Phase II clinical trial of nirogacestat in patients with relapsed ovarian granulosa cell tumours

This Phase II clinical trial of a rare tumour achieved its enrolment target in under 1 year and completed primary analysis within 2 years. 87% (46 of 53 patients who received nirogacestat) had fresh or archival biopsies that were analysed by next-generation sequencing for mutational profiling. Of the 3 patients with activating NOTCH1 mutations, all achieved 6-month progression-free survival (PFS6); 8 other patients also achieved PFS6 but did not share a common mutation.

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Publication Details

Journal
Clinical and Translational Medicine
Published
2026-01-01
DOI
https://doi.org/10.1002/ctm2.70568
Primary Topic
Ovarian cancer diagnosis and treatment
Type
article
Field-Weighted Citation Impact
0.00
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article

Phase II clinical trial of nirogacestat in patients with relapsed ovarian granulosa cell tumours

Patricia S. Braly, P. Konstantinopoulos, Thomas Reid, R.M. Wenham et al.
Clinical and Translational Medicine
Ovarian cancer diagnosis and treatment
article

Phase II clinical trial of nirogacestat in patients with relapsed ovarian granulosa cell tumours

Patricia S. Braly, P. Konstantinopoulos, Thomas Reid, R.M. Wenham, Rachel N. Grisham, R. Holloway, Kathryn P. Pennington, Koji Matsuo, Ritu Salani, Jocelyn Lewis, Elizabeth Hopp, Shinta Cheng, Mariusz Bidziński, Paul Celano, Mark Johnson, Robert DuBose, Sarah Ahn, Shelly Seward, Lauren Dockery, Carmelita Alvero
article en

Abstract

This Phase II clinical trial of a rare tumour achieved its enrolment target in under 1 year and completed primary analysis within 2 years. 87% (46 of 53 patients who received nirogacestat) had fresh or archival biopsies that were analysed by next-generation sequencing for mutational profiling. Of the 3 patients with activating NOTCH1 mutations, all achieved 6-month progression-free survival (PFS6); 8 other patients also achieved PFS6 but did not share a common mutation.

Clinical and Translational MedicineVol. 16(1)
University of Southern California (US), Greater Baltimore Medical Center (US), Research Triangle Park Foundation (US), Froedtert Hospital (US), Memorial Sloan Kettering Cancer Center (US), Orlando Health (US), AdventHealth Orlando (US), North Carolina Biotechnology Center (US), UCLA Health (US), AdventHealth East Orlando (US), University of Washington Medical Center (US), Moffitt Cancer Center (US), Florida Hospital Cancer Institute (US), Spring Bank Pharmaceuticals (United States) (US), Alaska Women's Cancer Care (US), Dana-Farber Cancer Institute (US), National Institute of Oncology (HU), The Maria Sklodowska-Curie National Research Institute of Oncology (PL), University of Oklahoma Health Sciences Center (US)
Good health and well-being
Openalex Percentile: Top 92%
Ovarian cancer diagnosis and treatment
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