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 (May 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
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
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 →
Could you qualify for this trial?
Enter your biomarker results to see how this trial's eligibility criteria match your specific cancer profile.
Check if I qualify