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Study on Metoprolol for Preventing Heart Disease in Boys with Duchenne Muscular Dystrophy Aged 8-17

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying the effects of the medication Metoprolol in young patients with Duchenne Muscular Dystrophy (DMD). DMD is a genetic disorder characterized by progressive muscle degeneration and weakness. The study aims to see if Metoprolol, when added to the usual heart care, can help prevent or slow down the development of heart problems, specifically a condition called cardiomyopathy, which affects the heart muscle. Cardiomyopathy can lead to the heart not pumping blood as well as it should, which is a concern for patients with DMD.

Participants in the study will be randomly assigned to receive either Metoprolol or a placebo, in addition to their standard heart treatment. The study is designed to be double-blind, meaning neither the participants nor the researchers will know who is receiving Metoprolol and who is receiving the placebo. This helps ensure that the results are not biased. The main goal is to determine if Metoprolol can slow the decline in heart function, measured by a test called echocardiography, which uses sound waves to create images of the heart. The study will also look at how long it takes for participants to develop cardiomyopathy or heart failure, which is when the heart can't pump enough blood to meet the body's needs.

The trial will involve regular check-ups and heart function tests over a period of time to monitor the effects of the treatment. The study is expected to continue until 2026, providing valuable information on whether Metoprolol can be an effective addition to the treatment plan for young patients with DMD to help protect their heart health.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initial assessment
Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes reviewing the diagnosis of Duchenne Muscular Dystrophy (DMD) and ensuring the ability to take oral medication.
The participant's parent or legal guardian provides written consent, and the participant provides assent if over 16 years old.
Step 2
Randomization
Participants are randomly assigned to receive either the study medication, metoprolol, or a placebo. This process is double-blind, meaning neither the participant nor the study team knows which treatment is being administered.
Step 3
Medication administration
Participants take the assigned medication orally. The study involves two forms of metoprolol: Betaloc ZOK 100 (95 mg) and Betaloc ZOK 25 (23.75 mg), both in prolonged-release tablet form.
The dosage and frequency are determined by the study protocol and are administered alongside standard cardiac care.
Step 4
Regular monitoring
Participants undergo regular monitoring to assess heart function. This includes echocardiography to measure the left ventricular ejection fraction (LVEF), which helps evaluate heart performance.
The primary goal is to observe any changes in LVEF over time, comparing it to baseline measurements.
Step 5
Interim and final analysis
Interim and final analyses are conducted to evaluate the effectiveness of the treatment. The primary endpoint is the change in LVEF, while secondary endpoints include the time to develop cardiomyopathy and heart failure.
These analyses help determine the impact of metoprolol on preventing cardiomyopathy in participants.
Step 6
Study completion
The study is estimated to conclude by March 31, 2026. Participants will have contributed valuable data to understand the role of metoprolol in managing heart health in DMD patients.

Who can join the trial?

8 criteria

The child's parent(s) or legal guardian(s) must provide written permission for the child to join the study. If the child is over 16 years old, they will also need to agree in writing or verbally.
The child and their family must agree to follow all the study rules and be available for the entire study period.
The child must be a boy, aged 8 to 16 years old, at the time they join the study.
The child must be able to take medicine by mouth and agree to follow the study's treatment plan.
The child must have a confirmed diagnosis of **Duchenne muscular dystrophy (DMD)**. This means:
- The child shows typical signs of DMD, and tests show a complete lack of a protein called **dystrophin**.
- There is a known change in the **DMD gene**, such as a missing or extra part, that affects the gene's function.
- Complete gene testing shows a change that stops the production of dystrophin, like a specific type of mutation.
- The child must be taking a type of heart medicine called **ACE inhibitors (ACEi)** at the required doses for at least 30 days before joining the study.

Who cannot join the trial?

9 criteria

Patients who have a known allergy or adverse reaction to the study medication or any of its components cannot participate.
Individuals who are currently participating in another clinical trial or have participated in one within the last 30 days are excluded.
Patients with a history of severe heart problems, such as a heart attack or heart failure, are not eligible.
Those with uncontrolled high blood pressure, which means their blood pressure is not well-managed with medication, cannot join the study.
Individuals with severe liver or kidney disease, which affects how the body processes medications, are excluded.
Patients who are unable to follow the study procedures or take the study medication as directed are not eligible.
Women who are pregnant or breastfeeding cannot participate in the study.
Individuals with a history of drug or alcohol abuse within the past year are excluded.
Patients with any other medical condition that the study doctors believe would make participation unsafe or affect the study results are not eligible.

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Uniwersyteckie Centrum Kliniczne
Gdansk, Poland

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Investigated drugs

Metoprolol is a medication used in this study to see if it can help prevent heart problems in boys with Duchenne Muscular Dystrophy. It is given in addition to the usual heart treatments to see if it can slow down the worsening of heart function.

Metoprolol Tartrate

What is already known about the treatment

Metoprolol – Metoprolol is administered orally, typically in tablet form, and is used in this clinical trial as an add-on treatment to standard cardiac care. It is currently being studied for its potential to prevent cardiomyopathy in patients with Duchenne Muscular Dystrophy (DMD), particularly in boys aged 8-17 years. The main therapeutic indication in this trial is to slow the progression of heart muscle weakening, which can lead to cardiomyopathy. Metoprolol works by blocking beta-adrenergic receptors, which reduces heart rate and the force of heart contractions, thereby decreasing the heart's workload. It is classified as a beta-blocker, a type of medication commonly used to manage heart conditions.

Investigated diseases

Duchenne Muscular Dystrophy
This is a genetic disorder characterized by progressive muscle weakness and degeneration. It primarily affects boys and symptoms usually begin in early childhood. Over time, muscle weakness spreads to other parts of the body, including the heart and respiratory muscles. As the disease progresses, individuals may experience difficulty walking, frequent falls, and challenges with motor skills. Eventually, the condition can lead to severe mobility issues and the need for assistive devices.
Cardiomyopathy
This is a disease of the heart muscle that affects its size, shape, and structure. It can lead to the heart becoming enlarged, thickened, or rigid, which affects its ability to pump blood effectively. Over time, this can result in symptoms such as fatigue, shortness of breath, and swelling in the legs and feet. The progression of cardiomyopathy can vary, with some individuals experiencing a gradual worsening of symptoms. It can affect people of all ages and may be inherited or develop due to other conditions.
Tachycardia
This condition is characterized by an abnormally fast heart rate, typically over 100 beats per minute in adults. It can occur in the upper or lower chambers of the heart and may be caused by various factors, including stress, heart disease, or an imbalance of electrolytes. Symptoms can include palpitations, dizziness, and shortness of breath. The condition can be temporary or persistent, and its progression depends on the underlying cause. In some cases, tachycardia may lead to more serious heart complications if not managed.