Ongoing, recruitingTherapeutic exploratory (Phase II)Interventional

A Phase 2, Randomized, Double-Blind, Placebo-Controlled, 48-Week, Parallel-Group Study of the Efficacy and Safety of Losmapimod in Treating Subjects with Facioscapulohumeral Muscular Dystrophy (FSHD) with Open-Label Extension (OLE)

This study is looking into a new medicine called Losmapimod for people who have a condition called Facioscapulohumeral Muscular Dystrophy (FSHD). This part of the study is called an 'Open-Label Extension' and follows on from an earlier trial. Researchers want to understand how safe Losmapimod is when taken for a long time. They will carefully check for any side effects using various medical tests like blood tests, heart checks (ECGs), and physical examinations. They will also look at how the body handles the medicine over time. The main goal is to make sure the treatment is safe for people with FSHD.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic exploratory (Phase II)

Sponsor

Fulcrum Therapeutics Inc.

Enrolment target

Start

24 Apr 2024

Facioscapulohumeral Muscular Dystrophy 1 (FSHD1)

What is this study about?

This research study is focused on a health condition called Facioscapulohumeral Muscular Dystrophy, often shortened to FSHD. It's a type of muscular dystrophy that causes muscles to weaken, mainly in the face, shoulders, and upper arms, but it can affect other parts of the body too. Scientists are testing a new medicine called Losmapimod to see if it can help people with FSHD. This particular study is a follow-up, an 'Open-Label Extension' (OLE), meaning participants who were in an earlier trial can continue taking the medicine.

The main purpose of this follow-up study is to understand the long-term safety of Losmapimod. When a new medicine is developed, it goes through several stages of testing. 'Phase 2' means that initial tests have shown promise, and now researchers need to gather more information, especially about safety and how the body copes with the medicine over longer periods. The study isn't primarily looking at whether the medicine cures FSHD, but rather if it's safe enough for people to use consistently.

Researchers will be carefully monitoring participants using various medical checks like blood tests, heart rhythm recordings (ECGs), and general health check-ups. They will also keep track of any unexpected reactions or side effects that people experience. Another important aspect is understanding how the medicine levels in the blood change over time with long-term use. All this information helps doctors decide if Losmapimod could be a safe and valuable treatment option for people living with FSHD in the future.

Key takeaways

This study is testing the long-term safety of Losmapimod for people with FSHD.
It's a follow-up study for those who were already in an earlier trial.
Participants will have regular health checks, including blood tests and ECGs.
The study aims to understand how the body handles the medicine over time.
Participation involves ongoing monitoring for about 48 weeks.
You can stop participating at any time.

Who may be eligible?

This study is looking for adult participants, meaning you must be 18 years old or older. There is no upper age limit, so older adults can also take part if they meet other health requirements.

The study is open to both men and women. Your gender does not affect whether you can join this research.

Crucially, this particular study is an 'Open-Label Extension'. This means that to be eligible, you would usually have already participated in the earlier part of this Losmapimod study (the 'Phase 2' trial). The details you were given when you joined the first part of the study would explain if you could continue into this extension phase.

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?
Have you previously participated in the earlier Losmapimod study for FSHD?
Are you able to attend regular clinic appointments for about 48 weeks?
Are you willing to undergo various medical tests, including blood draws and ECGs?

Answer every question to see your result.

What does participation involve?

If you are eligible and choose to take part in this study, you would primarily be continuing treatment with Losmapimod, as this is an extension phase of a previous study. You would attend regular appointments at a clinic, likely every 12 weeks, for about a year (48 weeks).

During these visits, healthcare professionals would carry out several assessments to monitor your health and the medicine's effects. These would include physical examinations, taking your vital signs (such as blood pressure and heart rate), blood tests, and possibly urine tests. You would also have electrocardiograms (ECGs) to check your heart health.

Throughout the study, you would continue to take the study medicine, Losmapimod, as directed. You would need to report any new or worsening health problems or side effects to the study team. Regular follow-up ensures your safety is prioritised while gathering important data about the long-term effects of the medication. The total duration of this specific extension study is 48 weeks.

Potential risks and benefits

Taking part in a study like this could offer potential benefits, such as continued access to a new medicine that might help manage your FSHD symptoms, even if it's primarily a safety study. You would also receive close medical attention and monitoring from a dedicated research team. However, there are potential risks, as with any medicine. Losmapimod might cause side effects, some of which could be mild, while others might be more serious or unexpected, and you would be asked to report these. There is no guarantee that the medicine will improve your condition. Crucially, your participation is completely voluntary; you have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (2)

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

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Unverified
Spain
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Unverified
France

Common questions

What is FSHD?

FSHD stands for Facioscapulohumeral Muscular Dystrophy. It's a condition that causes muscles to weaken, mostly in the face, shoulders, and upper arms.

What is Losmapimod?

Losmapimod is an investigational medicine being tested in this study to see if it can help people with FSHD.

What does 'Open-Label Extension' mean?

It means this study is a follow-up for people who were already in an earlier part of the study, and everyone in this phase knows they are receiving the study medicine.

How long will I be in this study?

This particular study phase lasts for 48 weeks, which is almost one year.

Will Losmapimod cure my FSHD?

This study is mainly looking at the long-term safety of Losmapimod, not whether it can cure FSHD. It's too early to say if it will cure the condition.