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Phase 2 study of lenvatinib monotherapy as second-line treatment in unresectable biliary tract cancer: primary
Makoto Ueno1, Masafumi Ikeda2, Takashi Sasaki3
1Kanagawa Cancer Centre Hospital, Yokohama, Japan.
Background:
Biliary tract cancer (BTC) has a poor prognosis and lacks a standardized second-line therapy. Vascular endothelial growth factor (VEGF), fibroblast growth factor receptor (FGFR) 4, and platelet-derived growth factor receptor (PDGFR) are highly expressed in BTC. Therefore, lenvatinib (a known inhibitor of VEGF receptors 1-3, FGFRs 1-4, and PDGFR-α) was evaluated for second-line treatment of BTC.
Methods:
In this single-arm, multicenter, open-label, phase 2 study, patients with BTC received lenvatinib 24 mg orally once daily in 28-day cycles. The primary endpoint was objective response rate (ORR). Secondary endpoints included overall survival (OS), progression-free survival (PFS), PFS rate at 12 weeks, disease control rate, clinical benefit rate, safety and pharmacokinetic profiles.
Results:
Twenty-six Japanese patients were enrolled and treated; 3 had a confirmed partial response per investigator assessment and per independent imaging review (IIR); ORR was 11.5% (90% confidence interval [CI]: 3.2-27.2). Median PFS was 3.19 months (95% CI: 2.79-7.23) per investigator assessment and 1.64 months (95% CI: 1.41-3.19) per IIR. Median OS was 7.35 months (95% CI: 4.50-11.27). Grade ≥ 3 treatment-emergent adverse events (TEAEs) occurred in 21 patients (80.8%) and included hypertension (n = 10 [38.5%]), proteinuria (n = 3 [11.5%]), palmar-plantar erythrodysesthesia (n = 3 [11.5%]), decreased appetite (n = 3 [11.5%]), and anemia (n = 3 [11.5%]). Two deaths occurred due to TEAEs between treatment initiation and 30 days after last dose, but neither were considered treatment related.
Conclusions:
Lenvatinib demonstrated antitumor activity in BTC, with a tolerable safety profile, and should be further evaluated as potential second-line therapy for this difficult to treat population.
Trial Registration:
ClinicalTrials.gov NCT02579616 . Date of registration: October 19, 2015.
Insights
Lenvatinib showed antitumor activity in biliary tract cancer (BTC) patients as a second-line treatment. While demonstrating some efficacy, further evaluation is needed for this challenging cancer.
Area of Science:
- Oncology
- Medical Research
- Clinical Trials
Background:
- Biliary tract cancer (BTC) presents a poor prognosis with limited standardized second-line treatment options.
- High expression of vascular endothelial growth factor (VEGF), fibroblast growth factor receptor (FGFR) 4, and platelet-derived growth factor receptor (PDGFR) is observed in BTC.
- Lenvatinib, an inhibitor of VEGF, FGFR, and PDGFR, was investigated for its potential in second-line BTC therapy.
Purpose of the Study:
- To evaluate the efficacy and safety of lenvatinib as a second-line treatment for patients with biliary tract cancer.
- To determine the objective response rate (ORR) as the primary endpoint.
- To assess secondary endpoints including overall survival (OS) and progression-free survival (PFS).
Main Methods:
- A single-arm, multicenter, open-label, phase 2 study.
- Twenty-six Japanese patients with BTC received lenvatinib 24 mg orally once daily.
- Primary endpoint: objective response rate (ORR). Secondary endpoints: OS, PFS, safety, and pharmacokinetics.
Main Results:
- The objective response rate (ORR) was 11.5% (90% CI: 3.2-27.2).
- Median progression-free survival (PFS) was 3.19 months (investigator) and 1.64 months (independent review). Median overall survival (OS) was 7.35 months.
- Grade ≥3 treatment-emergent adverse events (TEAEs) occurred in 80.8% of patients, with hypertension being the most common.
Conclusions:
- Lenvatinib demonstrated antitumor activity in biliary tract cancer.
- The drug exhibited a tolerable safety profile in this patient population.
- Further evaluation of lenvatinib as a second-line therapy for BTC is warranted.
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