A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)
This research study is about a new drug, VX-670, for adults living with Myotonic Dystrophy Type 1 (DM1). The main goal is to check how safe the drug is when given at different doses, and how well it's tolerated by the body. Researchers also want to understand how the body processes the medicine and if it has any effects on the condition. Participants will be aged between 18 and 64 years. Some will receive the active drug (VX-670), while others will get a placebo (a dummy treatment). This is a standard way to test new medicines fairly. The study is divided into two parts to gather important information on both safety and initial signs of effectiveness.
At a glance
What is this study about?
This study is looking into a new treatment called VX-670 for a condition called Myotonic Dystrophy Type 1, often shortened to DM1. DM1 is a type of muscular dystrophy that can affect many parts of the body, including muscles. We want to understand if this new medicine is safe for people with DM1 and if it can help with the condition.
The study will involve giving participants either the new medicine, VX-670, or a 'placebo'. A placebo looks just like the real medicine but contains no active drug. This is common in medical research and helps us fairly compare the effects of the new medicine against not having it. By giving different doses, we can work out the best and safest amount of the medicine.
The information gathered from this study, especially in its early phases, helps us decide whether VX-670 could be a helpful treatment for DM1 in the future. It's an important step in developing new ways to manage this condition.
Key takeaways
- Tests a new medicine (VX-670) for Myotonic Dystrophy Type 1 (DM1).
- Aims to check safety and how the body handles the medicine.
- Compares VX-670 to a placebo (dummy treatment).
- Open to adults aged 18-64 with a confirmed DM1 diagnosis.
- Requires regular clinic visits and health checks.
- Participation is voluntary, and you can withdraw at any time.
Who may be eligible?
To join this study, you must have a confirmed diagnosis of Myotonic Dystrophy Type 1 (DM1). This diagnosis needs to be supported by a genetic test showing a certain change (called a CTG repeat of at least 100). Also, your symptoms of DM1 must have started when you were older than one year old. You need to be between 18 and 64 years old to take part.
There are also some reasons why you might not be able to join. For example, if you have certain other health conditions or illnesses, you might not be eligible. This is to make sure the study is as safe as possible for everyone involved and that we can clearly see the effects of the new medicine.
Your study doctor will go through all the detailed requirements with you to see if this study is a good fit. It's important to be open about your full health history.
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.
- Do you have a personal diagnosis of Myotonic Dystrophy Type 1 (DM1)?
- Did your DM1 symptoms appear after you were one year old?
- Do you have a genetic test result confirming DM1 with at least 100 CTG repeats?
- Are you between 18 and 64 years old?
- Do you not have any other major health conditions that might prevent you from joining a study?
What does participation involve?
If you decide to take part, you will have regular visits to the study clinic. During these visits, the study team will carry out various checks and assessments to see how you are doing. This often includes things like physical exams, blood tests, and other specific tests related to your condition. You will be given either the study medicine (VX-670) or a placebo, which you'll take as instructed by the study team.
The study has two parts. The first part will look at different single doses of the medicine, and the second part will look at giving the medicine multiple times. You will be closely monitored throughout your participation to ensure your safety and track any changes. The total length of time you'd be involved in the study will be explained in detail by the study team, including how many visits and follow-up checks you'll have.
Potential risks and benefits
Locations (26)
- Stanford Neuromuscular ResearchVerified postcodeSan Carlos, United States· Recruiting
- University of Florida Clinical Research CenterVerified postcodeGainesville, United States· Recruiting
- University of Kansas Medical CenterVerified postcodeFairway, United States· Recruiting
- Boston Children's HospitalVerified postcodeBoston, United States· Recruiting
- Washington University School of Medicine / St. Louis Children's HospitalVerified postcodeSt Louis, United States· Recruiting
- Wake Forest Baptist HealthVerified postcodeWinston-Salem, United States· Recruiting
- University of PennsylvaniaVerified postcodePhiladelphia, United States· Recruiting
- Virginia Commonwealth University (Sanger Hall)Verified postcodeRichmond, United States· Recruiting
- Wesley Research InstituteVerified postcodeAuchenflower, Australia· Recruiting
- Neuroscience Clinical Trials Unit, Alfred BrainVerified postcodeMelbourne, Australia· Recruiting
- Universitaire Ziekenhuizen Leuven - Campus GasthuisbergVerified postcodeLeuven, Belgium· Recruiting
- Hopital de ChicoutimiVerified postcodeChicoutimi, Canada· Recruiting
Common questions
What is Myotonic Dystrophy Type 1 (DM1)?
It's a long-term muscle disease that can cause muscle weakness and slowness, and affect other body systems, including the heart and brain.
What is a 'placebo'?
A placebo looks like the study medicine but doesn't contain any active drug. It helps researchers fairly compare how much the new drug helps versus not taking it.
Will I know if I'm getting the real medicine or the placebo?
No, usually neither you nor the study team will know during the study. This helps ensure unbiased results.
Are there any costs involved for me?
Clinical trials generally cover all study-related medical costs. Check with the study team about travel or other expenses.
How long will I be in the study?
The total duration of your participation, including visits and follow-ups, will be clearly explained by the study team before you join.
How to find out more
Medical Information
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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