Sorafenib Tosylate in Treating Patients With Desmoid Tumors or Aggressive Fibromatosis
NCT ID: NCT02066181
Last Updated: 2023-06-07
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
87 participants
INTERVENTIONAL
2014-03-21
2022-12-01
Brief Summary
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Detailed Description
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I. To compare the progression-free survival (PFS) rates of patients with desmoid tumors (DT)/deep fibromatosis (DF) who receive either sorafenib (sorafenib tosylate) or placebo using a double-blinded randomized phase III study.
SECONDARY OBJECTIVES:
I. To assess toxicity. II. To assess time to surgical intervention. III. To assess tumor response rates and survival.
TERTIARY OBJECTIVES:
I. To evaluate changes in magnetic resonance imaging (MRI) Tesla (T)2 to predict (or correlate) with a biological effect such as tumor growth (by Response Evaluation Criteria in Solid Tumors \[RECIST\] version \[v\]1.1), and pain palliation. (Correlative companion study) II. The mechanism of action of sorafenib in DT/DF remains unknown. In patients consenting to undergo the paired tumor biopsies (A091105-ST1), treatment induced changes will be quantified by histology, gene expression profiling, proteomic changes and selected interrogation of key pathways by western blot and reverse transcription-polymerase chain reaction (RT-PCR). (Correlative companion study) III. To collect archival tissue, baseline (tumor, blood) and day 8 (tumor, blood) specimens for basic science research (A091105-ST1). (Correlative companion study) IV. To assess patient-reported adverse events and quality of life (QOL) as measured by the Patient-Reported Outcomes-Common Terminology Criteria for Adverse Events (PRO-CTCAE) and the single-item overall Linear Analogue Self-Assessment (LASA) (A091105-HO1). (Correlative companion study) V. To assess pain palliation measured by the "worst pain" item of the Brief Pain Inventory Short Form (A091105-HO1). (Correlative companion study)
OUTLINE: Patients are randomized to 1 of 2 treatment arms.
ARM I: Patients receive sorafenib tosylate orally (PO) once daily (QD) on days 1-28.
ARM II: Patients receive placebo PO QD on days 1-28. Patients may crossover to Arm I upon disease progression.
In both arms, courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed up annually for up to 3 years.
Conditions
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Study Design
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RANDOMIZED
CROSSOVER
TREATMENT
DOUBLE
Study Groups
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Arm I (sorafenib tosylate)
Patients receive sorafenib tosylate PO QD on days 1-28.
Laboratory Biomarker Analysis
Optional correlative studies
Quality-of-Life Assessment
Optional ancillary studies
Sorafenib Tosylate
Given PO
Arm II (placebo)
Patients receive placebo PO QD on days 1-28. Patients may crossover to Arm I upon disease progression.
Laboratory Biomarker Analysis
Optional correlative studies
Placebo Administration
Given PO
Quality-of-Life Assessment
Optional ancillary studies
Interventions
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Laboratory Biomarker Analysis
Optional correlative studies
Placebo Administration
Given PO
Quality-of-Life Assessment
Optional ancillary studies
Sorafenib Tosylate
Given PO
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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
18 Years
ALL
No
Sponsors
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National Cancer Institute (NCI)
NIH
Responsible Party
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Principal Investigators
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Mrinal M Gounder
Role: PRINCIPAL_INVESTIGATOR
Alliance for Clinical Trials in Oncology
Locations
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University of Alabama at Birmingham Cancer Center
Birmingham, Alabama, United States
University of Arkansas for Medical Sciences
Little Rock, Arkansas, United States
PCR Oncology
Arroyo Grande, California, United States
Yale University
New Haven, Connecticut, United States
Beebe Medical Center
Lewes, Delaware, United States
Christiana Gynecologic Oncology LLC
Newark, Delaware, United States
Delaware Clinical and Laboratory Physicians PA
Newark, Delaware, United States
Helen F Graham Cancer Center
Newark, Delaware, United States
Medical Oncology Hematology Consultants PA
Newark, Delaware, United States
Christiana Care Health System-Christiana Hospital
Newark, Delaware, United States
Beebe Health Campus
Rehoboth Beach, Delaware, United States
TidalHealth Nanticoke / Allen Cancer Center
Seaford, Delaware, United States
Christiana Care Health System-Wilmington Hospital
Wilmington, Delaware, United States
MedStar Georgetown University Hospital
Washington D.C., District of Columbia, United States
MedStar Washington Hospital Center
Washington D.C., District of Columbia, United States
Sibley Memorial Hospital
Washington D.C., District of Columbia, United States
Mayo Clinic in Florida
Jacksonville, Florida, United States
John B Amos Cancer Center
Columbus, Georgia, United States
Low Country Cancer Care
Savannah, Georgia, United States
Lewis Cancer and Research Pavilion at Saint Joseph's/Candler
Savannah, Georgia, United States
Hawaii Cancer Care Inc - Waterfront Plaza
Honolulu, Hawaii, United States
Queen's Medical Center
Honolulu, Hawaii, United States
Straub Clinic and Hospital
Honolulu, Hawaii, United States
University of Hawaii Cancer Center
Honolulu, Hawaii, United States
Hawaii Cancer Care Inc-Liliha
Honolulu, Hawaii, United States
Kuakini Medical Center
Honolulu, Hawaii, United States
Queen's Cancer Center - Kuakini
Honolulu, Hawaii, United States
Kapiolani Medical Center for Women and Children
Honolulu, Hawaii, United States
Wilcox Memorial Hospital and Kauai Medical Clinic
Lihue, Hawaii, United States
Pali Momi Medical Center
‘Aiea, Hawaii, United States
Queen's Cancer Center - Pearlridge
‘Aiea, Hawaii, United States
Northwestern University
Chicago, Illinois, United States
Loyola University Medical Center
Maywood, Illinois, United States
Good Samaritan Regional Health Center
Mount Vernon, Illinois, United States
Reid Health
Richmond, Indiana, United States
University of Iowa/Holden Comprehensive Cancer Center
Iowa City, Iowa, United States
Siouxland Regional Cancer Center
Sioux City, Iowa, United States
Oncology Hematology Care Inc-Crestview
Crestview Hills, Kentucky, United States
Johns Hopkins University/Sidney Kimmel Cancer Center
Baltimore, Maryland, United States
Brigham and Women's Hospital
Boston, Massachusetts, United States
Dana-Farber Cancer Institute
Boston, Massachusetts, United States
University of Michigan Comprehensive Cancer Center
Ann Arbor, Michigan, United States
Fairview Ridges Hospital
Burnsville, Minnesota, United States
Mercy Hospital
Coon Rapids, Minnesota, United States
Fairview Southdale Hospital
Edina, Minnesota, United States
Unity Hospital
Fridley, Minnesota, United States
Hutchinson Area Health Care
Hutchinson, Minnesota, United States
Minnesota Oncology Hematology PA-Maplewood
Maplewood, Minnesota, United States
Saint John's Hospital - Healtheast
Maplewood, Minnesota, United States
Abbott-Northwestern Hospital
Minneapolis, Minnesota, United States
Hennepin County Medical Center
Minneapolis, Minnesota, United States
Health Partners Inc
Minneapolis, Minnesota, United States
New Ulm Medical Center
New Ulm, Minnesota, United States
North Memorial Medical Health Center
Robbinsdale, Minnesota, United States
Mayo Clinic in Rochester
Rochester, Minnesota, United States
Metro Minnesota Community Oncology Research Consortium
Saint Louis Park, Minnesota, United States
Park Nicollet Clinic - Saint Louis Park
Saint Louis Park, Minnesota, United States
Regions Hospital
Saint Paul, Minnesota, United States
United Hospital
Saint Paul, Minnesota, United States
Saint Francis Regional Medical Center
Shakopee, Minnesota, United States
Lakeview Hospital
Stillwater, Minnesota, United States
Ridgeview Medical Center
Waconia, Minnesota, United States
Rice Memorial Hospital
Willmar, Minnesota, United States
Minnesota Oncology Hematology PA-Woodbury
Woodbury, Minnesota, United States
Central Care Cancer Center - Bolivar
Bolivar, Missouri, United States
Cox Cancer Center Branson
Branson, Missouri, United States
Siteman Cancer Center at Saint Peters Hospital
City of Saint Peters, Missouri, United States
Siteman Cancer Center at West County Hospital
Creve Coeur, Missouri, United States
Freeman Health System
Joplin, Missouri, United States
Mercy Hospital Joplin
Joplin, Missouri, United States
Delbert Day Cancer Institute at PCRMC
Rolla, Missouri, United States
Mercy Clinic-Rolla-Cancer and Hematology
Rolla, Missouri, United States
Mercy Hospital Springfield
Springfield, Missouri, United States
CoxHealth South Hospital
Springfield, Missouri, United States
Saint Louis Cancer and Breast Institute-South City
St Louis, Missouri, United States
Washington University School of Medicine
St Louis, Missouri, United States
Siteman Cancer Center-South County
St Louis, Missouri, United States
Mercy Hospital Saint Louis
St Louis, Missouri, United States
Nebraska Hematology and Oncology
Lincoln, Nebraska, United States
Nebraska Cancer Research Center
Lincoln, Nebraska, United States
Southeast Nebraska Cancer Center - 68th Street Place
Lincoln, Nebraska, United States
Faith Regional Health Services Carson Cancer Center
Norfolk, Nebraska, United States
Great Plains Health Callahan Cancer Center
North Platte, Nebraska, United States
Missouri Valley Cancer Consortium
Omaha, Nebraska, United States
Alegent Health Immanuel Medical Center
Omaha, Nebraska, United States
Alegent Health Bergan Mercy Medical Center
Omaha, Nebraska, United States
Nebraska Cancer Specialists - Omaha
Omaha, Nebraska, United States
Alegent Health Lakeside Hospital
Omaha, Nebraska, United States
Oncology Hematology West PC
Omaha, Nebraska, United States
Creighton University Medical Center
Omaha, Nebraska, United States
Regional West Medical Center Cancer Center
Scottsbluff, Nebraska, United States
Cancer and Blood Specialists-Henderson
Henderson, Nevada, United States
Comprehensive Cancer Centers of Nevada - Henderson
Henderson, Nevada, United States
Las Vegas Cancer Center-Henderson
Henderson, Nevada, United States
Comprehensive Cancer Centers of Nevada-Southeast Henderson
Henderson, Nevada, United States
GenesisCare USA - Henderson
Henderson, Nevada, United States
Cancer and Blood Specialists-Shadow
Las Vegas, Nevada, United States
Radiation Oncology Centers of Nevada Central
Las Vegas, Nevada, United States
GenesisCare USA - Las Vegas
Las Vegas, Nevada, United States
HealthCare Partners Medical Group Oncology/Hematology-Maryland Parkway
Las Vegas, Nevada, United States
HealthCare Partners Medical Group Oncology/Hematology-San Martin
Las Vegas, Nevada, United States
Radiation Oncology Centers of Nevada Southeast
Las Vegas, Nevada, United States
Cancer Therapy and Integrative Medicine
Las Vegas, Nevada, United States
Cancer and Blood Specialists-Tenaya
Las Vegas, Nevada, United States
Comprehensive Cancer Centers of Nevada - Northwest
Las Vegas, Nevada, United States
GenesisCare USA - Vegas Tenaya
Las Vegas, Nevada, United States
HealthCare Partners Medical Group Oncology/Hematology-Tenaya
Las Vegas, Nevada, United States
Comprehensive Cancer Centers of Nevada-Summerlin
Las Vegas, Nevada, United States
Summerlin Hospital Medical Center
Las Vegas, Nevada, United States
Las Vegas Cancer Center-Medical Center
Las Vegas, Nevada, United States
Comprehensive Cancer Centers of Nevada
Las Vegas, Nevada, United States
GenesisCare USA - Fort Apache
Las Vegas, Nevada, United States
OptumCare Cancer Care at Fort Apache
Las Vegas, Nevada, United States
HealthCare Partners Medical Group Oncology/Hematology-Centennial Hills
Las Vegas, Nevada, United States
Comprehensive Cancer Centers of Nevada - Central Valley
Las Vegas, Nevada, United States
Nevada Cancer Research Foundation NCORP
Las Vegas, Nevada, United States
Saint Mary's Regional Medical Center
Reno, Nevada, United States
Hackensack University Medical Center
Hackensack, New Jersey, United States
NYP/Columbia University Medical Center/Herbert Irving Comprehensive Cancer Center
New York, New York, United States
Memorial Sloan Kettering Cancer Center
New York, New York, United States
Duke University Medical Center
Durham, North Carolina, United States
Miami Valley Hospital South
Centerville, Ohio, United States
Oncology Hematology Care Inc-Eden Park
Cincinnati, Ohio, United States
Oncology Hematology Care Inc-Mercy West
Cincinnati, Ohio, United States
Oncology Hematology Care Inc-Anderson
Cincinnati, Ohio, United States
Oncology Hematology Care Inc-Kenwood
Cincinnati, Ohio, United States
Oncology Hematology Care Inc-Blue Ash
Cincinnati, Ohio, United States
Good Samaritan Hospital - Dayton
Dayton, Ohio, United States
Miami Valley Hospital
Dayton, Ohio, United States
Miami Valley Hospital North
Dayton, Ohio, United States
Dayton NCI Community Oncology Research Program
Dayton, Ohio, United States
Oncology Hematology Care Inc-Healthplex
Fairfield, Ohio, United States
Blanchard Valley Hospital
Findlay, Ohio, United States
Atrium Medical Center-Middletown Regional Hospital
Franklin, Ohio, United States
Wayne Hospital
Greenville, Ohio, United States
Kettering Medical Center
Kettering, Ohio, United States
Springfield Regional Cancer Center
Springfield, Ohio, United States
Springfield Regional Medical Center
Springfield, Ohio, United States
Upper Valley Medical Center
Troy, Ohio, United States
Wright-Patterson Medical Center
Wright-Patterson Air Force Base, Ohio, United States
University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, United States
Oklahoma Cancer Specialists and Research Institute-Tulsa
Tulsa, Oklahoma, United States
Christiana Care Health System-Concord Health Center
Chadds Ford, Pennsylvania, United States
Fox Chase Cancer Center
Philadelphia, Pennsylvania, United States
M D Anderson Cancer Center
Houston, Texas, United States
Huntsman Cancer Institute/University of Utah
Salt Lake City, Utah, United States
Sovah Health Martinsville
Martinsville, Virginia, United States
Medical College of Wisconsin
Milwaukee, Wisconsin, United States
Cancer Center of Western Wisconsin
New Richmond, Wisconsin, United States
University Health Network-Princess Margaret Hospital
Toronto, Ontario, Canada
Countries
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References
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Mazza GL, Petersen MM, Ginos B, Langlais BT, Heon N, Gounder MM, Mahoney MR, Zoroufy AJ, Schwartz GK, Rogak LJ, Thanarajasingam G, Basch E, Dueck AC. Missing data strategies for the Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) in Alliance A091105 and COMET-2. Qual Life Res. 2022 Apr;31(4):1069-1080. doi: 10.1007/s11136-021-02968-1. Epub 2021 Aug 21.
Basch E, Becker C, Rogak LJ, Schrag D, Reeve BB, Spears P, Smith ML, Gounder MM, Mahoney MR, Schwartz GK, Bennett AV, Mendoza TR, Cleeland CS, Sloan JA, Bruner DW, Schwab G, Atkinson TM, Thanarajasingam G, Bertagnolli MM, Dueck AC. Composite grading algorithm for the National Cancer Institute's Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE). Clin Trials. 2021 Feb;18(1):104-114. doi: 10.1177/1740774520975120. Epub 2020 Dec 1.
Gounder MM, Mahoney MR, Van Tine BA, Ravi V, Attia S, Deshpande HA, Gupta AA, Milhem MM, Conry RM, Movva S, Pishvaian MJ, Riedel RF, Sabagh T, Tap WD, Horvat N, Basch E, Schwartz LH, Maki RG, Agaram NP, Lefkowitz RA, Mazaheri Y, Yamashita R, Wright JJ, Dueck AC, Schwartz GK. Sorafenib for Advanced and Refractory Desmoid Tumors. N Engl J Med. 2018 Dec 20;379(25):2417-2428. doi: 10.1056/NEJMoa1805052.
Provided Documents
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Document Type: Study Protocol and Statistical Analysis Plan
Related Links
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Data Available: Select individual patient-level data from this trial can be requested from the NCTN/NCORP Data Archive.
Other Identifiers
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NCI-2014-00264
Identifier Type: REGISTRY
Identifier Source: secondary_id
PA091105_A04PAMDREVW01
Identifier Type: -
Identifier Source: secondary_id
A091105
Identifier Type: -
Identifier Source: secondary_id
A091105
Identifier Type: OTHER
Identifier Source: secondary_id
A091105
Identifier Type: OTHER
Identifier Source: secondary_id
NCI-2014-00264
Identifier Type: -
Identifier Source: org_study_id
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