Table 4.
Secondary efficacy and pharmacodynamic end points in the C3G cohort at week 48 (ITT population; n = 8)
| Parameter, mean (SD)a | Baselined | Week 48d | %CFBe |
|---|---|---|---|
| Serum albumin, n; g/dl | 8; 3.3 (0.7) | 7; 3.9 (0.5) | 7; 23.5 (36.7) |
| Stabilized or improved eGFRb, n; ≤25% decrease | (–) | 6; (75.0) | (–) |
| Serum C3, n; mg/dl | 8; 44.0 (42.3) | 7; 243.1 (115.2) | 7; 666.4 (477.6) |
| Serum C4, n; mg/dl | 8; 18.5 (7.3) | 6; 18.0 (4.1) | 6; 22.2 (78.8) |
| Plasma C5a, n; μg/l | 7; 7.5 (3.7) | 5; 6.4 (1.7) | 5; −18.7 (27.2) |
| Plasma sC5b-9, n; μg/l | 7; 1337.1 (1382.7) | 5; 553.0 (645.5) | 5; −57.3 (19.3) |
| Serum AH50c, n; units/ml | 8; 38.8 (53.9) | 6; 55.7 (42.8) | 3; −42.7 (49.8) |
| Serum CH50, n; units/ml | 8; 128.6 (121.9) | 6; 234.0 (37.6) | 6; 1216.8 (2557.8) |
AH50, alternative complement pathway hemolytic activity; C3G, complement 3 glomerulopathy; CFB, change from baseline; CH50, classical pathway hemolytic activity, eGFR, estimated glomerular filtration rate; ITT, intent-to-treat; sC5b-9, soluble C5b-9; SD, standard deviation.
Unless specified otherwise.
The eGFR was calculated using the Chronic Kidney Disease Epidemiology Collaboration Creatinine Equation.
Three patients with an AH50 value at baseline of 0 were excluded from the %CFB analysis.
Reference values: albumin, 3.5–5.5 g/dl; eGFR, ≥60 ml/min per 1.73 m2; C3, 90–180 mg/dl; C4, 10–40 mg/dl; C5b-9, 72–244 μg/l; AH50, 77–159 U/ml; CH50, 176–382 U/ml.
%CFB is the mean of individual percent changes from baseline due to missing data at week 48 (n = 8 at baseline and n = 7 at week 48 due to one participant who stopped study drug at Week 24).