ARGX-113-1801 (ADVANCE)

A Phase 3, Multicenter, Randomized, Double-Blinded, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Efgartigimod (ARGX 113) 10 mg/kg Intravenous in Adult Patients With Primary Immune Thrombocytopenia (ITP)

Study treatments

• Arm 1: efgartigimod 
• Arm 2: placebo

Inclusion criteria

• Male or female patient aged ≥18 years.
• Confirmed ITP diagnosis, at least 3 months before randomization and according to the American Society of Hematology Criteria, and no known other etiology for thrombocytopenia.
• Diagnosis supported by a response to a prior ITP therapy (other than thrombopoietin receptor agonists (TPO-RAs)), in the opinion of the investigator.
• Mean platelet count of <30×109/L (and no single platelet count of >35×109/L) from 3 qualifying counts, 2 during the screening period and the predose platelet count at visit 1. The 3 platelet counts must be over the course of 7 to 14 days, with at least 2 days between any 2 counts.

• At the start of the trial, the patient is either on concurrent ITP treatment(s) and has received at least 1 prior therapy for ITP in the past, or the patient is not on treatment for ITP but has received at least 2 prior treatments for ITP. Permitted concurrent ITP medications include oral corticosteroids, oral immunosuppressants, dapsone/danazol, fostamatinib, and/or oral TPO-RAs.

  Patients not receiving concurrent ITP therapy are also eligible for the trial if they have not received prior ITP therapy for at least 4 weeks prior to baseline, and 6 months in case of prior ITP therapy with an anti-CD20 therapy (eg, rituximab).

Exclusion criteria

• ITP/thrombocytopenia associated with another condition, eg, lymphoma, chronic lymphocytic leukemia, viral infection, hepatitis, induced or alloimmune thrombocytopenia, or thrombocytopenia associated with myeloid dysplasia.
• Use of anticoagulants (eg, vitamin K antagonists, direct oral anticoagulants) within 4 weeks prior to randomization.
• Use of any transfusions within 4 weeks prior to randomization.
• Use of Ig (IV, subcutaneous, or intramuscular route) or plasmapheresis (PLEX), 4 weeks prior to randomization.
• Use of anti-CD20 therapy (eg, rituximab) within 6 months prior to randomization.
• Use of romiplostim within 4 weeks prior to randomization.

Participating sites

• University Hospital Leuven Gasthuisberg