Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

A Phase 3 Global, Randomized, Double-Blind, Placebo-Controlled, 48-Week, Parallel-Group Study of the Efficacy and Safety of Losmapimod in Treating Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) (REACH)

This important study is looking into a new tablet called losmapimod for adults living with Facioscapulohumeral Muscular Dystrophy (FSHD). FSHD is a condition that causes muscle weakness, often in the face, shoulders, and upper arms. The main goal is to find out if losmapimod can help improve how well people can use their arms, specifically looking at wrist movement, after about a year of treatment. Researchers also want to understand if the medication is safe and well-tolerated when taken for a longer period. Participants will be given either the active drug or a dummy tablet (placebo) to ensure a fair test, and neither the participants nor their doctors will know who is getting which. The study will last for 48 weeks, making sure to track any changes in muscle strength and overall well-being.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Fulcrum Therapeutics Inc.

Enrolment target

144

Start

06 Aug 2024

Facioscapulohumeral Muscular Dystrophy (FSHD)

What is this study about?

This study, called REACH, is a significant worldwide research project investigating a new medication called losmapimod. It's designed for people who have Facioscapulohumeral Muscular Dystrophy, or FSHD. FSHD is a genetic condition that causes muscles to weaken and waste away over time, often starting in the face, shoulders, and upper arms.

The main purpose of this study is to see if losmapimod, taken as a tablet, can help improve how people use their arms, particularly focusing on wrist movements, after about a year of treatment. The researchers will also be closely watching to make sure the medication is safe to use and doesn't cause too many side effects over a longer period. To get reliable results, some participants will receive the active drug, while others will receive a 'dummy' tablet that looks identical to the real one. This approach, called 'double-blind,' means that neither you nor your study doctor will know whether you're getting the active treatment or the dummy tablet until after the study is complete. This helps to make sure the results are as unbiased as possible.

This type of study, known as 'Phase 3,' is usually the last step before a new medicine can be considered for approval. It means that earlier studies have shown some promise, and now the researchers need to confirm these findings in a larger group of people. The information gathered from this study will be crucial in understanding whether losmapimod could become a new treatment option for people with FSHD.

Key takeaways

This study evaluates a potential new tablet treatment (losmapimod) for FSHD.
It aims to see if the drug improves arm function and is safe over nearly a year.
Participants will receive either the active drug or a dummy tablet (placebo).
It's a large, global study, so the results will be very important.
Participation involves regular clinic visits for checks, tests, and assessments.
All adults with FSHD (age 18+) are welcome to check if they are eligible.

Who may be eligible?

The study is looking for adults who have been diagnosed with Facioscapulohumeral Muscular Dystrophy (FSHD). You must be at least 18 years old to take part; there is no upper age limit, meaning older adults are also welcome to participate.

Participants can be of any sex – both men and women are invited to join the study if they meet the other requirements. The most important thing is that you have a confirmed diagnosis of FSHD.

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.

I have been diagnosed with Facioscapulohumeral Muscular Dystrophy (FSHD).
I am 18 years old or older.
I am able to attend regular clinic appointments.
I am living in the UK and can travel to a study site.
I am willing to take a tablet twice a day for nearly a year.
I am comfortable with my study team and I not knowing if I am taking the active drug or a dummy tablet.

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would primarily be taking a tablet, either the study drug (losmapimod) or a placebo (a dummy tablet), twice a day for 48 weeks (around 11 months). You wouldn't know which one you are taking. You would have regular visits to the study clinic where the medical team would: perform physical examinations; check your vital signs (like blood pressure and heart rate); take blood samples for lab tests; measure your arm and shoulder strength with special tools; and ask you to fill out questionnaires about your well-being and how you feel your condition is changing. There will also be assessments of your arm function, such as how you move your wrist with a small weight. Your heart activity will be monitored with ECGs (heart tracings). After the 48 weeks, there might be a follow-up period to check on your health.

Potential risks and benefits

Taking part in a clinical trial could potentially offer you access to a new treatment before it's widely available. While losmapimod is being carefully studied, it's possible it might improve your arm function or slow down the progression of your FSHD, but there's no guarantee. There may also be risks involved, such as experiencing side effects from the medication. These could be mild, like an upset stomach, or more serious. The study team will closely monitor you for any side effects and explain all known potential risks before you decide to join. Remember, you can choose to leave the study at any time, for any reason, without it affecting your usual medical care.

Locations (6)

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

—
Unverified
Italy
—
Unverified
Spain
—
Unverified
France
—
Unverified
Germany
—
Unverified
Netherlands
—
Unverified
Denmark

Common questions

What is FSHD?

FSHD stands for Facioscapulohumeral Muscular Dystrophy. It's a genetic condition that causes muscles to weaken over time, often affecting the face, shoulders, and upper arms.

What is losmapimod?

Losmapimod is a new drug being tested in this study. It's a tablet you take by mouth, and researchers want to see if it can help people with FSHD.

What does 'placebo' mean?

A placebo is a 'dummy' tablet that looks exactly like the actual study drug but contains no active medicine. It helps researchers fairly compare how well the real drug works.

How long will the study last?

The main treatment part of the study will last for 48 weeks, which is almost a year. There might be some follow-up appointments after that.

Will I know if I'm getting the real drug or the placebo?

No, neither you nor your study doctor will know if you're taking losmapimod or the placebo until after the study is finished. This is to ensure the results are unbiased.