NCT02066181

Brief 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. \[Funding Source - FDA OOPD\]

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
87

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Mar 2014

Longer than P75 for phase_3

Geographic Reach
2 countries

150 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

February 17, 2014

Completed
2 days until next milestone

First Posted

Study publicly available on registry

February 19, 2014

Completed
1 month until next milestone

Study Start

First participant enrolled

March 21, 2014

Completed
5.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 3, 2019

Completed
3 months until next milestone

Results Posted

Study results publicly available

September 20, 2019

Completed
3.2 years until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2022

Completed
Last Updated

June 7, 2023

Status Verified

June 1, 2023

Enrollment Period

5.3 years

First QC Date

February 17, 2014

Results QC Date

March 22, 2019

Last Update Submit

June 6, 2023

Conditions

Outcome Measures

Primary Outcomes (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.

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

Secondary Outcomes (5)

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

    Up to 3 years

  • Time to Surgical Intervention During Treatment

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

  • Overall Survival

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

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

    Up to 3 years

  • Duration of Response

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

Other Outcomes (12)

  • Percent Change in Tumor Size by Response Evaluation Criteria in Solid Tumors Version 1.1 (Correlative Companion Study-Imaging Study)

    Baseline up to 3 years

  • Percent Changes in MRI T2 Signal (Correlative Companion Study-Imaging Study)

    Baseline up to 3 years

  • Time to Pain Progression Measured by the ?Worst Pain? Item of the Brief Pain Inventory Short Form (Correlative Companion Study- A091105-H01 QOL Study)

    Date of randomization to the earliest date that pain progression is observed, assessed up to 12 weeks

  • +9 more other outcomes

Study Arms (2)

Arm I (sorafenib tosylate)

EXPERIMENTAL

Patients receive sorafenib tosylate PO QD on days 1-28.

Other: Laboratory Biomarker AnalysisOther: Quality-of-Life AssessmentDrug: Sorafenib Tosylate

Arm II (placebo)

PLACEBO COMPARATOR

Patients receive placebo PO QD on days 1-28. Patients may crossover to Arm I upon disease progression.

Other: Laboratory Biomarker AnalysisOther: Placebo AdministrationOther: Quality-of-Life Assessment

Interventions

Optional correlative studies

Arm I (sorafenib tosylate)Arm II (placebo)

Given PO

Arm II (placebo)

Optional ancillary studies

Also known as: Quality of Life Assessment
Arm I (sorafenib tosylate)Arm II (placebo)

Given PO

Also known as: BAY 43-9006 Tosylate, BAY 54-9085, Nexavar, sorafenib
Arm I (sorafenib tosylate)

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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
  • +30 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (150)

University of Alabama at Birmingham Cancer Center

Birmingham, Alabama, 35233, United States

Location

University of Arkansas for Medical Sciences

Little Rock, Arkansas, 72205, United States

Location

PCR Oncology

Arroyo Grande, California, 93420, United States

Location

Yale University

New Haven, Connecticut, 06520, United States

Location

Beebe Medical Center

Lewes, Delaware, 19958, United States

Location

Christiana Gynecologic Oncology LLC

Newark, Delaware, 19713, United States

Location

Delaware Clinical and Laboratory Physicians PA

Newark, Delaware, 19713, United States

Location

Helen F Graham Cancer Center

Newark, Delaware, 19713, United States

Location

Medical Oncology Hematology Consultants PA

Newark, Delaware, 19713, United States

Location

Christiana Care Health System-Christiana Hospital

Newark, Delaware, 19718, United States

Location

Beebe Health Campus

Rehoboth Beach, Delaware, 19971, United States

Location

TidalHealth Nanticoke / Allen Cancer Center

Seaford, Delaware, 19973, United States

Location

Christiana Care Health System-Wilmington Hospital

Wilmington, Delaware, 19801, United States

Location

MedStar Georgetown University Hospital

Washington D.C., District of Columbia, 20007, United States

Location

MedStar Washington Hospital Center

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

Location

Sibley Memorial Hospital

Washington D.C., District of Columbia, 20016, United States

Location

Mayo Clinic in Florida

Jacksonville, Florida, 32224-9980, United States

Location

John B Amos Cancer Center

Columbus, Georgia, 31904, United States

Location

Low Country Cancer Care

Savannah, Georgia, 31404, United States

Location

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

Savannah, Georgia, 31405, United States

Location

Hawaii Cancer Care Inc - Waterfront Plaza

Honolulu, Hawaii, 96813, United States

Location

Queen's Medical Center

Honolulu, Hawaii, 96813, United States

Location

Straub Clinic and Hospital

Honolulu, Hawaii, 96813, United States

Location

University of Hawaii Cancer Center

Honolulu, Hawaii, 96813, United States

Location

Hawaii Cancer Care Inc-Liliha

Honolulu, Hawaii, 96817, United States

Location

Kuakini Medical Center

Honolulu, Hawaii, 96817, United States

Location

Queen's Cancer Center - Kuakini

Honolulu, Hawaii, 96817, United States

Location

Kapiolani Medical Center for Women and Children

Honolulu, Hawaii, 96826, United States

Location

Wilcox Memorial Hospital and Kauai Medical Clinic

Lihue, Hawaii, 96766, United States

Location

Pali Momi Medical Center

‘Aiea, Hawaii, 96701, United States

Location

Queen's Cancer Center - Pearlridge

‘Aiea, Hawaii, 96701, United States

Location

Northwestern University

Chicago, Illinois, 60611, United States

Location

Loyola University Medical Center

Maywood, Illinois, 60153, United States

Location

Good Samaritan Regional Health Center

Mount Vernon, Illinois, 62864, United States

Location

Reid Health

Richmond, Indiana, 47374, United States

Location

University of Iowa/Holden Comprehensive Cancer Center

Iowa City, Iowa, 52242, United States

Location

Siouxland Regional Cancer Center

Sioux City, Iowa, 51101, United States

Location

Oncology Hematology Care Inc-Crestview

Crestview Hills, Kentucky, 41017, United States

Location

Johns Hopkins University/Sidney Kimmel Cancer Center

Baltimore, Maryland, 21287, United States

Location

Brigham and Women's Hospital

Boston, Massachusetts, 02115, United States

Location

Dana-Farber Cancer Institute

Boston, Massachusetts, 02215, United States

Location

University of Michigan Comprehensive Cancer Center

Ann Arbor, Michigan, 48109, United States

Location

Fairview Ridges Hospital

Burnsville, Minnesota, 55337, United States

Location

Mercy Hospital

Coon Rapids, Minnesota, 55433, United States

Location

Fairview Southdale Hospital

Edina, Minnesota, 55435, United States

Location

Unity Hospital

Fridley, Minnesota, 55432, United States

Location

Hutchinson Area Health Care

Hutchinson, Minnesota, 55350, United States

Location

Minnesota Oncology Hematology PA-Maplewood

Maplewood, Minnesota, 55109, United States

Location

Saint John's Hospital - Healtheast

Maplewood, Minnesota, 55109, United States

Location

Abbott-Northwestern Hospital

Minneapolis, Minnesota, 55407, United States

Location

Hennepin County Medical Center

Minneapolis, Minnesota, 55415, United States

Location

Health Partners Inc

Minneapolis, Minnesota, 55454, United States

Location

New Ulm Medical Center

New Ulm, Minnesota, 56073, United States

Location

North Memorial Medical Health Center

Robbinsdale, Minnesota, 55422, United States

Location

Mayo Clinic in Rochester

Rochester, Minnesota, 55905, United States

Location

Metro Minnesota Community Oncology Research Consortium

Saint Louis Park, Minnesota, 55416, United States

Location

Park Nicollet Clinic - Saint Louis Park

Saint Louis Park, Minnesota, 55416, United States

Location

Regions Hospital

Saint Paul, Minnesota, 55101, United States

Location

United Hospital

Saint Paul, Minnesota, 55102, United States

Location

Saint Francis Regional Medical Center

Shakopee, Minnesota, 55379, United States

Location

Lakeview Hospital

Stillwater, Minnesota, 55082, United States

Location

Ridgeview Medical Center

Waconia, Minnesota, 55387, United States

Location

Rice Memorial Hospital

Willmar, Minnesota, 56201, United States

Location

Minnesota Oncology Hematology PA-Woodbury

Woodbury, Minnesota, 55125, United States

Location

Central Care Cancer Center - Bolivar

Bolivar, Missouri, 65613, United States

Location

Cox Cancer Center Branson

Branson, Missouri, 65616, United States

Location

Siteman Cancer Center at Saint Peters Hospital

City of Saint Peters, Missouri, 63376, United States

Location

Siteman Cancer Center at West County Hospital

Creve Coeur, Missouri, 63141, United States

Location

Freeman Health System

Joplin, Missouri, 64804, United States

Location

Mercy Hospital Joplin

Joplin, Missouri, 64804, United States

Location

Delbert Day Cancer Institute at PCRMC

Rolla, Missouri, 65401, United States

Location

Mercy Clinic-Rolla-Cancer and Hematology

Rolla, Missouri, 65401, United States

Location

Mercy Hospital Springfield

Springfield, Missouri, 65804, United States

Location

CoxHealth South Hospital

Springfield, Missouri, 65807, United States

Location

Saint Louis Cancer and Breast Institute-South City

St Louis, Missouri, 63109, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

Siteman Cancer Center-South County

St Louis, Missouri, 63129, United States

Location

Mercy Hospital Saint Louis

St Louis, Missouri, 63141, United States

Location

Nebraska Hematology and Oncology

Lincoln, Nebraska, 68506, United States

Location

Nebraska Cancer Research Center

Lincoln, Nebraska, 68510, United States

Location

Southeast Nebraska Cancer Center - 68th Street Place

Lincoln, Nebraska, 68516, United States

Location

Faith Regional Health Services Carson Cancer Center

Norfolk, Nebraska, 68701, United States

Location

Great Plains Health Callahan Cancer Center

North Platte, Nebraska, 69101, United States

Location

Missouri Valley Cancer Consortium

Omaha, Nebraska, 68106, United States

Location

Alegent Health Immanuel Medical Center

Omaha, Nebraska, 68122, United States

Location

Alegent Health Bergan Mercy Medical Center

Omaha, Nebraska, 68124, United States

Location

Nebraska Cancer Specialists - Omaha

Omaha, Nebraska, 68124, United States

Location

Alegent Health Lakeside Hospital

Omaha, Nebraska, 68130, United States

Location

Oncology Hematology West PC

Omaha, Nebraska, 68130, United States

Location

Creighton University Medical Center

Omaha, Nebraska, 68131, United States

Location

Regional West Medical Center Cancer Center

Scottsbluff, Nebraska, 69361, United States

Location

Cancer and Blood Specialists-Henderson

Henderson, Nevada, 89052, United States

Location

Comprehensive Cancer Centers of Nevada - Henderson

Henderson, Nevada, 89052, United States

Location

Las Vegas Cancer Center-Henderson

Henderson, Nevada, 89052, United States

Location

Comprehensive Cancer Centers of Nevada-Southeast Henderson

Henderson, Nevada, 89074, United States

Location

GenesisCare USA - Henderson

Henderson, Nevada, 89074, United States

Location

Cancer and Blood Specialists-Shadow

Las Vegas, Nevada, 89106, United States

Location

Radiation Oncology Centers of Nevada Central

Las Vegas, Nevada, 89106, United States

Location

GenesisCare USA - Las Vegas

Las Vegas, Nevada, 89109, United States

Location

HealthCare Partners Medical Group Oncology/Hematology-Maryland Parkway

Las Vegas, Nevada, 89109, United States

Location

HealthCare Partners Medical Group Oncology/Hematology-San Martin

Las Vegas, Nevada, 89113, United States

Location

Radiation Oncology Centers of Nevada Southeast

Las Vegas, Nevada, 89119, United States

Location

Cancer Therapy and Integrative Medicine

Las Vegas, Nevada, 89121, United States

Location

Cancer and Blood Specialists-Tenaya

Las Vegas, Nevada, 89128, United States

Location

Comprehensive Cancer Centers of Nevada - Northwest

Las Vegas, Nevada, 89128, United States

Location

GenesisCare USA - Vegas Tenaya

Las Vegas, Nevada, 89128, United States

Location

HealthCare Partners Medical Group Oncology/Hematology-Tenaya

Las Vegas, Nevada, 89128, United States

Location

Comprehensive Cancer Centers of Nevada-Summerlin

Las Vegas, Nevada, 89144, United States

Location

Summerlin Hospital Medical Center

Las Vegas, Nevada, 89144, United States

Location

Las Vegas Cancer Center-Medical Center

Las Vegas, Nevada, 89148-2405, United States

Location

Comprehensive Cancer Centers of Nevada

Las Vegas, Nevada, 89148, United States

Location

GenesisCare USA - Fort Apache

Las Vegas, Nevada, 89148, United States

Location

OptumCare Cancer Care at Fort Apache

Las Vegas, Nevada, 89148, United States

Location

HealthCare Partners Medical Group Oncology/Hematology-Centennial Hills

Las Vegas, Nevada, 89149, United States

Location

Comprehensive Cancer Centers of Nevada - Central Valley

Las Vegas, Nevada, 89169, United States

Location

Nevada Cancer Research Foundation NCORP

Las Vegas, Nevada, 89169, United States

Location

Saint Mary's Regional Medical Center

Reno, Nevada, 89503, United States

Location

Hackensack University Medical Center

Hackensack, New Jersey, 07601, United States

Location

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

New York, New York, 10032, United States

Location

Memorial Sloan Kettering Cancer Center

New York, New York, 10065, United States

Location

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

Miami Valley Hospital South

Centerville, Ohio, 45459, United States

Location

Oncology Hematology Care Inc-Eden Park

Cincinnati, Ohio, 45202, United States

Location

Oncology Hematology Care Inc-Mercy West

Cincinnati, Ohio, 45211, United States

Location

Oncology Hematology Care Inc-Anderson

Cincinnati, Ohio, 45230, United States

Location

Oncology Hematology Care Inc-Kenwood

Cincinnati, Ohio, 45236, United States

Location

Oncology Hematology Care Inc-Blue Ash

Cincinnati, Ohio, 45242, United States

Location

Good Samaritan Hospital - Dayton

Dayton, Ohio, 45406, United States

Location

Miami Valley Hospital

Dayton, Ohio, 45409, United States

Location

Miami Valley Hospital North

Dayton, Ohio, 45415, United States

Location

Dayton NCI Community Oncology Research Program

Dayton, Ohio, 45459, United States

Location

Oncology Hematology Care Inc-Healthplex

Fairfield, Ohio, 45014, United States

Location

Blanchard Valley Hospital

Findlay, Ohio, 45840, United States

Location

Atrium Medical Center-Middletown Regional Hospital

Franklin, Ohio, 45005-1066, United States

Location

Wayne Hospital

Greenville, Ohio, 45331, United States

Location

Kettering Medical Center

Kettering, Ohio, 45429, United States

Location

Springfield Regional Cancer Center

Springfield, Ohio, 45504, United States

Location

Springfield Regional Medical Center

Springfield, Ohio, 45505, United States

Location

Upper Valley Medical Center

Troy, Ohio, 45373, United States

Location

Wright-Patterson Medical Center

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

Location

University of Oklahoma Health Sciences Center

Oklahoma City, Oklahoma, 73104, United States

Location

Oklahoma Cancer Specialists and Research Institute-Tulsa

Tulsa, Oklahoma, 74146, United States

Location

Christiana Care Health System-Concord Health Center

Chadds Ford, Pennsylvania, 19317, United States

Location

Fox Chase Cancer Center

Philadelphia, Pennsylvania, 19111, United States

Location

M D Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Huntsman Cancer Institute/University of Utah

Salt Lake City, Utah, 84112, United States

Location

Sovah Health Martinsville

Martinsville, Virginia, 24115, United States

Location

Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

Cancer Center of Western Wisconsin

New Richmond, Wisconsin, 54017, United States

Location

University Health Network-Princess Margaret Hospital

Toronto, Ontario, M5G 2M9, Canada

Location

Related Publications (3)

  • 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.

Related Links

MeSH Terms

Conditions

Desmoid Tumors

Interventions

Sorafenib

Condition Hierarchy (Ancestors)

FibromaNeoplasms, Fibrous TissueNeoplasms, Connective TissueNeoplasms, Connective and Soft TissueNeoplasms by Histologic TypeNeoplasms

Intervention Hierarchy (Ancestors)

Phenylurea CompoundsUreaAmidesOrganic ChemicalsBenzene DerivativesHydrocarbons, AromaticHydrocarbons, CyclicHydrocarbonsNiacinamideNicotinic AcidsAcids, HeterocyclicHeterocyclic CompoundsPyridinesHeterocyclic Compounds, 1-Ring

Results Point of Contact

Title
Mrinal Murugesan Gounder M.D.
Organization
Memorial Sloan-Kettering Cancer Center

Study Officials

  • Mrinal M Gounder

    Alliance for Clinical Trials in Oncology

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
LTE60
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
DOUBLE
Who Masked
PARTICIPANT, INVESTIGATOR
Purpose
TREATMENT
Intervention Model
CROSSOVER
Sponsor Type
NIH
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 17, 2014

First Posted

February 19, 2014

Study Start

March 21, 2014

Primary Completion

July 3, 2019

Study Completion

December 1, 2022

Last Updated

June 7, 2023

Results First Posted

September 20, 2019

Record last verified: 2023-06

Locations