OncoMatch/Clinical Trials/NCT04833894
Evaluating the Pharmacokinetics, Pharmacodynamics, and Safety of Efgartigimod Administered Intravenously in Children With Generalized Myasthenia Gravis
Is NCT04833894 recruiting? Yes, currently enrolling (Jun 2026). This Phase 2/3 trial studies Efgartigimod IV for generalized myasthenia gravis.
Treatment: Efgartigimod IV — The purpose of this trial is to investigate the PK, PD, safety, and activity of efgartigimod IV in children and adolescents aged from 2 to less than 18 years of age with gMG. Trial details include: * The maximum trial duration for each individual participant will be approximately 28 weeks * The treatment duration will be 8 weeks for the dose-confirmatory part (Part A) and 18 weeks for the treatment response-confirmatory part (Part B)
Check if I qualifyExtracted eligibility criteria
Treatments studied
Other
Biomarker criteria
Required: CHRNA1 anti-AChR antibody positive
Positive serologic test for acetylcholine receptor (anti-AChR) antibodies at screening (for younger participants (<15kg) historical values can be used).
Disease stage
Required: Stage MGFA CLASS II, MGFA CLASS III, MGFA CLASS IVA
Excluded: Stage MGFA CLASS I, MGFA CLASS IVB, MGFA CLASS V
Demographics
Prior therapy
Must have received: immunosuppressant
unsatisfactory response (efficacy and/or safety) to immunosuppressants, steroids or acetylcholinesterase (AChE) inhibitors and should be on stable concomitant gMG therapy of adequate duration before screening
Cannot have received: monoclonal antibody
Use of any monoclonal antibody within the 6 months before the first dose of IMP
Cannot have received: intravenous immunoglobulin
Use of intravenous immunoglobulin (IVIg), administered subcutaneously or intramuscularly, or PLEX within 4 weeks before screening
Cannot have received: plasma exchange
Use of intravenous immunoglobulin (IVIg), administered subcutaneously or intramuscularly, or PLEX within 4 weeks before screening
Cannot have received: investigational product
Use of an investigational product within 3 months or 5 half-lives (whichever is longer) before the first dose of IMP
Cannot have received: efgartigimod
previous participation in an efgartigimod trial with at least 1 dose of IMP received
Structured fields extracted by AI. May contain errors — verify against the official protocol.
US trial sites
- Ann and Robert H Lurie Children's Hospital of Chicago - Main Hospital · Chicago, Illinois
- University of North Carolina at Chapel Hill · Chapel Hill, North Carolina
- University of Virginia (UVA) Health - Developmental Pediatrics Clinic · Charlottesville, Virginia
Showing up to 5 US sites.
See all sites on ClinicalTrials.gov →Frequently asked questions
Is NCT04833894 currently recruiting?
Yes, this trial is currently recruiting patients.
Are there prior therapy exclusions?
Yes. Prior monoclonal antibody, intravenous immunoglobulin, plasma exchange disqualifies patients from enrollment.
Does this trial require CHRNA1?
Yes, CHRNA1 anti-AChR antibody positive is a required biomarker for enrollment.
What disease stage is eligible?
Stage MGFA CLASS II or MGFA CLASS III or MGFA CLASS IVA is required.
Is there an age limit?
Yes. Patients must be 18 years or younger and at least 2 years old.
Could you qualify for this trial?
Enter your biomarker results to see how this trial's eligibility criteria match your specific cancer profile.
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