Phase 3 Trial of Cabozantinib to Treat Advanced Neuroendocrine Tumors
Authors' conclusion
Cabozantinib, as compared with placebo, significantly improved progression-free survival in patients with previously treated, progressive advanced extrapancreatic or pancreatic neuroendocrine tumors. Adverse events were consistent with the known safety profile of cabozantinib.
Study context
- Population
- Patients with previously treated (PRRT or targeted therapy or both), progressive advanced extrapancreatic or pancreatic NETcohorts of 203 and 95
- Comparison
- Cabozantinib 60 mg daily vs placebo
- Primary endpoint
- PFS by blinded independent central review
Methodological detailsphase 3, placebo-controlled, two independent cohorts, randomized 2:1
- Phase 3
- Placebo-controlled
- Two independent cohorts
- Randomized 2:1
Primary result
As reported in the abstract
Cabozantinib
- Median PFS, extrapancreatic NET cohort
- 8.4 months
- Median PFS, pancreatic NET cohort
- 13.8 months
Placebo
- Median PFS, extrapancreatic NET cohort
- 3.9 months
- Median PFS, pancreatic NET cohort
- 4.4 months
Median PFS, extrapancreatic NET cohort
Cabozantinib8.4 months
Placebo3.9 months
Median PFS, pancreatic NET cohort
Cabozantinib13.8 months
Placebo4.4 months
Cabozantinib vs PlaceboEstimate (95% CI)P value
Extrapancreatic NET cohort (n = 203)
stratified HR for progression or death 0.38(0.25–0.59)
P < 0.001Pancreatic NET cohort (n = 95)
stratified HR 0.23(0.12–0.42)
P < 0.001Secondary outcomes
- Confirmed objective response — Placebo
- 0%
- Confirmed objective response: Cabozantinib (extrapancreatic NET) 5%
- Confirmed objective response: Cabozantinib (pancreatic NET) 19%
Safety
| Outcome | Cabozantinib | Placebo |
|---|---|---|
| Grade ≥3 adverse events | 62 to 65% | 23 to 27% |
Grade ≥3 adverse events
- Common treatment-related grade ≥3 AEs: hypertension, fatigue, diarrhea, thromboembolic events
Original publication
Phase 3 Trial of Cabozantinib to Treat Advanced Neuroendocrine Tumors. N Engl J Med. Published 16 September 2024. doi:10.
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