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Study on DMD06-MAB Injection for Duchenne Muscular Dystrophy in Non-Ambulant Patients

Verified siteInvestigationalNo placebo

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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 Duchenne Muscular Dystrophy (DMD), a genetic disorder characterized by progressive muscle degeneration and weakness. The treatment being tested is called DMD06-MAB, which involves a single injection of specially prepared cells known as mesoangioblasts (MABs) into specific muscles. These cells are genetically modified to help correct a part of the dystrophin gene, which is responsible for muscle function. The aim of the study is to evaluate the safety of this treatment and its ability to restore the production of dystrophin, a protein that is lacking in individuals with DMD.

The study will involve five participants who are unable to walk due to DMD. Each participant will receive an injection of the genetically corrected MABs into a muscle in the foot. The researchers will monitor the participants for any adverse effects and will also check if the treatment successfully increases the levels of dystrophin in the muscle. The study will last for one year, during which the safety and effectiveness of the treatment will be closely observed.

Participants will be monitored for any local or systemic adverse events following the injection. The primary goal is to ensure the treatment is safe and to determine if there is an increase in dystrophin levels in the muscle, which will be assessed through muscle biopsies. The study is designed to provide valuable information on the potential of this innovative treatment for improving muscle function in individuals with Duchenne Muscular Dystrophy.

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 verifying age, non-ambulant status, and a confirmed diagnosis of Duchenne Muscular Dystrophy with specific genetic mutations.
A quantitative MRI is performed to assess muscle degeneration, ensuring it is within the required range.
Step 2
Informed consent
Written informed consent is obtained from caregivers, and the patient's assent is required to proceed with the study.
Step 3
Pre-injection preparation
Pre-clinical safety assessments are conducted on the treatment product, DMD06-MAB, to ensure it is safe for use.
Step 4
Treatment administration
A single injection of DMD06-MAB, a suspension for injection, is administered intramuscularly into the foot muscle known as the Extensor Digitorum Brevis (EDB).
The treatment involves genetically corrected cells designed to help restore dystrophin production in the muscle.
Step 5
Post-injection monitoring
Monitoring for adverse events occurs over the course of one year following the injection. This includes both local reactions at the injection site and any systemic effects.
Regular assessments are conducted to evaluate the presence of dystrophin in the muscle, aiming for levels comparable to at least 10% of a healthy individual.
Step 6
Efficacy evaluation
Three months after the injection, a muscle biopsy is performed to assess the presence of dystrophin using various laboratory techniques.
The goal is to confirm the effectiveness of the treatment in restoring dystrophin production in the treated muscle.

Who can join the trial?

5 criteria

Must be a boy aged between 12 and 17 years old at the time of joining the study.
Cannot walk at the time of joining the study.
Must have a confirmed diagnosis of Duchenne Muscular Dystrophy (DMD) with specific changes in the dystrophin gene that can be treated by skipping exon 51. The dystrophin gene is important for muscle function.
Muscle loss should not be more than 50%, as measured by a special scan called quantitative MRI. This scan helps to see how much muscle is left.
Caregivers must provide written permission for the patient to join the study, and the patient must agree to participate.

Who cannot join the trial?

4 criteria

Patients who do not have Duchenne Muscular Dystrophy cannot participate. This is a genetic disorder that affects muscles.
Females cannot participate in this study. Only males are eligible.
Patients who are not within the specified age range for the study cannot participate. The exact age range is not provided here.
Patients who are part of a vulnerable population, which means they might need special protection or care, cannot participate.

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Verified
Ospedale San Raffaele S.r.l.
Milan, Italy

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Investigated drugs

Autologous Mesoangioblasts (MABs): These are special cells taken from the patient's own body. In this trial, they are genetically corrected to help produce dystrophin, a protein that is missing or not working properly in patients with Duchenne Muscular Dystrophy. The corrected cells are injected into specific muscles to see if they can help restore the production of dystrophin and improve muscle function.

Dmd06-Mab

What is already known about the treatment

Autologous Mesoangioblasts (MABs) – This medication is administered through a single injection directly into individual skeletal muscles. It is currently being studied in clinical trials for its potential to treat Duchenne Muscular Dystrophy, a severe muscle-wasting condition. The main therapeutic goal is to restore the synthesis of dystrophin, a protein lacking in patients with this condition. The mechanism involves using genetically corrected cells that skip exon 51 of the dystrophin gene, allowing for the production of functional dystrophin. This approach falls under the category of gene therapy, aiming to correct genetic defects at the molecular level.

Investigated diseases

Duchenne Muscular Dystrophy – Duchenne Muscular Dystrophy is a genetic disorder characterized by progressive muscle weakness and degeneration. It primarily affects boys and is caused by mutations in the dystrophin gene, which is crucial for maintaining muscle cell structure. Symptoms usually begin in early childhood, with affected individuals experiencing difficulty in walking, running, and jumping. As the disease progresses, muscle weakness spreads to the arms, neck, and other areas, leading to increased difficulty in performing daily activities. Over time, individuals may lose the ability to walk and require the use of a wheelchair. The progression of muscle weakness can also affect the heart and respiratory muscles.