Table 2.
Tumor-associated assessments based on investigator and central radiology reviews (Primary efficacy populations)
| Investigator radiology review | Central radiology review | |||||||||
|---|---|---|---|---|---|---|---|---|---|---|
| BTH1677 (N = 46) | Control (N = 26) | BTH1677 (N = 41) | Control (N = 26) | |||||||
| n (%) | 95% CI | n (%) | 95% CI | P value | n (%) | 95% CI | n (%) | 95% CI | P value | |
| Objective response rate | 22 (47.8) | (32.9, 63.1) | 6 (23.1) | (9.0, 43.6) | 0.0468 | 15 (36.6) | (22.1, 53.1) | 6 (23.1) | (9.0, 43.6) | 0.2895 |
| Disease control rate | 36 (78.3) | (63.6, 89.1) | 20 (76.9) | (56.4, 91.0) | 1.000 | 35 (85.4) | (70.8, 94.4) | 21 (80.8) | (60.6, 93.4) | 0.7385 |
| Best observed response | ||||||||||
| Complete response | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | ||
| Partial response | 22 (47.8) | (32.9, 63.1) | 6 (23.1) | (9.0, 43.6) | 15 (36.6) | (22.1, 53.1) | 6 (23.1) | (9.0, 43.6) | ||
| Stable disease | 14 (30.4) | (17.7, 45.8) | 14 (53.8) | (33.4, 73.4) | 20 (48.8) | (32.9, 64.9) | 15 (57.7) | (36.9, 76.6) | ||
| Progressive disease | 10 (21.7) | (10.9, 36.4) | 6 (23.1) | (9.0, 43.6) | 6 (14.6) | (5.6, 29.2) | 5 (19.2) | (6.6, 39.4) | ||
| Duration of objective tumor response | BTH1677 (N = 22) | Control (N = 6) | HR (95% CI)a/ Log-rank P value | BTH1677 (N = 15) | Control (N = 6) | HR (95% CI)a/ Log-rank P value | ||||
| Number of patients (%) with objective response (CR + PR) | 22 (100.0) | 6 (100.0) | 15 (100.0) | 6 (100.0) | ||||||
| Number of patients (%) with known duration (uncensored) | 18 (81.8) | 5 (83.3) | 8 (53.3) | 3 (50.0) | ||||||
| Number of patients (%) with unknown duration (censored) | 4 (18.2) | 1 (16.7) | 7 (46.7) | 3 (50.0) | ||||||
| Duration of objective response (months)b | ||||||||||
| Median (95% CI) | 3.8 (2.8, 4.2) | 4.7 (1.4, NE) | 1.563 (0.574, 4.253) 0.3670 | 4.4 (2.8, 6.5) | 4.1 (1.4, 5.4) | 0.594 (0.148, 2.382) 0.4456 | ||||
| Time-to-Progression | BTH1677 (N = 46) | Control (N = 26) | HR (95% CI)a Log-rank P value | BTH1677 (N = 41) | Control (N = 26) | HR (95% CI)a Log-rank P value | ||||
| Number of patients with progressive disease, n (%) | 38 (82.6) | 21 (80.8) | 20 (48.8) | 14 (53.8) | ||||||
| Number of patients censored, n (%) | 8 (17.4) | 5 (19.2) | 21 (51.2) | 12 (46.2) | ||||||
| Time-to-progression (months)c | ||||||||||
| Median (95% CI) | 4.3 (3.6, 5.6) | 4.4 (3.2, 5.9) | 1.10 (0.65, 1.90) 0.7305 | 6.4 (4.3, 8.3) | 6.0 (4.3, 7.1) | 0.83 (0.42, 1.69) 0.6044 | ||||
Tumor response data reported as the number (n) and percent (%) of patients and the 95% exact binomial confidence interval
CI confidence interval, CR complete response, PR partial response, HR hazard ratio, NE not estimable
aHazard ratio and 95% CI based on Cox proportional hazards model with treatment as a factor
bDuration of objective response (months) based on Kaplan-Meier estimates
cTime-to-progression (TTP) (months) based on Kaplan-Meier estimates. TTP was defined as the time from the date of randomization to the first date of documented progressive disease. Patients who died on study from other causes (not related to study disease) and patients who were lost to follow up or who were alive without documented progressive disease as of the data cut-off date for the analysis were censored at the last tumor assessment date