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Can a pill stop desmoid tumors from growing?

NCT ID NCT03785964

Disease control Sponsor: SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Aug 20, 2026 · Last updated Aug 21, 2026 · Updated 1 time

Summary

This phase 3 trial investigates whether nirogacestat, an oral drug, can slow the growth of desmoid tumors (also called aggressive fibromatosis) in adults whose tumors are progressing. Half the participants receive nirogacestat, while the other half receive a placebo, in a double-blind design. The study measures how long people live without their disease worsening, and whether tumors shrink. If it works, nirogacestat could offer a less invasive option for managing this rare soft-tissue condition.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
nirogacestat, an oral tablet being tested against a placebo
What this could lead to
If successful, nirogacestat could become a non-surgical treatment option to control desmoid tumors, potentially delaying progression and reducing the need for aggressive interventions.
What could go wrong
This is a phase 3 trial, but results are not yet known. The drug may not outperform placebo, and side effects could limit its use. Desmoid tumors can also behave unpredictably, making outcomes hard to generalize.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

142 people

The number who actually took part.

Started

Apr 2019

Finished

Oct 2024

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Double-Blind Key Inclusion Criteria: * Participant has histologically confirmed DT/AF (by local pathologist prior to informed consent) that has progressed by ≥ 20% as measured by RECIST v1.1 within 12 months of the screening visit scan. * Participant has: 1. Treatment naïve, measurably progressing DT/AF that is deemed not amenable to surgery without the risk of significant morbidity; OR 2. Recurrent, measurably progressing DT/AF following at least one line of therapy; OR 3. Refractory, measurably progressing DT/AF following at least one line of therapy. * Participant has a DT/AF tumor where continued progressive disease will not result in immediate significant risk to the participant. * Participant agrees to provide archival or new tumor tissue for re-confirmation of disease. * If participant is currently being treated with any therapy for the treatment of DT/AF, this must be completed at least 28 days (or 5 half-lives, whichever is longer) prior to first dose of study treatment. All toxicities from prior therapy must be resolved to ≤Grade 1 or clinical baseline. * Participants who are receiving chronic nonsteroidal anti-inflammatory drugs (NSAIDs) as treatment for conditions other than DT/AF must be receiving them prior to the documented DT/AF progressive disease (inclusion criteria 2) and on a stable dose for at least 28 days prior to first dose of study treatment. * Participant has an Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2 at screening. * Participant has adequate organ and bone marrow function. Double-Blind Key Exclusion Criteria: * Participant has known malabsorption syndrome or preexisting gastrointestinal conditions that may impair absorption of nirogacestat. * Participant has experienced any of the following within 6 months of signing informed consent: clinically significant cardiac disease (New York Heart Association Class III or IV), myocardial infarction, severe/unstable angina, coronary/peripheral artery bypass graft, symptomatic congestive heart failure, cerebrovascular accident, transient ischemic attack, or symptomatic pulmonary embolism. * Participant has an abnormal QT interval at screening. * Participant is using concomitant medications that are known to prolong the QT/QTcF interval including Class Ia and Class III antiarrhythmics at the time of informed consent. Non-antiarrhythmic medications which may prolong the QT/QTcF interval are allowed provided the participant does not have additional risk factors for Torsades de Pointes (TdP) * Participant has congenital long QT syndrome. * Participant has a history of additional risk factors for Torsades de Pointes (TdP) (e.g., heart failure, hypokalemia, family history of Long QT Syndrome). * Participant has had lymphoma, leukemia, or any malignancy within the past 5 years at the time of informed consent, except for any locally recurring cancer that has been treated curatively (e.g., resected basal or squamous cell skin cancer, superficial bladder cancer, carcinoma in situ of the cervix or breast), with no evidence of metastatic disease for 3 years at the time of informed consent. * Participant has current or chronic history of liver disease or known hepatic or biliary abnormalities (except for Gilbert's syndrome or asymptomatic gallstones). * Participant previously received or is currently receiving therapy with GS inhibitors or anti-Notch antibody therapy. * Participant is currently using any treatment for DT/AF including tyrosine kinase inhibitors (TKIs), NSAIDs (chronic daily use) or any investigational treatment 28 days (or 5 half-lives, whichever is longer) prior to the first dose of study treatment. OR Participant has started any treatment for DT/AF after the documented DT/AF progressive disease. * Participant is currently using or anticipates using food or drugs that are known strong/moderate cytochrome P450 3A4 (CYP3A4) inhibitors, or strong CYP3A inducers within 14 days prior to the first dose of study treatment. * Participant has a positive human immunodeficiency virus antibody test. * Participant has presence of Hepatitis B surface antigen at screening. * Participant has a positive Hepatitis C antibody or Hepatitis C ribonucleic acid (RNA) test result at screening or within 3 months prior to starting study treatment. * Participant is unable to tolerate MRI or for whom MRI is contraindicated. * Participant with active or chronic infection at the time of informed consent and during the screening period. * Participant has experienced other severe acute or chronic medical or psychiatric conditions within 1 year of signing informed consent. * Participant is unable to comply with study related procedures (including, but not limited to, the completion of electronic patient report outcomes (ePROs), or the ePRO questionnaires are not available in the participant's preferred language). Open-Label Key Inclusion * Participant is enrolled in the double-blind phase when the estimated number of PFS events have been observed and the primary PFS analysis has been completed; OR * Participant is randomized to receive placebo in the double-blind phase and Central Imaging Review determines that the participant has radiographic progressive disease; OR * Participant is randomized to receive nirogacestat in the double-blind phase and Central Imaging Review determines that the participant has radiographic progressive disease but the participant is deriving clinical benefit without significant toxicity (as determined by the investigator). * Participant has adequate organ and bone marrow function Open-Label Key Exclusion * Participant requires surgery to prevent organ dysfunction. * Participant has prematurely discontinued from the double-blind phase for any reason other than radiographic progressive disease (as determined by Central Imaging Review). * Participant developed a concurrent illness/condition that, in the opinion of the investigator, would represent a risk to overall health if they enroll in this study. * Participant has initiated a new treatment for DT/AF after the Central Imaging Review determines that a participant has radiographic progressive disease.

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Conditions

The condition(s) this trial relates to.

desmoid tumor

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Aggressive fibromatosis Desmoid tumor

Contacts and locations

Locations

  • Abramson Cancer Center at Pennsylvania Hospital

    Philadelphia, Pennsylvania, 19106, United States

  • Arkansas Children's Hospital

    Little Rock, Arkansas, 72202, United States

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229, United States

  • Cliniques Universitaires Saint-Luc, Institut Roi Albert II

    Brussels, 1200, Belgium

  • Columbia University Medical Center

    New York, New York, 10032, United States

  • DUMC/Duke Cancer Center

    Durham, North Carolina, 27710, United States

  • Dana-Farber Cancer Institute (DFCI)

    Boston, Massachusetts, 02215, United States

  • Department of Oncology, University College of London Hospital

    London, NW12PG, United Kingdom

  • Fondazione IRCCS Instituto Nazionale dei Tumori di Milano

    Milan, 20133, Italy

  • Fox Chase Cancer Center

    Philadelphia, Pennsylvania, 19111, United States

  • Froedtert Hospital & the Medical College of Wisconsin

    Milwaukee, Wisconsin, 53226, United States

  • Helios Klinikum Berlin-Buch

    Berlin, 13125, Germany

  • Henry-Joyce Cancer Clinic

    Nashville, Tennessee, 37232, United States

  • IRCCS Istituto Ortopedico Rizzoli

    Bologna, 40136, Italy

  • Institut Jules Bordet-Medical Oncology

    Brussels, 1000, Belgium

  • Istituto di Candiolo IRCCS Oncologia Medica

    Candiolo, 10060, Italy

  • James Cancer Hospital and Solove Research Institute at The Ohio State University Comprehensive Cancer Center

    Columbus, Ohio, 43210, United States

  • Johns Hopkins Hospital

    Baltimore, Maryland, 21287, United States

  • Leiden University Medical Center (LUMC)

    Leiden, 23333, Netherlands

  • Massachusetts General Hosptial (MGH)

    Boston, Massachusetts, 02114, United States

  • Mayo Clinic Florida

    Jacksonville, Florida, 32224, United States

  • Mayo Clinic Rochester

    Rochester, Minnesota, 55905, United States

  • McGill University Health Centre

    Montreal, Quebec, H4A3JI, Canada

  • Memorial Sloan Kettering Cancer Center

    New York, New York, 10065, United States

  • Northwell Health

    Lake Success, New York, 11042, United States

  • Northwestern Memorial Hospital

    Chicago, Illinois, 60611, United States

  • Oregon Health & Science University-Center for Health & Healing

    Portland, Oregon, 97201, United States

  • Policlinico Unvrsitario Campus Bio-Medico

    Roma, 00128, Italy

  • Princess Margaret Cancer Centre

    Toronto, Ontario, M5G2M9, Canada

  • Radboud University Medical Centre

    Nijmegen, Gelderland, 6525GA, Netherlands

  • Ronald Regan UCLA Medical Center

    Los Angeles, California, 90095, United States

  • Sarcoma Oncology Research Center

    Santa Monica, California, 90403, United States

  • Seattle Cancer Care Alliance

    Seattle, Washington, 98109, United States

  • Smilow Cancer Hospital at Yale-New Haven

    New Haven, Connecticut, 06510, United States

  • Stanford Cancer Center

    Palo Alto, California, 94304, United States

  • Sylvester Comprehensive Cancer Center

    Miami, Florida, 33136, United States

  • The Netherlands Cancer Institute

    Amsterdam, 1066, Netherlands

  • The Royal Marsden NHS Foundation Trust

    London, SW36JJ, United Kingdom

  • The University of Texas MD Anderson Cancer Center

    Houston, Texas, 77030, United States

  • UCSF Mission Bay

    San Francisco, California, 94158, United States

  • UPMC Hillman Cancer Center

    Pittsburgh, Pennsylvania, 15232, United States

  • USC/Norris Comprehensive Cancer Center

    Los Angeles, California, 90033, United States

  • UT Southwestern Medical Center

    Dallas, Texas, 75390, United States

  • UZ Gent

    Ghent, 9000, Belgium

  • UZ Leuven

    Leuven, 3000, Belgium

  • Universitaetsklinikum Hamburg-Eppendorf

    Hamburg, 20246, Germany

  • University of Colorado Hospital-Anschutz Cancer Pavilion (ACP)

    Aurora, Colorado, 80045, United States

  • University of Michigan

    Ann Arbor, Michigan, 48109, United States

  • University of Wisconsin Clinical Science Center

    Madison, Wisconsin, 53792, United States

  • Universitätsmedizin Mannheim

    Mannheim, D-68167, Germany

  • Washington Cancer Institute at MedStar Washington Hospital Center

    Washington D.C., District of Columbia, 20010, United States

  • Washington University School of Medicine

    St Louis, Missouri, 63110, United States

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