A Phase 3 Study of Avacopan in Active ANCA-associated Vasculitis (AAV)

  • Research type

    Research Study

  • Full title

    A Phase 3, Open-label, Uncontrolled Single-arm Study to Evaluate the Efficacy, Pharmacokinetics, and Safety of Avacopan in Combination With a Rituximab or a Cyclophosphamide-containing Regimen in Children from 6 Years to <18 Years of Age with Active ANCA-associated Vasculitis (AAV)

  • IRAS ID

    1011286

  • Contact name

    Matthew Rodaway

  • Contact email

    gbinfoline@amgen.com

  • Sponsor organisation

    Amgen Inc.

  • Eudract number

    2024-514779-16

  • Clinicaltrials.gov Identifier

    NCT06321601

  • Research summary

    This study is being done to learn more about avacopan in participants aged 6 years to less than 18 years with active anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV; a rare disease where the body’s immune system mistakenly attacks small to medium sized blood vessels, causing them to become inflamed and damaged). AAV includes different subtypes. This study focuses on two subtypes of AAV: granulomatosis with polyangiitis (GPA), which mainly affects the respiratory system and kidneys among other organs and microscopic polyangiitis (MPA), which primarily affects the kidneys among other organs.
    This study will explore if avacopan combined with rituximab or cyclophosphamide containing regimen (a planned regular treatment) works well to treat AAV. This study will also look at how avacopan moves throughout the body and whether avacopan is safe to take. Avacopan, rituximab or cyclophosphamide that will be received as part of this study will be called study product.
    Patients who participate in the study will undergo a screening period of up to 30 days. The main study period will last for about 60 weeks, which includes treatment period of about 52 weeks, and safety follow-up of about 8 weeks after the patient receives the last dose of avacopan.

  • REC name

    North of Scotland Research Ethics Committee 1

  • REC reference

    25/NS/0127

  • Date of REC Opinion

    8 Dec 2025

  • REC opinion

    Further Information Favourable Opinion