An Open-Label, Single-arm Study to Evaluate the Pharmacokinetics (PK), Pharmacodynamics (PD), Safety, and Efficacy of Gefurulimab in Pediatric Patients (6 to < 18 years of age) with Generalized Myasthenia Gravis (gMG) Who Express Acetylcholine Receptor Antibodies (AChR+)

Generalized Myasthenia Gravis with autoantibodies against acetylcholine receptor (AChR)

Primary PK Estimand: Variables: Serum gefurulimab concentrations from Day 1 predose through Week 18 predose (Cmax, Ctrough) Summary measures: Summary statistics of gefurulimab concentrations at Day 1 predose through Week 18 predose Primary PD Estimand: Variables: Serum free C5 concentrations from Day 1 predose through Week 18 predose Summary measures: Summary statistics of serum free C5 concentrations at Day 1 predose through Week 18 predose

"• Incidence of TEAEs and TESAEs • Change from Baseline in vital signs, ECG, physical examination, and laboratory assessments • Tolerability of the subcutaneous injection", "• Change from Baseline in QMG total score from Baseline through Week 18 • Proportion of participants with ≥ 5-point reduction compared to Baseline in the QMG total score over time through Week 18 • Change in MG-ADL total score from Baseline through Week 18 • Proportion of participants with ≥ 3-point reduction compared to Baseline in the MG-ADL total score over time through Week 18"

No related papers found yet.

Poland