RecruitingPHASE4INTERVENTIONAL

Nirogacestat in Premenopausal Females With Desmoid Tumor/Aggressive Fibromatosis (DT/AF)

This study is for adult women, aged 18 to 40, who still have their period and have desmoid tumours that are growing. Desmoid tumours are rare, non-spreading growths that can still cause problems. The study is investigating a tablet called nirogacestat, which is already approved in the USA for this condition and works by slowing tumour growth. Researchers want to see how nirogacestat affects the ovaries, how well it treats the tumour, and if it causes any side effects. This study aims to help us better understand the safety and effectiveness of nirogacestat for women with desmoid tumours. It's important to remember that this medicine is approved in the USA, but may not be in the UK, and this study seeks to gather more information.

At a glance

Status

Recruiting

Phase

PHASE4

Sponsor

SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany

Enrolment target

Start

17 Sep 2025

Estimated completion

24 Apr 2031

Desmoid Tumor Aggressive Fibromatosis

What is this study about?

Desmoid tumours, sometimes called aggressive fibromatosis, are rare growths that develop in soft tissues like muscles and ligaments. While they are not cancerous in the way that they spread to other parts of the body, they can grow into surrounding tissues and cause a lot of pain and problems. These tumours can be quite serious for some people, even though they are considered 'benign' because they don't spread.

This study is looking at a new medicine called nirogacestat. This medicine is a tablet that you take by mouth, and it works by slowing down or stopping the growth of tumour cells. It has already been approved in the USA for adults with desmoid tumours that are getting worse and need treatment, but we need more information, especially for women in the UK.

The main aim of this study is to understand how nirogacestat might affect the ovaries of women who haven't yet reached menopause. Researchers also want to see how well it works to shrink or stop the growth of desmoid tumours, and to monitor any side effects people might experience. By taking part, you would be helping doctors learn more about this treatment and improve care for others with desmoid tumours in the future.

Key takeaways

The study is for premenopausal women (18-40) with growing desmoid tumours.
It tests nirogacestat tablets to see how they affect ovaries and tumour growth.
Nirogacestat is already approved in the USA for this condition.
Participation involves regular clinic visits, tests, and taking the study medicine.
You can withdraw from the study at any time without affecting your normal care.
This study helps doctors learn more about desmoid tumour treatment.

Who may be eligible?

To join this study, you need to be a woman between 18 and 40 years old who still has regular periods. You must have a desmoid tumour that has been confirmed by a doctor and is causing symptoms or growing. You also need to be generally well enough to take part in a study, as assessed by a doctor.

There are certain reasons why you might not be able to join. For example, if you have severe heart problems, certain recent serious health events like a heart attack or stroke, or other types of cancer within the last five years (unless it was a very common skin cancer or similar that has been fully treated). You also can't take part if you have problems absorbing medicines from your gut or if your liver isn't working properly.

To ensure your safety, you'll need to use effective non-hormonal contraception during the study and for a period afterwards. You also cannot be pregnant or breastfeeding. These rules help make sure the study gathers accurate information and that you are as safe as possible.

Quick self-check

Am I a woman aged between 18 and 40?
Do I still have regular periods (not gone through menopause)?
Have I been diagnosed with a desmoid tumour that is causing problems or growing?
Am I willing to use effective non-hormonal contraception during the study?
Do I have any serious heart conditions or other cancers in the past 5 years (excluding common treated skin cancers)?
Do I have any problems absorbing medicines or severe liver issues?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you decide to take part in this study, you would be taking the study medicine, nirogacestat, as a tablet by mouth. The study involves a series of visits to the clinic over time. At these visits, doctors and nurses will carry out various checks, including blood tests, physical examinations, and scans to monitor your desmoid tumour and your overall health. They will pay close attention to your ovarian function to see how the medicine might be affecting your periods and hormone levels. You'll also be asked to report any side effects you experience.

The total duration of your participation in the study, including follow-up, will be decided by the researchers. It's important to remember that throughout the study, you'll have regular contact with the study team who will support you and answer any questions. You are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Potential risks and benefits

Taking part in this study might offer a potential benefit by providing you with a treatment for your desmoid tumour that could slow its growth or reduce its size. However, it's possible that the treatment may not work for everyone, or it might not work as well as expected. All medicines, including nirogacestat, come with potential risks and side effects. Some common side effects in other studies of nirogacestat have included diarrhoea, feeling sick, and tiredness. Because this study is specifically looking at how the medicine affects ovaries, there's a risk of changes to your menstrual cycle or ovarian health. The study team will explain all known and potential side effects in detail before you decide to join. You have the right to withdraw from the study at any time, for any reason, without penalty or affecting your ongoing medical care.

Locations (21)

Cliniques Universitaires Saint-Luc (CUSL)
Brussels, Belgium· Recruiting
Universitätsklinikum Mannheim, Mannheim Cancer Center
Mannheim, Germany· Recruiting
Istituto Romagnolo per lo Studio dei Tumori "Dino Amadori" IRCCS IRST. S.r.l.
Meldola, Italy· Recruiting
Fondazione del Piemonte per l'Oncologia Istituto di Candiolo IRCCS
Candiolo, Italy· Recruiting
IRCCS Istituto Ortopedico Rizzoli
Bologna, Italy· Recruiting
IRCCS Azienda Ospedaliero-Universitaria S. Orsola
Bologna, Italy· Recruiting
Istituto Nazionale Tumori I.R.C.C.S- Fondazione G. Pascale
Naples, Italy· Recruiting
Fondazione Policlinico Unversitario Campus Bio-Medico di Roma
Roma, Italy· Recruiting
Nederlands Kanker Instituut - Antoni van Leeuwenhoek Ziekenhuis (NKI-AVL)
Amsterdam, Netherlands· Recruiting
LUMC
Leiden, Netherlands· Recruiting
Hospital de la Santa Creu i Sant Pau
Barcelona, Spain· Recruiting
Hospital General Universitario Gregorio Maranon
Madrid, Spain· Recruiting

+9 more sites — see the official record for the full list.

Common questions

What is a desmoid tumour?

A desmoid tumour is a rare growth that doesn't spread like cancer but can grow into surrounding tissues and cause problems.

What is nirogacestat?

Nirogacestat is a tablet medicine that aims to slow down or stop the growth of desmoid tumour cells.

Why is this study focusing on women's ovaries?

The study wants to understand if nirogacestat affects the ovaries and periods in women who haven't reached menopause yet.

Will I have to pay to be in the study?

No, all treatments, tests, and care directly related to the study are provided free of charge.

Can I leave the study if I change my mind?

Yes, you can leave the study at any time, for any reason, without it affecting your other medical care.

How to find out more

US Medical Information

eMediUSA@emdserono.com 888-275-7376 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.