ARGX-113-1803: A Phase 3, Multicenter, Open-label, Long-term Trial to Evaluate the Safety and Efficacy of Efgartigimod (ARGX-113) 10 mg/kg Intravenous in Adult Patients With Primary Immune Thrombocytopenia

Primary immune thrombocytopenia

Frequency and severity of AEs, vital signs, and laboratory assessments.

First 52-week treatment period only: 1. Extent of disease control defined as the percentage of weeks in the trial with platelet counts of ≥50×10^9/L., First 52-week treatment period only: Percentage of patients with overall platelet count response defined as achieving a platelet count of ≥50×10^9/L on at least 4 occasions at any time during the 52-week treatment period., First 52-week treatment period only: Mean change from baseline in platelet count at each visit., First 52-week treatment period only: For patients rolling-over from the ARGX-113-1801 trial with a platelet count of <30×10^9/L: time to response is defined as the time to achieve 2 consecutive platelet counts of ≥50×10^9/L., First 52-week treatment period only: The percentage of weeks in the trial with platelet counts of ≥ 30×10^9/L and at least 20×10^9/L above baseline., First 52-week treatment period only: In patients with baseline platelet count of <15×10^9/L in the current trial (ARGX-113-1803), the percentage of weeks in the trial with platelet counts of ≥30×10^9/L and at least 20×10^9/L above baseline., First 52-week treatment period only: In patients with first exposure to efgartigimod: proportion of patients who achieve a sustained platelet response defined as achieving platelet counts of at least 50×10^9/L for at least 4 of the 6 visits between week 19 and 24 of the trial., First 52-week treatment period only: In patients with first exposure to efgartigimod: proportion of patients in the overall population achieving platelet counts of at least 50×10^9/L for at least 6 of the 8 visits between week 17 and 24 of the trial., First 52-week treatment period only: Rate of receipt of rescue therapy (rescue per patient per month)., First 52-week treatment period only: Reduction in concurrent ITP therapy., First 52-week treatment period only: Incidence and severity of the WHO-classified bleeding events., First 52-week treatment period only: Change from baseline in PRO (FACIT-Fatigue, Fact-Th6) and QoL (SF- 36) at planned visits., First 52-week treatment period only: Pharmacokinetic parameter of efgartigimod: serum concentration observed predose (Ctrough)., First 52-week treatment period only: Pharmacodynamics markers: total IgG., First 52-week treatment period and additional 52-week treatment periods: Incidence of anti-drug antibodies (ADA) to efgartigimod.

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