Study on the Safety and Effects of Losmapimod for Patients with Facioscapulohumeral Muscular Dystrophy 1 (FSHD1)

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What is this study about?

This clinical trial is focused on studying Facioscapulohumeral Muscular Dystrophy 1 (FSHD1), 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 FSHD1.

Participants in the study will take Losmapimod orally and will be monitored over a period of time to assess how well they tolerate the medication and to observe any changes in their condition. The study will also look at specific markers in the body that might indicate how the disease is progressing or responding to the treatment. Some participants may receive a placebo, which is a tablet that looks like the medication but does not contain the active ingredient.

The study will involve regular visits to the clinic for check-ups, which may include tests like blood work, heart monitoring (ECG), and other assessments to ensure the safety of the participants. The trial aims to gather important information that could help in understanding the effects of Losmapimod on FSHD1 and potentially improve treatment options for those affected by this condition.

1 joining the study

Participation begins after signing an informed consent form. This confirms understanding and agreement to the study’s terms.

Eligibility is confirmed through a genetic test for Facioscapulohumeral Muscular Dystrophy 1 (FSHD1). This involves assessing the size of the D4Z4 array on chromosome 4.

2 screening phase

A screening phase ensures all criteria are met. This includes a genetic confirmation, which may use previous test results if verified.

During this phase, a muscle biopsy and MRI are scheduled to confirm eligibility.

3 treatment phase

The treatment involves taking Losmapimod tablets orally. The dosage and frequency are determined by the study protocol.

Participants must adhere to the treatment plan, including scheduled visits and laboratory tests.

4 monitoring and assessments

Regular assessments are conducted to monitor safety and tolerability. This includes checking for any adverse effects, conducting laboratory tests, and recording vital signs.

Participants undergo whole-body MRI and muscle biopsies as part of the monitoring process.

5 completion of main study

The main study concludes after the Week 60 visit. Participants must complete this phase to be eligible for the extension study.

Completion involves final assessments and documentation of outcomes.

6 extension study

Participants who complete the main study may enter an extension phase. This continues the evaluation of Losmapimod over a longer period.

The extension study follows similar procedures for monitoring and assessments as the main study.

Who Can Join the Study?

Patients must be between 18 and 65 years old.
Patients must have completed the main study up to the Week 60 visit to join the extension.
Patients need to sign and date a form that shows they agree to participate in the study.
Patients must have a confirmed diagnosis of Facioscapulohumeral Muscular Dystrophy 1 (FSHD1), which is a genetic condition. This confirmation can come from previous tests if properly documented. If a patient has a close family member with a confirmed diagnosis, they may also be eligible for the study.
Patients should be willing and able to attend scheduled visits, follow the treatment plan, and comply with study rules, including laboratory tests and other procedures.
Both men and women can participate. Women who can have children must use effective birth control during the study and for 90 days after the last dose. Men must also agree to use birth control during the same period.
Patients must have a Clinical Severity Score between 2 and 4 on a specific scale used to measure the severity of their condition. Patients who use a wheelchair or walker cannot join the study.
Patients must agree to complete two visits for muscle biopsies and all visits for whole-body MRI scans.
Patients should be able to complete certain physical tests and questionnaires at the screening visit.
Patients must have a muscle that is suitable for biopsy, as determined by a specialist.

Who Cannot Join the Study?

Patients who have a different type of muscular dystrophy other than Facioscapulohumeral Muscular Dystrophy 1 (FSHD1) cannot participate.
Individuals who are not within the specified age range for the study are excluded. The age range is typically defined in the study details.
Participants who are not able to follow the study procedures or take the study medication as required will be excluded.
People with certain medical conditions that might interfere with the study or pose a risk to their health if they participate will not be allowed to join.
Individuals who are currently participating in another clinical trial or have recently participated in one may be excluded.
Pregnant or breastfeeding women are usually not allowed to participate in clinical trials to avoid any potential risks to the baby.
Patients who have a history of allergic reactions to the study medication or similar drugs will be excluded.
Individuals with severe mental health issues that might affect their ability to participate in the study will not be included.
People who have a history of substance abuse, which could interfere with the study results, may be excluded.

Where you can join this trial?

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Other Sites

Site Name	City	Country	Status
Stichting Radboud University Medical Center	Nijmegen	The Netherlands

Trial status

Country	Status	Recruitment Start
The Netherlands	Not recruiting	23.08.2019

Trial locations

Investigated drugs:

Losmapimod

Losmapimod is a medication being studied for its potential to help people with Facioscapulohumeral Muscular Dystrophy 1 (FSHD1). This study aims to see if taking losmapimod over a long period is safe and if it is well-tolerated by patients. Researchers are also looking at how it might affect certain biomarkers and clinical outcomes related to 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, leading to difficulties in facial expressions, lifting the arms, and other movements. The progression of muscle weakness can vary significantly among individuals, with some experiencing mild symptoms and others facing more severe challenges. Over time, the weakness may extend to other muscle groups, including those in the lower body. The onset of symptoms typically occurs in adolescence or early adulthood, but it can also appear later in life. The rate of progression and severity of symptoms can differ widely, even among members of the same family.

Trial ID:

2024-512736-29-00

Protocol code:

FIS-001-2019

NCT ID:

NCT04004000

Trial Phase:

Therapeutic exploratory (Phase II)

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Study on the Safety and Effects of Losmapimod for Patients with Facioscapulohumeral Muscular Dystrophy 1 (FSHD1)

What is this study about?

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