Efgartigimod for children with generalized myasthenia gravis
Treatments studied
Part of Brain & nervous system, Cancer, Immune system & allergy clinical trials.
This trial studies how efgartigimod (given through a vein) behaves in children and whether it is safe and effective for generalized myasthenia gravis. It is designed for children who have not done well enough with standard treatments like steroids or other immune medicines.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Child is age 2 up to younger than 18, with consent/assent from the child or caregiver
- Doctor-confirmed generalized myasthenia gravis, in MGFA class II, III, or IVa
- Not adequately helped (or had unacceptable side effects) by treatments like steroids, other immune medicines, or acetylcholinesterase medicines, and still on stable gMG treatment
- Blood test shows positive acetylcholine receptor antibodies (anti-AChR)
- Family agrees to follow the visit schedule and trial rules, including birth control rules if relevant
- No major exclusion issues, like recent certain infections, certain medicines/vaccines, or prior use of the study drug
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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This trial checks the long-term safety of efgartigimod given through an IV and as a quick under-the-skin injection in children with generalized myasthenia gravis (gMG). It may help your care team understand how safely this treatment can be used over time for kids who already took related study treatments.
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