Granulosa Cell ovarian carcinoma is an infrequent subtype of neoplasia well differentiated from epithelial tumors. They account for 5% of all ovarian malignancies and, with an incidence of 0.4-1.2 cases per 100000 habitants, is considered as a rare disease. Though most cases are identified at initial stages and can be cured through surgical resection, distant recurrences have been documented even 10 years after resecting the primary tumor. At advanced stage it is a lethal disease. Unfortunately because of the low incidence of this disease randomized clinical trials are lacking. In fact current evidence for treatment is provided by case reports, retrospective studies and phase II clinical trials performed one decade ago. Orteronel, a novel, orally active, selective inhibitor of 17,20-lyase, is being developed as an endocrine therapy for relevant hormone-sensitive cancers such as prostate cancer and breast cancer. Orteronel is expected to suppress sex hormone levels in both circulation and relevant hormone-dependent malignant tissue. Since sex hormone overproduction has been demonstrated in granulosa cell ovarian tumors and seems to play a major role in this disease, this study will assess the efficacy or orteronel treating such tumors.
Name: Orteronel 300mg BIDType: Drug
Orteronel 300mg b.i.d.
Description: Clinical benefit is defined as the average of patients with radiological response (partial or complete) plus stable disease longer than 6 months by RECIST 1.1 criteriaMeasure: Clinical benefit at 6 months Time: 6 months
Description: Overall Response Rate according to RECIST 1.1 criteria.Measure: Overall Response Rate Time: Every 8 weeks, during 6 months
Description: Progression Free Survival defined as the time from the administration of the first dose of treatment to disease progression or death from any cause.Measure: Progression free survival Time: Every 8 weeks, during 6 months
Description: Overall Survival defined as the time from first dose of treatment to patient death from any causeMeasure: Overall Survival Time: Every 12 weeks, untill death
Description: Significant reduction of sex hormones production will be considered as at least a reduction to half the basal level confirmed in one determination one month apart.Measure: Reduction of sex hormones production. Time: Every 8 weeks, during 6 months
Description: Frequency of each adverse event per patientMeasure: Toxicity profile Time: Every 4 weeks, untill end of treatment (6 months estimated)
Single Group Assignment
There is one SNP
- Availability of sufficient biopsy material to confirm the malignant diagnosis of granulosa cell ovarian tumor by a centralized pathologist and to perform the determine the FOXL2 402C mutation → G (C134W). --- C134W ---