Nirogacestat for Adults With Desmoid Tumor/Aggressive Fibromatosis


Trial name: Nirogacestat for Adults With Desmoid Tumor/Aggressive Fibromatosis
Agents: Nirogacestat vs. placebo
Phase III
Status  Closed, active
Sponsor SpringWorks Therapeutics, Inc.


This randomized, double-blind, placebo-controlled, phase 3 trial evaluates nirogacestat versus placebo in the treatment of adult patients with progressing desmoid tumors/aggressive fibromatosis. Further information:



WHO is the trial for?

  • Patients with measurably progressing DT/AF within the last 12 months that is not amenable to surgical resection or radiation therapy
  • Overall health status of the patient has to be good (ECOG performance status 0-2)

WHAT is the key question that this trial is attempting to answer?

This study assesses the efficacy, safety and tolerability as well as quality of life (by patient-reported outcomes, PRO) of nirogacestat in patients with a progressing desmoid tumor / aggressive fibromatosis.

WHY patients might want to participate?

This clinical trial offers patients an opportunity to access a new therapy strategy. This trial will further support the research in desmoid tumors/aggressive fibromatosis and potentially help other patients with this disease.

The trial might or might not have benefit in your individual case.

More about the importance and benefits of joining clinical trials: Please click here.

WHEN will the trial be open?

 The study is closed, but active

WHERE is the trial available?

The trial is being conducted in several study centers in the US, Belgium, Canada and the UK. Further study sites are planned for Germany, the Netherlands and Italy. For further information please check here.

Study contact:

Nicole H Leedom
Phone: +1-984-204-8065

STUDY DESIGN: What does the study look like?

There are 2 study arms (groups): all participating patients will be randomly divided (by a computer) in two groups. Patients in one group will be assigned to nirogacestat, the other group will receive placebo.

Nirogacestat will be given at a dosage of 150 mg, taken as a tablet (orally) twice daily.

.SPAEN Study Registry nirogacestat phase III

HOW do I get more information?

Patient organisations supporting desmoid (or sarcoma) patients in your country may offer additional information about the trial, current recruitment status, and key contacts. Click here for a list of patient organisations serving desmoid, GIST and/or sarcoma patients. If there are no such organisation in your country, please email us for more information:

SHARE your experience

You want to share your experience on this trial?
Send us an e-mail to:
Note that your experience would be helpful for other patients and patient organisations.

RESULTS of the study

No results are available at this time. Future results will be linked here.


Disclaimer: This is a patient-friendly summary of the clinical trial which has been provided for informational purposes only. Patients should consult their physician about any clinical trial opportunity.


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