Table 2.

Efficacy results for 12-wk treatment perioda

VariablePlacebo plus 60 mg Prednisone Control (n=20)Avacopan plus 20 mg Prednisone (n=22)Avacopan without Prednisone (n=21)
BVAS and vasculitis damage index end points
 Primary end point: treatment responseb at week 12, no. (%)14 (70.0)19 (86.4)17 (81.0)
  Difference in percentage compared  with control16.411.0
  Two-sided 90% confidence interval  for difference in percentage,  avacopan minus control−4.3 to 37.1−11.0 to 32.9
 BVAS
  Baseline
    Actual13.6±1.414.3±1.313.6±1.4
   % Change
  Week 4
    Actual6.6±1.24.9±0.75.3±1.3
   % Change−40±12−64±5−61±9
  Week 12
    Actual5.0±1.62.6±0.73.6±1.1
   % Change−56±14−79±5−73±7
 Vasculitis damage index
  Baseline
   Actual1.2±0.30.9±0.30.5±0.3
   Change
  Week 12
   Actual1.8±0.41.2±0.30.8±0.3
   Change0.7±0.20.3±0.10.2±0.1
 Remission (BVAS 0) at week 12, no. (%)8 (40)10 (45)7 (33)
 Remission at week 4 sustained through week 12, no. (%)1 (5)3 (14)6 (29)c
Renal end points
No. patients201818
 Renal responsed at week 12, no. (%)8 (40)10 (56)6 (33)
 UACR (milligram per gram creatinine) in patients with albuminuria at baseline
  No. patients202220
  Baseline
   Actual318279280
   % Change
  Week 4
   Actual355168142
   % Change15−40e−47f
  Week 12
   Actual252127158
   % Change−21−56e−43
 eGFR, ml/min per 1.73 m2
  Baseline
   Actual47.2±3.552.5±5.754.8±4.4
   Change
  Week 4
   Actual45.8±3.154.5±4.553.5±4.8
   Change−0.8±2.02.0±2.3−1.2±3.7
  Week 12
   Actual52.8±3.656.2±4.356.1±5.2
   Change5.6±2.36.0±2.30.8±2.2
 Urinary red blood cell count (cells per high-power field) in patients with hematuria at baseline
  No. patients202019
  Baseline
   Actual222617
   % Change
  Week 4
   Actual576
   % Change−76−72−65
  Week 12
   Actual253
   % Change−92−83−85
Inflammation markers
 Urinary MCP-1–to-creatinine (pg/mg)
  Baseline
   Actual7521266806
   % Change
  Week 4
   Actual641588557
   % Change−15−54e−30
  Week 12
   Actual426373374
   % Change−43−70f−50
 C-reactive protein (mg/L)
  Baseline
   Actual9.35.95.0
   % Change
  Week 4
   Actual2.11.94.0
   % Change−76−68−17c
  Week 12
   Actual3.61.32.7
   % Change−61−77−41
Health-related quality-of-life end points
 No. patients10149
 SF-36 version 2 Physical Functioning
  Baseline
   Actual67±768±874±11
   % Change
  Week 4
   Actual71±575±690±6
   % Change31±3061±5168±71c
  Week 12
   Actual72±484±493±3
   % Change33±3183±6561±57c
 SF-36 version 2 Mental Health
  Baseline
   Actual66±562±582±7
   % Change
  Week 4
   Actual68±573±679±6
   % Change4±322±8−8±8c
  Week 12
   Actual65±579±689±4
   % Change1±834±11f3±6e
 EQ-5D-5L visual analog scale
  Baseline
   Actual69±565±569±6
   % Change
  Week 4
   Actual66±570±580±4
   % Change−3±520±157±7c
  Week 12
   Actual66±575±578±4
   % Change−3±628±145±2
  • –, not applicable.

  • a Plus-minus values are mean±SEM; UACR, urinary red blood cell count, and urinary MCP-1–to-creatinine ratio actual values are geometric means; and percentage change from baseline are ratios of geometric means of visit over baseline. Number of patients per group for each end point is as indicated in the first row unless otherwise indicated.

  • b Primary end point: treatment response on the basis of BVAS decrease of at least 50% from baseline and no worsening in any body system. Avacopan plus 20 mg prednisone group was statistically noninferior to control (P=0.002) and avacopan without prednisone was also noninferior to control (P=0.01).

  • c P<0.05 for comparison of avacopan versus control.

  • d Renal response was assessed in patients with hematuria and albuminuria at baseline, and was defined as an improvement in renal parameters, i.e., an increase in eGFR, a decrease in urinary red blood cell count, and a decrease in UACR.

  • e P<0.01 for comparison of avacopan versus control.

  • f P<0.001 for comparison of avacopan versus control.