The Efficacy and Safety of Metoprolol as add-on Treatment to Standard of Care in Preventing Cardiomyopathy in Patients With DMD

Launched by MEDICAL UNIVERSITY OF GDANSK · Sep 23, 2021

Keywords

ClinConnect Summary

This clinical trial is studying the effectiveness and safety of a medication called metoprolol when added to standard heart treatment in boys aged 8 to 16 with Duchenne Muscular Dystrophy (DMD). The goal is to see if metoprolol can help prevent heart problems associated with this condition. To be eligible, participants must have a confirmed diagnosis of DMD and be receiving another heart medication for at least a month before joining the study.

If your child participates, they will be randomly placed in one of two groups: one will receive metoprolol, and the other will receive a placebo (a treatment that looks the same but has no active medication). The study will last for up to 5 years, with regular check-ups to monitor your child’s heart health and overall well-being. Parents will also be involved in tracking health indicators at home, like heart rate and blood pressure, using telemedicine tools. This trial is important as it may provide new insights into managing heart health in children with DMD.

Gender

MALE

Eligibility criteria

• Inclusion Criteria:
• • 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)
• • Stated willingness to comply with all study procedures and availability for the duration of the study
• • Ability to take oral medication and be willing to adhere to the study intervention regimen
• • Subject has confirmed diagnosis of DMD, as defined as clinical picture consistent with typical DMD and: i) Dystrophin immunofluorescence and/or immunoblot showing complete dystrophin deficiency, or ii) Identifiable mutation within the DMD gene (deletion/duplication of one or more exons), where reading frame can be predicted as 'out-of-frame' or, iii) 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)
• • Taking ACEi treatment at minimum required doses for at least 30 days
• Exclusion Criteria:
• • Current or previous permanent use of any beta-blocker medication
• • Treatment with another investigational drug or other intervention within 3 months prior to screening
• • 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
• • Presence of pacemaker or ICD
• • Clinical signs or symptoms of heart failure
• • Left ventricular Ejection Fraction (LVEF) \<57% (assessed by Teichholtz echocardiography)
• • Inability to obtain adequate quality echocardiography images (necessary to monitor for primary endpoint and safety)
• • Known allergic reactions to components of the IMPs