Table 3.
Best Clinical Response by Dose-Level Cohort (N = 56)
| Cohort | Dose | No. of Patients | CR | PR |
|---|---|---|---|---|
| I | 1.25 mg/kg/d | 7 | 0 | 2 |
| II | 2.5 mg/kg/d | 9 | 1 | 3 |
| III | 5.0 mg/kg/d | 6 | 2 | 1 |
| IV | 8.3 mg/kg/d | 8 | 3 | 1 |
| CD-I | 8.3 mg/kg/d | 10 | 1 | 5 |
| V | 12.5 mg/kg/d | 7 | 0 | 4 |
| CD-II | 560 mg/d | 9 | 1 | 6 |
| Total | 56* | |||
| No. | 8 | 22 | ||
| % | 14 | 39 |
Abbreviations: CD, continuous dosing; CR, complete response; PR, partial response.
*
Six of 56 patients were not evaluable. This includes three patients who withdrew consent after 7, 15, and 35 days in the study, respectively; one patient with a serious adverse event of failure to thrive after 14 days in the study; and two patients with dose-limiting toxicities.