Promising drug may shrink rare tumors in kids without surgery

NCT ID NCT04195399

Disease control Sponsor: Children's Oncology Group Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jul 31, 2026 · Updated 2 times

Summary

This study tests a drug called nirogacestat in children and teens with desmoid tumors that have grown despite treatment and cannot be surgically removed. The goal is to see if the drug can stop or slow tumor growth and to check for side effects. About 31 participants aged 1 to 18 will take the drug by mouth, and researchers will monitor their health and tumor changes over time.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

31 people

The number who actually took part.

Started

Oct 2020

Expected to finish

Dec 2032

An estimate. End dates often move.

Lead sponsor

A research network

The lead sponsor is a research network or cooperative group.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

12 months to 18 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Patients must be \> 12 months and \< 18 years of age at the time of enrollment * Patients must have a body surface area of \> 0.3 m\^2 at the time of enrollment * 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 * Patients must have had histologic verification of the desmoid tumor * Patients must have measurable disease by RECIST v1.1 criteria * Patient must have received at least one prior course of systemic therapy for desmoid tumor * 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 * 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 * Cytotoxic chemotherapy: must not have received within 2 weeks of entry onto this study (4 weeks if prior nitrosourea) * 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 * 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 * Biologic (anti-neoplastic agent): at least 7 days since the completion of therapy with a biologic agent * Local regional tumor directed therapy, including, but not limited to small port radiation therapy (RT), radiofrequency ablation, cryotherapy, surgery: at least 2 weeks since these therapies and all toxicity must have resolved to grade =\< 1. If prior craniospinal RT or if \>= 50% radiation of pelvis then \>= 6 months must have elapsed. If other substantial bone marrow (BM) radiation, then \>= 6 weeks must have elapsed * Stem cell transplant (SCT): No evidence of active graft versus (vs.) host disease. For allogeneic SCT, \>= 6 months must have elapsed * No prior gamma-secretase, Notch or beta-catenin inhibitor * Investigational drugs: must not have received investigational drug within 4 weeks of study entry, and all toxicities related to prior therapy must be resolved to grade =\< 1 or baseline * Concomitant Medication Restrictions * Growth factor(s): must not have received within 1 week of entry onto this study * Patients who are currently receiving drugs that are strong inducers or moderate or strong inhibitors of CYP3A4 are not eligible. Strong inducers or moderate or strong inhibitors of CYP3A4 are not allowed from 14 days prior to enrollment to the end of protocol therapy. Note: CYP3A4 inducing anti-epileptic drugs on a stable dose, are allowed * Must not be receiving non-steroidal anti-inflammatory drugs (NSAIDs) as treatment for desmoid tumor after the observed progression and patient agrees to not use NSAIDs while on study. Occasional use (defined as =\< 3 times per week) for treatment of pain is permitted * Peripheral absolute neutrophil count (ANC) \>= 1000/uL (within 7 days prior to enrollment) * Platelet count \>= 100,000/uL (transfusion independent) (within 7 days prior to enrollment) * Hemoglobin \>= 9.0 g/dL (may receive red blood cell \[RBC\] transfusions) (within 7 days prior to enrollment) * Creatinine clearance or radioisotope glomerular filtration rate (GFR) \>= 70 mL/min/1.73 m\^2 or a serum creatinine based on age/sex as follows (within 7 days prior to enrollment): * Age: Maximum serum creatinine (mg/dL) * Age: 1 to \< 2 years; Maximum serum creatinine (mg/dL): 0.6 (male and female) * Age: 2 to \< 6 years; Maximum serum creatinine (mg/dL): 0.8 (male and female) * Age: 6 to \< 10 years; Maximum serum creatinine (mg/dL): 1 (male and female) * Age: 10 to \< 13 years; Maximum serum creatinine (mg/dL): 1.2 (male and female) * Age: 13 to \< 16 years; Maximum serum creatinine (mg/dL): 1.5 (male); 1.4 (female) * Age: \>= 16 years; Maximum serum creatinine (mg/dL): 1.7 (male); 1.4 (female) * Adequate liver function defined as: * Total bilirubin =\< 1.5 x upper limit of normal (ULN) for age (unless secondary to previously diagnosed Gilbert's syndrome) (within 7 days prior to enrollment) * Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase \[ALT\]) =\< 135 U/L * Note: For the purpose of this study, the ULN for SGPT (ALT) has been set to the value of 45 U/L (within 7 days prior to enrollment) * Adequate cardiac function defined as: * Corrected QT (QTc) interval \< 470 ms * No history of congenital or acquired prolonged QTc syndrome * No history of clinically significant cardiac arrhythmias, congestive heart failure, stroke or myocardial infarction within 6 months prior to study entry * All patients and/or their parents or legal guardians must sign a written informed consent * All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met Exclusion Criteria: * Active or chronic infection within 7 days prior to study entry * Presence of non-healing fracture * Note: patients with pathologic fracture related to tumor are eligible * Use of corticosteroids within 21 days of enrollment, except in the following situations: * Physiologic steroid replacement for adrenal insufficiency * Topical, ocular, intra-articular, intranasal, or inhaled corticosteroid with minimal systemic absorption * Short course (=\< 7 days) of corticosteroid prescribed prophylactically (eg, for contrast dye allergy) or for the treatment of a non-autoimmune condition (eg, delayed-type hypersensitivity reaction caused by contact allergen), or exacerbation of asthma * Patients with gastrointestinal conditions that might predispose for drug intolerability or poor drug absorption (e.g., inability to take oral medication, prior surgical procedures affecting absorption (e.g., gastric bypass), malabsorption syndrome, and active peptic ulcer disease) * Patients with ulcerative colitis, inflammatory bowel disease, or a partial or complete small bowel obstruction * Known active infection with hepatitis B, hepatitis C or human immunodeficiency virus (HIV) * Patients with a prior history of malignancy, with the exceptions of desmoid tumor(s) and non-melanoma skin cancer, who are not in remission for more than 3 years * Patients who are unable to swallow tablets. Tablets must not be crushed or chewed. Administration of nirogacestat via gastrostomy tube or nasogastric tube is not allowed * Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study * Sexually active female patients of reproductive potential who have not agreed to use 1 method of highly effective contraceptive (including copper-containing intrauterine device, condom with spermicidal foam/gel/film/cream/suppository, bilateral tubal ligation, established use of inserted, injected or implanted hormonal method of contraception, abstinence, or male sterilization) for the duration of their study participation and for at least 6 months after last dose of nirogacestat. A second form of contraception (i.e. barrier method) is required for patients who are using hormonal contraception as nirogacestat may reduce the efficacy of hormonal contraceptives * Sexually active male patients of reproductive potential who have not agreed to use a condom and their female partner who have not agreed to use one of the highly effective methods of contraception mentioned above during treatment and for at least 90 days after the last dose of nirogacestat * Female patients who are breastfeeding * Female patients who are pregnant. These patients are excluded because there is no available information regarding the effects of nirogacestat on the developing human fetus and inhibition of gamma-secretase is known to be teratogenic * Female patients of childbearing potential unless a negative pregnancy test result has been obtained

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Conditions

The condition(s) this trial relates to.

desmoid tumor

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Desmoid fibromatosis Recurrent desmoid fibromatosis Unresectable desmoid fibromatosis

Contacts and locations

Locations

AdventHealth Orlando

Orlando, Florida, 32803, United States
Albany Medical Center

Albany, New York, 12208, United States
Alberta Children's Hospital

Calgary, Alberta, T3B 6A8, Canada
Alfred I duPont Hospital for Children

Wilmington, Delaware, 19803, United States
Alliance for Childhood Diseases/Cure 4 the Kids Foundation

Las Vegas, Nevada, 89135, United States
Arkansas Children's Hospital

Little Rock, Arkansas, 72202-3591, United States
BI-LO Charities Children's Cancer Center

Greenville, South Carolina, 29605, United States
Banner Children's at Desert

Mesa, Arizona, 85202, United States
Bronson Methodist Hospital

Kalamazoo, Michigan, 49007, United States
C S Mott Children's Hospital

Ann Arbor, Michigan, 48109, United States
Carilion Children's

Roanoke, Virginia, 24014, United States
Carolinas Medical Center/Levine Cancer Institute

Charlotte, North Carolina, 28203, United States
Children's Healthcare of Atlanta - Arthur M Blank Hospital

Atlanta, Georgia, 30329, United States
Children's Hospital Colorado

Aurora, Colorado, 80045, United States
Children's Hospital Los Angeles

Los Angeles, California, 90027, United States
Children's Hospital Medical Center of Akron

Akron, Ohio, 44308, United States
Children's Hospital New Orleans

New Orleans, Louisiana, 70118, United States
Children's Hospital of Alabama

Birmingham, Alabama, 35233, United States
Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States
Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, 15224, United States
Children's Hospital of The King's Daughters

Norfolk, Virginia, 23507, United States
Children's Hospital of Wisconsin

Milwaukee, Wisconsin, 53226, United States
Children's Mercy Hospitals and Clinics

Kansas City, Missouri, 64108, United States
Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States
Christchurch Hospital

Christchurch, 8011, New Zealand
Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States
Cleveland Clinic Foundation

Cleveland, Ohio, 44195, United States
Connecticut Children's Medical Center

Hartford, Connecticut, 06106, United States
Cook Children's Medical Center

Fort Worth, Texas, 76104, United States
Corewell Health Grand Rapids Hospitals - Helen DeVos Children's Hospital

Grand Rapids, Michigan, 49503, United States
Dana-Farber Cancer Institute

Boston, Massachusetts, 02215, United States
Dayton Children's Hospital

Dayton, Ohio, 45404, United States
Dell Children's Medical Center of Central Texas

Austin, Texas, 78723, United States
East Tennessee Childrens Hospital

Knoxville, Tennessee, 37916, United States
El Paso Children's Hospital

El Paso, Texas, 79905, United States
Hackensack University Medical Center

Hackensack, New Jersey, 07601, United States
Johns Hopkins All Children's Hospital

St. Petersburg, Florida, 33701, United States
Johns Hopkins University/Sidney Kimmel Cancer Center

Baltimore, Maryland, 21287, United States
Kaiser Permanente Downey Medical Center

Downey, California, 90242, United States
Kaiser Permanente-Oakland

Oakland, California, 94611, United States
Kapiolani Medical Center for Women and Children

Honolulu, Hawaii, 96826, United States
Loma Linda University Medical Center

Loma Linda, California, 92354, United States
Lucile Packard Children's Hospital Stanford University

Palo Alto, California, 94304, United States
Lurie Children's Hospital-Chicago

Chicago, Illinois, 60611, United States
Massachusetts General Hospital Cancer Center

Boston, Massachusetts, 02114, United States
Mattel Children's Hospital UCLA

Los Angeles, California, 90095, United States
MedStar Georgetown University Hospital

Washington D.C., District of Columbia, 20007, United States
Medical University of South Carolina

Charleston, South Carolina, 29425, United States
Memorial Sloan Kettering Cancer Center

New York, New York, 10065, United States
Mercy Hospital Saint Louis

St Louis, Missouri, 63141, United States
Michigan State University

East Lansing, Michigan, 48823, United States
Montefiore Medical Center - Moses Campus

The Bronx, New York, 10467, United States
NYP/Columbia University Medical Center/Herbert Irving Comprehensive Cancer Center

New York, New York, 10032, United States
Nationwide Children's Hospital

Columbus, Ohio, 43205, United States
Nemours Children's Clinic-Jacksonville

Jacksonville, Florida, 32207, United States
Nemours Children's Hospital

Orlando, Florida, 32827, United States
Norton Children's Hospital

Louisville, Kentucky, 40202, United States
OSF Children's Hospital of Illinois

Peoria, Illinois, 61637, United States
Ochsner Medical Center Jefferson

New Orleans, Louisiana, 70121, United States
Oregon Health and Science University

Portland, Oregon, 97239, United States
Penn State Children's Hospital

Hershey, Pennsylvania, 17033, United States
Perth Children's Hospital

Perth, Western Australia, 6009, Australia
Primary Children's Hospital

Salt Lake City, Utah, 84113, United States
Queensland Children's Hospital

South Brisbane, Queensland, 4101, Australia
Rady Children's Hospital - San Diego

San Diego, California, 92123, United States
Renown Regional Medical Center

Reno, Nevada, 89502, United States
Rhode Island Hospital

Providence, Rhode Island, 02903, United States
Riley Hospital for Children

Indianapolis, Indiana, 46202, United States
Rocky Mountain Hospital for Children-Presbyterian Saint Luke's Medical Center

Denver, Colorado, 80218, United States
Roswell Park Cancer Institute

Buffalo, New York, 14263, United States
Saint Joseph's Hospital/Children's Hospital-Tampa

Tampa, Florida, 33607, United States
Saint Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States
Saint Luke's Cancer Institute - Boise

Boise, Idaho, 83712, United States
Starship Children's Hospital

Grafton, Auckland, 1145, New Zealand
State University of New York Upstate Medical University

Syracuse, New York, 13210, United States
Summerlin Hospital Medical Center

Las Vegas, Nevada, 89144, United States
Sunrise Hospital and Medical Center

Las Vegas, Nevada, 89109, United States
The Children's Hospital at TriStar Centennial

Nashville, Tennessee, 37203, United States
The Children's Hospital at Westmead

Westmead, New South Wales, 2145, Australia
The Montreal Children's Hospital of the MUHC

Montreal, Quebec, H3H 1P3, Canada
UCSF Benioff Children's Hospital Oakland

Oakland, California, 94609, United States
UCSF Medical Center-Mission Bay

San Francisco, California, 94158, United States
UF Health Cancer Institute - Gainesville

Gainesville, Florida, 32610, United States
USA Health Strada Patient Care Center

Mobile, Alabama, 36604, United States
UT MD Anderson Cancer Center

Houston, Texas, 77030, United States
UT Southwestern/Simmons Cancer Center-Dallas

Dallas, Texas, 75390, United States
University Medical Center of Southern Nevada

Las Vegas, Nevada, 89102, United States
University Pediatric Hospital

San Juan, 00926, Puerto Rico
University of Alberta Hospital

Edmonton, Alberta, T6G 2B7, Canada
University of California Davis Comprehensive Cancer Center

Sacramento, California, 95817, United States
University of Chicago Comprehensive Cancer Center

Chicago, Illinois, 60637, United States
University of Mississippi Medical Center

Jackson, Mississippi, 39216, United States
University of Oklahoma Health Sciences Center

Oklahoma City, Oklahoma, 73104, United States
University of Texas Health Science Center at San Antonio

San Antonio, Texas, 78229, United States
University of Wisconsin Carbone Cancer Center - University Hospital

Madison, Wisconsin, 53792, United States
Vanderbilt University/Ingram Cancer Center

Nashville, Tennessee, 37232, United States
Walter Reed National Military Medical Center

Bethesda, Maryland, 20889-5600, United States
Washington University School of Medicine

St Louis, Missouri, 63110, United States

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