A Study of the Efficacy of ABT-199 in Subjects With Relapsed/Refractory or Previously Untreated Chronic Lymphocytic Leukemia With the 17p Deletion
Phase 2N = 158NCT01889186
- Population
- Adults with CLL with 17p deletion, relapsed or refractory (the full population also includes 5 previously untreated patients).
- Design
- Single-arm multicentre phase 2 study of venetoclax 400 mg daily after a ramp-up of 4 to 5 weeks, in 31 centres. The pivotal cohort was 107 patients (Lancet Oncology 2016); a safety expansion brought the full population to 158, including 5 previously untreated patients (JCO 2018). ClinicalTrials.gov lists 158 actual enrolments.
- Primary endpoint
- ORR
- Arms
- Venetoclax (experimental, n = 158): Venetoclax 400 mg daily after a weekly ramp-up (20, 50, 100, 200, 400 mg) until progression or discontinuation
- People
- Stilgenbauer S
Show the numbers
| Endpoint | Analysis | Venetoclax | Control | Effect |
|---|---|---|---|---|
| ORRPatients who received at least one dose of venetoclax (107) | primarymedian follow-up 12.1 monthsStilgenbauer 2016 | 79.4% (70.5–86.6%) | — | 85 of 107 patients; overall response assessed by an independent review committee; interquartile range of follow-up 10.1 to 14.2 months. |
| ORRAll 158 enrolled patients (5 previously untreated) | long term-updateStilgenbauer 2018 | 77% | — | Investigator-assessed; 122 of 158 patients, 20% with complete remission. In 16 patients with prior kinase inhibitors the response rate was 63% (10 of 16). |
| PFSAll 158 enrolled patients (5 previously untreated) | long term-updateStilgenbauer 2018 | 54% at 2 y (45–62%) | — | In the 16 patients with prior kinase inhibitors the 24-month estimate was 50% (95% CI 25 to 71). |
| Undetectable MRD rateAll 158 enrolled patients (intention-to-treat analysis as stated in the abstract)MRD below 1 CLL cell per 10,000 leukocytes (10^-4) in blood, measured by flow cytometry | long term-updateStilgenbauer 2018 | 30% | — | 48 of 158 patients. |
| Endpoint | Analysis | Venetoclax | Control | Effect |
|---|---|---|---|---|
| ORRPatients who received at least one dose of venetoclax (107) | primarymedian follow-up 12.1 monthsStilgenbauer 2016 | 79.4% (70.5–86.6%) | — | 85 of 107 patients; overall response assessed by an independent review committee; interquartile range of follow-up 10.1 to 14.2 months. |
| ORRAll 158 enrolled patients (5 previously untreated) | long term-updateStilgenbauer 2018 | 77% | — | Investigator-assessed; 122 of 158 patients, 20% with complete remission. In 16 patients with prior kinase inhibitors the response rate was 63% (10 of 16). |
| PFSAll 158 enrolled patients (5 previously untreated) | long term-updateStilgenbauer 2018 | 54% at 2 y (45–62%) | — | In the 16 patients with prior kinase inhibitors the 24-month estimate was 50% (95% CI 25 to 71). |
| Undetectable MRD rateAll 158 enrolled patients (intention-to-treat analysis as stated in the abstract)MRD below 1 CLL cell per 10,000 leukocytes (10^-4) in blood, measured by flow cytometry | long term-updateStilgenbauer 2018 | 30% | — | 48 of 158 patients. |
Appears in
Sources
Stilgenbauer S, Eichhorst B, Schetelig J, Coutre S, Seymour JF, Munir T, et al. Venetoclax in relapsed or refractory chronic lymphocytic leukaemia with 17p deletion: a multicentre, open-label, phase 2 study. Lancet Oncol. 2016;17(6):768-778.
Stilgenbauer S, Eichhorst B, Schetelig J, Hillmen P, Seymour JF, Coutre S, et al. Venetoclax for Patients With Chronic Lymphocytic Leukemia With 17p Deletion: Results From the Full Population of a Phase II Pivotal Trial. J Clin Oncol. 2018;36(19):1973-1980.