RecruitingRare disease

Study of Gefurulimab for Children and Teens with Generalized Myasthenia Gravis

Fast replyInvestigationalNo placebo

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a condition called Generalized Myasthenia Gravis (gMG), which is a chronic autoimmune disease that causes muscle weakness. The study is specifically looking at children and teenagers aged 6 to under 18 who have a type of gMG that involves antibodies against a part of the body called the acetylcholine receptor (AChR). The treatment being tested is a medication called Gefurulimab, which is given as a solution for injection under the skin. This medication is also known by its code name, ALXN1720.

The purpose of the study is to understand how Gefurulimab works in the body, how safe it is, and how effective it is in treating children with this specific type of gMG. Participants in the study will receive the medication and be monitored over a period of time to see how their bodies respond to the treatment. The study will also look at how the medication affects the symptoms of gMG and whether it helps improve muscle strength and daily activities.

Throughout the study, participants will have regular check-ups to monitor their health and the effects of the medication. These check-ups will include assessments of vital signs, physical examinations, and laboratory tests. The study aims to gather important information that could help improve treatment options for children with Generalized Myasthenia Gravis in the future.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initial visit
Upon joining the study, you will attend an initial visit at the study site. During this visit, your eligibility for the study will be confirmed. This includes checking your age, weight, and medical history related to generalized myasthenia gravis (gMG).
You will undergo a series of tests to confirm the presence of acetylcholine receptor antibodies (AChR+), which are necessary for participation in the study.
Step 2
Vaccination check
Before starting the study treatment, it is important to ensure that you have received vaccinations for certain infections. These include vaccinations for S pneumoniae, Hib, and meningococcal infections from serogroups A, C, W, Y, and B (if available).
If you have not been vaccinated within the last three years, you may need to receive these vaccinations before the study begins. If the vaccination occurs less than two weeks before starting the study, you will receive antibiotics for at least two weeks to reduce the risk of infection.
Step 3
Treatment administration
The study involves receiving a medication called gefurulimab. This medication is given as a subcutaneous injection, which means it is injected under the skin.
The dosage and frequency of the injections will be determined based on your weight and other factors. The treatment will continue for a specified duration, with regular monitoring to assess its effects.
Step 4
Regular monitoring
Throughout the study, you will have regular visits to the study site for monitoring. These visits will include assessments of your health, including physical examinations, blood tests, and other necessary evaluations.
The study team will monitor the levels of gefurulimab in your blood and assess its effects on your condition. This will help determine the safety and effectiveness of the treatment.
Step 5
Completion of study
At the end of the study period, you will have a final visit to assess your overall health and the effects of the treatment.
The study team will review all the data collected during the study to evaluate the outcomes and determine the next steps.

Who can join the trial?

13 criteria

Participant must be between 6 and 17 years old at the time of signing the informed consent or assent.
All participants must have received vaccinations for S pneumoniae (a type of bacteria that can cause infections) and Hib (Haemophilus influenzae type b, a bacteria that can cause severe infections), unless they have already been vaccinated according to current national or local guidelines.
To reduce the risk of meningococcal infection (a serious bacterial infection), all participants must be vaccinated against meningococcal infection from serogroups A, C, W, Y (and B where available) within 3 years before the study starts. If the vaccination occurs less than 2 weeks before the study starts, participants will receive preventive antibiotics for at least 2 weeks after the vaccination.
A legal guardian or primary caregiver must be available to help with study visits, follow the treatment plan, and provide information about the participant during the study.
A legal guardian or primary caregiver must be able to accurately maintain the child's health records at home.
Participants must have a documented diagnosis of generalized Myasthenia Gravis (gMG) for at least 3 months before the study, confirmed by specific tests or previous treatment response.
Participants must have a positive test for AChR autoantibodies (antibodies against a specific receptor in the body) confirmed by a central laboratory.
Participants must be classified as MGFA Class II to IV at the start of the study. This is a way to describe the severity of the condition.
Participants aged 12 to 17 must have a QMG total score of 12 or higher at the start of the study. This score measures the severity of muscle weakness. Participants aged 6 to 11 do not have a minimum score requirement but must have documented weakness in at least one limb.
Participants taking certain medications must be on a stable dose for a specified time before the study starts. If they recently stopped taking these medications, a certain amount of time must pass before they can join the study.
Participants must weigh at least 20 kg and are expected to maintain this weight during the study.
Participants who can have children must follow specific contraception guidelines during the study.
The study investigator will obtain written informed consent from each participant's legal guardian and the participant's assent, if applicable, before any study activities begin. All legal guardians and participants should be fully informed about the study in understandable language.

Who cannot join the trial?

4 criteria

Patients who do not have **Generalized Myasthenia Gravis** with autoantibodies against **acetylcholine receptor (AChR)** cannot participate. This means the study is only for those diagnosed with this specific condition.
Patients who are not within the specified age range for the study cannot participate. The study is for a specific age group.
Patients who are not either **male** or **female** cannot participate. The study includes both genders.
Patients who are not considered part of a **vulnerable population** cannot participate. This refers to groups that may need special protection or consideration in research.

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Responsive
Uniwersyteckie Centrum Kliniczne Warszawskiego Uniwersytetu Medycznego
Warsaw, Poland

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Investigated drugs

Gefurulimab is a medication being studied for its effects on children and teenagers who have a condition called generalized myasthenia gravis (gMG). This condition affects the muscles, making them weak and tired because the body's immune system mistakenly attacks them. Gefurulimab works by targeting specific antibodies in the body that are involved in this process. The goal of using gefurulimab in this study is to understand how it moves through the body, how it affects the body, and to see if it is safe and effective for young patients with this condition.

Gefurulimab

What is already known about the treatment

Gefurulimab – Gefurulimab is administered as an injection and is currently being studied in clinical trials for its effects on children aged 6 to under 18 years with a condition called Generalized Myasthenia Gravis (gMG), which involves antibodies against the acetylcholine receptor. This medication is still under investigation, so it is not yet widely used in medical practice. Its main purpose is to help manage symptoms of gMG by targeting specific antibodies that interfere with nerve signals to muscles. Gefurulimab belongs to a class of drugs that work by modulating the immune system to reduce the harmful effects of these antibodies.

Investigated diseases

Generalized Myasthenia Gravis with autoantibodies against acetylcholine receptor (AChR) – Generalized Myasthenia Gravis is a chronic autoimmune disorder where the body's immune system mistakenly attacks the communication between nerves and muscles. This condition is characterized by the presence of autoantibodies against the acetylcholine receptor, which is crucial for muscle contraction. As the disease progresses, individuals experience muscle weakness that worsens with activity and improves with rest. The weakness often affects muscles that control eye and eyelid movement, facial expression, chewing, talking, and swallowing. Over time, the weakness can spread to other muscle groups, including those involved in breathing and limb movement. The severity and progression of symptoms can vary widely among individuals.