ECEESPE2025 Poster Presentations Endocrine Related Cancer (76 abstracts)
Cabozantinib in advanced adrenocortical carcinoma – first preliminary results of the prospective phase II caboACC study
Miriam Reuter 1 , Marie-Elisabeth Goebeler 2 , 3 , Otilia Kimpel 1 , Lydia Kürzinger 1 , Felix Megerle 1 , Carmina Fuß 1 , Johanna Werner 1 , Cyrus Sayehli 3 , Horst-Dieter Hummel 2 , Laura-Sophie Landwehr 1 , Max Kurlbaum 1 , Nicole Reisch 4 , Oliver Scherf-Clavel 5 , Cornelia Fiessler 6 , 7 , Martin Fassnacht 2 , 8 & Matthias Kroiss 1 & 4
1University Hospital of Wuerzburg, Department of Internal Medicine I, Division of Endocrinology and Diabetes, Wuerzburg, Germany; 2University Hospital of Wuerzburg, Comprehensive Cancer Center Mainfranken, Interdisciplinary Clinical Trials Center with Early Clinical Trials Unit, Wuerzburg, Germany; 3University Hospital of Wuerzburg, Department of Internal Medicine II, Interdisciplinary Clinical Trials Center with Early Clinical Trials Unit, Wuerzburg, Germany; 4University Hospital of LMU Munich, Department of Internal Medicine IV, Munich, Germany; 5LMU Munich, Department of Clinical Pharmacy, Munich, Germany; 6University Hospital of Wuerzburg, Clinical Trial Center, Wuerzburg, Germany; 7University Hospital of Wuerzburg, Institute for medical Data Science, Wuerzburg, Germany; 8University Hospital of Wuerzburg, Comprehensive Cancer Center Mainfranken, Wuerzburg, Germany
JOINT2731
Background: Adrenocortical carcinoma (ACC) is a rare malignancy with a poor prognosis in advanced stages and limited therapeutic options. In a retrospective analysis, we previously observed clinical activity of the multityrosine kinase inhibitor cabozantinib (cabo; Kroiss, J. Clin. Endocrinol. Metab, 2020). A recently published phase II study conducted in parallel with this trial reported that 13 out of 18 patients with advanced ACC achieved progression-free survival at four months (Campbell, Lancet Oncol., 2024).
Objective: To prospectively evaluate the clinical activity of cabo in patients with unresectable/metastatic ACC after failure of standard-of-care therapy.
Study Design: The prospective, open-label, multicenter, single-arm, phase II study (NCT03612232) followed a two-stage accrual design. Patients received oral cabo at an initial dose of 60 mg/d. Tumor response was evaluated radiologically every 8 weeks. To minimize potential drug-drug interactions, patients with mitotane plasma concentration >5 mg/l were excluded.
Primary Outcome Measure: The proportion of patients with progression-free survival after four months of treatment.
Results: Of 45 patients screened, 37 were enrolled, with 36 undergoing at least one follow-up imaging. Two patients discontinued cabo due to treatment-related toxicity despite stable disease prior to primary endpoint assessment. Among 34 evaluable patients, 14 (41. 2%) met the primary endpoint, maintaining stable disease for at least four months (preliminary data). The median duration of treatment was 22 weeks. Two patients achieved partial remission, and four additional patients experienced disease control for more than ten months. No new safety aspects were detected.
Conclusion: This investigator-initiated study confirms the promising clinical activity of cabo in heavily pre-treated ACC patients in a patient population larger than both previous retrospective and prospective studies combined. The lower response rate compared to the smaller parallel study may be due to differences in disease characteristics, prior treatments, and the larger sample size. Cabo may have greater efficacy in earlier lines of treatment for advanced ACC or in combination regimens, such as with immunotherapy.
Outlook: Secondary endpoints, markers of response and the potential impact of prior treatment, including interaction with prior mitotane, are currently under investigation.
Funding: This study was sponsored by the University Hospital of Würzburg and funded by Ipsen Pharma. The study team was solely responsible for the study conduct and interpretation of the data.
Volume 110
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Endocrine Abstracts
ISSN 1470-3947 (print) | ISSN 1479-6848 (online)
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