About the study

The MeMDM study evaluates whether early initiation of metoprolol treatment can slow disease progression and delay the development of cardiomyopathy in patients with Duchenne muscular dystrophy (DMD).

The project is designed as a randomised, double-blind, placebo-controlled clinical trial, allowing an objective assessment of both treatment efficacy and safety.

What is Duchenne muscular dystrophy?

Duchenne muscular dystrophy (DMD) is a severe X-linked genetic disorder characterised by progressive damage to skeletal muscles, respiratory muscles and the heart muscle.

Cardiac involvement is one of the most important complications of the disease and has a significant impact on patient prognosis. For this reason, regular cardiology care and early detection of cardiac abnormalities are essential.

Routine monitoring of patients may include:

• electrocardiography (ECG);
• Holter ECG monitoring;
• echocardiography (ECHO);
• cardiac magnetic resonance imaging (CMR).

Why is a new approach needed?

Despite advances in the care of patients with DMD, there is still a lack of treatment strategies that effectively delay the development of cardiomyopathy.

Early introduction of cardioprotective therapy may play a key role in improving both life expectancy and quality of life.

Why metoprolol?

Metoprolol belongs to a group of medicines known as beta-blockers, which are commonly used in the treatment of cardiovascular diseases.

These medicines reduce heart rate and cardiac workload. The study evaluates whether early use of metoprolol in patients with DMD can delay cardiac damage and improve long-term outcomes.

Study design

Approximately 140 patients aged 8–18 years with genetically confirmed DMD are expected to participate in the study.

Participants are randomly assigned to receive either metoprolol or placebo. The study is conducted in a double-blind manner, meaning that neither the patient nor the treating physician knows which treatment is being administered.

Patients are followed for up to five years, with regular diagnostic assessments performed throughout the study to monitor cardiac function and disease progression.

Study procedures

Following eligibility assessment, participants undergo screening procedures and randomisation.

Some patients may first receive standard cardioprotective therapy with an ACE inhibitor (ACEi) or angiotensin receptor blocker (ARB). The main study phase then begins, during which participants receive either metoprolol or placebo in an identical formulation.

Regular follow-up visits and diagnostic examinations are conducted throughout the study. Additional medical care is provided if adverse events occur.

Study objectives

The primary objective is to determine whether early metoprolol treatment can slow disease progression and delay the development of cardiomyopathy in patients with DMD.

In the long term, the project aims to improve quality of life and prolong survival.

Study population

Eligible participants are patients aged 8–18 years with genetically confirmed Duchenne muscular dystrophy.

Detailed inclusion and exclusion criteria were not available in the project materials and should be confirmed directly with the study centre.

Centre experience

The University Clinical Centre in Gdańsk has been providing comprehensive care for patients with Duchenne muscular dystrophy for many years, including regular cardiological assessments in accordance with current clinical guidelines.

Nearly 100 patients from across Poland are under the centre’s care, making it one of the leading referral centres for DMD management in the country.

Organisational information

The project is funded by the Polish state budget through the Medical Research Agency (MRA).

Project duration: up to 60 months.