40 Participants Needed

Nirogacestat for Aggressive Fibromatosis

KT
Overseen ByKeila Torres, MD,PHD
Age: 18+
Sex: Any
Trial Phase: Phase 2
Sponsor: M.D. Anderson Cancer Center
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human trial
Breakthrough TherapyThis drug has been fast-tracked for approval by the FDA given its high promise

What You Need to Know Before You Apply

What is the purpose of this trial?

To learn about the safety and effects of an investigational drug called nirogacestat when given to participants with a desmoid tumor/aggressive fibromatosis

Will I have to stop taking my current medications?

The trial requires that participants stop their prior therapy for at least 28 days or 5 half-lives of the drug, whichever is longer, before starting the study treatment. However, the protocol does not specify if you need to stop all current medications, so it's best to discuss your specific situation with the study team.

How is the drug Nirogacestat different from other treatments for aggressive fibromatosis?

Nirogacestat is unique because it is a gamma-secretase inhibitor, which targets a specific pathway involved in tumor growth, and has shown promising results in treating desmoid tumors, a type of aggressive fibromatosis, especially in cases where traditional treatments like surgery and chemotherapy are not effective.12345

Who Is on the Research Team?

KT

Keila Torres, MD, PHD

Principal Investigator

M.D. Anderson Cancer Center

Are You a Good Fit for This Trial?

This trial is for adults with desmoid tumors/aggressive fibromatosis who have either not been treated or whose tumors grew after treatment. They must be able to swallow tablets, sign consent, and have a tumor that can be measured. Participants need proper liver, kidney, and bone marrow function and agree to use contraception. Excluded are pregnant/breastfeeding women, those with certain health conditions or treatments that could interfere with the study.

Inclusion Criteria

My organs and bone marrow are working well.
I have a known heart condition or symptoms.
I can understand and am willing to sign the consent form.
See 14 more

Exclusion Criteria

Known hypersensitivity to nirogacestat or any of its excipients
Patients with psychiatric illness/social situations limiting compliance with study requirements
I cannot have repeated tissue samples taken.
See 12 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive nirogacestat by mouth every day of each 28-day study cycle

12 months
Monthly visits for assessment

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

  • Nirogacestat
Trial Overview The trial is testing nirogacestat's safety and effects on patients with desmoid tumors. About 40 participants will receive this investigational drug to see how well it works against their tumors which have shown growth despite previous treatments or in those untreated.
How Is the Trial Designed?
1Treatment groups
Experimental Treatment
Group I: nirogacestatExperimental Treatment1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

M.D. Anderson Cancer Center

Lead Sponsor

Trials
3,107
Recruited
1,813,000+

SpringWorks Therapeutics, Inc.

Industry Sponsor

Trials
14
Recruited
900+

Published Research Related to This Trial

In a study of 90 children and adolescents with aggressive fibromatosis, systemic therapy (ST) was found to be effective, particularly when primary resection was not possible, leading to a 5-year overall survival rate of 100%.
The most common initial response to ST was stable disease at 3 months and partial response at 6 months, indicating that while ST can be beneficial, the 5-year event-free survival rate was only 44%, highlighting the need for ongoing treatment strategies.
Systemic therapy of aggressive fibromatosis in children and adolescents: Report of the Cooperative Weichteilsarkom Studiengruppe (CWS).Sparber-Sauer, M., Seitz, G., von Kalle, T., et al.[2019]
A three-year-old patient successfully underwent conservative surgery for aggressive fibromatosis of the mandible, remaining free of recurrence for nine years post-surgery.
The case suggests that conservative surgical approaches may be more beneficial than radical surgeries in children, as they minimize the impact on facial growth while effectively managing the tumor.
[Mandibular aggressive fibromatosis].Jeblaoui, Y., Bouguila, J., Haddad, S., et al.[2007]
Wider surgical resection in patients with aggressive fibromatosis led to a lower recurrence rate, highlighting the importance of surgical intervention in managing this condition.
Two-thirds of the tumors exhibited estrogen or progesterone receptors, and all tumors showed inappropriate expression of c-sis and PDGF, suggesting these factors may play a significant role in the growth and behavior of aggressive fibromatosis.
Aggressive fibromatosis.Alman, BA., Goldberg, MJ., Naber, SP., et al.[2004]

Citations

Systemic therapy of aggressive fibromatosis in children and adolescents: Report of the Cooperative Weichteilsarkom Studiengruppe (CWS). [2019]
[Mandibular aggressive fibromatosis]. [2007]
Aggressive fibromatosis. [2004]
Safety and efficacy of gamma-secretase inhibitor nirogacestat (PF-03084014) in desmoid tumor: Report of four pediatric/young adult cases. [2021]
Aggressive Fibromatosis, Clinicopathologic Findings of 25 Cases; A Single-Center Experience and Review of the Literature. [2022]
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