Table 2.
Objective Response and Long-Term Stable Disease per Independent Central Review (response population).
| Parameter | HGG n = 19 | DIPG n = 9 | Ependymoma n = 9 | Medulloblastoma n = 9 |
|---|---|---|---|---|
| Rate of objective response or long-term stable disease a | ||||
| n (%) | 2 (10.5) | 0 | 1 (11.1) | 0 |
| 95% CI | (1.3-33.1) | (0.0-33.6) | (0.3-48.2) | (0.0-33.6) |
| Objective response, n (%) | 1 (5.3) | 0 | 0 | 0 |
| Long-term stable disease, n (%) a | 1 (5.3) | 0 | 1 (11.1) | 0 |
| Best overall response, n (%) | ||||
| Complete response | 0 | 0 | 0 | 0 |
| Partial response | 1 (5.3) | 0 | 0 | 0 |
| Stable disease | 1 (5.3) | 0 | 3 (33.3) | 1 (11.1) |
| ≥ 3 cycles | 1 (5.3) | 0 | 3 (33.3) | 1 (11.1) |
| ≥ 6 cycles | 1 (5.3) | 0 | 1 (11.1) | 0 |
| Disease progression | 11 (57.9) | 6 (66.7) | 6 (66.7) | 5 (55.6) |
| Not evaluable b | 6 (31.6) | 3 (33.3) | 0 | 3 (33.3) |
DIPG, diffuse intrinsic pontine glioma; HGG, high-grade glioma.
a
Long-term stable disease was defined as stable disease maintained for ≥ 6 cycles (≥ 3 cycles for DIPG).
b
Patients who discontinued study treatment due to disease progression or relapse prior to disease assessment were considered not evaluable with regard to the primary endpoint.