Clinical Trial to Evaluate Safety and Efficacy of CCX168 in ANCA-Associated Vasculitis
Clinical Trial to Evaluate Safety and Efficacy of CCX168 in ANCA-Associated Vasculitis
The aim of this trial is to test the safety and efficacy of two dose regimens of the
 complement C5a receptor CCX168 in patients with anti-neutrophil cytoplasmic antibody
 (ANCA)-associated vasculitis (AAV).

 Funding Source - FDA OOPD
Complement 5a and its receptor C5aR (CD88) is involved in the pathogenesis of anti-neutrophil
 cytoplasmic antibody (ANCA)-associated vasculitis. This is a randomized, double-blind,
 placebo-controlled Phase 2 study to evaluate the safety and efficacy of the C5aR inhibitor
 CCX168 in subjects with ANCA-associated vasculitis. The aim of this trial is to test the
 safety and efficacy of two dose regimens of the complement C5a receptor CCX168 in patients
 with anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV).
Inclusion Criteria:

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

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

 -  Use of adequate contraception during, and for at least the three months after, any
 administration of study medication is 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

 -  Have at least one "major" item, or at least 3 other items, or at least 2 renal items
 on the Birmingham Vasculitis Activity Score (BVAS) version 3

 -  Estimated glomerular filtration rate (eGFR) ≥ 20 mL per minute

 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 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 withdrawn prior to receiving the cyclophosphamide or rituximab dose on Day 1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 corticosteroids, >3000 mg methylprednisolone equivalent, within
 12 weeks prior to screening

 -  Received an oral daily dose of a corticosteroid of more than 10 mg
 prednisone-equivalent for more than 6 weeks continuously prior to the screening visit

 -  Received rituximab or other B-cell antibody within 52 weeks of screening or 26 weeks
 provided B cell reconstitution has occurred; received anti-tumor necrosis factor (TNF)
 treatment, abatacept, alemtuzumab, intravenous immunoglobulin (IVIg), belimumab,
 tocilizumab, or plasma exchange within 12 weeks prior to screening