RecruitingRare disease

Study on Nipocalimab for Children Aged 2-17 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, which is a disease that causes muscle weakness. The study is specifically for children and teenagers aged 2 to less than 18 years who have this condition. The treatment being tested is a medication called nipocalimab, which is given as a solution through an infusion into a vein. The purpose of the study is to evaluate how well nipocalimab works, how safe it is, and how it behaves in the body of young patients with generalized myasthenia gravis who have not responded well to their current treatment.

Participants in the study will receive nipocalimab and will be monitored over a period of time to see how their bodies respond to the treatment. The study will look at various aspects, such as the effect of nipocalimab on certain proteins in the blood and any side effects that may occur. The study aims to gather information on how the medication is processed in the body and its overall impact on the disease.

This trial is open-label, meaning that both the participants and the researchers know what treatment is being given. The study does not involve a placebo, and all participants will receive the active medication. The information collected from this study will help in understanding the potential benefits and risks of using nipocalimab in treating generalized myasthenia gravis in children and teenagers.

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
Joining the study
Upon joining the study, you will be informed about the purpose and procedures involved. This study is designed to evaluate the effects of a medication called nipocalimab in children and adolescents aged 2 to less than 18 years with a condition known as generalized myasthenia gravis (gMG).
Step 2
Initial assessment
An initial assessment will be conducted to confirm your eligibility. This includes verifying your age, diagnosis of gMG, and response to current treatments. A blood test will be performed to check for specific antibodies related to your condition.
Step 3
Treatment administration
You will receive the study medication, nipocalimab, through an intravenous infusion. This means the medication will be given directly into your vein using a needle. The dosage and frequency of administration will be determined by the study team based on your specific needs.
Step 4
Monitoring and follow-up
Throughout the study, regular visits will be scheduled to monitor your health and the effects of the medication. These visits will include physical examinations, blood tests, and assessments of your muscle strength and overall well-being.
Step 5
Completion of the study
At the end of the study period, a final assessment will be conducted to evaluate the overall impact of the treatment. This will include a review of your health status and any changes in your condition.

Who can join the trial?

7 criteria

Must be between 2 and 17 years old.
Must have a diagnosis of myasthenia gravis (MG) with generalized muscle weakness. This means the muscles are weaker than normal.
Must have a positive blood test for specific antibodies: acetylcholine receptor (anti-AChR) antibodies or muscle-specific tyrosine kinase (anti-MuSK) antibodies. These are proteins in the blood that are checked to confirm the condition.
Must not be responding well enough to current treatment for MG, according to the doctor.
If the participant has had a splenectomy (surgery to remove the spleen), it must have been done at least 3 months before joining the study. The participant must also be vaccinated according to local health guidelines.
Should be up to date on all recommended vaccinations, like those for diphtheria and tetanus, before joining the study. It is strongly recommended to have completed a COVID-19 vaccination at least 2 weeks before study visits.
If female and able to have children, must have a negative pregnancy test before starting the study.

Who cannot join the trial?

10 criteria

Patients who are not between the ages of 2 and 17 years old.
Patients who do not have a diagnosis of Myasthenia Gravis (MG). This is a condition that causes weakness in the muscles.
Patients who have a good response to their current treatment and do not need additional help.
Patients who are not on a stable treatment plan for their condition.
Patients who are not able to follow the study procedures or instructions.
Patients who have other health conditions that might interfere with the study.
Patients who are pregnant or breastfeeding.
Patients who have participated in another clinical trial recently.
Patients who have allergies or reactions to the study medication or similar drugs.
Patients who have a history of drug or alcohol abuse.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Academisch Ziekenhuis Leiden
Responsive
Leiden, The Netherlands
Uniwersyteckie Centrum Kliniczne
Verified
Gdansk, Poland

Trial locations

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Investigated drugs

Nipocalimab is a medication being studied for its potential to help children and teenagers with a condition called generalized myasthenia gravis, which affects the muscles and makes them weak. This medication works by targeting specific parts of the immune system that may be causing the muscle weakness. The goal of using nipocalimab in this study is to see if it can improve muscle strength and overall health in young patients who haven't responded well to other treatments they are already taking. Researchers are also looking at how safe the medication is and how it behaves in the body over time.

Nipocalimab

What is already known about the treatment

Nipocalimab – Nipocalimab is administered through an intravenous infusion, meaning it is delivered directly into the bloodstream via a vein. It is currently being studied in clinical trials and is not yet widely available in medical practice. The main therapeutic use being explored is for treating generalized myasthenia gravis (MG) in children and adolescents who do not respond well to standard treatments. Nipocalimab works by targeting and blocking a specific protein involved in the immune response, which helps reduce the symptoms of MG. It is classified as an immunomodulatory agent, which means it helps regulate the immune system.

Investigated diseases

Myasthenia gravis – Myasthenia gravis is a chronic autoimmune neuromuscular disorder characterized by weakness and rapid fatigue of voluntary muscles. It occurs when the immune system produces antibodies that block or destroy many of the muscles' receptor sites for a neurotransmitter called acetylcholine. This leads to a breakdown in communication between nerves and muscles. The condition often affects muscles that control eye and eyelid movement, facial expression, and swallowing. Muscle weakness tends to worsen with activity and improve after periods of rest. The progression of the disease can vary, with symptoms ranging from mild to more severe muscle weakness.