A Study of a New Drug, Nirogacestat, for Treating Desmoid Tumors That Cannot be Removed by Surgery (NCT04195399)

🧪 Interventions in This Study

• Nirogacestat Hydrobromide (DRUG)

Check every box below. If you can check all of them, you may meet the study's basic screening requirements (final eligibility is confirmed by the site team).

1. Patients must be \> 12 months and \< 18 years of age at the time of enrollment
2. Patients must have a body surface area of \> 0.3 m\^2 at the time of enrollment
3. Existing or recurrent desmoid tumor that is deemed not amenable to surgery without significant morbidity and progressed by \>= 10% as assessed by RECIST version (v)1.1 within the 6-month period prior to study enrollment
4. Patients must have had histologic verification of the desmoid tumor
5. Patients must have measurable disease by RECIST v1.1 criteria
6. Patient must have received at least one prior course of systemic therapy for desmoid tumor
7. Patients must have a Lansky (for patients =\< 16 years of age) or Karnofsky (for patients \> 16 years of age) performance status score of \>= 50. Patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing performance score
8. Patients must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, surgery or radiotherapy prior to entering this study. Patients may not be using or anticipate using these treatments after the observed progression or within the time period stated below
9. Cytotoxic chemotherapy: must not have received within 2 weeks of entry onto this study (4 weeks if prior nitrosourea)
10. Small molecule tyrosine kinase inhibitors (e.g., sorafenib, pazopanib, imatinib), rapalogs (e.g., temsirolimus, everolimus, sirolimus) or anti estrogen therapy (e.g., tamoxifen): may not have received within 28 days prior to the first dose of study treatment
11. Antibodies: \>= 21 days must have elapsed from infusion of last dose of antibody, and toxicity related to prior antibody therapy must be recovered to grade =\< 1
12. Biologic (anti-neoplastic agent): at least 7 days since the completion of therapy with a biologic agent

🔍 Extracted verbatim from the official NIH eligibility criteria. Always confirm with the study coordinator.

📍 All Participating Trial Locations (98 Sites)

Showing the first 25 of 98 sites.

• Children's Hospital of Alabama — Birmingham, Alabama
• USA Health Strada Patient Care Center — Mobile, Alabama
• Banner Children's at Desert — Mesa, Arizona
• Arkansas Children's Hospital — Little Rock, Arkansas
• Kaiser Permanente Downey Medical Center — Downey, California
• Loma Linda University Medical Center — Loma Linda, California
• Children's Hospital Los Angeles — Los Angeles, California
• Mattel Children's Hospital UCLA — Los Angeles, California
• UCSF Benioff Children's Hospital Oakland — Oakland, California
• Kaiser Permanente-Oakland — Oakland, California
• Lucile Packard Children's Hospital Stanford University — Palo Alto, California
• University of California Davis Comprehensive Cancer Center — Sacramento, California
• Rady Children's Hospital - San Diego — San Diego, California
• UCSF Medical Center-Mission Bay — San Francisco, California
• Children's Hospital Colorado — Aurora, Colorado
• Rocky Mountain Hospital for Children-Presbyterian Saint Luke's Medical Center — Denver, Colorado
• Connecticut Children's Medical Center — Hartford, Connecticut
• Alfred I duPont Hospital for Children — Wilmington, Delaware
• MedStar Georgetown University Hospital — Washington D.C., District of Columbia
• Children's National Medical Center — Washington D.C., District of Columbia
• UF Health Cancer Institute - Gainesville — Gainesville, Florida
• Nemours Children's Clinic-Jacksonville — Jacksonville, Florida
• AdventHealth Orlando — Orlando, Florida
• Nemours Children's Hospital — Orlando, Florida
• Johns Hopkins All Children's Hospital — St. Petersburg, Florida

💰 Cost, Insurance & Patient Rights

• 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
• Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
• Voluntary Participation: You may withdraw at any time without affecting your standard medical care.

More studies in Desmoid Fibromatosis

• Observing People With Desmoid-Type Fibromatosis — New York, New York

Explore by condition: All Desmoid Fibromatosis clinical trials

💡 How to Participate

1. Check the checklist above to see if you may qualify.
2. Review the official NIH record below to find the coordinating site.
3. Ask about the visit schedule, what is covered at no cost, and any travel support.
4. Confirm with your own doctor before making a decision.