Cancer drug shows promise in slowing rare tumor growth

NCT ID NCT02066181

Disease control Sponsor: National Cancer Institute (NCI) Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This Phase 3 trial tested the drug sorafenib in 87 people with desmoid tumors, a rare type of growth that can be painful and invasive. The study compared sorafenib to a placebo to see if it could slow tumor progression. Sorafenib works by blocking proteins that help tumors grow. The main goal was to see how long patients lived without their disease getting worse.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance: sorafenib (Nexavar)
What this could lead to: If successful, sorafenib could offer a new treatment option to slow or stop the growth of desmoid tumors, potentially avoiding the need for surgery or other invasive procedures.
What could go wrong: This is a relatively small Phase 3 trial (87 participants), and sorafenib can cause side effects like fatigue, diarrhea, and skin reactions. The drug may not work for everyone, and long-term benefits are still uncertain.

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 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

87 people

The number who actually took part.

Started

Mar 2014

Finished

Dec 2022

Lead sponsor

A government research agency

The lead sponsor is the US National Institutes of Health.

Who can take part

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

Ages

18 years and older

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 have confirmation of DT/DF by local pathologist prior to registration * Patients may have been treated with locoregional therapies such as major surgery, radiation, radiofrequency ablation, or cryosurgery provided this has been completed at least 4 weeks prior to registration and recovered from therapy related toxicity to less than CTCAE grade 2 * Patients may have been treated with cytotoxic, biologic (antibody), immune or experimental therapy, tyrosine kinase inhibitors, hormone inhibitors or nonsteroidal anti-inflammatory drugs (NSAIDs) provided this has been completed at least 4 weeks prior to registration (6 weeks for mitomycin and nitrosoureas) and recovered from any therapy related toxicity to less than CTCAE grade 2 * Patients with prior or current treatment of sorafenib are excluded * No concomitant treatment, in therapeutic doses, with anticoagulants such as warfarin or warfarin-related agents, heparin, thrombin or Factor Xa inhibitors, or antiplatelet agents (e.g., clopidogrel); low dose aspirin (=\< 81 mg/day), low-dose warfarin (=\< 1 mg/day), and prophylactic low molecular weight heparin (LMWH) are permitted; please note that drugs that strongly induce or inhibit cytochrome P450, family 3, subfamily A, polypeptide 4 (CYP3A4) or are associated with a risk of torsades are not allowed; chronic concomitant treatment of CYP3A4 inducers is not allowed (e.g., dexamethasone, phenytoin, carbamazepine, rifampin, rifabutin, rifapentin, phenobarbital, and St. John's wort); as part of the enrollment/informed consent procedures, the patient will be counseled on the risk of interactions with other agents, and what to do if new medications need to be prescribed or if the patient is considering a new over-the-counter medicine or herbal product; the following drugs are strong inhibitors of CYP3A4 and are not allowed during the treatment with sorafenib: * Boceprevir * Indinavir * Nelfinavir * Lopinavir/ritonavir * Saquinavir * Telaprevir * Ritonavir * Clarithromycin * Conivaptan * Itraconazole * Ketoconazole * Mibefradil * Nefazodone * Posaconazole * Voriconazole * Telithromycin * Drugs with possible or conditional risk of torsades should be used with caution knowing that sorafenib could prolong the QT interval * Chronic daily NSAID use as treatment for controlling desmoid tumors is not allowed, and should be stopped \>= 3 days prior to registration; NSAIDS are allowed when used for desmoid tumor-related pain or for symptoms that are unrelated to desmoid disease (eg. headache, arthritis) * Patients must have measurable disease * Patients have to meet one of the following criteria to be eligible: * Disease determined unresectable or entailing unacceptably morbid surgery based on 1 or more of the following characteristics: * Multifocal disease * Disease in which there is involvement or inadequate plane from: neurovascular bundle, bone, skin, or viscera * Large size in relationship to location OR multi-compartment involvement * Progression by radiographic imaging (10% increase in size by RECIST v1.1 within 6 months of registration) * Patients with symptomatic disease which meets the following criteria Brief Pain Inventory (BPI) score greater than or equal to 3 AND one of the following: * Inability to control pain with NSAIDs and considering addition of narcotics OR * \> 30% increase in current use of narcotics OR * Addition of a new opioid narcotic * Eastern Cooperative Oncology Group (ECOG) performance status =\< 2 * Patients who are pregnant or nursing are not eligible * No patients with a history of cardiac disease: congestive heart failure \> class II New York Heart Association (NYHA); active coronary artery disease (CAD) (myocardial infarction or unstable angina within 6 months prior to study entry) * No patients with inadequately controlled hypertension (defined as a blood pressure of \>= 150 mmHg systolic and/or \>= 90 mmHg diastolic), or any prior history of hypertensive crisis or hypertensive encephalopathy * No patients with clinically significant gastrointestinal (GI) bleeding or bleeding diathesis within 30 days prior to registration * Absolute neutrophil count \>= 1,500/mm\^3 * Hemoglobin \>= 8 g/dl * Platelets \>= 75,000/mm\^3 * Total bilirubin =\< 1.5 x upper limits of normal (ULN) * Serum glutamic oxaloacetic transaminase (SGOT) (aspartate aminotransferase \[AST\])/serum glutamate pyruvate transaminase (SGPT) (aspartate aminotransferase \[ALT\]) =\< 1.5 x ULN * Calculated creatinine clearance \>= 50 mL/min using the Cockcroft-Gault equation

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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

Contacts and locations

Locations

Abbott-Northwestern Hospital

Minneapolis, Minnesota, 55407, United States
Alegent Health Bergan Mercy Medical Center

Omaha, Nebraska, 68124, United States
Alegent Health Immanuel Medical Center

Omaha, Nebraska, 68122, United States
Alegent Health Lakeside Hospital

Omaha, Nebraska, 68130, United States
Atrium Medical Center-Middletown Regional Hospital

Franklin, Ohio, 45005-1066, United States
Beebe Health Campus

Rehoboth Beach, Delaware, 19971, United States
Beebe Medical Center

Lewes, Delaware, 19958, United States
Blanchard Valley Hospital

Findlay, Ohio, 45840, United States
Brigham and Women's Hospital

Boston, Massachusetts, 02115, United States
Cancer Center of Western Wisconsin

New Richmond, Wisconsin, 54017, United States
Cancer Therapy and Integrative Medicine

Las Vegas, Nevada, 89121, United States
Cancer and Blood Specialists-Henderson

Henderson, Nevada, 89052, United States
Cancer and Blood Specialists-Shadow

Las Vegas, Nevada, 89106, United States
Cancer and Blood Specialists-Tenaya

Las Vegas, Nevada, 89128, United States
Central Care Cancer Center - Bolivar

Bolivar, Missouri, 65613, United States
Christiana Care Health System-Christiana Hospital

Newark, Delaware, 19718, United States
Christiana Care Health System-Concord Health Center

Chadds Ford, Pennsylvania, 19317, United States
Christiana Care Health System-Wilmington Hospital

Wilmington, Delaware, 19801, United States
Christiana Gynecologic Oncology LLC

Newark, Delaware, 19713, United States
Comprehensive Cancer Centers of Nevada

Las Vegas, Nevada, 89148, United States
Comprehensive Cancer Centers of Nevada - Central Valley

Las Vegas, Nevada, 89169, United States
Comprehensive Cancer Centers of Nevada - Henderson

Henderson, Nevada, 89052, United States
Comprehensive Cancer Centers of Nevada - Northwest

Las Vegas, Nevada, 89128, United States
Comprehensive Cancer Centers of Nevada-Southeast Henderson

Henderson, Nevada, 89074, United States
Comprehensive Cancer Centers of Nevada-Summerlin

Las Vegas, Nevada, 89144, United States
Cox Cancer Center Branson

Branson, Missouri, 65616, United States
CoxHealth South Hospital

Springfield, Missouri, 65807, United States
Creighton University Medical Center

Omaha, Nebraska, 68131, United States
Dana-Farber Cancer Institute

Boston, Massachusetts, 02215, United States
Dayton NCI Community Oncology Research Program

Dayton, Ohio, 45459, United States
Delaware Clinical and Laboratory Physicians PA

Newark, Delaware, 19713, United States
Delbert Day Cancer Institute at PCRMC

Rolla, Missouri, 65401, United States
Duke University Medical Center

Durham, North Carolina, 27710, United States
Fairview Ridges Hospital

Burnsville, Minnesota, 55337, United States
Fairview Southdale Hospital

Edina, Minnesota, 55435, United States
Faith Regional Health Services Carson Cancer Center

Norfolk, Nebraska, 68701, United States
Fox Chase Cancer Center

Philadelphia, Pennsylvania, 19111, United States
Freeman Health System

Joplin, Missouri, 64804, United States
GenesisCare USA - Fort Apache

Las Vegas, Nevada, 89148, United States
GenesisCare USA - Henderson

Henderson, Nevada, 89074, United States
GenesisCare USA - Las Vegas

Las Vegas, Nevada, 89109, United States
GenesisCare USA - Vegas Tenaya

Las Vegas, Nevada, 89128, United States
Good Samaritan Hospital - Dayton

Dayton, Ohio, 45406, United States
Good Samaritan Regional Health Center

Mount Vernon, Illinois, 62864, United States
Great Plains Health Callahan Cancer Center

North Platte, Nebraska, 69101, United States
Hackensack University Medical Center

Hackensack, New Jersey, 07601, United States
Hawaii Cancer Care Inc - Waterfront Plaza

Honolulu, Hawaii, 96813, United States
Hawaii Cancer Care Inc-Liliha

Honolulu, Hawaii, 96817, United States
Health Partners Inc

Minneapolis, Minnesota, 55454, United States
HealthCare Partners Medical Group Oncology/Hematology-Centennial Hills

Las Vegas, Nevada, 89149, United States
HealthCare Partners Medical Group Oncology/Hematology-Maryland Parkway

Las Vegas, Nevada, 89109, United States
HealthCare Partners Medical Group Oncology/Hematology-San Martin

Las Vegas, Nevada, 89113, United States
HealthCare Partners Medical Group Oncology/Hematology-Tenaya

Las Vegas, Nevada, 89128, United States
Helen F Graham Cancer Center

Newark, Delaware, 19713, United States
Hennepin County Medical Center

Minneapolis, Minnesota, 55415, United States
Huntsman Cancer Institute/University of Utah

Salt Lake City, Utah, 84112, United States
Hutchinson Area Health Care

Hutchinson, Minnesota, 55350, United States
John B Amos Cancer Center

Columbus, Georgia, 31904, United States
Johns Hopkins University/Sidney Kimmel Cancer Center

Baltimore, Maryland, 21287, United States
Kapiolani Medical Center for Women and Children

Honolulu, Hawaii, 96826, United States
Kettering Medical Center

Kettering, Ohio, 45429, United States
Kuakini Medical Center

Honolulu, Hawaii, 96817, United States
Lakeview Hospital

Stillwater, Minnesota, 55082, United States
Las Vegas Cancer Center-Henderson

Henderson, Nevada, 89052, United States
Las Vegas Cancer Center-Medical Center

Las Vegas, Nevada, 89148-2405, United States
Lewis Cancer and Research Pavilion at Saint Joseph's/Candler

Savannah, Georgia, 31405, United States
Low Country Cancer Care

Savannah, Georgia, 31404, United States
Loyola University Medical Center

Maywood, Illinois, 60153, United States
M D Anderson Cancer Center

Houston, Texas, 77030, United States
Mayo Clinic in Florida

Jacksonville, Florida, 32224-9980, United States
Mayo Clinic in Rochester

Rochester, Minnesota, 55905, United States
MedStar Georgetown University Hospital

Washington D.C., District of Columbia, 20007, United States
MedStar Washington Hospital Center

Washington D.C., District of Columbia, 20010, United States
Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States
Medical Oncology Hematology Consultants PA

Newark, Delaware, 19713, United States
Memorial Sloan Kettering Cancer Center

New York, New York, 10065, United States
Mercy Clinic-Rolla-Cancer and Hematology

Rolla, Missouri, 65401, United States
Mercy Hospital

Coon Rapids, Minnesota, 55433, United States
Mercy Hospital Joplin

Joplin, Missouri, 64804, United States
Mercy Hospital Saint Louis

St Louis, Missouri, 63141, United States
Mercy Hospital Springfield

Springfield, Missouri, 65804, United States
Metro Minnesota Community Oncology Research Consortium

Saint Louis Park, Minnesota, 55416, United States
Miami Valley Hospital

Dayton, Ohio, 45409, United States
Miami Valley Hospital North

Dayton, Ohio, 45415, United States
Miami Valley Hospital South

Centerville, Ohio, 45459, United States
Minnesota Oncology Hematology PA-Maplewood

Maplewood, Minnesota, 55109, United States
Minnesota Oncology Hematology PA-Woodbury

Woodbury, Minnesota, 55125, United States
Missouri Valley Cancer Consortium

Omaha, Nebraska, 68106, United States
NYP/Columbia University Medical Center/Herbert Irving Comprehensive Cancer Center

New York, New York, 10032, United States
Nebraska Cancer Research Center

Lincoln, Nebraska, 68510, United States
Nebraska Cancer Specialists - Omaha

Omaha, Nebraska, 68124, United States
Nebraska Hematology and Oncology

Lincoln, Nebraska, 68506, United States
Nevada Cancer Research Foundation NCORP

Las Vegas, Nevada, 89169, United States
New Ulm Medical Center

New Ulm, Minnesota, 56073, United States
North Memorial Medical Health Center

Robbinsdale, Minnesota, 55422, United States
Northwestern University

Chicago, Illinois, 60611, United States
Oklahoma Cancer Specialists and Research Institute-Tulsa

Tulsa, Oklahoma, 74146, United States
Oncology Hematology Care Inc-Anderson

Cincinnati, Ohio, 45230, United States
Oncology Hematology Care Inc-Blue Ash

Cincinnati, Ohio, 45242, United States
Oncology Hematology Care Inc-Crestview

Crestview Hills, Kentucky, 41017, United States
Oncology Hematology Care Inc-Eden Park

Cincinnati, Ohio, 45202, United States
Oncology Hematology Care Inc-Healthplex

Fairfield, Ohio, 45014, United States
Oncology Hematology Care Inc-Kenwood

Cincinnati, Ohio, 45236, United States
Oncology Hematology Care Inc-Mercy West

Cincinnati, Ohio, 45211, United States
Oncology Hematology West PC

Omaha, Nebraska, 68130, United States
OptumCare Cancer Care at Fort Apache

Las Vegas, Nevada, 89148, United States
PCR Oncology

Arroyo Grande, California, 93420, United States
Pali Momi Medical Center

‘Aiea, Hawaii, 96701, United States
Park Nicollet Clinic - Saint Louis Park

Saint Louis Park, Minnesota, 55416, United States
Queen's Cancer Center - Kuakini

Honolulu, Hawaii, 96817, United States
Queen's Cancer Center - Pearlridge

‘Aiea, Hawaii, 96701, United States
Queen's Medical Center

Honolulu, Hawaii, 96813, United States
Radiation Oncology Centers of Nevada Central

Las Vegas, Nevada, 89106, United States
Radiation Oncology Centers of Nevada Southeast

Las Vegas, Nevada, 89119, United States
Regional West Medical Center Cancer Center

Scottsbluff, Nebraska, 69361, United States
Regions Hospital

Saint Paul, Minnesota, 55101, United States
Reid Health

Richmond, Indiana, 47374, United States
Rice Memorial Hospital

Willmar, Minnesota, 56201, United States
Ridgeview Medical Center

Waconia, Minnesota, 55387, United States
Saint Francis Regional Medical Center

Shakopee, Minnesota, 55379, United States
Saint John's Hospital - Healtheast

Maplewood, Minnesota, 55109, United States
Saint Louis Cancer and Breast Institute-South City

St Louis, Missouri, 63109, United States
Saint Mary's Regional Medical Center

Reno, Nevada, 89503, United States
Sibley Memorial Hospital

Washington D.C., District of Columbia, 20016, United States
Siouxland Regional Cancer Center

Sioux City, Iowa, 51101, United States
Siteman Cancer Center at Saint Peters Hospital

City of Saint Peters, Missouri, 63376, United States
Siteman Cancer Center at West County Hospital

Creve Coeur, Missouri, 63141, United States
Siteman Cancer Center-South County

St Louis, Missouri, 63129, United States
Southeast Nebraska Cancer Center - 68th Street Place

Lincoln, Nebraska, 68516, United States
Sovah Health Martinsville

Martinsville, Virginia, 24115, United States
Springfield Regional Cancer Center

Springfield, Ohio, 45504, United States
Springfield Regional Medical Center

Springfield, Ohio, 45505, United States
Straub Clinic and Hospital

Honolulu, Hawaii, 96813, United States
Summerlin Hospital Medical Center

Las Vegas, Nevada, 89144, United States
TidalHealth Nanticoke / Allen Cancer Center

Seaford, Delaware, 19973, United States
United Hospital

Saint Paul, Minnesota, 55102, United States
Unity Hospital

Fridley, Minnesota, 55432, United States
University Health Network-Princess Margaret Hospital

Toronto, Ontario, M5G 2M9, Canada
University of Alabama at Birmingham Cancer Center

Birmingham, Alabama, 35233, United States
University of Arkansas for Medical Sciences

Little Rock, Arkansas, 72205, United States
University of Hawaii Cancer Center

Honolulu, Hawaii, 96813, United States
University of Iowa/Holden Comprehensive Cancer Center

Iowa City, Iowa, 52242, United States
University of Michigan Comprehensive Cancer Center

Ann Arbor, Michigan, 48109, United States
University of Oklahoma Health Sciences Center

Oklahoma City, Oklahoma, 73104, United States
Upper Valley Medical Center

Troy, Ohio, 45373, United States
Washington University School of Medicine

St Louis, Missouri, 63110, United States
Wayne Hospital

Greenville, Ohio, 45331, United States
Wilcox Memorial Hospital and Kauai Medical Clinic

Lihue, Hawaii, 96766, United States
Wright-Patterson Medical Center

Wright-Patterson Air Force Base, Ohio, 45433, United States
Yale University

New Haven, Connecticut, 06520, United States

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