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.

简要总结

主要结局

次要结局

• Extent of disease control defined as the percentage of weeks in the trial with platelet counts of ≥50×10E9/L.(Over the 52 weeks of treatment)
• Mean change from baseline in platelet count at each visit.(Up to 60 weeks, at each visit)
• In patients with first exposure to efgartigimod: proportion of patients in the overall population achieving platelet counts of at least 50x10^9/L for at least 6 of the 8 visits between week 17 and 24 of the trial.(Up to 7 weeks, between visit 17 and 24 of the trial)
• Rate of receipt of rescue therapy (rescue per patient per month).(Up to 60 weeks, at each visit)
• Incidence of anti-drug antibodies (ADA) to efgartigimod.(Up to 216 weeks)
• 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(Up to 60 weeks, at each visit)
• The percentage of weeks in the trial with platelet counts of ≥30×109/L and at least 20×10E9/L above baseline.(Over the 52 weeks of treatment)
• 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.(Over the 52 weeks of treatment)
• In patients with baseline platelet count of <15×10E9/L in the current trial (ARGX-113-1803), the percentage of weeks in the trial with platelet counts of ≥30×10E9/L and at least 20×10E9/L above baseline.(Over the 52 weeks of treatment)
• Incidence and severity of the WHO-classified bleeding events.(Up to 60 weeks, at each visit)
• Change from baseline in Patient reported Outcomes (Fact-Th6) at planned visits.(Up to 52 weeks)
• 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.(Up to 5 weeks, between visit 19 and 24 of the trial)
• Change from baseline in Patient reported Outcomes (FACIT-Fatigue) at planned visits.(Up to 52 weeks)
• Reduction in concurrent ITP therapy.(Up to 60 weeks, at each visit)
• Change from baseline in Quality of Life (SF-36) at planned visits.(Up to 52 weeks)
• Pharmacokinetic parameter of efgartigimod: serum concentration observed predose (Ctrough).(Up to 60 weeks)
• Pharmacodynamics markers: total IgG.(Up to 60 weeks)