Nirogacestat in Ovarian Granulosa Cell Tumors

NCT ID: NCT05348356

Last Updated: 2025-12-15

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

53 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-08-30

Study Completion Date

2025-07-14

Brief Summary

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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.

Detailed Description

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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.

Conditions

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Ovarian Granulosa-Stromal Tumor Ovarian Granulosa Cell Tumor Ovarian Cancer

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Nirogacestat Open-Label

Nirogacestat 150 mg by mouth, twice daily

Nirogacestat oral tablet: Nirogacestat tablet

Group Type EXPERIMENTAL

Nirogacestat

Intervention Type DRUG

nirogacestat oral tablet

Interventions

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Nirogacestat

nirogacestat oral tablet

Intervention Type DRUG

Other Intervention Names

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PF-03084014

Eligibility Criteria

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Inclusion Criteria

* Has histologically confirmed recurrent adult-type granulosa cell tumor of the ovary prior to first dose of study treatment
* Have documented radiological evidence of relapse after at least one systemic therapy that is not amenable to surgery, or radiation and have measurable disease by RECIST v1.1 criteria
* Have adequate bone marrow, renal and hepatic function as defined by screening visit laboratory values

Exclusion Criteria

* Has signs of bowel obstruction requiring parenteral nutrition, malabsorption syndrome or preexisting gastrointestinal conditions that may impair absorption of nirogacestat
* Has had a major cardiac or thrombo-embolic event within 6 months of signing informed consent
* Has abnormal QT interval at Screening, or has congenital or acquired long QT syndrome or a history of additional risk factors for Torsades de Pointes
* Has current or chronic history of liver disease or known hepatic or biliary abnormalities
* Has received bevacizumab treatment or other monoclonal antibody therapy with targeted anti-angiogenic activity for OvGCT within 28 days (or 5 half-lives, whichever is shorter) prior to the first dose of study treatment;
* Has received treatment for OvGCT including but not limited to the following within 28 days (or 5 half-lives, whichever is longer) prior to the first dose of study treatment: hormonal therapy, chemotherapy, immunotherapy, targeted therapy or any investigational treatment
Minimum Eligible Age

18 Years

Eligible Sex

FEMALE

Accepts Healthy Volunteers

No

Sponsors

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SpringWorks Therapeutics, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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USC/Norris Comprehensive Cancer Center

Los Angeles, California, United States

Site Status

UCLA-JCCC Dept. of OBGYN - Women's Health Clinical Research Unit

Los Angeles, California, United States

Site Status

AdventHealth Orlando

Orlando, Florida, United States

Site Status

Orlando Health Cancer Institute

Orlando, Florida, United States

Site Status

H. Lee Moffitt Cancer Center and Research Institute

Tampa, Florida, United States

Site Status

Women's Cancer Care

Covington, Louisiana, United States

Site Status

Greater Baltimore Medical Center

Baltimore, Maryland, United States

Site Status

Dana-Farber Cancer Institute

Boston, Massachusetts, United States

Site Status

David C. Pratt Cancer Center

St Louis, Missouri, United States

Site Status

University of New Mexico Comprehensive Cancer Center

Albuquerque, New Mexico, United States

Site Status

Columbia University Medical Center

New York, New York, United States

Site Status

Memorial Sloan Kettering Cancer Institute

New York, New York, United States

Site Status

Women's Cancer Center at Kettering

Kettering, Ohio, United States

Site Status

OU Health Stephenson Cancer Center

Oklahoma City, Oklahoma, United States

Site Status

UW/Fred Hutch Cancer Center

Seattle, Washington, United States

Site Status

Froedtert and Medical College of Wisconsin

Milwaukee, Wisconsin, United States

Site Status

Cross Cancer Institute

Edmonton, Alberta, Canada

Site Status

Maria Sklodowska-Curie Bialystok Oncology Center

Bialystok, , Poland

Site Status

Jagiellonian Innovation Centre Clinical Research Centre

Krakow, , Poland

Site Status

University Teaching Hospital Poznan, Department of Oncological Gynaecology

Poznan, , Poland

Site Status

Maria Sklodowska-Curie National Institute of Oncology-National Research Institute, Clinic of Oncological Gynecology

Warsaw, , Poland

Site Status

Countries

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United States Canada Poland

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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NIR-OGT-201

Identifier Type: -

Identifier Source: org_study_id

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