A Phase 3, Randomized, Double-Blind, 48-Week Placebo-Controlled Study to Assess the Efficacy, Safety, and Tolerability of DYNE-101 Administered to Participants with Myotonic Dystrophy Type 1
This research study is looking into a new treatment called DYNE-101 for Myotonic Dystrophy Type 1 (DM1). DM1 is a condition that causes muscle weakness and other problems. Scientists want to see if DYNE-101 can help people with DM1 and if it's safe to use. Participants will be randomly given either DYNE-101 or a placebo (a dummy treatment that looks like the real medicine but has no active ingredients). No one, not even the doctors or the patients, will know who is getting which treatment until the study ends. This helps make the results fair. The study will last for 48 weeks, and researchers will mainly look at how walking and daily tasks are affected.
At a glance
What is this study about?
This clinical trial is designed to investigate a new potential treatment called DYNE-101 for individuals living with Myotonic Dystrophy Type 1, often referred to as DM1. DM1 is a lifelong condition that affects muscles, causing them to weaken and not relax properly after use. It can also impact other parts of the body.
The main goal of this study is to determine if DYNE-101 can improve daily functions and overall well-being for people with DM1, while also checking if it's safe and well-tolerated. Participants will receive either DYNE-101 or a placebo, which looks identical but contains no active medicine. This method is called 'double-blind,' meaning neither the patients nor the study doctors will know who is receiving which treatment. This is a common and important way to ensure the study results are reliable and unbiased.
The study will carefully track various aspects of participants' health over 48 weeks. For example, they will measure how long it takes to complete certain walking tests, as this can indicate changes in muscle strength and function. They will also monitor side effects, changes in general health, and how the body handles the medicine. This detailed information will help researchers understand if DYNE-101 could be a valuable new option for managing DM1.
Key takeaways
- This study is testing a new medicine (DYNE-101) for Myotonic Dystrophy Type 1.
- It aims to see if the medicine is safe and can improve daily activities.
- Participants will receive either the new medicine or a dummy treatment (placebo).
- Neither you nor the doctors will know which treatment you are getting.
- The study lasts for about 48 weeks with regular clinic visits.
- You can stop participating at any time.
Who may be eligible?
This study is looking for adults aged 18 years and older who have been diagnosed with Myotonic Dystrophy Type 1. Both men and women are invited to take part.
There might be other health conditions or medications that could prevent someone from joining, as these could affect the safety or results of the study. For instance, sometimes certain heart conditions, liver problems, or other serious illnesses mean a person cannot participate.
When you talk to the study team, they will go through a full list of these types of requirements to make sure the study is a safe and suitable option for you.
- Are you 18 years old or older?
- Have you been diagnosed with Myotonic Dystrophy Type 1?
- Are you able to attend regular appointments for about a year?
- Are you willing to potentially receive a placebo instead of the active drug?
- Are you currently not taking any medications that might interfere with the study drug?
This is a guide only — the research team will confirm whether you can take part.
What does participation involve?
If you decide to take part, you will be in the study for about 48 weeks (just under a year). You will have regular visits to the clinic where the study team will carry out various assessments. These might include blood tests, heart checks (ECGs), checking your vital signs like blood pressure, and specific tests to measure muscle strength and how you perform daily activities, such as walking tests.
During these visits, you will receive doses of either the study medicine (DYNE-101) or the placebo. You won't know which one you are getting. The study team will explain how and when to take your medication. You will also be asked to answer questionnaires about your health and how you are feeling. All these assessments help the researchers understand the effects of the treatment. After the 48 weeks, there might be a follow-up call or visit.
Potential risks and benefits
Locations (7)
- —Netherlands
- —Spain
- —Italy
- —Denmark
- —Germany
- —Belgium
- —France
Common questions
What is Myotonic Dystrophy Type 1 (DM1)?
DM1 is a genetic condition that causes muscles to become weak and waste away over time. It can also affect other body systems, like the heart and digestive system.
What is a 'placebo'?
A placebo is a dummy treatment that looks exactly like the real medicine but does not contain any active ingredients. It's used to compare against the new medicine to see if it actually works better than no treatment.
What does 'double-blind' mean?
Double-blind means that neither the patient nor the doctors and study staff know whether a person is receiving the active drug or the placebo. This helps make the study fair and stops anyone's beliefs from influencing the results.
How long will I be in the study?
The main part of the study will last for 48 weeks, which is just under a year. There might be some follow-up checks after that.
What kind of tests will I have?
You'll have regular physical exams, blood tests, heart checks (ECGs), and tests to measure your muscle strength and ability to do daily activities, like walking.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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