Using Metoprolol to Prevent Heart Issues in Young Patients with Duchenne Muscular Dystrophy
The Efficacy and Safety of Metoprolol as add-on Treatment to Standard of Care in Preventing Cardiomyopathy in Patients With Duchenne Muscular Dystrophy Aged 8-16 Years. A Randomized, Double-blind, Placebo-controlled Study
This study is testing if adding metoprolol to standard care can help prevent heart problems in young patients with Duchenne Muscular Dystrophy.
Quick facts
| Phase | Phase 3 |
|---|---|
| Study type | Interventional |
| Enrollment | 150 (estimated) |
| Ages | 8 Years to 17 Years |
| Sex | Male |
| Sponsor | Medical University of Gdansk Academic / other |
| Locations | 1 site (Gdansk, Pomeranian Voivodeship) |
| Trial ID | NCT05066633 on ClinicalTrials.gov |
What this trial studies
This clinical trial investigates the efficacy and safety of metoprolol as an additional treatment to standard care for preventing cardiomyopathy in patients diagnosed with Duchenne Muscular Dystrophy (DMD). It involves 150 participants aged 8 to 16 years who will be randomly assigned to receive either metoprolol or a placebo for up to 5 years, alongside standard cardiac therapy. The study includes regular monitoring of heart rate, blood pressure, and overall well-being using telemedicine technology, ensuring comprehensive oversight of patient health throughout the trial. Participants will undergo various diagnostic examinations to evaluate disease progression and treatment effects.
Who should consider this trial
Good fit: Ideal candidates are boys aged 8 to 16 years with a confirmed diagnosis of Duchenne Muscular Dystrophy who are already receiving standard cardiac therapy.
Not a fit: Patients who do not have a confirmed diagnosis of Duchenne Muscular Dystrophy or are outside the age range of 8 to 16 years may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly reduce the risk of heart complications in young patients with Duchenne Muscular Dystrophy.
How similar studies have performed: While there have been studies on heart treatments in DMD, the specific use of metoprolol as an add-on therapy in this context is relatively novel.
Eligibility criteria
Show full inclusion / exclusion 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
Where this trial is running
Gdansk, Pomeranian Voivodeship
- University Clinical Centre in Gdańsk, Clinic of Paediatric Cardiology and Congenital Heart Defects — Gdansk, Pomeranian Voivodeship, Poland (Recruiting)
Study contacts
- Study coordinator: Joanna Kwiatkowska, MD, PhD
- Email: joanna.kwiatkowska@gumed.edu.pl
- Phone: 58349 2899
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.