Nirogacestat for Desmoid Tumors
Trial Summary
What is the purpose of this trial?
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.
Will I have to stop taking my current medications?
The trial requires that you stop taking certain medications before starting, such as strong inducers or inhibitors of CYP3A4, and NSAIDs for treating the tumor. You should discuss your current medications with the trial team to see if any need to be stopped or adjusted.
What data supports the effectiveness of the drug Nirogacestat for Desmoid Tumors?
Research shows that Nirogacestat, a gamma-secretase inhibitor, has been effective in treating desmoid tumors, with some patients experiencing partial or complete responses and stable disease. In a study with 17 patients, 29% had a partial response, and the drug was well tolerated, indicating promising results for this condition.12345
Is Nirogacestat safe for humans?
Nirogacestat, also known as PF-03084014, has been generally well tolerated in studies with both adults and children with desmoid tumors. In one study, no severe side effects were reported in pediatric patients, and in another study with adults, it was well tolerated with participants experiencing improvements in symptoms.12367
How is the drug Nirogacestat different from other treatments for desmoid tumors?
Nirogacestat is unique because it is a gamma-secretase inhibitor, which targets a specific pathway involved in the growth of desmoid tumors. Unlike other treatments, it has shown promising results in both adults and young patients, with a good safety profile and significant clinical benefits, such as partial and complete responses in some cases.12348
Research Team
Fariba Navid
Principal Investigator
Children's Oncology Group
Eligibility Criteria
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.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive nirogacestat orally twice daily on days 1-28. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.
Pharmacokinetics and Safety Monitoring
Pharmacokinetic parameters are assessed, and adverse events are monitored. Imaging and blood sample collection are conducted.
Follow-up
Participants are monitored for safety and effectiveness after treatment completion.
Treatment Details
Interventions
- Nirogacestat
Find a Clinic Near You
Who Is Running the Clinical Trial?
Children's Oncology Group
Lead Sponsor
SpringWorks Therapeutics, Inc.
Industry Sponsor
National Cancer Institute (NCI)
Collaborator