An Open-label, Phase 4 Study of Nirogacestat in Adult Premenopausal Females with DT AF
Research type
Research Study
Full title
A SINGLE-ARM, OPEN-LABEL PHASE 4 STUDY OF NIROGACESTAT IN ADULT PREMENOPAUSAL FEMALES WITH DESMOID TUMORS/AGGRESSIVE FIBROMATOSIS (DT/AF)
IRAS ID
1011677
Contact name
Cindy Garrison
Contact email
Sponsor organisation
SpringWorks Therapeutics, Inc.
Research summary
Desmoids tumours, also referred to as aggressive fibromatosis (or DT/AF), are rare, locally invasive, slow growing soft tissue tumours. DT/AF develop more often in young adults with a peak age of around 30 years. They are more common in women than men.
This research study is being conducted to study how nirogacestat may affect the ovarian function of adult premenopausal women with progressing DT/AF.
Nirogacestat is a tumour inhibitor that works by slowing or stopping the growth of tumour cells. Nirogacestat is a tablet taken by mouth and has been approved in the USA for adult patients with progressing desmoid tumours who require systemic treatment. In the UK, nirogacestat is considered an investigational drug. This means that it has not yet been approved by the Medicines and Healthcare products Regulatory Agency (MHRA). However, it can be used in this type of research study (known as a clinical trial).
Approximately 50 women, aged 18 to 40 years old, will take part in this research study from medical centres in the UK, and additional countries in the EU. Participants are expected to be enrolled in the study for up to 4 years. This study will consist of three parts; a Screening period (that will take approximately 28 days), a Treatment period (up to 2 years of nirogacestat treatment) and a Clinical Follow-up period (up to 2 years following the last dose of nirogacestat). This is an open-label study, and all women taking part in the study will take 150 mg of nirogacestat twice a day during the Treatment period.
As part of this study, the participants will attend visits at their medical centre. During these visits, participants will be asked to describe any changes in their health. They will also complete clinical assessments such as CT/MRI scans, blood tests and physical examinations to continually monitor their health.
SpringWorks Therapeutics is the “Sponsor” of this research study and is responsible for the development and oversight of the study.REC name
Wales REC 3
REC reference
25/WA/0141
Date of REC Opinion
9 Jun 2025
REC opinion
Further Information Favourable Opinion