eligibility_summary
Eligibility: Children 6-<18 with GPA or MPA, newly diagnosed or relapsed AAV, anti-PR3 or anti-MPO positive, activity: >=1 PVAS major, >=3 PVAS nonmajor, or proteinuria+hematuria, eGFR >15 mL/min/1.73 m2, weight >=15 kg. Exclude: other multisystem autoimmune diseases (e.g., EGPA, SLE, IgA vasculitis, rheumatoid vasculitis, Sjogren, anti-GBM, cryoglobulinemia), alveolar hemorrhage needing prolonged invasive ventilation, ongoing immunosuppressives/corticosteroids.
trial_source
clinical_trials.gov from Dec 2, 2025
annotation_status
ai
ai_summary
Phase 3, open-label, single-arm pediatric AAV (GPA/MPA) study testing avacopan with background rituximab or cyclophosphamide. Avacopan (AMG 569, Tavneos) is a small-molecule oral C5a receptor 1 (C5aR1/CD88) antagonist that blocks C5a-driven neutrophil activation, chemotaxis, and degranulation, aiming to reduce ANCA-mediated vascular inflammation and serve as steroid-sparing therapy. Rituximab is an anti-CD20 monoclonal antibody that depletes CD20+ B cells, lowering ANCA (PR3/MPO) autoantibody production. Cyclophosphamide is an alkylating immunosuppressant that cross-links DNA and depletes proliferating lymphocytes (B and T cells). Targeted cells/pathways: complement C5a–C5aR axis and neutrophil effector functions, B-cell compartment driving ANCA production, broader lymphocyte proliferation with cyclophosphamide.