Abigail Schwaede, MD, Assistant Professor of Pediatrics at Northwestern University Feinberg School of Medicine, discusses results on intravenous efgartigimod in juvenile generalized myasthenia gravis (gMG).
gMG is a chronic autoimmune neuromuscular disease characterized by weakness of the skeletal muscles. It mostly develops in adults but children can also develop this rare condition. Common symptoms include weakness of the muscles that control the eye and eyelid, facial expressions, chewing, talking, and swallowing. The condition results from a defect in the transmission of nerve impulses to muscles due to the presence of antibodies against the acetylcholine receptor. The exact reason this occurs is not known.
The ADAPT JR (NCT04833894) clinical trial is a phase 2/3, open-label, uncontrolled study evaluating the pharmacokinetics, pharmacodynamics, safety, and activity of efgartigimod in children ages 2 to 18 years of age with acetylcholine receptor antibody–positive (AChR-Ab+) gMG. Efgartigimod is a human immunoglobulin G1 antibody Fc fragment, which blocks the neonatal Fc receptor. Phase 3 ADAPT/ADAPT+ trials demonstrated that efgartigimod IV is efficacious and well tolerated in adults with gMG.
During dose-confirmatory part A, participants received 1 efgartigimod infusion and pharmacokinetic, pharmacodynamic, and safety endpoints were monitored over 8 weeks. During treatment response–confirmatory part B, participants received 1 or 2 cycles of 4 once-weekly efgartigimod infusionsand additional endpoints, including MG Activities of Daily Living (MG-ADL) scores, were assessed over 18 weeks.
In the adolescent cohort, 6 participants enrolled in part A and continued to part B, and 5 participants enrolled directly in part B. Efgartigimod treatment resulted in IgG and AChR-Ab decreases similar to those observed in previous studies of adults with gMG. Safety profile and MG-ADL improvements were also similar to previous observations.
ADAPT JR child cohort enrollment is ongoing.
To read the abstract presented at AAN, click here.
To learn more about gMG and other rare neurological disorders, visit https://checkrare.com/diseases/neurology-nervous-system-diseases/