A Phase 3 Clinical Trial of CCX168 (Avacopan) in Patients With ANCA-Associated Vasculitis
A Phase 3 Clinical Trial of CCX168 (Avacopan) in Patients With ANCA-Associated Vasculitis
The aim of the trial is to assess the safety and efficacy of the orally-administered,
 selective complement C5a receptor inhibitor CCX168 (avacopan) in inducing and sustaining
 remission in patients with anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis
 (AAV).
Complement 5a and its receptor C5aR (CD88) are involved in the pathogenesis of
 anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis.

 This is a randomized, double-blind, active-controlled Phase 3 study to evaluate the safety
 and efficacy of the orally-administered, selective C5aR inhibitor CCX168 (avacopan) in
 inducing and sustaining remission in patients with anti-neutrophil cytoplasmic antibody
 (ANCA)-associated vasculitis (AAV) treated concomitantly with Rituximab or
 Cyclophosphamide/Azathioprine.
Inclusion Criteria:

 -  Clinical diagnosis of granulomatosis with polyangiitis (Wegener's) or microscopic
 polyangiitis

 -  Male and female subjects, aged at least 18 years, with newly-diagnosed or relapsed AAV
 where treatment with cyclophosphamide or rituximab is needed; where approved by
 Regulatory Agencies, adolescents (12-17 year old) may be enrolled

 -  Use of adequate contraception

 -  Positive test for anti-PR3 or anti-MPO

 -  At least 1 major item, or at least 3 non-major items, or at least the 2 renal items of
 proteinuria and hematuria on 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 be
 withdrawn prior to receiving the cyclophosphamide or rituximab dose on Day 1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 mg
 prednisone-equivalent for more than 6 weeks continuously prior to screening

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

 -  For patients scheduled to receive cyclophosphamide treatment, urinary outflow
 obstruction, active infection (especially varicella zoster infection), or platelet
 count <50,000/μL before start of dosing

 -  Participated previously in a CCX168 study