A Phase 3, Multicenter, Open-Label, Long-Term Trial to Evaluate the Safety and Efficacy of Efgartigimod (ARGX-113) PH20 Subcutaneous in Adult Patients With Primary Immune Thrombocytopenia

简要总结

主要结局

次要结局

• Severity of the World Health Organization (WHO)-classified bleeding events(52 weeks)
• Mean change from baseline in platelet count at each visit(52 weeks)
• Change from baseline in PRO (QoL (Short Form-36 [SF-36]) at planned visits(52 weeks)
• Number of patients who performed self-administration at home over time(52 weeks)
• Percentage of caregivers who administered the injection to the patient at home over time(52 weeks)
• In patients with a baseline platelet count of <15×10E9/L in the current trial (ARGX-113-2005), the percentage of weeks in the trial with platelet counts of ≥30×10E9/L and ≥20×10E9/L above baseline(52 weeks)
• For patients rolling over from the ARGX-113-2004 trial with a platelet count of <30×10E9/L: time to response defined as the time to achieve 2 consecutive platelet counts of ≥50×10E9/L(52 weeks)
• In patients with the first exposure to efgartigimod PH20 SC, the proportion of patients achieving platelet counts of ≥50×10E9/L for at least 6 of the 8 visits between week 17 and week 24(7 weeks (week 17-24))
• Proportion of patients with overall platelet count response defined as achieving a platelet count of ≥50×10E9/L on at least 4 occasions at any time during the 52-week treatment period(52 weeks)
• Extent of disease control defined as the percentage of weeks in the trial with platelet counts of ≥50×10E9/L(52 weeks)
• The percentage of weeks in the trial with platelet counts of ≥30×10E9/L and ≥20×10E9/L above baseline(52 weeks)
• In patients with the first exposure to efgartigimod PH20 SC, the proportion of patients who achieve a sustained platelet response defined as achieving platelet counts of ≥50×10E9/L for at least 4 of the 6 visits between week 19 and week 24(5 weeks (week 19-24))
• Rate of receipt of rescue therapy (rescue per patient per month)(52 weeks)
• Percentage of patients who performed self-administration at home over time(52 weeks)
• Proportion of patients for whom dose and/or frequency of concurrent ITP therapies have been reduced compared to baseline(52 weeks)
• Number of caregivers who administered the injection to the patient at home over time(52 weeks)
• Number of self- or caregiver-supported administrations at home(52 weeks)
• Presence of neutralizing antibodies (NAb) against efgartigimod(216 weeks)
• Incidence of the World Health Organization (WHO)-classified bleeding events(52 weeks)
• Change from baseline in PRO (Functional Assessment of Chronic Illness Therapy Fatigue Scale [FACIT-fatigue]) at planned visits(52 weeks)
• Pharmacodynamics markers: total IgG(52 weeks)
• Number of training visits needed for the participant or caregiver to be competent to start administering efgartigimod PH20 SC(52 weeks)
• Percentage of self- or caregiver-supported administrations at home(52 weeks)
• Incidence and prevalence of antibodies to efgartigimod(216 weeks)
• Titers of antibodies to efgartigimod(216 weeks)
• Serum efgartigimod concentration observed predose (Ctrough)(52 weeks)
• Change from baseline in PRO (Functional Assessment of Cancer Therapy questionnaire-Th6 [FACT-Th6]) at planned visits(52 weeks)