AzTrials
AzTrials

Desmoid FibromatosisPhase 3NCT02066181

Sorafenib Tosylate in Treating Patients With Desmoid Tumors or Aggressive Fibromatosis

A Phase 3 study testing Laboratory Biomarker Analysis, Quality-of-Life Assessment and Sorafenib Tosylate in people with Desmoid Fibromatosis, run by National Cancer Institute (NCI) at 150 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

This randomized phase III trial compares the effects, good and/or bad, of sorafenib tosylate in treating patients with desmoid tumors or aggressive fibromatosis. Sorafenib tosylate may stop the growth of tumor cells by blocking some of the proteins needed for cell growth. [Funding Source - FDA OOPD] Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Desmoid Fibromatosis
What is being tested?
Laboratory Biomarker Analysis, Quality-of-Life Assessment and Sorafenib Tosylate
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
87 participants
Where
150 sitesin 2 countries
Study dates
21 Mar 2014to 1 Dec 2022
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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
Show all 45Show fewer
  • 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

Who cannot join

None listed.

Trial locations and contacts

150 sites in 2 countries.

150 sites in 2 countries

United States149 sites

  • Pali Momi Medical Center

    ‘Aiea, Hawaii, 96701

  • Queen's Cancer Center - Pearlridge

    ‘Aiea, Hawaii, 96701

  • University of Michigan Comprehensive Cancer Center

    Ann Arbor, Michigan, 48109

  • PCR Oncology

    Arroyo Grande, California, 93420

  • Johns Hopkins University/Sidney Kimmel Cancer Center

    Baltimore, Maryland, 21287

  • University of Alabama at Birmingham Cancer Center

    Birmingham, Alabama, 35233

  • Central Care Cancer Center - Bolivar

    Bolivar, Missouri, 65613

  • Brigham and Women's Hospital

    Boston, Massachusetts, 02115

  • Dana-Farber Cancer Institute

    Boston, Massachusetts, 02215

  • Cox Cancer Center Branson

    Branson, Missouri, 65616

  • Fairview Ridges Hospital

    Burnsville, Minnesota, 55337

  • Miami Valley Hospital South

    Centerville, Ohio, 45459

  • Christiana Care Health System-Concord Health Center

    Chadds Ford, Pennsylvania, 19317

  • Northwestern University

    Chicago, Illinois, 60611

  • Oncology Hematology Care Inc-Eden Park

    Cincinnati, Ohio, 45202

  • Oncology Hematology Care Inc-Mercy West

    Cincinnati, Ohio, 45211

  • Oncology Hematology Care Inc-Anderson

    Cincinnati, Ohio, 45230

  • Oncology Hematology Care Inc-Kenwood

    Cincinnati, Ohio, 45236

  • Oncology Hematology Care Inc-Blue Ash

    Cincinnati, Ohio, 45242

  • Siteman Cancer Center at Saint Peters Hospital

    City of Saint Peters, Missouri, 63376

  • John B Amos Cancer Center

    Columbus, Georgia, 31904

  • Mercy Hospital

    Coon Rapids, Minnesota, 55433

  • Oncology Hematology Care Inc-Crestview

    Crestview Hills, Kentucky, 41017

  • Siteman Cancer Center at West County Hospital

    Creve Coeur, Missouri, 63141

  • Good Samaritan Hospital - Dayton

    Dayton, Ohio, 45406

  • Miami Valley Hospital

    Dayton, Ohio, 45409

  • Miami Valley Hospital North

    Dayton, Ohio, 45415

  • Dayton NCI Community Oncology Research Program

    Dayton, Ohio, 45459

  • Duke University Medical Center

    Durham, North Carolina, 27710

  • Fairview Southdale Hospital

    Edina, Minnesota, 55435

  • Oncology Hematology Care Inc-Healthplex

    Fairfield, Ohio, 45014

  • Blanchard Valley Hospital

    Findlay, Ohio, 45840

  • Atrium Medical Center-Middletown Regional Hospital

    Franklin, Ohio, 45005-1066

  • Unity Hospital

    Fridley, Minnesota, 55432

  • Wayne Hospital

    Greenville, Ohio, 45331

  • Hackensack University Medical Center

    Hackensack, New Jersey, 07601

  • Cancer and Blood Specialists-Henderson

    Henderson, Nevada, 89052

  • Comprehensive Cancer Centers of Nevada - Henderson

    Henderson, Nevada, 89052

  • Las Vegas Cancer Center-Henderson

    Henderson, Nevada, 89052

  • Comprehensive Cancer Centers of Nevada-Southeast Henderson

    Henderson, Nevada, 89074

  • GenesisCare USA - Henderson

    Henderson, Nevada, 89074

  • Hawaii Cancer Care Inc - Waterfront Plaza

    Honolulu, Hawaii, 96813

  • Queen's Medical Center

    Honolulu, Hawaii, 96813

  • Straub Clinic and Hospital

    Honolulu, Hawaii, 96813

  • University of Hawaii Cancer Center

    Honolulu, Hawaii, 96813

  • Hawaii Cancer Care Inc-Liliha

    Honolulu, Hawaii, 96817

  • Kuakini Medical Center

    Honolulu, Hawaii, 96817

  • Queen's Cancer Center - Kuakini

    Honolulu, Hawaii, 96817

  • Kapiolani Medical Center for Women and Children

    Honolulu, Hawaii, 96826

  • M D Anderson Cancer Center

    Houston, Texas, 77030

  • Hutchinson Area Health Care

    Hutchinson, Minnesota, 55350

  • University of Iowa/Holden Comprehensive Cancer Center

    Iowa City, Iowa, 52242

  • Mayo Clinic in Florida

    Jacksonville, Florida, 32224-9980

  • Freeman Health System

    Joplin, Missouri, 64804

  • Mercy Hospital Joplin

    Joplin, Missouri, 64804

  • Kettering Medical Center

    Kettering, Ohio, 45429

  • Cancer and Blood Specialists-Shadow

    Las Vegas, Nevada, 89106

  • Radiation Oncology Centers of Nevada Central

    Las Vegas, Nevada, 89106

  • GenesisCare USA - Las Vegas

    Las Vegas, Nevada, 89109

  • HealthCare Partners Medical Group Oncology/Hematology-Maryland Parkway

    Las Vegas, Nevada, 89109

  • HealthCare Partners Medical Group Oncology/Hematology-San Martin

    Las Vegas, Nevada, 89113

  • Radiation Oncology Centers of Nevada Southeast

    Las Vegas, Nevada, 89119

  • Cancer Therapy and Integrative Medicine

    Las Vegas, Nevada, 89121

  • Cancer and Blood Specialists-Tenaya

    Las Vegas, Nevada, 89128

  • Comprehensive Cancer Centers of Nevada - Northwest

    Las Vegas, Nevada, 89128

  • GenesisCare USA - Vegas Tenaya

    Las Vegas, Nevada, 89128

  • HealthCare Partners Medical Group Oncology/Hematology-Tenaya

    Las Vegas, Nevada, 89128

  • Comprehensive Cancer Centers of Nevada-Summerlin

    Las Vegas, Nevada, 89144

  • Summerlin Hospital Medical Center

    Las Vegas, Nevada, 89144

  • Las Vegas Cancer Center-Medical Center

    Las Vegas, Nevada, 89148-2405

  • Comprehensive Cancer Centers of Nevada

    Las Vegas, Nevada, 89148

  • GenesisCare USA - Fort Apache

    Las Vegas, Nevada, 89148

  • OptumCare Cancer Care at Fort Apache

    Las Vegas, Nevada, 89148

  • HealthCare Partners Medical Group Oncology/Hematology-Centennial Hills

    Las Vegas, Nevada, 89149

  • Comprehensive Cancer Centers of Nevada - Central Valley

    Las Vegas, Nevada, 89169

  • Nevada Cancer Research Foundation NCORP

    Las Vegas, Nevada, 89169

  • Beebe Medical Center

    Lewes, Delaware, 19958

  • Wilcox Memorial Hospital and Kauai Medical Clinic

    Lihue, Hawaii, 96766

  • Nebraska Hematology and Oncology

    Lincoln, Nebraska, 68506

  • Nebraska Cancer Research Center

    Lincoln, Nebraska, 68510

  • Southeast Nebraska Cancer Center - 68th Street Place

    Lincoln, Nebraska, 68516

  • University of Arkansas for Medical Sciences

    Little Rock, Arkansas, 72205

  • Minnesota Oncology Hematology PA-Maplewood

    Maplewood, Minnesota, 55109

  • Saint John's Hospital - Healtheast

    Maplewood, Minnesota, 55109

  • Sovah Health Martinsville

    Martinsville, Virginia, 24115

  • Loyola University Medical Center

    Maywood, Illinois, 60153

  • Medical College of Wisconsin

    Milwaukee, Wisconsin, 53226

  • Abbott-Northwestern Hospital

    Minneapolis, Minnesota, 55407

  • Hennepin County Medical Center

    Minneapolis, Minnesota, 55415

  • Health Partners Inc

    Minneapolis, Minnesota, 55454

  • Good Samaritan Regional Health Center

    Mount Vernon, Illinois, 62864

  • Yale University

    New Haven, Connecticut, 06520

  • Cancer Center of Western Wisconsin

    New Richmond, Wisconsin, 54017

  • New Ulm Medical Center

    New Ulm, Minnesota, 56073

  • NYP/Columbia University Medical Center/Herbert Irving Comprehensive Cancer Center

    New York, New York, 10032

  • Memorial Sloan Kettering Cancer Center

    New York, New York, 10065

  • Christiana Gynecologic Oncology LLC

    Newark, Delaware, 19713

  • Delaware Clinical and Laboratory Physicians PA

    Newark, Delaware, 19713

  • Helen F Graham Cancer Center

    Newark, Delaware, 19713

  • Medical Oncology Hematology Consultants PA

    Newark, Delaware, 19713

  • Christiana Care Health System-Christiana Hospital

    Newark, Delaware, 19718

  • Faith Regional Health Services Carson Cancer Center

    Norfolk, Nebraska, 68701

  • Great Plains Health Callahan Cancer Center

    North Platte, Nebraska, 69101

  • University of Oklahoma Health Sciences Center

    Oklahoma City, Oklahoma, 73104

  • Missouri Valley Cancer Consortium

    Omaha, Nebraska, 68106

  • Alegent Health Immanuel Medical Center

    Omaha, Nebraska, 68122

  • Alegent Health Bergan Mercy Medical Center

    Omaha, Nebraska, 68124

  • Nebraska Cancer Specialists - Omaha

    Omaha, Nebraska, 68124

  • Alegent Health Lakeside Hospital

    Omaha, Nebraska, 68130

  • Oncology Hematology West PC

    Omaha, Nebraska, 68130

  • Creighton University Medical Center

    Omaha, Nebraska, 68131

  • Fox Chase Cancer Center

    Philadelphia, Pennsylvania, 19111

  • Beebe Health Campus

    Rehoboth Beach, Delaware, 19971

  • Saint Mary's Regional Medical Center

    Reno, Nevada, 89503

  • Reid Health

    Richmond, Indiana, 47374

  • North Memorial Medical Health Center

    Robbinsdale, Minnesota, 55422

  • Mayo Clinic in Rochester

    Rochester, Minnesota, 55905

  • Delbert Day Cancer Institute at PCRMC

    Rolla, Missouri, 65401

  • Mercy Clinic-Rolla-Cancer and Hematology

    Rolla, Missouri, 65401

  • Metro Minnesota Community Oncology Research Consortium

    Saint Louis Park, Minnesota, 55416

  • Park Nicollet Clinic - Saint Louis Park

    Saint Louis Park, Minnesota, 55416

  • Regions Hospital

    Saint Paul, Minnesota, 55101

  • United Hospital

    Saint Paul, Minnesota, 55102

  • Huntsman Cancer Institute/University of Utah

    Salt Lake City, Utah, 84112

  • Low Country Cancer Care

    Savannah, Georgia, 31404

  • Lewis Cancer and Research Pavilion at Saint Joseph's/Candler

    Savannah, Georgia, 31405

  • Regional West Medical Center Cancer Center

    Scottsbluff, Nebraska, 69361

  • TidalHealth Nanticoke / Allen Cancer Center

    Seaford, Delaware, 19973

  • Saint Francis Regional Medical Center

    Shakopee, Minnesota, 55379

  • Siouxland Regional Cancer Center

    Sioux City, Iowa, 51101

  • Mercy Hospital Springfield

    Springfield, Missouri, 65804

  • CoxHealth South Hospital

    Springfield, Missouri, 65807

  • Springfield Regional Cancer Center

    Springfield, Ohio, 45504

  • Springfield Regional Medical Center

    Springfield, Ohio, 45505

  • Saint Louis Cancer and Breast Institute-South City

    St Louis, Missouri, 63109

  • Washington University School of Medicine

    St Louis, Missouri, 63110

  • Siteman Cancer Center-South County

    St Louis, Missouri, 63129

  • Mercy Hospital Saint Louis

    St Louis, Missouri, 63141

  • Lakeview Hospital

    Stillwater, Minnesota, 55082

  • Upper Valley Medical Center

    Troy, Ohio, 45373

  • Oklahoma Cancer Specialists and Research Institute-Tulsa

    Tulsa, Oklahoma, 74146

  • Ridgeview Medical Center

    Waconia, Minnesota, 55387

  • MedStar Georgetown University Hospital

    Washington D.C., District of Columbia, 20007

  • MedStar Washington Hospital Center

    Washington D.C., District of Columbia, 20010

  • Sibley Memorial Hospital

    Washington D.C., District of Columbia, 20016

  • Rice Memorial Hospital

    Willmar, Minnesota, 56201

  • Christiana Care Health System-Wilmington Hospital

    Wilmington, Delaware, 19801

  • Minnesota Oncology Hematology PA-Woodbury

    Woodbury, Minnesota, 55125

  • Wright-Patterson Medical Center

    Wright-Patterson Air Force Base, Ohio, 45433

Other trials in United States

Canada1 site

  • University Health Network-Princess Margaret Hospital

    Toronto, Ontario, M5G 2M9

Other trials in Canada

About this study

Official title: A Phase III, Double Blind, Randomized, Placebo-Controlled Trial of Sorafenib in Desmoid Tumors or Aggressive Fibromatosis (DT/DF)

This randomized phase III trial compares the effects, good and/or bad, of sorafenib tosylate in treating patients with desmoid tumors or aggressive fibromatosis. Sorafenib tosylate may stop the growth of tumor cells by blocking some of the proteins needed for cell growth. [Funding Source - FDA OOPD]

Read the detailed description

PRIMARY OBJECTIVES:

  1. 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:

  1. To assess toxicity. II. To assess time to surgical intervention. III. To assess tumor response rates and survival.

TERTIARY OBJECTIVES:

  1. 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.

Being tested
Laboratory Biomarker Analysis, Placebo Administration, Quality-of-Life Assessment, Sorafenib Tosylate
Medical subject headings
Desmoid Tumors

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Laboratory Biomarker AnalysisOther

    Optional correlative studies

  • Placebo AdministrationOther

    Given PO

  • Quality-of-Life AssessmentOther

    Also called Quality of Life Assessment

    Optional ancillary studies

  • Sorafenib TosylateDrug

    Also called BAY 43-9006 Tosylate, BAY 54-9085, Nexavar, sorafenib

    Given PO

Study groups in NCT02066181
Study groupRoleWhat they receive
Arm I (sorafenib tosylate)ExperimentalPatients receive sorafenib tosylate PO QD on days 1-28.
Receives: Other: Laboratory Biomarker Analysis; Other: Quality-of-Life Assessment; Drug: Sorafenib Tosylate
Arm II (placebo)Placebo comparatorPatients receive placebo PO QD on days 1-28. Patients may crossover to Arm I upon disease progression.
Receives: Other: Laboratory Biomarker Analysis; Other: Placebo Administration; Other: Quality-of-Life Assessment

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Progression-free Survival(PFS) Rate

    PFS is defined as the time from randomization to the first occurrence of progression or death due to any cause. If no event exists, the PFS will be censored at the last disease assessment. Data following cross over will be analyzed and summarized separately from the data from the main course of treatment for these patients in an exploratory and hypothesis generating manner. Intention to treat principles will be used. Patient disease status was evaluated using RECSIT v1.1. Patients ending treatment for symptomatic deterioration without radiographic evidence of PD, were classified as having PD. Otherwise, patients not yet showing disease progression were classified as having no progression at the most recent disease assessment and in the following cases: crossing over to receive sorafenib, date of first non-protocol directed anti-cancer therapy, lost to follow-up, withdrawal of consent, and changing imaging methods from that which was used at study entry.

    Measured: Time from randomization to the first occurrence of progression or death due to any cause, assessed up to 3 years

Secondary outcomes (5)

  1. Incidence of Adverse Events, Using the Patient Reported Outcomes-Common Terminology Criteria in Adverse Events Version 4.0

    INCLUDED IN THE ADVERSE EVENTS PORTION OF THE RESULTS SECTION. Frequency tables, summary statistics, and categorical analysis will be used to compare the distributions of toxicity for patients treated with sorafenib tosylate vs placebo. Data for patients who have crossed over or having received surgical or radiotherapy intervention will be summarized independently from their primary course of study treatment in an exploratory and hypothesis generating manner.

    Measured: Up to 3 years

  2. Time to Surgical Intervention During Treatment

    A log rank test will be used to compare the distributions of time to surgical intervention between the two arms using a 2-sided test and alpha=0.05 level of significance. Kaplan-Meier methodology will be used to estimate various time points and 95% confidence intervals will be calculated for these estimates. Surgery will be classified by outcome (eg, complete-macroscopic, complete-microscopic, or partial), type, location (eg, limb), thereafter analyzed by categorical analysis and descriptive statistics. Non-parametric methods will be used, as appropriate. Too few patients had surgery during treatment to perform analysis.

    Measured: Time between randomization to the patient undergoing therapeutic surgical resection for this disease, assessed up to 3 years

  3. Overall Survival

    Kaplan-Meier methodology and log rank tests will be used to compare overall survival between the groups at various time points (eg, 1 year rate, 2 year rate, etc) and 95% confidence intervals will be calculated for these estimates. Data following crossover will be analyzed and summarized separately from the main course of treatment for these patients in an exploratory and hypothesis generating manner.

    Measured: Time between the date of randomization to until death, assessed up to 3 years

  4. Best Objective Status Between the Two Treatment Arms According to Response Evaluation Criteria in Solid Tumors Version 1.1

    Compared between the two treatment arms and using the Cochran-Mantel-Haenszel test. Complete Response (CR): All of the following must be true: a. Disappearance of all target lesions. b. Each target lymph node must have reduction in short axis to <1.0 cm. Partial Response (PR): At least a 30% decrease in PBSD (sum of the longest diameter for all target lesions plus the sum of the short axis of all the target lymph nodes at current evaluation) taking as reference the BSD. Patients on Arm II (placebo) who crossover are censored for Best Objective Status at the time of crossover.

    Measured: Up to 3 years

  5. Duration of Response

    Kaplan Meier methodology will be used to estimate the distribution of duration of response and the log-rank test will be used to test for a difference in duration of response between the two arms. Patients on Arm II (placebo) who crossover are censored for Duration of Response at the time of crossover.

    Measured: Time between first tumor response and progression, assessed up to 3 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted19 Feb 2014 (estimated)
  2. Study start21 Mar 2014
  3. Primary completion3 Jul 2019
  4. Study completion1 Dec 2022

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Crossover
Masking
Double blind
Who is masked
Participant, Investigator
Enrolment
87 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02066181
Sponsor's protocol ID
NCI-2014-00264
Other IDs
NCI-2014-00264, PA091105_A04PAMDREVW01, A091105, A091105, A091105, R01FD005105, U10CA180821, U10CA031946
Status last verified
May 2026
First submitted
17 Feb 2014
Last updated
24 Jun 2026
Data monitoring committee
No

Publications

  • 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.

    PubMed 34420143

  • 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.

    PubMed 33258687

  • 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.

    PubMed 30575484

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Laboratory Biomarker Analysis, Quality-of-Life Assessment and Sorafenib Tosylate in people with Desmoid Fibromatosis, run by National Cancer Institute (NCI) at 150 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 45 inclusion and 0 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 24 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 150 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 20 Sep 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Progression-free Survival(PFS) Rate (time frame: Time from randomization to the first occurrence of progression or death due to any cause, assessed up to 3 years). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02066181, which also lists 3 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Jun 2026.

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