This phase 2 clinical trial will study the effectiveness of nirogacestat in ovarian granulosa cell tumors (OvGCTs). Nirogacestat is a gamma secretase inhibitor (GSI) which is hypothesized to decrease the growth and activity of ovarian granulosa tumors.
Ovarian granulosa cell tumors (OvGCTs) represent 5-7% of all ovarian cancers (\~1.5 to 2k newly diagnosed patients/year in the United States) and are the most common subtype of ovarian sex cord tumors (70%). Treatment of granulosa cell tumors with nirogacestat is expected to inhibit Notch-induced granulosa cell proliferation. This is a multi-center, single-arm, Phase 2 open label treatment study to determine the efficacy, safety, tolerability, and pharmacokinetics of nirogacestat in adult participants with relapsed/refractory OvGCT. The participants will continue treatment until disease progression as determined by Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 (unless the participants meet criteria for continued treatment) or unacceptable toxicity.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
53
nirogacestat oral tablet
USC/Norris Comprehensive Cancer Center
Los Angeles, California, United States
Objective Response Rate (ORR)
The proportion of participants with complete response (CR) + partial response (PR) assessed using RECIST v1.1 criteria.
Time frame: 2.5 years
Progression Free Survival at 6 months (PFS-6)
The number of participants without progression according to RECIST v1.1 or death at 6 months.
Time frame: First day of cycle 7 (approximately 6 months after the first dose of study treatment). Each cycle is 28 days.
Overall Survival
The number of participants who have not died of any cause.
Time frame: 2 years after first dose of study treatment
Participant reported ovarian cancer symptoms
Change from baseline as measured by Functional Assessment of Cancer Therapy - Ovarian Symptom Index (FOSI).
Time frame: At each study visit until study completion (estimated to be an average of 2.5 years)
Duration of Response
The time from response (CR + PR using RECIST v.1.) to disease progression and/or death
Time frame: First day of every other cycle (each cycle is 28 days) for the first year, and then every 3 cycles thereafter until study completion (estimated to be an average of 2.5 years).
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UCLA-JCCC Dept. of OBGYN - Women's Health Clinical Research Unit
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...and 11 more locations