Ongoing, recruitingTherapeutic use (Phase IV)Interventional

COMPIS - Congenital myopathy intervention study. An open-label, cross over, randomized, controlled study using salbutamol

This study is investigating whether a common medicine called salbutamol, often used for asthma and known by the brand name Ventolin, could help people with a condition called congenital myopathy. Congenital myopathy affects muscle strength from birth. The study wants to see if taking salbutamol for six months can improve muscle function and make everyday tasks easier. Researchers will compare people who take salbutamol with those who don't, to understand its effects. This type of study (Phase IV) means the medicine is already approved for other uses but is being looked at for a new purpose to learn more about its long-term benefits.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic use (Phase IV)

Sponsor

Vaestra Goetalandsregionen, Vaestra Goetalandsregionen

Enrolment target

Start

02 Apr 2024

Congenital myopathy

What is this study about?

This study, called COMPIS, is looking into how a medicine called salbutamol might help people living with congenital myopathy. Congenital myopathy is a condition that affects the muscles, making them weaker right from birth. Salbutamol is a well-known medicine, often used as an inhaler for asthma, but this study is exploring if it can also help improve muscle strength and daily activities in people with congenital myopathy.

The main goal is to find out if taking salbutamol for six months can lead to better muscle function, as measured by a specific test called MFM32. They will compare people who take the medicine with those who don't, to clearly see any differences. The researchers will also look at other things, such as how far people can walk in six minutes, how tired they feel during physical activities, how quickly they can do certain movements like walking 10 metres or going up and down steps, and even their hand strength.

Ultimately, the study hopes to understand if salbutamol can improve people's overall quality of life by making everyday tasks easier and reducing tiredness. This is a "Phase IV" study, which means the medicine is already approved for other conditions, but doctors are now studying it further to see if it can help people with congenital myopathy.

Key takeaways

The study is investigating a common asthma medicine (salbutamol) for muscle weakness.
It aims to see if salbutamol can improve muscle function and daily activities in congenital myopathy.
The main treatment period for the study is six months.
You need to be at least 18 years old to take part.
The study measures changes in muscle function, walking, tiredness, and quality of life.

Who may be eligible?

To be part of this study, you need to be at least 18 years old. There's no upper age limit, meaning older adults can also take part. The study is open to both men and women.

Since this information is a summary, it's really important to talk to your doctor or the study team. They will have a full list of things they need to check to make sure the study is a good fit for you and that taking part would be safe.

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 congenital myopathy?
Are you able to attend regular clinic visits?
Are you prepared to take the study medicine or no treatment for six months?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you'll be given either salbutamol (Ventolin) as a liquid or tablets, or no treatment, for six months. Because it's a 'crossover' study, it's possible you might switch treatments part-way through. Throughout the study, you'll have several visits to the clinic.

During these visits, you'll have different tests to check your muscle strength and how easily you can do certain activities. These might include walking tests, timed activities like getting up and down, checking hand strength, and even breathing tests. They will also ask about how you feel day-to-day to understand your quality of life. The total length of your participation, including follow-up visits, will be explained by the study team.

Potential risks and benefits

Taking part in any medical study has potential benefits and potential risks. You might find that salbutamol helps improve your muscle strength and makes daily activities easier, which could improve your quality of life. However, it's also possible you might not experience any benefit, or you could experience side effects from the medicine, as all medicines can have them. You have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (1)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Sweden

Common questions

What is congenital myopathy?

Congenital myopathy is a group of conditions that affect muscle strength from birth or early childhood, making muscles weaker than usual.

What is salbutamol (Ventolin)?

Salbutamol is a medicine often used to help people with asthma breathe more easily. It's available as a liquid or tablets in this study.

What does 'crossover study' mean?

It means that during the study, participants might switch between getting the medicine and not getting the medicine, or vice-versa, so researchers can compare the effects within the same person.

Will I know if I'm getting the medicine?

This is an 'open-label' study, which means you and the study team will know if you are taking salbutamol or not.

How long will the treatment last?

The treatment period in the study is for six months.

How to find out more

Official trial record

Always speak to your GP or specialist before deciding to take part in a study.