What side effects are associated with this type of intervention?

Nirogacestat, an enzyme inhibitor being studied for the treatment of desmoid tumors, may cause side effects such as gastrointestinal issues, fatigue, and potential liver enzyme abnormalities. Enzyme inhibitors in general can also lead to skin rashes, hypertension, and hematologic abnormalities. Patients should discuss these potential side effects with their healthcare provider to manage and mitigate risks effectively.

What prior FDA approvals exist?

As of now, there are no specific FDA-approved drugs exclusively for the treatment of Desmoid Tumors. However, treatments that involve enzyme inhibition, similar to the investigational drug Nirogacestat, are being explored. Nirogacestat works by inhibiting gamma-secretase, an enzyme involved in cell growth pathways. Other treatments for Desmoid Tumors have included non-specific chemotherapy agents, nonsteroidal anti-inflammatory drugs (NSAIDs), and hormone therapy, but these do not specifically target enzyme inhibition like Nirogacestat.

What other types of research exist?

Promising alternatives to Nirogacestat for Desmoid Tumor patients include Pazopanib, a tyrosine kinase inhibitor, and Sorafenib, another kinase inhibitor. Both have shown efficacy in treating desmoid tumors by inhibiting pathways involved in tumor growth and angiogenesis.

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Gamma Secretase Inhibitor

Nirogacestat for Desmoid Tumors

Phase 2

Waitlist Available

Led By Fariba Navid

Research Sponsored by Children's Oncology Group

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be younger than 65 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 2 years

Awards & highlights

No Placebo-Only Group

Summary

This trial studies how well nirogacestat works in children and adolescents with desmoid tumors that have not responded to other treatments and cannot be surgically removed. Nirogacestat is a medication taken by mouth that stops tumor growth by blocking necessary enzymes.

Who is the study for?

This trial is for patients under 18 with aggressive desmoid tumors that can't be surgically removed and have grown despite previous treatment. Participants must not use NSAIDs for tumor treatment, avoid certain drugs affecting liver enzymes, and meet specific health criteria including organ function tests.

What is being tested?

The study is testing Nirogacestat's effectiveness on children with inoperable desmoid tumors. It involves monitoring the drug's impact through various assessments like echocardiography, MRI, CT scans, X-rays, biospecimen collection, questionnaires and quality-of-life evaluations.

What are the potential side effects?

Potential side effects of Nirogacestat may include issues related to blocking enzymes needed for cell growth which could affect normal cells as well. Specific side effects are not listed but generally could involve digestive disturbances or blood count changes.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 2 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 2 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 2 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Incidence of adverse events

PK parameter: drug clearance

PK parameter: half-life

+2 more

Secondary study objectives

Objective response rate

Other study objectives

CTNNB1 and APC gene mutations and genomic signatures

Changes in the levels of each of the lymphocyte subsets and immunoglobulins

Patient reported outcomes (PROs)

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Treatment (nirogacestat)Experimental Treatment8 Interventions

Patients receive nirogacestat PO BID on days 1-28. Cycles repeats every 28 days in the absence of disease progression or unacceptable toxicity. Patients undergo ECHO and CT or MRI on study. Patients may also undergo x-ray imaging and blood sample collection on study.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Biospecimen Collection

2004

Completed Phase 3

~2020

Echocardiography

2013

Completed Phase 4

~11580

Computed Tomography

2017

Completed Phase 2

~2740

Magnetic Resonance Imaging

2017

Completed Phase 3

~1160

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Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Desmoid Tumors are often treated with enzyme inhibitors like Nirogacestat, which work by blocking specific enzymes necessary for tumor cell growth. Nirogacestat inhibits gamma-secretase, an enzyme involved in the Notch signaling pathway, which is crucial for cell proliferation and survival. By inhibiting this enzyme, Nirogacestat can potentially halt the growth of tumor cells. This mechanism is particularly important for Desmoid Tumor patients because these tumors are locally aggressive and can be difficult to remove surgically. Enzyme inhibition offers a non-surgical option that targets the tumor's growth mechanisms directly, providing a promising treatment avenue for patients with inoperable tumors.

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