A Phase II Multicenter Trial of Rucaparib and Nivolumab as Maintenance Therapy in Patients with Advanced Biliary Tract Cancer: BilT-02.
Arathi Mohan, Kent A Griffith, Laura W Goff et al.
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In 30 seconds
BilT-02 was a multicenter phase II trial of rucaparib plus nivolumab as maintenance therapy for patients with advanced biliary tract cancer whose disease had not progressed after 4 to 6 months of first-line platinum-based chemotherapy. Among 31 patients, the primary endpoint was not met: the 4-month progression-free survival rate was 54.8% (95% CI, 36-72.7), below the prespecified 63% null benchmark. Rates were 83.3% in patients with DNA damage response mutations and 100% in those with IDH1 mutations; these subgroup findings support further investigation, not established benefit.
Key findings
- The 4-month progression-free survival rate was 54.8% (95% CI, 36-72.7), and the trial did not meet its primary endpoint.
- Four-month progression-free survival was 83.3% in the DNA damage response cohort and 100% in the IDH1 cohort.
- Median progression-free survival and overall survival from study treatment were 4.6 months (95% CI, 3.7-6.2) and 15.9 months (95% CI, 9.8-24.1), respectively.
- Two patients (6.4%) had a partial response; common grade ≥3 treatment-related events included anemia (12.9%), neutropenia (9.7%), and elevated liver enzymes (12.9%).
Why it matters
Maintenance strategies after platinum-based chemotherapy are relevant in advanced biliary tract cancer, where this trial tested combined PARP inhibition and immune checkpoint inhibition. The overall primary endpoint was not achieved, but the reported subgroup progression-free survival rates in patients with DNA damage response or IDH1 mutations provide a rationale for further study of biomarker-defined populations.
What to keep in mind
The trial was a phase II study with 31 patients; the DNA damage response and IDH1 subgroups were small, and the abstract does not report a comparator arm.