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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
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
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 moreSecondary 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)
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
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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Find a Location
Who is running the clinical trial?
SpringWorks Therapeutics, Inc.Industry Sponsor
12 Previous Clinical Trials
931 Total Patients Enrolled
Children's Oncology GroupLead Sponsor
457 Previous Clinical Trials
239,795 Total Patients Enrolled
National Cancer Institute (NCI)NIH
13,842 Previous Clinical Trials
41,002,965 Total Patients Enrolled
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Eligibility Criteria:
This trial includes the following eligibility criteria:- I haven't taken certain cancer drugs like sorafenib or tamoxifen in the last 28 days.I am currently breastfeeding.I am using effective birth control and will continue for 6 months after my last dose.I can do most activities or at least move around in a wheelchair.I haven't had chemotherapy in the last 2 weeks, or 4 weeks if it was nitrosourea.My body surface area is more than 0.3 square meters.I am a man who agrees to use contraception during and 90 days after treatment.I can swallow tablets without crushing or using a tube.I have not had an infection in the week before joining the study.I have a fracture that hasn't healed.I do not have an active hepatitis B, C, or HIV infection.I am currently pregnant.My heart's electrical activity is within a healthy range.I am a woman who can have children and have a negative pregnancy test.I agree not to use NSAIDs for my desmoid tumor treatment while in the study, except for occasional pain relief.My desmoid tumor was confirmed through a tissue examination.It has been over a week since my last biologic treatment.I haven't taken growth factor medications in the last week.I haven't taken any experimental drugs in the last 4 weeks and any side effects from past treatments have mostly gone away.My kidney function is good based on tests.I don't have a history of long QT syndrome.I haven't had serious heart issues or a stroke in the last 6 months.I am between 12 months and 18 years old.I have recovered from previous cancer treatments and am not planning any soon.My desmoid tumor can't be removed by surgery without high risk and has grown by 10% or more recently.I am not taking any strong medication that affects liver enzymes.My liver is working well, based on recent tests.I have had at least one treatment for my desmoid tumor.I haven't taken corticosteroids in the last 21 days, except for specific reasons.It's been weeks since my last local tumor treatment and any side effects are mild.I have not taken any gamma-secretase, Notch, or beta-catenin inhibitors.I have a stomach or intestine condition that affects how I absorb medicine.It's been over 21 days since my last antibody treatment and any side effects are mild.My platelet count is at least 100,000 and I haven't needed a transfusion.I had a stem cell transplant over 6 months ago with no current graft vs. host disease.I have had cancer before, but it wasn't skin cancer or desmoid tumors, and I've been in remission for less than 3 years.I have ulcerative colitis, inflammatory bowel disease, or a bowel obstruction.My hemoglobin level is at least 9.0 g/dL.
Research Study Groups:
This trial has the following groups:- Group 1: Treatment (nirogacestat)
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
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