This single-arm Phase II trial evaluated darolutamide (600 mg BID), exemestane (25 mg daily), and leuprolide acetate (7.5 mg IM monthly) in 17 patients with recurrent adult-type ovarian granulosa cell tumor (AGCT) who had previously progressed on an aromatase inhibitor; accrual ran January–April 2024.
Of 16 evaluable patients, ORR was 6.25% (1 partial response); 62.5% had stable disease and 31.25% had progressive disease. Median PFS was 8.5 months (95% CI: 3.1–12.0 months); median OS was not reached. No grade 4 or 5 adverse events were attributed to treatment. The trial did not advance to stage 2.
- Very small sample size (n=16 evaluable) limits generalizability. - Single-arm design with no comparator group makes it difficult to attribute PFS benefit to treatment. - Accrual was completed in just 3 months, raising questions about patient selection at participating sites.
This triplet hormonal regimen did not meet its prespecified ORR threshold in AI-pretreated recurrent AGCT and cannot be recommended as a standard approach. However, the disease control rate (62.5% stable disease) and 8.5-month median PFS may still inform future combination strategies for this rare tumor.
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