Global Study of Del-desiran for the Treatment of DM1
This study, called the Global Study of Del-desiran, is looking at a new treatment for Myotonic Dystrophy Type 1 (DM1), also known as Steinert's disease. Researchers want to find out if the medicine, named del-desiran, is safe and effective in improving this condition. Participants will be randomly assigned to receive either del-desiran or a placebo (a treatment with no active medicine) through an IV drip every eight weeks for about a year. Doctors and patients won't know who is getting which treatment. The study will last around 60 weeks in total. Afterwards, there might be an option to continue receiving the medicine in a long-term study, if approved.
At a glance
What is this study about?
Myotonic Dystrophy Type 1 (DM1), sometimes called Steinert's disease, is a condition that affects muscles, causing them to weaken and have trouble relaxing after use. This can impact many daily activities. Currently, there isn't a cure, and treatments mainly help manage the symptoms. This study aims to see if a new investigative medicine, called del-desiran, could be a helpful treatment for people living with DM1.
This is a 'Phase 3' study, which means it's one of the final stages of testing a new medicine before it might be considered for wider use. It's designed to be 'randomised', meaning participants are put into groups by chance, and 'double-blind', meaning neither the patient nor the doctor knows if they're receiving the new medicine or a placebo (a dummy treatment). This helps ensure the study results are as fair and accurate as possible.
The main goal of this research is to see if del-desiran is safe for people with DM1 and if it can improve their symptoms or slow the progression of the disease. An independent group of experts will regularly check on the study's progress to make sure everyone is safe.
Key takeaways
- Tests a new medicine (del-desiran) for Myotonic Dystrophy Type 1 (DM1).
- Compares the medicine to a placebo (dummy treatment).
- Participation involves IV infusions every 8 weeks for about a year.
- Aims to check if del-desiran is safe and effective.
- For adults aged 16-65 who can walk independently.
- You won't know if you're getting the active drug or placebo.
Who may be eligible?
This study is looking for adults aged 16 to 65 who have an official diagnosis of Myotonic Dystrophy Type 1 (DM1), confirmed by genetic testing. You also need to be able to walk by yourself, even if you use aids like ankle braces, for at least 10 metres.
There are also some reasons why you might not be able to join. For example, if you are pregnant, breastfeeding, or planning to become pregnant during the study. You also can't take part if you have serious uncontrolled diabetes, certain heart conditions, or if you've recently taken other experimental medicines.
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 confirmed diagnosis of Myotonic Dystrophy Type 1 (DM1)?
- Am I between 16 and 65 years old?
- Can I walk on my own for at least 10 metres, even with walking aids?
- Am I not pregnant, breastfeeding, or planning to become pregnant while in the study?
- Do I have generally stable health conditions?
What does participation involve?
If you join this study, you'll first have a screening period that could last up to six weeks to check if you're suitable. The treatment part of the study will then last about 54 weeks. Overall, your involvement will be for around 60 weeks, which is a bit over a year.
During the treatment period, you'll visit the study site every eight weeks to receive either del-desiran or a placebo through an IV drip. You'll receive a total of seven doses, with the last one at Week 48. There will be a final check-up at Week 54. After this, if you meet the requirements and it's approved, you might have the option to join a long-term study where everyone receives the active medicine.
Potential risks and benefits
Locations (34)
- Stanford UniversityVerified postcodeStanford, United States
- University of ColoradoVerified postcodeDenver, United States
- University of FloridaVerified postcodeGainesville, United States
- University Research Center of South FloridaVerified postcodeTampa, United States
- Indiana University (IU)Verified postcodeIndianapolis, United States
- Kansas University Medical CenterVerified postcodeKansas City, United States
- Kennedy Krieger InstituteVerified postcodeBaltimore, United States
- Beth Israel Deaconess Medical CenterVerified postcodeBoston, United States
- University of MinnesotaVerified postcodeMinneapolis, United States
- University of Rochester Medical CenterVerified postcodeRochester, United States
- Duke University Medical CenterVerified postcodeDurham, United States
- Wake ForestVerified postcodeWinston-Salem, United States
Common questions
What is Myotonic Dystrophy Type 1 (DM1)?
DM1 is a genetic condition that causes muscles to become weak and have difficulty relaxing. It can affect many parts of the body.
What is a 'placebo'?
A placebo is a dummy treatment that looks exactly like the real medicine but contains no active ingredients. It's used to compare against the new medicine.
Will I know if I'm getting the drug or placebo?
No, this is a 'double-blind' study, meaning neither you nor your doctors will know if you're receiving del-desiran or the placebo until after the study ends.
How often do I need to visit the clinic?
After screening, you'll need to visit the clinic every eight weeks for IV infusions, for a total of seven doses, and a final check-up.
What happens after the study finishes?
After 54 weeks, if you meet certain criteria and it's allowed, you might have the option to join a separate long-term study where you would receive the active medicine.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
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