Study of SRP-1003 in Participants With Type 1 Myotonic Dystrophy
This research study is looking at a new potential medicine, SRP-1003, for people living with Type 1 Myotonic Dystrophy (DM1). The main goals are to check if the medicine is safe and if the body tolerates it well. Scientists also want to understand how the medicine moves through the body and what effects it has. The study involves giving different doses of SRP-1003, either into a vein or under the skin, or a dummy medicine (placebo). This helps researchers compare the real medicine's effects. Participants will be carefully monitored to see how they react to the treatment. This is an early-stage study, meaning it's one of the first times this medicine is being tested in people with DM1.
At a glance
What is this study about?
This study is an important step in developing new treatments for Type 1 Myotonic Dystrophy (DM1). DM1 is a condition that affects your muscles, making them weak and sometimes making it hard for them to relax after you use them. The new medicine being tested, called SRP-1003, aims to tackle some of the problems caused by DM1.
In this study, researchers want to answer several key questions. Firstly, is SRP-1003 safe for people with DM1, and can their bodies handle it without too many side effects? Secondly, they want to understand how the medicine works in the body – for example, how much of it gets into your system and how long it stays there. Thirdly, they're hoping to see if the medicine has any positive effects on the body's processes related to DM1.
To find these answers, some people in the study will receive SRP-1003, while others will receive a 'placebo', which looks exactly like the medicine but contains no active drug. This is a common way to compare results fairly and find out if any changes are truly due to the new medicine. Because this is an early-stage study, it's about gathering initial information to guide future research.
Key takeaways
- This study is testing a new medicine (SRP-1003) for Type 1 Myotonic Dystrophy.
- It's an early-stage study focusing on safety, how the body handles the medicine, and potential effects.
- Participants will receive either the new medicine or a dummy medicine (placebo).
- The medicine will be given into a vein or as an injection under the skin.
- Strict rules apply for who can and cannot join, including age and medical history.
Who may be eligible?
To join this study, you need to have a confirmed diagnosis of Type 1 Myotonic Dystrophy (DM1) through a genetic test. Your doctor also needs to have noticed clear signs of DM1, including muscle stiffness (myotonia). Your DM1 symptoms must have started after you turned 12 years old, and you must be able to walk at least 10 metres on your own before the study starts. You'll need to be between 18 and 65 years old.
There are also some reasons why you might not be able to join. For example, if you have diabetes that's not well controlled, very high blood pressure that's not under control, or serious heart, liver, or kidney problems. If you have congenital DM1 (a type of DM1 that starts very early in life), or certain infections like HIV or hepatitis B or C, you wouldn't be able to participate. You also shouldn't have had a muscle biopsy in your lower leg in the last three months, or be planning to have one during the study.
If you could become pregnant or make someone pregnant, you would need to use very effective contraception throughout the study and for 90 days after your last dose. You also wouldn't be able to donate sperm or eggs during this time.
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 I have a genetically confirmed diagnosis of Type 1 Myotonic Dystrophy?
- Did my DM1 symptoms start after I turned 12 years old?
- Can I walk at least 10 meters on my own?
- Am I between 18 and 65 years old?
- Do I have any serious uncontrolled medical conditions like diabetes, high blood pressure, or heart/liver/kidney disease?
- Am I willing to use effective contraception if I could become pregnant or make someone pregnant?
What does participation involve?
If you decide to take part, you'll first have screening visits to make sure you meet all the study requirements. Once enrolled, you'll either receive SRP-1003 or a placebo, which looks identical but has no active drug. The medicine will be given either into a vein (like a drip) or as an injection under the skin.
Some participants will receive a single dose, while others will receive multiple doses over time. You will have regular appointments at the study clinic for check-ups, blood tests, and other assessments to see how you are doing and how your body is responding to the treatment. These visits help the research team monitor your safety and the medicine's effects. The total length of your involvement in the study will depend on which part of the study you are in (single or multiple doses), but it will involve follow-up after your last dose. You will be told the exact schedule of visits and procedures if you are considering joining.
Potential risks and benefits
Locations (35)
- Liverpool HospitalVerified postcodeLiverpool, Australia· Recruiting
- Sunshine Coast University HospitalVerified postcodeBirtinya, Australia· Recruiting
- Royal Brisbane And Women's HospitalVerified postcodeHerston, Australia· Recruiting
- Alfred HealthVerified postcodeMelbourne, Australia· Recruiting
- Nucleus Network Brisbane ClinicVerified postcodeHerston, Australia· Recruiting
- Mater Hospital BrisbaneVerified postcodeSouth Brisbane, Australia· Recruiting
- Universitair Ziekenhuis GentVerified postcodeGhent, Belgium· Recruiting
- UZ LeuvenVerified postcodeLeuven, Belgium· Recruiting
- University Of CalgaryVerified postcodeCalgary, Canada· Recruiting
- University Of AlbertaVerified postcodeEdmonton, Canada· Recruiting
- McGill UniversityVerified postcodeMontreal, Canada· Recruiting
- University Hospital of AngersVerified postcodeAngers, France· Recruiting
Common questions
What is Type 1 Myotonic Dystrophy (DM1)?
DM1 is a genetic condition that causes muscles to become weak and can affect how they relax. It can also cause problems in other parts of the body.
What does 'placebo-controlled' mean?
It means some participants will receive the active medicine, while others will receive a 'dummy' medicine (placebo) that looks the same but has no drug in it. This helps researchers compare the true effects of the new medicine.
What does 'Phase 1/2' mean for a study?
These are early stages of research. Phase 1 mainly checks if the medicine is safe and what dose can be given. Phase 2 starts looking at whether the medicine actually works for the condition, as well as continuing to check safety.
Will I know if I'm getting the active medicine or the placebo?
No, this is a 'double-blinded' study, meaning neither you nor the study team will know who is getting the active medicine and who is getting the placebo. This ensures the results are fair and unbiased.
How is the medicine given?
The medicine SRP-1003 will be given either into a vein (like a drip in your arm) or as an injection just under the skin.
How to find out more
Sarepta Therapeutics Inc. For Clinical Trial Information, Select Option 4
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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