Home › Trials › NCT06321601

Evaluating Avacopan for treating pediatric vasculitis

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)

Phase 3 Interventional Amgen · NCT06321601

This study is testing if a new medication called avacopan, combined with other treatments, can help children with certain types of vasculitis feel better and stay safe.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment20 (estimated)
Ages6 Years to 17 Years
SexAll
SponsorAmgen Industry-sponsored
Locations31 sites (Atlanta, Georgia and 30 other locations)
Trial IDNCT06321601 on ClinicalTrials.gov

What this trial studies

This study aims to evaluate the efficacy of avacopan in children aged 6 to less than 18 years who are diagnosed with antineutrophil cytoplasmic antibody-associated vasculitis (AAV), specifically granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA). Participants will receive avacopan in combination with either rituximab or cyclophosphamide as part of their treatment regimen. The study will assess the clinical outcomes and safety of this combination therapy in a pediatric population. The trial is designed to provide insights into the effectiveness of avacopan in managing AAV in children.

Who should consider this trial

Good fit: Ideal candidates are children and adolescents aged 6 to less than 18 years with a clinical diagnosis of GPA or MPA and specific laboratory findings.

Not a fit: Patients with other known multisystem autoimmune diseases or severe complications like alveolar hemorrhage may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve outcomes for children suffering from AAV.

How similar studies have performed: Other studies have shown promising results with similar approaches in adult populations, but this specific pediatric application is novel.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* Male and female children and adolescents from 6 to \< 18 years of age
* Clinical diagnosis of granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA), consistent with Chapel-Hill Consensus Conference definitions (Jennette et al, 2013)
* Newly diagnosed or relapsed AAV with positive test for anti-PR3 or anti-MPO antibodies
* At least 1 PVAS major item, at least 3 PVAS nonmajor items, or at least the 2 renal items of proteinuria and hematuria.
* eGFR \> 15 mL/min/1.73 m2 (using modified Schwartz equation per central lab guidelines)
* Participants must have a bodyweight of ≥ 15 kg at day 1.

Exclusion Criteria:

* Any other known multisystem autoimmune disease including eosinophilic granulomatosis with polyangiitis (Churg-Strauss), systemic lupus erythematosus , IgA vasculitis (Henoch-Schönlein), rheumatoid vasculitis, Sjogren's syndrome, anti-glomerular basement membrane disease, or cryoglobulinemic vasculitis
* Alveolar hemorrhage requiring invasive pulmonary ventilation support anticipated to last beyond the screening period of the study
* Any medical condition requiring or expected to require continued use of immunosuppressive treatments, including corticosteroids that may cause confoundment with study assessments and study conclusions.

Where this trial is running

Atlanta, Georgia and 30 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions VasculitisPediatricAvacopanTavneos®
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.