A Phase 3, Randomized, Multicenter, Open-label Study to Evaluate the Efficacy and Safety of Romiplostim Plus Predniso(lo)ne vs. Predniso(lo)ne Alone for the Treatment of Adults With Previously Untreated Primary Immune Thrombocytopenia (ITP)

Condition studied

Diagnosis of primary ITP according to the 2019 International Consensus Report that is previously untreated and requires treatment

Investigational medicinal product(s)

Eligibility

Primary endpoint

Durable Platelet Response (DPR), defined as achieving at least 9999 platelet responses of ≥ 50 × 109/L during the 9999, with at least 1 platelet response of ≥ 50 × 109/L 9999. Qualifying platelet counts must be measured at least 5 days apart.

Endpoint detail

Time to next treatment (TTNT), defined as time from randomization to the earliest of the initiation of the second-line of ITP therapy, receiving rescue medication/therapy 9999, continuing predniso(lo)ne 9999 restarting romiplostim or predniso(lo)ne after the taper, use of antifibrinolytics, or death., Cumulative corticosteroid exposure during study 9999., Changes from baseline at each assessment in Immune Thrombocytopenia – Patient Assessment Questionnaire (ITP-PAQ) scales and sub-scales, Summary scores at each assessment and changes from baseline of visual analogue scale (VAS) scores as measured by EQ-5D-5L, Hospitalization and rescue medication during study part 1 9999, Incidence and severity of treatment emergent adverse events (TEAEs), treatment emergent serious adverse events (TESAEs), treatment emergent adverse events of interest (EOI), and fatal TEAEs, Clinically significant bleeding in the Immune Thrombocytopenia-specific Bleeding Assessment Tool (ITP-BAT), defined as Grade ≥ 2 in the skin domain, or Grade ≥ 1 in the mucosal domain, or Grade ≥ 1 in the organ domain, at each assessment, Serum trough levels (Ctrough) before dosing of romiplostim at select timepoints, Incidence of anti-romiplostim antibodies and anti-thrombopoietin (TPO) antibodies