular ejection fraction drop that leads to cardiomyopathy in males with DMD compared to placebo.
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 1. Subject's parent(s) or legal guardian(s) has (have) provided written informed consent, where applicable, prior to any study-related procedures; participants will be asked to give written or verbal assent according to requirements (>16 years old) 2. Stated willingness to comply with all study procedures and availability for the duration of the study 3. Male, ≥ 8 years and <17 years of age at time of enrolment in the study 4. Ability to take oral medication and be willing to adhere to the study intervention regimen 5. Subject has confirmed diagnosis of DMD, as defined as clinical picture consistent with typical DMD and: • Dystrophin immunofluorescence and/or immunoblot showing complete dystrophin deficiency, or • Identifiable mutation within the DMD gene (deletion/duplication of one or more exons), where reading frame can be predicted as 'out-offrame' or • Complete dystrophin gene sequencing showing an alteration (point mutation, duplication, other) that is expected to preclude production of the dystrophin protein (i.e., nonsense mutation, deletion/duplication leading to a downstream stop codon) 6. Taking ACEi treatment at minimum required doses as outlined in the protocol for at least 30 days
You likely can't join if
- 1. Current or previous permanent use of any beta-blocker medication 2. Treatment with another investigational drug or other intervention within 3 months prior to screening 3. Clinically significant bradycardia at rest or by Holter ECG, based on age and sex adjusted normal values, atrioventricular block higher than first degree at rest, or second degree Wenckebach at night, pauses longer than 2.5 seconds 4. Presence of pacemaker or ICD 5. Clinical signs or symptoms of heart failure 6. Left ventricular Ejection Fraction (LVEF) <57% (assessed by Teichholtz echocardiography) 7. Inability to obtain adequate quality echocardiography images (necessary to monitor for primary endpoint and safety) 8. Known allergic reactions to components of the IMPs
The study team makes the final eligibility decision.
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years, 0-17 years. The study team makes the final eligibility decision.
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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.