Adrenocortical carcinoma (ACC) is an uncommon kind of endocrine cancer with few available therapies. For a study, researchers sought to examine carbozantinib’s function in ACC treatment.

The phase II trial was launched by the investigator and is examining the effectiveness and safety of cabozantinib in individuals with advanced ACC. With the aid of The Response Evaluation Criteria in Solid Tumors (RECIST)v1.1, the effectiveness of the treatment was evaluated objectively. Progression-free survival (PFS) at 4 months (mo) (PFS4) was the major end objective. Median PFS, overall response rate (ORR), median overall survival (OS), pharmacokinetic studies, and safety were considered secondary objectives. In addition, studies using related tissues were carried out.

The trial was completed by recruiting 18 patients with advanced ACC, 8 of whom are female (44%). About 19.4 months were the average follow-up (range 2.9-45.6). PFS4 was 72% (13/18), median PFS was 7.2 months (95% CI: 3.3, 9.2 months), and PFS4 was 72%. Of the 18 patients, 16 underwent restaging with ORR, with the greatest response being a partial response in two patients, 12 patients with stable illness, and two patients with progressive disease; the disease control rate was 78% (95% CI, 52% to 94%). With a 95% CI of 3.3 to 9.2 months, the median PFS was 7.2 months, while the median OS was 23.9 months. A minimum of 13 patients (72.2%) experienced potentially treatment-related grade 3 or 4 adverse events. Cabozantinib’s median day 29 pre-dose level was 505mg/mL. (range 279-1,810).

The majority of ACC patients who use cabozantinib get persistent disease management with a reasonable safety profile.