A Phase 3 Clinical Trial of CCX168 (Avacopan) in Patients With ANCA-Associated Vasculitis

Inclusion Criteria:

• Clinical diagnosis of granulomatosis with polyangiitis (Wegener's) or microscopicpolyangiitis

• Male and female subjects, aged at least 18 years, with newly-diagnosed or relapsedassociated vasculitis (AAV) where treatment with cyclophosphamide or rituximab isneeded; where approved by Regulatory Agencies, adolescents (12-17 year old) may beenrolled

• Use of adequate contraception

• Positive test for anti-proteinase 3 (PR3) or anti-myeloperoxidase (MPO)

• At least 1 major item, or at least 3 non-major items, or at least the 2 renal itemsof proteinuria and hematuria on Birmingham Vasculitis Activity Score (BVAS)

• Estimated glomerular filtration rate ≥15 mL/minute/1.73 m^2 at screening

Exclusion Criteria:

• Pregnant or breast-feeding

• Alveolar hemorrhage requiring pulmonary ventilation support at screening

• Any other known multi-system autoimmune disease

• Required dialysis or plasma exchange within 12 weeks prior to screening

• Have a kidney transplant

• Received cyclophosphamide within 12 weeks prior to screening; if on azathioprine,mycophenolate mofetil or methotrexate at the time of screening, these drugs must bewithdrawn prior to receiving the cyclophosphamide or rituximab dose on Day 1

• Received intravenous glucocorticoids, >3000 mg methylprednisolone equivalent, within 4 weeks prior to screening

• Have been taking an oral daily dose of a glucocorticoid of more than 10 mgprednisone-equivalent for more than 6 weeks continuously prior to screening

• Received rituximab or other B-cell antibody within 52 weeks of screening or 26 weeksprovided B cell reconstitution has occurred (i.e., CD19 count > 0.01x10^9/L);received anti-tumor necrosis factor (TNF) treatment, abatacept, alemtuzumab,intravenous immunoglobulin (IVIg), belimumab, tocilizumab, or eculizumab within 12weeks prior to screening

• For patients scheduled to receive cyclophosphamide treatment, urinary outflowobstruction, active infection (especially varicella zoster infection), or plateletcount <50,000/μL before start of dosing

• Participated previously in a CCX168 study