A Study to Evaluate the Safety and Efficacy of CCX168 in Subjects With ANCA-Associated Vasculitis
A Study to Evaluate the Safety and Efficacy of CCX168 in Subjects With ANCA-Associated Vasculitis
The aim of this trial is to optimize the treatment to induce remission for patients with
 non-life-threatening anti-neutrophil cytoplasmic antibody vasculitis (AAV). The intent is to
 reduce the toxicity of induction therapy by reducing the overall exposure to or eliminating
 entirely the use of systemic corticosteroids during the induction period with an inhibitor of
 the complement C5a receptor plus cyclophosphamide or rituximab.
The primary safety objective of this study is to evaluate the safety and tolerability of
 CCX168 in subjects with AAV on background cyclophosphamide or rituximab treatment.

 The primary efficacy objective is to evaluate the efficacy of CCX168 based on the Birmingham
 Vasculitis Activity Score (BVAS) version 3.

 The secondary objectives of this study include assessment of the feasibility of reducing or
 eliminating the use of corticosteroids in the treatment of subjects with ANCA-associated
 vasculitis without the need for rescue corticosteroid measures and the effect of CCX168 on
 several disease parameters.
Key Inclusion Criteria:

 -  Clinical diagnosis of granulomatosis with polyangiitis (Wegener's), microscopic
 polyangiitis or renal limited vasculitis

 -  Male and postmenopausal or surgically sterile female subjects aged at least 18 years
 with new or relapsed AAV where treatment with cyclophosphamide or rituximab would be
 required

 -  Positive indirect immunofluorescence (IIF) test for P-ANCA or C-ANCA, or positive
 ELISA test for anti-proteinase-3 (PR3) or anti-myeloperoxidase (MPO) at screening

 -  Estimated glomerular filtration rate (eGFR) ≥ 20mL/min

 -  Have at least one "major" item, or at least 3 non-major items, or at least 2 renal
 items on the BVAS version 3

 Key Exclusion Criteria:

 -  Severe disease as determined by rapidly progressive glomerulonephritis, alveolar
 hemorrhage, hemoptysis, rapid-onset mononeuritis multiplex or central nervous system
 involvement

 -  Any other multi-system autoimmune disease

 -  Medical history of coagulopathy or bleeding disorder

 -  Received cyclophosphamide within 12 weeks of 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 withdrawn prior to receiving the cyclophosphamide or rituximab dose on Day 1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 high-dose intravenous corticosteroids within 4 weeks of screening

 -  On an oral dose of a corticosteroid of more than 10mg prednisone-equivalent at the
 time of screening

 -  Received rituximab or other B-cell antibody within 52 weeks of screening or 26 weeks
 provided B cell reconstitution has occurred; received anti-TNF treatment, abatacept,
 alemtuzumab, IVIg or plasma exchange within 12 weeks of screening