Study on the Effectiveness and Safety of Satralizumab for Patients with Type 1 Facioscapulohumeral Muscular Dystrophy

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What is this study about?

This clinical trial is focused on studying a condition called Type 1 facioscapulohumeral muscular dystrophy (FSHD1). FSHD1 is a genetic disorder that causes muscle weakness, primarily affecting the face, shoulders, and upper arms. The study will explore the effects of a treatment called satralizumab, which is a type of medication known as a monoclonal antibody. Monoclonal antibodies are proteins designed to target specific substances in the body. In this study, satralizumab will be compared to a placebo, which looks like the treatment but does not contain the active ingredient.

The purpose of the study is to evaluate the effectiveness and safety of satralizumab in patients with FSHD1. Participants will receive either satralizumab or a placebo through an injection under the skin. The study will last for a total of 96 weeks, with the first 48 weeks being a double-blind period, meaning neither the participants nor the researchers will know who is receiving the actual treatment or the placebo. After this period, there will be an open-label phase where all participants may receive satralizumab. Throughout the study, various assessments will be conducted to monitor changes in muscle strength, physical abilities, and overall health.

Participants will undergo regular check-ups, including MRI scans to examine muscle condition, and will be asked to maintain their usual level of physical activity. The study aims to provide valuable insights into how satralizumab can help manage symptoms of FSHD1 and improve the quality of life for those affected by this condition. The safety of the treatment will also be closely monitored by tracking any side effects or adverse reactions experienced by participants.

1 initial assessment

Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes a review of medical history and a physical examination.

A whole-body MRI is performed to assess muscle condition. This helps in determining the presence of muscle changes required for participation.

2 randomization

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

3 treatment administration

The treatment involves a subcutaneous injection of Enspryng 120 mg solution or placebo. The injections are administered at regular intervals as specified by the study protocol.

4 double blind period

The double-blind period lasts for 48 weeks. During this time, the effects of the treatment are monitored through various assessments.

These assessments include changes in muscle condition via MRI, clinical severity scores, and muscle strength measurements.

5 open-label period

Following the double-blind period, an open-label period extends the study to 96 weeks. During this phase, all participants receive satralizumab.

The focus remains on monitoring efficacy and safety, with continued assessments similar to those in the double-blind period.

6 final evaluation

At the end of the study, a final evaluation is conducted. This includes a comprehensive review of all collected data to assess the overall impact of the treatment.

Participants undergo a final MRI and other assessments to measure any changes in muscle condition and overall health.

Who Can Join the Study?

Must be able to understand and sign a written consent form.
Must be willing and able to attend scheduled visits, follow the treatment plan, and adhere to study guidelines and procedures.
For women who can have children: Must use effective birth control during the treatment period and until stopping the treatment.
Must be between 18 and 65 years old.
Must be part of a European social security system (only for the Nice center).
Must have a confirmed genetic diagnosis of typical FSHD1. This involves checking a specific part of the DNA called D4Z4 repeats on chromosome 4. If a family member has a confirmed diagnosis, you may be eligible for screening.
Must have a clinical severity score between 2 and 4 on the RICCI score, which measures the severity of the condition.
Must weigh 100 kg or less.
On an initial whole-body MRI, must have at least one muscle in the lower limbs showing specific signals (STIR or T2-Dixon positive signal). There should also be evidence of muscle fat replacement, with certain conditions on the amount of muscle fat.
Must be able to walk without support.
Must be willing to keep the same level of exercise (how often and how intense) during the study.

Who Cannot Join the Study?

Patients with any other type of muscular dystrophy besides Type 1 facioscapulohumeral muscular dystrophy cannot participate. This is a specific muscle disorder that affects the face, shoulder blades, and upper arms.
Patients who are not within the specified age range for the study cannot participate. The study is open to certain age groups only.
Patients who are part of a vulnerable population, which means they might need special protection or care, are not eligible for this study.

Where you can join this trial?

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Site Name	City	Country	Status
Centre Hospitalier Universitaire De Nice	Nice	France

Trial status

Country	Status	Recruitment Start
France	Not recruiting	01.01.2024

Trial locations

Investigated drugs:

Satralizumab

Satralizumab is a medication being studied for its potential to help patients with FSHD1, a type of muscular dystrophy. The trial aims to see how effective and safe satralizumab is for these patients. It works by targeting specific pathways in the immune system that may be involved in the progression of the disease.

Investigated diseases:

Facioscapulohumeral muscular dystrophy

Type 1 Facioscapulohumeral Muscular Dystrophy – 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 the rate of progression can differ widely. The condition is caused by genetic changes that affect the DUX4 gene, leading to muscle cell damage.

Trial ID:

2023-504507-81-00

Protocol code:

22-PP-24

Trial Phase:

Therapeutic exploratory (Phase II)

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Study on the Effectiveness and Safety of Satralizumab for Patients with Type 1 Facioscapulohumeral Muscular Dystrophy

What is this study about?

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