Table 6.
Not-approved use of sorafenib, lenvatinib, vandetanib and cabozantinib.
| Drug | Study design / ClinicalTrial.gov ID | Enrolled patients | Treatment arm(s) | Results | Reference |
|---|---|---|---|---|---|
| Sorafenib | Phase II, open label |
Hereditary or sporadic MTC: 16 | sorafenib 400 mg orally twice daily | PR: 6.3% SD: 87.6% mPFS: 17.9 months |
(81) |
| Lenvatinib | Phase II, multicenter, open-label, single-arm [NCT00784303] |
MTC: 59 | lenvatinib 24 mg orally once daily | ORR: 36% DCR: 80% SD: 44% mPFS: 9 months |
(82) |
| Vandetanib | Phase II, randomized, Double-Blind, Placebo-Controlled [NCT00537095] |
DTC (advanced or RAI-R): 145 | vandetanib 300 mg per day (n=72) vs placebo (n=73) | mPFS: 11.1 (vandetanib) vs 5.9 (placebo) months | (83) |
| Phase III, randomized, Double-Blind, Placebo-Controlled, Multi-Centre [NCT01876784] |
DTC (advanced or RAI-R): 238 | vandetanib 300 mg once daily vs placebo (119 patients for each group) | N/A Estimated end date 12/2022 |
||
| Cabozantinib | Phase II, single arm, open label [NCT02041260] |
RAI-R DTC: 35 | cabozantinib 60 mg orally once a day | PR: 54% SD: 43% durable SD (≥6 months): 26% mPFS not reached |
(84) |
DCR, disease control rate; DTC, differentiated thyroid cancer; MTC, medullary thyroid cancer; N/A, not reported; ORR, objective response rate; PFS, progression-free survival; PR, partial response; RAI-R, resistant to 131I treatment; SD, stable disease.