Table 3.
Efficacy and tolerability of pegloticase in randomized controlled trials
| Study | Treatment, dosage | No. of patients | Duration | sUr < 6 mg/dL (0.36 mmol/L)a | Gout flares | Tophus dissolution (final visit) | Withdrawal due to ADR |
|---|---|---|---|---|---|---|---|
| Phase I43 | 4–24 mg sc | 13 | single dose | – | – | – | – |
| Phase I44 | 0.5–12 mg iv | 24 | single dose | – | – | – | – |
| Phase II45,46 | 4 mg iv q2w | 41 | 3 mo | 4/7 (57.1%) | 86% | Not available | 15 (37%) |
| 8 mg iv q2w | 7/8 (87.5%) | 63% | Case reports48 | ||||
| 8 mg iv q4w | 7/13 (53.8%) | 92% | |||||
| 12 mg iv q4w | 8/13 (61, 5%) | 100% | |||||
| GOUT 1 and GOUT 249,50 | 8 mg iv q2w | 43 + 42 | 26 wk | 20/43 (46.5%) (P < 0.001)b and 16/42 (38.1%) (P < 0.001)b | mo 1–3: 77%; mo 4–6: 41% (P = 0.007)b | 21/52 (40.4%) | 1 (2%) |
| 8 mg iv q4w | 41 + 43 | 8/41 (19.5%) (P = 0.044)b and 21/43 (48.8%) (P < 0.001)b | mo 1–3: 81%; mo 4–6: 57% | 11/52 (21.2%) | 16 (19%) | ||
| Placebo | 20 + 23 | 0/20 (0%) and 0/23 (0%) | mo 1–3: 54%; mo 4–6: 67% | 2/29 (6.9%) | 17 (20%) |
a
Notes: Proportion of subjects maintaining a PUA concentration <6 mg/dL for at least 80% of the time during the treatment period (Phase II ) or during the months 3 and 6 (Phase II);
b
Pegloticase vs placebo.
Abbreviations: ADR, adverse drug reactions; PAU, plasma uric acid.