A PHASE 1/2, OPEN-LABEL, EXPLORATORY CLINICAL TRIAL TO EVALUATE THE SAFETY AND EFFICACY OF DT-DEC01 THERAPY IN PATIENTS WITH DUCHENNE MUSCULAR DYSTROPHY
This study is a clinical trial looking into a new treatment called DT-DEC01 for Duchenne Muscular Dystrophy (DMD). It's designed to find out how safe the treatment is and if it can help improve muscle strength and movement in people with DMD. The study involves two main parts, Phase 1 and Phase 2. Researchers will closely monitor for any side effects and will measure how well patients can move, walk, and perform daily tasks. They will also look at muscle health using special tests. Both patients who can walk (ambulatory) and those who cannot (non-ambulatory) will be included. This is an exploratory study, meaning it's a first step to understand the treatment's potential.
At a glance
What is this study about?
This clinical trial is about a new treatment called DT-DEC01 for Duchenne Muscular Dystrophy, often just called DMD. DMD is a condition that causes muscles to become weaker over time. The main goal of this study is to see if DT-DEC01 is safe for people with DMD and if it can help improve their muscle function and overall well-being.
The study is divided into two parts, known as Phase 1 and Phase 2. In Phase 1, the main focus is on safety: researchers will carefully watch for any side effects or unexpected reactions to the treatment. They also start to look at how well the treatment might be working by checking muscle strength and movement. In Phase 2, they continue monitoring safety but specifically focus more on how effective the treatment is, by observing changes in daily activities and muscle health over time.
Researchers will be looking at several things to understand the treatment's effects. This includes how easily patients can move, such as their ability to walk a certain distance or stand up from the floor. For those who cannot walk, they will use other tests to measure arm and hand function. They'll also use a special test called an EMG to look at how well muscles are working at a microscopic level. These measurements will help them see if the treatment helps slow down the progression of muscle weakness in people with DMD.
Key takeaways
- This study is testing a new treatment (DT-DEC01) for Duchenne Muscular Dystrophy.
- The main goals are to check if the treatment is safe and if it helps improve muscle function.
- Both patients who can walk and those who cannot will be included.
- Participation involves regular clinic visits and tests over 12 months.
- It's an early-stage study, meaning it's one of the first times this treatment is given to people.
- You can stop participating in the study at any time.
Who may be eligible?
To join this study, people need tohave been diagnosed with Duchenne Muscular Dystrophy. The study is specifically looking for male patients, but there isn't a strict age limit mentioned, meaning a wide range of ages might be considered.
Since this is an early-stage study, there will be other important health requirements to make sure it's safe for people to take part. These usually include things like having stable health conditions and not taking certain medications that could interfere with the study treatment.
It's really important to talk to your doctor or the study team to understand all the specific reasons why someone might be able or not able to join. They can explain the full list of criteria and answer any questions you have.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Are you male?
- Do you have a confirmed diagnosis of Duchenne Muscular Dystrophy?
- Are you and your doctor comfortable with you trying an experimental treatment?
- Are you able to attend regular clinic appointments for a year?
What does participation involve?
If you join this study, you'll receive the new treatment called DT-DEC01. You'll have regular visits to the clinic over a period of 12 months. During these visits, the study team will carry out various assessments. These include checks for any side effects, and tests to measure your muscle strength and how well you can move. For example, you might be asked to walk for a certain distance if you can, stand up, or perform tasks to assess your arm and hand function. There will also be a special test called an EMG to look at your muscles. These visits will happen at month 1, 3, 6, and 12 from when you start the treatment. The total duration of active participation in the study involves these regular check-ups and the treatment throughout the year.
Potential risks and benefits
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Common questions
What is Duchenne Muscular Dystrophy?
Duchenne Muscular Dystrophy (DMD) is a genetic condition that causes muscles to become weak and waste away over time.
What is DT-DEC01?
DT-DEC01 is a new, experimental treatment being tested for Duchenne Muscular Dystrophy in this study.
What does 'Phase 1/2' mean?
It means the study is in its early stages. Phase 1 focuses on safety, and Phase 2 starts to look at how well the treatment works, while still checking for safety.
Will I have to stay in hospital?
The information provided suggests regular clinic visits for tests and treatment, rather than a hospital stay, but you should confirm this with the study team.
How long will the study last?
The main part of the study involves assessments and treatment for 12 months.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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