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

Primary Immune Thrombocytopenia

Incidence, frequency, and severity of adverse events (AEs), AEs of special interest (AESIs), and serious AEs (SAEs), Vital signs, electrocardiogram (ECG), and laboratory safety evaluations

First 52-week treatment period only: 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: Proportion of participants 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 participants rolling over from the ARGX-113-2004 trial with a platelet count of <30×10^9/L: time to response 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 ≥20×10^9/L above baseline, First 52-week treatment period only: In patients with a baseline platelet count of <15×10^9/L in the current trial (ARGX-113-2005), the percentage of weeks in the trial with platelet counts of ≥30×10^9/L and ≥20×10^9/L above baseline, First 52-week treatment period only: In participants with the first exposure to efgartigimod PH20 SC, the proportion of participants who achieve a sustained platelet response defined as achieving platelet counts of ≥50×10^9/L for at least 4 of the 6 visits between week 19 and week 24, First 52-week treatment period only: In participants with the first exposure to efgartigimod PH20 SC, the proportion of participants achieving platelet counts of ≥50×10^9/L for at least 6 of the 8 visits between week 17 and week 24, First 52-week treatment period only: Proportion of participants for whom dose and/or frequency of concurrent ITP therapies have been reduced compared to baseline, First 52-week treatment period only: Rate of receipt of rescue therapy (rescue per participant per month), First 52-week treatment period only: Incidence and severity of the World Health Organization (WHO)-classified bleeding events, First 52-week treatment period only: Serum efgartigimod concentration observed predose (Ctrough), First 52-week treatment period only: Change from baseline in PRO (Functional Assessment of Chronic Illness Therapy Fatigue Scale [FACIT-fatigue], Functional Assessment of Cancer Therapy questionnaire-Th6 [FACT-Th6]), QoL (Short Form-36 [SF-36]), and ITP-Patient Assessment Questionnaire [ITP-PAQ] at planned visits, First 52-week treatment period only: PD markers: total IgG, First 52-week treatment period only: Number and percentage of participants who performed self-administration at home over time, First 52-week treatment period only: Number and percentage of caregivers who administered the injection to the participant at home over time, First 52-week treatment period only: Number of training visits needed for the participant or caregiver to become competent in the (self-)administration of efgartigimod PH20 SC, First 52-week treatment period only: Number and percentage of self-administrations or caregiver-supported administrations at home First 52-week treatment period and additional 52-week treatment periods, First 52-week treatment period and additional 52-week treatment periods: Incidence and prevalence of antibodies to efgartigimod, First 52-week treatment period and additional 52-week treatment periods: Titers of antibodies to efgartigimod, First 52-week treatment period and additional 52-week treatment periods: Presence of neutralizing antibodies (NAb) against efgartigimod

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