Study on the Long-Term Safety and Effectiveness of Losmapimod for Patients with Facioscapulohumeral Muscular Dystrophy (FSHD)

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What is this study about?

This clinical trial is focused on studying a condition called Facioscapulohumeral Muscular Dystrophy (FSHD), which is a genetic disorder that causes muscle weakness, particularly in the face, shoulders, and upper arms. The study is testing a treatment called Losmapimod, which is taken as a film-coated tablet. The purpose of the study is to evaluate the safety and tolerability of long-term use of Losmapimod in individuals with FSHD.

Participants in the study will be randomly assigned to receive either Losmapimod or a placebo. The study is designed to be double-blind, meaning neither the participants nor the researchers will know who is receiving the actual medication or the placebo. The study will last for 48 weeks, with an option for an open-label extension, where all participants may receive Losmapimod. Throughout the study, participants will have regular check-ups to monitor their health and the effects of the treatment.

The main focus of the study is to ensure that Losmapimod is safe and well-tolerated over a long period. Researchers will also measure the levels of Losmapimod in the blood at regular intervals to understand how the body processes the medication. This study aims to provide valuable information about the potential benefits and safety of Losmapimod for people living with FSHD.

1 joining the study

Upon joining the study, the participant will be required to provide written informed consent. This involves understanding the study details and agreeing to participate.

Eligibility is confirmed through a genetic test for Facioscapulohumeral Muscular Dystrophy 1 (FSHD1), which must be completed before any further steps.

2 screening and baseline assessments

A screening period of up to four weeks is conducted to confirm eligibility. This includes a clinical severity assessment and an MRI to identify a suitable muscle for biopsy.

Participants must have a clinical severity score between 2 and 4 and must not require a wheelchair or walker.

3 randomization and initial treatment phase

Participants are randomly assigned to receive either the study drug, losmapimod, or a placebo. This is a double-blind process, meaning neither the participant nor the study team knows which treatment is being administered.

The medication is provided in the form of a film-coated tablet for oral use.

4 treatment administration

The treatment phase lasts for 48 weeks. Participants take the assigned medication as directed, with regular monitoring of health and any side effects.

The primary focus is on the safety and tolerability of long-term use of losmapimod.

5 open-label extension (OLE)

After the initial 48-week period, participants may enter an open-label extension phase where all receive losmapimod.

This phase continues to monitor safety and plasma concentrations of the drug every 12 weeks.

6 end of study

The study is estimated to conclude by October 31, 2025. Participants will undergo final assessments to evaluate the long-term effects of the treatment.

The primary endpoint is the safety and tolerability of losmapimod, while secondary endpoints include measuring drug levels in the blood.

Who Can Join the Study?

Must be able to understand and sign a written consent form.
Must be between 18 and 65 years old.
Must have a confirmed diagnosis of Facioscapulohumeral Muscular Dystrophy 1 (FSHD1), which is a genetic condition affecting muscles. This confirmation can be from previous tests if verified by a certified lab.
Must have a clinical severity score between 2 and 4, which measures the impact of the condition. Cannot use a wheelchair or walker for any activity.
Must have a muscle suitable for a biopsy, as determined by a specialist using an MRI scan.
Must be willing and able to attend scheduled visits, follow the treatment plan, and adhere to study guidelines, including laboratory tests and contraceptive use.
Must agree to use an approved method of birth control during the study and for 90 days after the last dose of the study drug. This applies to both male and female participants.

Who Cannot Join the Study?

Individuals who do not have Facioscapulohumeral Muscular Dystrophy 1 (FSHD1) cannot participate. This is a specific type of muscle disorder.
Participants must be within a certain age range, which is not specified here, but if you are outside this range, you cannot join.
Both males and females can participate, but if you do not identify as either, you may not be eligible.
If you are considered part of a vulnerable population, which means you might need extra protection or care, you may not be able to participate.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Hospital Universitario Y Politecnico La Fe	Valencia	Spain

Other Sites

Site Name	City	Country	Status
Centre Hospitalier Universitaire De Nice	Nice	France
Hfyvbxxk Dh Lk Seclc Cccd I Sdcx Plt	Barcelona	Spain

Trial status

Country	Status	Recruitment Start
France	Not recruiting	19.12.2019
Spain	Not recruiting	19.12.2019

Trial locations

Investigated drugs:

Losmapimod

Losmapimod is a medication being studied for its potential to treat facioscapulohumeral muscular dystrophy (FSHD). This condition is a genetic muscle disorder that causes progressive muscle weakness. The trial aims to assess how safe and well-tolerated losmapimod is when taken over a long period by individuals with FSHD. The study is designed to determine if losmapimod can help improve muscle function and slow down the progression of the disease.

Investigated diseases:

Facioscapulohumeral muscular dystrophy

Facioscapulohumeral Muscular Dystrophy 1 (FSHD1) – This is a genetic muscle disorder characterized by progressive muscle weakness and wasting. It primarily affects the muscles of the face, shoulders, and upper arms. The disease often begins with weakness in the facial muscles, leading to difficulties in smiling, whistling, or closing the eyes. As it progresses, it can cause weakness in the shoulder and upper arm muscles, making it difficult to lift objects or raise the arms. Over time, the weakness may extend to other muscles, including those in the legs and abdomen. The rate of progression and severity can vary widely among individuals.

Trial ID:

2024-512732-30-00

Protocol code:

FIS-002-2019

Trial Phase:

Therapeutic exploratory (Phase II)

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Study on the Long-Term Safety and Effectiveness of Losmapimod for Patients with Facioscapulohumeral Muscular Dystrophy (FSHD)

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?