Actively Recruiting
The Efficacy and Safety of Metoprolol as add-on Treatment to Standard of Care in Preventing Cardiomyopathy in Patients With DMD
Led by Medical University of Gdansk · Updated on 2021-10-04
150
Participants Needed
1
Research Sites
253 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
The study includes 150 patients with DMD diagnosis confirmed by genetic testing, 8-16 years old (≥8 and \<17) at the study entry with a follow-up of up to 5 years. Random enrollment of a patient to one of two groups (intervention or control) takes place after pre-screening and screening stage starts the first phase of the trial. To be eligible for participation in the study, patients must receive standard of care cardiac therapy, which is an Angiotensin-converting-enzyme inhibitor (ACEi) for at least one-month prior to enrollment. A major part of the trial is equal for all patients - who will be receiving indistinguishable investigational medicinal products (IMPs), the drug metoprolol succinate or placebo. As a part of the clinical trial, diagnostic examinations evaluating progression of the disease, will be performed periodically. In addition, all patients will be monitored at home. Heart rate, blood pressure and patients' personal well-being will be controlled using telemedicine technologies. Additional visits in the research center will be provided if any adverse events occur. This model will be continued for 30 months from the enrollment of a first patient. After this period the first drug efficiency analysis will be performed. After that, the intervention may be continued or in case of negative impact of the intervention on patients' health and well-being, terminated with further patients monitoring.
CONDITIONS
Official Title
The Efficacy and Safety of Metoprolol as add-on Treatment to Standard of Care in Preventing Cardiomyopathy in Patients With DMD
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Parent or legal guardian has provided written informed consent; assent obtained if applicable (over 16 years old)
- Willingness to comply with all study procedures and availability for the study duration
- Ability to take oral medication and follow the study treatment plan
- Confirmed diagnosis of Duchenne Muscular Dystrophy by genetic testing or dystrophin analysis
- Currently taking ACE inhibitor treatment at required doses for at least 30 days
You will not qualify if you...
- Current or previous permanent use of any beta-blocker medication
- Treatment with another investigational drug or intervention within 3 months prior to screening
- Clinically significant bradycardia or atrioventricular block above first degree at rest
- Presence of pacemaker or implantable cardioverter defibrillator (ICD)
- Signs or symptoms of heart failure
- Left ventricular ejection fraction below 57% by echocardiography
- Inability to obtain adequate quality echocardiography images
- Known allergy to any components of the investigational drugs
AI-Screening
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Trial Site Locations
Total: 1 location
1
University Clinical Centre in Gdańsk, Clinic of Paediatric Cardiology and Congenital Heart Defects
Gdansk, Pomeranian Voivodeship, Poland, 80-211
Actively Recruiting
Research Team
J
Joanna Kwiatkowska, MD, PhD
CONTACT
J
Jarosław Meyer-Szary, MD, PhD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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