Study of Efgartigimod PH20 SC for Children Aged 2 to 17 with Generalized Myasthenia Gravis

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What is this study about?

This clinical trial is focused on studying a condition called Generalized Myasthenia Gravis, which is a chronic autoimmune disease that causes muscle weakness. The study involves a treatment called efgartigimod PH20 SC, which is a solution for injection. This treatment is being tested in children aged 2 to less than 18 years who have this condition. The purpose of the study is to confirm the appropriate dose of this treatment for young patients with Generalized Myasthenia Gravis.

Participants in the study will receive the treatment through a subcutaneous injection, which means it is injected under the skin. The study will monitor how the body processes the treatment and its effects on the disease. This includes checking the levels of certain proteins in the blood and observing any changes in the symptoms of Generalized Myasthenia Gravis. The study will also look at the safety of the treatment by recording any side effects and changes in health indicators like heart rate and blood tests.

The study will take place over a period of time, during which participants will have regular check-ups to assess their response to the treatment. The goal is to gather information that will help determine the best way to use efgartigimod PH20 SC in treating children with Generalized Myasthenia Gravis. This research is important for understanding how to manage this condition in younger patients and improve their quality of life.

1 joining the study

Upon joining the study, the participant must provide written informed consent or assent, which indicates understanding of the study requirements.

The participant must be between 2 and less than 18 years old and have a diagnosis of generalized myasthenia gravis confirmed by a physical examination and seropositivity for anti-acetylcholine receptor antibodies.

2 initial assessment

An initial assessment will be conducted to ensure the participant has had an unsatisfactory response to previous treatments such as immunosuppressants, corticosteroids, or acetylcholinesterase inhibitors.

The participant must be on a stable dose of these medications for at least one month before the screening.

3 medication administration

The participant will receive efgartigimod PH20 SC through a subcutaneous injection. The specific dosage and frequency will be determined during the study to confirm an appropriate dose for pediatric participants.

The medication is provided as a solution for injection under the product name Vyvgart 1,000 mg.

4 monitoring and evaluation

Throughout the study, the participant’s efgartigimod serum concentrations will be monitored to analyze age and size dependency.

Total IgG levels and anti-acetylcholine receptor antibody levels will be measured for pharmacokinetic and pharmacodynamic modeling.

5 safety and side effects

The study will track the incidence and severity of any adverse events, including serious adverse events and those of special interest.

Changes in vital signs, electrocardiograms, and clinical laboratory tests will be observed to ensure participant safety.

6 quality of life assessment

The participant’s quality of life will be evaluated using various tools, such as the Myasthenia Gravis Activities of Daily Living score and the Quality of Life in Neurological Disorders Questionnaire.

Changes in fatigue levels and overall improvement will be assessed using the Clinical Global Impression of Improvement scale.

7 study duration

The study is expected to continue until September 30, 2026, with recruitment starting on September 1, 2024.

Participants will be involved in the study for its entire duration, with regular assessments and monitoring.

Who Can Join the Study?

The participant, or their legally authorized representative, must understand what the study involves and be able to give written permission to take part.
The participant must be between 2 and less than 18 years old at the time they agree to join the study.
The participant must have been diagnosed with Generalized Myasthenia Gravis, a condition confirmed by a physical exam and a positive test for specific antibodies called anti-acetylcholine receptor antibodies.
The participant must have tried treatments like immunosuppressants, corticosteroids, or acetylcholinesterase inhibitors but did not have a satisfactory response. They must be on a stable treatment plan for Myasthenia Gravis for at least one month before the study starts.
The participant must agree to use birth control as required by local rules. If they can have children, they must have a negative blood pregnancy test at the start and a negative urine pregnancy test before receiving the study medication.

Who Cannot Join the Study?

Patients who have a history of severe allergic reactions to any of the study medications.
Patients who are currently participating in another clinical trial.
Patients who have a serious infection that requires treatment with antibiotics.
Patients who have received a live vaccine within the last 4 weeks. A live vaccine is a type of vaccine that uses a weakened form of the germ that causes a disease.
Patients who have a history of drug or alcohol abuse within the past year.
Patients who have any other medical condition that the study doctors believe would make it unsafe for them to participate.
Patients who are pregnant or breastfeeding.
Patients who have had a major surgery within the last 3 months.
Patients who have a history of cancer, except for certain types of skin cancer that have been treated successfully.
Patients who have a history of heart problems, such as a heart attack or heart failure.

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
IRCCS Istituto Giannina Gaslini	Genoa	Italy
Universitair Ziekenhuis Gent	Gent	Belgium
Uniwersyteckie Centrum Kliniczne Warszawskiego Uniwersytetu Medycznego	Warsaw	Poland
Fakultni Nemocnice Brno	Brno	Czechia
University Hospital Ostrava	Ostrava	Czechia
Neurologia Śląska Centrum Medyczne	Katowice	Poland
Usakjluylksivytpxzndw Eruvh All	Essen	Germany
Lmucs Ulclhwvmlpom Mctplne Cjnhrgf (akjzp	Leiden	The Netherlands
Ahltkppwhp Pclbgyjc Htijjhkq Dz Midbwjisb	Marseille	France
Upoorpbqayxdye Ckbgagi Kudrteyvl	Gdansk	Poland

Trial status

Country	Status	Recruitment Start
Belgium	Recruiting	01.09.2024
Czechia	Recruiting	01.09.2024
France	Recruiting	01.09.2024
Germany	Recruiting	01.09.2024
Italy	Recruiting	01.09.2024
Poland	Recruiting	01.09.2024
Spain	Recruiting	01.09.2024
The Netherlands	Recruiting	01.09.2024

Trial locations

Investigated drugs:

Efgartigimod Alfa

Efgartigimod PH20 SC is a medication being studied for its effects on children and teenagers with generalized myasthenia gravis, a condition that causes muscle weakness. This medication is designed to help improve muscle strength by targeting specific parts of the immune system that are involved in the disease. The study aims to find the right dose for young patients to ensure it is safe and effective.

Investigated diseases:

Myasthenia gravis

Generalized Myasthenia Gravis – This is a chronic autoimmune disorder that affects the communication between nerves and muscles, leading to muscle weakness. It occurs when the immune system mistakenly attacks the connections between nerves and muscles, specifically targeting the acetylcholine receptors. This disruption results in fluctuating muscle weakness and fatigue, which can affect various muscle groups, including those responsible for eye movement, facial expression, chewing, talking, and swallowing. The severity of muscle weakness can vary throughout the day and may worsen with activity. Symptoms often improve with rest, and the condition can affect individuals of any age, though it is more common in women under 40 and men over 60.

Trial ID:

2023-506159-12-00

Protocol code:

ARGX-113-2207

Trial Phase:

Therapeutic use (Phase IV)

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Study of Efgartigimod PH20 SC for Children Aged 2 to 17 with Generalized Myasthenia Gravis

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?