A Phase I/II Trial of Sapanisertib in Advanced Anaplastic and Radioiodine Refractory Differentiated Thyroid Carcinoma
Authors' conclusion
Sapanisertib monotherapy did not meet the primary endpoint of this trial (proportion progression-free at 4 months) in ATC and did not show clinically meaningful activity. Clinical trials with alternative therapeutic strategies are needed.
Study context
- Population
- Patients with recurrent/metastatic anaplastic thyroid carcinoma (ATC) and radioiodine refractory (RAIR) differentiated thyroid carcinoma (DTC) refractory to multikinase inhibitors, treated with sapanisertib (oral mTORC1/2 inhibitor)40 received 5 mg daily
- Participants
- 46 patients
- Exposure
- ATC vs DTC
- ATCn = 20
- DTCn = 26
- Outcome
- Proportion of patients with ATC without disease progression
- Follow-up
- 4 months
Methodological detailsmulticenter, safety run-in phase I, nonrandomized phase II, exploratory RAIR DTC cohort
- Multicenter
- Safety run-in phase I
- Nonrandomized phase II
- Exploratory RAIR DTC cohort
Main finding
As reported in the abstract
Progression-free at 4 months (ATC)
ATC
Eleven percent
2 of 20
Observational data: associations, not effects.
Secondary outcomes
Median PFS
- Stable disease as best response (ATC)
- 22.2% · 4
- Response (RAIR DTC)
- 4.5% · 1
PI3K/AKT/mTOR genomic alterations
- Not associated with response or PFS
Safety
Grade 3 treatment-related AEs (phase II dose)
- Grade 3 treatment-related AEs
- 30%
- Most common: anorexia, nausea, diarrhea, fatigue, skin rash, hyperglycemia
Original publication
A Phase I/II Trial of Sapanisertib in Advanced Anaplastic and Radioiodine Refractory Differentiated Thyroid Carcinoma. J Clin Endocrinol Metab. Published 29 June 2024. doi:10.
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