A Phase 2, Randomized, Double-Blind, Placebo-Controlled Trial to Assess the Efficacy and Safety of surlorian (ARM210, S48168) in Adults with Autosomal Dominant RYR1-Related Myopathy
This research study is looking at a new medication called Surlorian for adults who have a specific muscle weakening condition known as Autosomal Dominant RYR1-Related Myopathy. This condition causes muscles to be weaker than they should be. The study is a Phase 2 trial, meaning it's an early but important step in checking if Surlorian works and is safe. Participants will be given either Surlorian or a dummy medicine (placebo) without knowing which one they receive. Researchers will then measure changes in muscle strength and how well people can do daily tasks over about a month. They'll also carefully watch for any side effects.
At a glance
What is this study about?
This study is investigating a new medicine called Surlorian for adults living with a condition called Autosomal Dominant RYR1-Related Myopathy. This is a genetic muscle condition, meaning it's passed down through families, and it can cause muscles to be weaker than they should be, affecting everyday activities.
The main goal of this research is to see if Surlorian can help improve muscle strength and overall physical abilities in people with this condition. The study will also carefully check for any side effects the medicine might cause, to make sure it's safe. It's a 'Phase 2' study, which means it's one of the first times this medicine is being tested in a larger group of people to understand how well it works and whether it has any unwanted effects.
Participants will be placed into two groups: one group will receive Surlorian, and the other will receive a 'placebo', which looks exactly like Surlorian but contains no active medicine. Neither the participants nor the doctors will know who is getting what, which helps ensure the results are as fair and unbiased as possible. Over about 28 days, researchers will measure muscle strength, how easily people can move, and how they feel about their energy levels and physical abilities. They will compare the results between the two groups to see if Surlorian makes a difference.
Key takeaways
- Tests a new medicine, Surlorian, for a specific muscle condition.
- Aims to improve muscle strength and physical abilities.
- Compares Surlorian to a dummy medicine (placebo).
- Participation lasts about 28 days with regular clinic visits.
- Carefully monitors for safety and side effects.
- Helps advance understanding of Autosomal Dominant RYR1-Related Myopathy.
Who may be eligible?
To join this study, you need to be an adult, aged 18 or older. There is no upper age limit, meaning older adults are also welcome to take part.
You must have been diagnosed with Autosomal Dominant RYR1-Related Myopathy, as this study is specifically designed for people with this particular muscle condition.
Both men and women can participate in the study. The medical team will check other specific health details to make sure the study is a good fit for you and that it's safe for you to take part.
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 18 years old or older?
- Do you have a diagnosis of Autosomal Dominant RYR1-Related Myopathy?
- Are you able to attend regular clinic visits for about a month?
- Are you comfortable with taking either the study medicine or a placebo?
What does participation involve?
If you decide to join this study, you would be taking part for approximately 28 days. During this time, you would regularly take either the study medicine (Surlorian) or a dummy medicine (placebo). You won't know which one you're receiving, and neither will your study doctor, to keep the results fair.
You'll have several visits to the study clinic during the month. These visits will involve various checks, such as measuring your muscle strength and how well you can move. For example, some tests might look at how quickly you can walk, how easily you can stand up, or how strong your handgrip is. You might also be asked to fill out questionnaires about your energy levels, how you feel, and your daily activities.
The study team will also perform regular health checks, including measuring your heart rate, blood pressure, and taking blood and urine samples to monitor your general health and safety. You will be closely monitored for any side effects or changes to your health throughout the study.
Potential risks and benefits
Locations (4)
- —UnverifiedNetherlands
- —UnverifiedSpain
- —UnverifiedFrance
- —UnverifiedGermany
Common questions
What is Autosomal Dominant RYR1-Related Myopathy?
It's a genetic muscle condition where muscles are weaker than they should be, passed down in families.
What is a 'Phase 2' study?
It's an early but important stage of testing a new medicine in a small group of people to see if it works and is safe.
What is a 'placebo'?
A placebo looks like the real medicine but doesn't contain any active ingredients; it's used to compare results fairly.
How long will I be in the study?
Participation in this study will last for approximately 28 days.
Can I leave the study if I change my mind?
Yes, you can leave the study at any time without giving a reason, and it won't affect your regular medical care.
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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