Study on the Safety and Effectiveness of DT-DEC01 for Patients with Duchenne Muscular Dystrophy

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What is this study about?

This clinical trial is focused on studying a condition known as Duchenne Muscular Dystrophy (DMD), a genetic disorder characterized by progressive muscle weakness and degeneration. The trial will explore the safety and effectiveness of a new treatment called DT-DEC01. This treatment involves a special type of cell therapy where cells from a healthy donor are combined with cells from a person with DMD. These combined cells are then injected into the patient to potentially improve muscle function.

The purpose of the study is to evaluate how safe and effective this new treatment is for patients with DMD. Participants in the study will receive the DT-DEC01 treatment through an injection into the bone. The study will monitor participants over time to observe any side effects and to assess changes in muscle strength and function. This will include tests like the Six-Minute Walk Test, which measures how far a person can walk in six minutes, and other assessments tailored to whether the patient can walk or not.

The study is divided into two phases. In the first phase, the focus is on observing any adverse effects and initial signs of improvement in muscle function. The second phase continues to monitor safety and looks more closely at how well the treatment works over a longer period. Throughout the study, various tests will be conducted to measure muscle strength and quality of life, providing a comprehensive view of the treatment’s impact on patients with Duchenne Muscular Dystrophy.

1 initial assessment and consent

The patient and their legal representative must understand and agree to participate in the study by signing the informed consent form.

Eligibility is confirmed for boys aged 5 to 18 years with Duchenne Muscular Dystrophy (DMD) confirmed by genetic testing.

2 preparation for treatment

The patient must have been receiving glucocorticosteroids for at least 6 months before the muscle tissue biopsy.

The patient must be cleared by an anesthesiologist for the biopsy and the DT-DEC01 injection, which will be performed under anesthesia.

3 treatment administration

The treatment involves the administration of DT-DEC01, a suspension for injection, through intraosseous use.

The procedure is performed under anesthesia to ensure comfort and safety.

4 monitoring and assessments

The primary focus is on evaluating safety by observing any adverse events, including serious ones.

Efficacy is assessed through functional tests adjusted to the disease stage, such as the Six-Minute Walk Test (6MWT) for ambulatory patients and PUL 2.0 for non-ambulatory patients.

Electromyography (EMG) assessments are conducted to evaluate muscle function.

5 follow-up visits

Follow-up assessments occur at months 1, 3, 6, and 12 to monitor changes in muscle strength and function.

Quality of life is assessed using the PODCI questionnaire at these intervals.

Who Can Join the Study?

The patient and their legal representative or guardian must understand and agree to participate in the study by signing an approved consent form.
The patient must be a boy aged between 5 and 18 years old at the time of screening, with a diagnosis of Duchenne Muscular Dystrophy (DMD) confirmed by genetic testing.
The patient must have been taking glucocorticosteroids (a type of medication) for at least 6 months before a muscle tissue biopsy is done.
The patient must have ongoing, symmetrical muscle weakness in the arms and legs, which means the weakness is similar on both sides of the body.
The patient must be willing and able to attend scheduled visits, undergo a muscle tissue biopsy under anesthesia, follow the drug administration plan, complete laboratory tests, adhere to study restrictions, and participate in functional testing that matches the stage of their disease.
If the patient is sexually active, they must be willing to use acceptable forms of contraception.
The patient must be approved by an anesthesiologist to undergo the muscle tissue biopsy and DT-DEC01 intraosseous injection procedures, which will be performed under anesthesia (local, general, or with sedation).

Who Cannot Join the Study?

Only male patients can participate. Female patients are not eligible.
Patients must have a diagnosis of Duchenne Muscular Dystrophy, a genetic disorder that affects muscles.
Patients must be within a specific age range, typically starting from a young age.
Patients who are part of a vulnerable population, meaning they may need special protection or care, are considered for the study.

Where you can join this trial?

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Verified Sites

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Other Sites

Site Name	City	Country	Status
Med Polonia Sp. z o.o.	Poznan	Poland

Trial status

Country	Status	Recruitment Start
Poland	Not yet recruiting	12.12.2027

Trial locations

DT-DEC01 is an investigational therapy being studied for its safety and effectiveness in patients with Duchenne Muscular Dystrophy. This therapy is being tested to see how well it works in improving muscle strength and function. The trial aims to observe any side effects and determine how the therapy affects the ability of patients to perform certain physical tasks, such as walking or moving from a lying to a standing position. The therapy is being evaluated in both patients who can walk and those who cannot, using various tests to measure muscle function and strength.

Investigated diseases:

Duchenne muscular dystrophy

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 gene responsible for producing dystrophin, a protein essential for muscle function. The disease typically begins in early childhood, with symptoms such as difficulty walking, frequent falls, and challenges in climbing stairs. As the condition progresses, muscle weakness spreads to other parts of the body, including the arms and trunk. Over time, individuals may lose the ability to walk and require assistance with daily activities. The progression of muscle weakness can also affect the heart and respiratory muscles, leading to additional health challenges.

Trial ID:

2024-519004-27-00

Protocol code:

DT-DEC01-DMD-CT-1/2

Trial Phase:

Phase I and Phase II (Integrated) – First administration to humans

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Study on the Safety and Effectiveness of DT-DEC01 for Patients with Duchenne Muscular Dystrophy

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