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CompletedNCT02066181Updated Jun 24, 2026Results posted

Sorafenib Tosylate in Treating Patients With Desmoid Tumors or Aggressive Fibromatosis

A Phase 3 interventional study of Laboratory Biomarker Analysis and Placebo Administration in Desmoid Fibromatosis, sponsored by National Cancer Institute (NCI). Completed at 150 sites in 2 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-06-24.

Sponsored by National Cancer Institute (NCI) · Phase 3, Interventional, and Treatment

Phase
Phase 3
Study type
Interventional
Enrollment
87
Allocation
Randomized
Ages
18 Years and older
Sex
All
01

Study 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 the detailed description

PRIMARY OBJECTIVES:

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.

02

Conditions studied

  • Desmoid Fibromatosis

Browse trials for

03

In context

Desmoid Tumors

33 studies on the registry are indexed under Desmoid Tumors; 17 are open to participants now.

This study's enrollment of 87 is above the median of 50 across 21 interventional studies indexed under Desmoid Tumors.

Browse Desmoid Tumors studies →

Lead sponsor

National Cancer Institute (NCI) is the lead sponsor of 3,506 studies on the registry; 334 are open to participants now.

Of its 402 completed or terminated interventional studies of FDA-regulated products, 365 (91%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Eligibility criteria

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
05

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Crossover assignment
Masking
Double (Participant, Investigator)
Enrollment
87 participants (actual)

Study arms

  • Experimental
    Arm I (sorafenib tosylate)

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

    Other: Laboratory Biomarker Analysis · Other: Quality-of-Life Assessment · Drug: Sorafenib Tosylate

  • Placebo comparator
    Arm II (placebo)

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

    Other: Laboratory Biomarker Analysis · Other: Placebo Administration · Other: Quality-of-Life Assessment

Interventions

  • OtherLaboratory Biomarker Analysis

    Optional correlative studies

  • OtherPlacebo Administration

    Given PO

  • OtherQuality-of-Life Assessment

    Optional ancillary studies

    Also known as: Quality of Life Assessment

  • DrugSorafenib Tosylate

    Given PO

    Also known as: BAY 43-9006 Tosylate, BAY 54-9085, Nexavar, sorafenib

06

What researchers measure

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

Secondary outcomes

  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.

    Time frame: 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.

    Time frame: 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.

    Time frame: 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.

    Time frame: 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.

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

Other outcomes

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

    Best response (ordinal variable) and percent T2 signal change (continuous) will be correlated by Spearman?s rho.

    Time frame: Baseline up to 3 years

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

    Percent T2 signal change (continuous) will be correlated by Spearman?s rho. The percent changes in MRI T2 signal from Week 8 to subsequent imaging will be compared between groups with \> 30% pain palliation using t-test or a nonparametric alternative (e.g., Wilcoxon rank-sum test).

    Time frame: Baseline up to 3 years

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

    Defined as a \>= 30% increase compared with baseline in the worst pain intensity score (BPI-SF ?worst pain? item) or either a \>= 30% increase in the average daily use of any type of opioid narcotic or the addition of a new opioid narcotic compared with baseline. Estimated for each arm using Kaplan-Meier estimates and will be compared between arms using a log- rank test. Descriptive statistics will include means, standard deviations, medians, and ranges for each continuous or ordinal scale/subscale/item by group at each time point.

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

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

    Defined at each time point as \>= 30% decrease from baseline in the worst pain intensity score (BPI-SF ?worst pain? item), with neither a concomitant \>= 30% increase in average daily use of any opioid narcotic, nor addition of any new opioid narcotic, relative to baseline. Estimated for each arm using Kaplan-Meier estimates and will be compared between arms using a log- rank test. Descriptive statistics will include means, standard deviations, medians, and ranges for each continuous or ordinal scale/subscale/item by group at each time point.

    Time frame: Date of randomization to the earliest date that confirmed pain palliation is observed, assessed up to 12 weeks

  5. Duration of Pain Palliation Measured by the ?Worst Pain? Item of the Brief Pain Inventory Short Form (Correlative Companion Study- A091105-H01 QOL Study)

    Defined for all patients who experience confirmed pain palliation. Descriptive statistics will include means, standard deviations, medians, and ranges for each continuous or ordinal scale/subscale/item by group at each time point. Descriptive graphical techniques will include mean plots by group for each continuous or ordinal scale/subscale/item. Relative frequencies of responses for each ordinal item will also be generated at each time point by group.

    Time frame: Time from the earliest date that confirmed pain palliation is observed to the earliest date that pain progression is observed, assessed up to 12 weeks

  6. Rate of Pain Palliation Measured by the ?Worst Pain? Item of the Brief Pain Inventory Short Form (Correlative Companion Study-A091105-H01 QOL Study)

    Rate of pain palliation at week 8 confirmed as week 12 will be compared between arms using a two-sided alpha=0.05 chi-squared tests at the time of the final analysis. Descriptive statistics will include means, standard deviations, medians, and ranges for each continuous or ordinal scale/subscale/item by group at each time point. Descriptive graphical techniques will include mean plots by group for each continuous or ordinal scale/subscale/item. Relative frequencies of responses for each ordinal item will also be generated at each time point by group.

    Time frame: Baseline up to 12 weeks

  7. Cadherin-associated Protein, Beta 1 (CTNNB1) Genotype (Correlative Companion Study-A091105-ST1 Study)

    Associations among the possible predictors of response to sorafenib and CTNNBI mutations will be examined using Fisher?s exact test, Kruskal-Wallis test, or Spearman?s correlation coefficient as appropriate. Strata will be compared by using the log-rank test. Multivariate models will be constructed by introducing all variables aforementioned simultaneously into the model and then eliminating variables using the backward selection method. P values will be two-tailed and considered significant at alpha 0.05.

    Time frame: Up to 3 years

  8. False Discovery Rate (Correlative Companion Study- A091105-ST1 Study)

    Permutation testing of the same selection will be performed 1000 times and the sample group labels switched around in each permutation. A two-sided t-test will be used to identify differentially expressed genes on log transformed data and those with a twofold change. False discovery rate will be assessed by permutation testing (n = 1000) of the sample group labels. Enrichment will be assessed by one-sided Fisher?s exact test with estimated false discovery rate.

    Time frame: Up to day 8

  9. Treatment-specific Gene Expression Signature (Correlative Companion Study- A091105-ST1 Study)

    The analysis will identify over- and under-expressed genes in pre-treatment and day 8 biopsies as compared to all control samples.

    Time frame: Up to day 8

  10. Changes in Immunohistochemistry Score of Vascular Endothelial Growth Factor (Correlative Companion Study- A091105-ST1 Study)

    Compared by paired t-test. Quantitative changes will be correlated with disease status at 1 year by Fishers exact test.

    Time frame: Baseline up to day 8

  11. Changes in Immunohistochemistry Score of Platelet-derived Growth Factor Receptor (Correlative Companion Study- A091105-ST1 Study)

    Compared by paired t-test. Quantitative changes will be correlated with disease status at 1 year by Fishers exact test.

    Time frame: Baseline up to day 8

  12. Changes in Immunohistochemistry Score of Beta-catenin Cytoplasm/Nuclear Ratio (Correlative Companion Study- A091105-ST1 Study)

    Compared by paired t-test. Quantitative changes will be correlated with disease status at 1 year by Fishers exact test.

    Time frame: Baseline up to day 8

07

Results

Posted Sep 20, 2019

Participant flow

Participant flow — Overall Study
MilestoneArm I (Sorafenib Tosylate)Arm II (Placebo)
Started5037
Crossed over028
Completed4936
Not completed11
Withdrew: Withdrawal by subject11

Outcome measures

PrimaryProgression-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 frame:
Time from randomization to the first occurrence of progression or death due to any cause, assessed up to 3 years
Reported as:
Count of participants · Participants
Progression-free Survival(PFS) Rate
ParticipantsArm I (Sorafenib Tosylate)Arm II (Placebo)
Progressed or Died722
Alive and Progression Free4213
Statistical analysis
  • Arm I (Sorafenib Tosylate) vs Arm II (Placebo) · Log Rank · p = <0.001 · Hazard ratio (hr): 11.3
SecondaryIncidence 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.

Time frame:
Up to 3 years
Reported as:
Count of participants · Participants
Incidence of Adverse Events, Using the Patient Reported Outcomes-Common Terminology Criteria in Adverse Events Version 4.0
ParticipantsArm I (Sorafenib Tosylate)Arm II (Placebo)Crossover Patients
Incidence of Adverse Events, Using the Patient Reported Outcomes-Common Terminology Criteria in Adverse Events Version 4.049360
SecondaryTime 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.

Time frame:
Time between randomization to the patient undergoing therapeutic surgical resection for this disease, assessed up to 3 years
Reported as:
Count of participants · Participants
Time to Surgical Intervention During Treatment
ParticipantsArm I (Sorafenib Tosylate)Arm II (Placebo)Crossover Patients
Had surgery during treatment110
Didn't have surgery during treatment48350
SecondaryOverall 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.

Time frame:
Time between the date of randomization to until death, assessed up to 3 years
Reported as:
Count of participants · Participants
Overall Survival
ParticipantsArm I (Sorafenib Tosylate)Arm II (Placebo)Crossover Patients
Deaths100
Alive48350
SecondaryBest 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.

Time frame:
Up to 3 years
Reported as:
Count of participants · Participants
Best Objective Status Between the Two Treatment Arms According to Response Evaluation Criteria in Solid Tumors Version 1.1
ParticipantsArm I (Sorafenib Tosylate)Arm II (Placebo)
CR10
PR157
Stable Diseaes or Progression3328
SecondaryDuration 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.

Time frame:
Time between first tumor response and progression, assessed up to 3 years
Reported as:
Median · Months
Duration of Response
MonthsArm I (Sorafenib Tosylate)Arm II (Placebo)
Duration of Response14.7 (8.7 to 18.6)11.0 (4.8 to 12.8)
Other pre-specifiedPercent Change in Tumor Size by Response Evaluation Criteria in Solid Tumors Version 1.1 (Correlative Companion Study-Imaging Study)

Best response (ordinal variable) and percent T2 signal change (continuous) will be correlated by Spearman?s rho.

Time frame:
Baseline up to 3 years

Results for this outcome have not been posted.

Other pre-specifiedPercent Changes in MRI T2 Signal (Correlative Companion Study-Imaging Study)

Percent T2 signal change (continuous) will be correlated by Spearman?s rho. The percent changes in MRI T2 signal from Week 8 to subsequent imaging will be compared between groups with \> 30% pain palliation using t-test or a nonparametric alternative (e.g., Wilcoxon rank-sum test).

Time frame:
Baseline up to 3 years

Results for this outcome have not been posted.

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

Defined as a \>= 30% increase compared with baseline in the worst pain intensity score (BPI-SF ?worst pain? item) or either a \>= 30% increase in the average daily use of any type of opioid narcotic or the addition of a new opioid narcotic compared with baseline. Estimated for each arm using Kaplan-Meier estimates and will be compared between arms using a log- rank test. Descriptive statistics will include means, standard deviations, medians, and ranges for each continuous or ordinal scale/subscale/item by group at each time point.

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

Results for this outcome have not been posted.

Other pre-specifiedTime to Pain Palliation Measured by the ?Worst Pain? Item of the Brief Pain Inventory Short Form (Correlative Companion Study-A091105-H01 QOL Study)

Defined at each time point as \>= 30% decrease from baseline in the worst pain intensity score (BPI-SF ?worst pain? item), with neither a concomitant \>= 30% increase in average daily use of any opioid narcotic, nor addition of any new opioid narcotic, relative to baseline. Estimated for each arm using Kaplan-Meier estimates and will be compared between arms using a log- rank test. Descriptive statistics will include means, standard deviations, medians, and ranges for each continuous or ordinal scale/subscale/item by group at each time point.

Time frame:
Date of randomization to the earliest date that confirmed pain palliation is observed, assessed up to 12 weeks

Results for this outcome have not been posted.

Other pre-specifiedDuration of Pain Palliation Measured by the ?Worst Pain? Item of the Brief Pain Inventory Short Form (Correlative Companion Study- A091105-H01 QOL Study)

Defined for all patients who experience confirmed pain palliation. Descriptive statistics will include means, standard deviations, medians, and ranges for each continuous or ordinal scale/subscale/item by group at each time point. Descriptive graphical techniques will include mean plots by group for each continuous or ordinal scale/subscale/item. Relative frequencies of responses for each ordinal item will also be generated at each time point by group.

Time frame:
Time from the earliest date that confirmed pain palliation is observed to the earliest date that pain progression is observed, assessed up to 12 weeks

Results for this outcome have not been posted.

Other pre-specifiedRate of Pain Palliation Measured by the ?Worst Pain? Item of the Brief Pain Inventory Short Form (Correlative Companion Study-A091105-H01 QOL Study)

Rate of pain palliation at week 8 confirmed as week 12 will be compared between arms using a two-sided alpha=0.05 chi-squared tests at the time of the final analysis. Descriptive statistics will include means, standard deviations, medians, and ranges for each continuous or ordinal scale/subscale/item by group at each time point. Descriptive graphical techniques will include mean plots by group for each continuous or ordinal scale/subscale/item. Relative frequencies of responses for each ordinal item will also be generated at each time point by group.

Time frame:
Baseline up to 12 weeks

Results for this outcome have not been posted.

Other pre-specifiedCadherin-associated Protein, Beta 1 (CTNNB1) Genotype (Correlative Companion Study-A091105-ST1 Study)

Associations among the possible predictors of response to sorafenib and CTNNBI mutations will be examined using Fisher?s exact test, Kruskal-Wallis test, or Spearman?s correlation coefficient as appropriate. Strata will be compared by using the log-rank test. Multivariate models will be constructed by introducing all variables aforementioned simultaneously into the model and then eliminating variables using the backward selection method. P values will be two-tailed and considered significant at alpha 0.05.

Time frame:
Up to 3 years

Results for this outcome have not been posted.

Other pre-specifiedFalse Discovery Rate (Correlative Companion Study- A091105-ST1 Study)

Permutation testing of the same selection will be performed 1000 times and the sample group labels switched around in each permutation. A two-sided t-test will be used to identify differentially expressed genes on log transformed data and those with a twofold change. False discovery rate will be assessed by permutation testing (n = 1000) of the sample group labels. Enrichment will be assessed by one-sided Fisher?s exact test with estimated false discovery rate.

Time frame:
Up to day 8

Results for this outcome have not been posted.

Other pre-specifiedTreatment-specific Gene Expression Signature (Correlative Companion Study- A091105-ST1 Study)

The analysis will identify over- and under-expressed genes in pre-treatment and day 8 biopsies as compared to all control samples.

Time frame:
Up to day 8

Results for this outcome have not been posted.

Other pre-specifiedChanges in Immunohistochemistry Score of Vascular Endothelial Growth Factor (Correlative Companion Study- A091105-ST1 Study)

Compared by paired t-test. Quantitative changes will be correlated with disease status at 1 year by Fishers exact test.

Time frame:
Baseline up to day 8

Results for this outcome have not been posted.

Other pre-specifiedChanges in Immunohistochemistry Score of Platelet-derived Growth Factor Receptor (Correlative Companion Study- A091105-ST1 Study)

Compared by paired t-test. Quantitative changes will be correlated with disease status at 1 year by Fishers exact test.

Time frame:
Baseline up to day 8

Results for this outcome have not been posted.

Other pre-specifiedChanges in Immunohistochemistry Score of Beta-catenin Cytoplasm/Nuclear Ratio (Correlative Companion Study- A091105-ST1 Study)

Compared by paired t-test. Quantitative changes will be correlated with disease status at 1 year by Fishers exact test.

Time frame:
Baseline up to day 8

Results for this outcome have not been posted.

Adverse events

Collected over Up to 3 years. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Arm I (Sorafenib Tosylate)1/49 (2%)11/49 (22.4%)49/49 (100%)
Arm II (Placebo)0/36 (0%)6/36 (16.7%)34/36 (94.4%)
Crossover Group0/28 (0%)5/28 (17.9%)27/28 (96.4%)
Most frequent serious events
Showing 10 of 30
Most frequent serious events
EventArm I (Sorafenib Tosylate)Arm II (Placebo)Crossover Group
Small intestinal obstructionGastrointestinal disorders0/492/360/28
DiarrheaGastrointestinal disorders2/490/360/28
FatigueGeneral disorders2/490/360/28
Palmar-plantar erythrodysesthesia syndromeSkin and subcutaneous tissue disorders2/490/360/28
MalabsorptionGastrointestinal disorders0/490/361/28
AppendicitisInfections and infestations0/490/361/28
SepsisInfections and infestations0/490/361/28
Injury, poisoning and procedural complications - Other, specifyInjury, poisoning and procedural complications0/490/361/28
Alanine aminotransferase increasedInvestigations0/490/361/28
HypertensionVascular disorders1/491/361/28
Most frequent other events
Showing 10 of 154
Most frequent other events
EventArm I (Sorafenib Tosylate)Arm II (Placebo)Crossover Group
FatigueGeneral disorders36/4923/3621/28
Palmar-plantar erythrodysesthesia syndromeSkin and subcutaneous tissue disorders35/499/3619/28
HypertensionVascular disorders31/4914/3616/28
Papulopustular rashInfections and infestations30/497/3610/28
DiarrheaGastrointestinal disorders27/4912/3617/28
NauseaGastrointestinal disorders25/4915/3617/28
Abdominal painGastrointestinal disorders16/4913/3616/28
ArthralgiaMusculoskeletal and connective tissue disorders17/499/3614/28
MyalgiaMusculoskeletal and connective tissue disorders19/4912/3612/28
AlopeciaSkin and subcutaneous tissue disorders18/494/367/28

Baseline characteristics

Age, Continuous
Age, Continuous(years)Arm I (Sorafenib Tosylate)Arm II (Placebo)Total
Median37 (18 to 72)37 (21 to 67)37 (18 to 72)
Sex: Female, Male
Sex: Female, Male(Participants)Arm I (Sorafenib Tosylate)Arm II (Placebo)Total
Female342660
Male161127
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Arm I (Sorafenib Tosylate)Arm II (Placebo)Total
American Indian or Alaska Native011
Asian000
Native Hawaiian or Other Pacific Islander000
Black or African American459
White412970
More than one race000
Unknown or Not Reported527
08

Study locations

150 sites
  • University of Alabama at Birmingham Cancer Center
    Birmingham, Alabama 35233, United States
  • University of Arkansas for Medical Sciences
    Little Rock, Arkansas 72205, United States
  • PCR Oncology
    Arroyo Grande, California 93420, United States
  • Yale University
    New Haven, Connecticut 06520, United States
  • Beebe Medical Center
    Lewes, Delaware 19958, United States
  • Christiana Gynecologic Oncology LLC
    Newark, Delaware 19713, United States
  • Delaware Clinical and Laboratory Physicians PA
    Newark, Delaware 19713, United States
  • Helen F Graham Cancer Center
    Newark, Delaware 19713, United States
  • Medical Oncology Hematology Consultants PA
    Newark, Delaware 19713, United States
  • Christiana Care Health System-Christiana Hospital
    Newark, Delaware 19718, United States
  • Beebe Health Campus
    Rehoboth Beach, Delaware 19971, United States
  • TidalHealth Nanticoke / Allen Cancer Center
    Seaford, Delaware 19973, United States
  • Christiana Care Health System-Wilmington Hospital
    Wilmington, Delaware 19801, 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
  • Sibley Memorial Hospital
    Washington D.C., District of Columbia 20016, United States
  • Mayo Clinic in Florida
    Jacksonville, Florida 32224-9980, United States
  • John B Amos Cancer Center
    Columbus, Georgia 31904, United States
  • Low Country Cancer Care
    Savannah, Georgia 31404, United States
  • Lewis Cancer and Research Pavilion at Saint Joseph's/Candler
    Savannah, Georgia 31405, United States
  • Hawaii Cancer Care Inc - Waterfront Plaza
    Honolulu, Hawaii 96813, United States
  • Queen's Medical Center
    Honolulu, Hawaii 96813, United States
  • Straub Clinic and Hospital
    Honolulu, Hawaii 96813, United States
  • University of Hawaii Cancer Center
    Honolulu, Hawaii 96813, United States
  • Hawaii Cancer Care Inc-Liliha
    Honolulu, Hawaii 96817, United States
  • Kuakini Medical Center
    Honolulu, Hawaii 96817, United States
  • Queen's Cancer Center - Kuakini
    Honolulu, Hawaii 96817, United States
  • Kapiolani Medical Center for Women and Children
    Honolulu, Hawaii 96826, United States
  • Wilcox Memorial Hospital and Kauai Medical Clinic
    Lihue, Hawaii 96766, United States
  • Pali Momi Medical Center
    ‘Aiea, Hawaii 96701, United States
  • Queen's Cancer Center - Pearlridge
    ‘Aiea, Hawaii 96701, United States
  • Northwestern University
    Chicago, Illinois 60611, United States
  • Loyola University Medical Center
    Maywood, Illinois 60153, United States
  • Good Samaritan Regional Health Center
    Mount Vernon, Illinois 62864, United States
  • Reid Health
    Richmond, Indiana 47374, United States
  • University of Iowa/Holden Comprehensive Cancer Center
    Iowa City, Iowa 52242, United States
  • Siouxland Regional Cancer Center
    Sioux City, Iowa 51101, United States
  • Oncology Hematology Care Inc-Crestview
    Crestview Hills, Kentucky 41017, United States
  • Johns Hopkins University/Sidney Kimmel Cancer Center
    Baltimore, Maryland 21287, United States
  • Brigham and Women's Hospital
    Boston, Massachusetts 02115, United States
  • Dana-Farber Cancer Institute
    Boston, Massachusetts 02215, United States
  • University of Michigan Comprehensive Cancer Center
    Ann Arbor, Michigan 48109, United States
  • Fairview Ridges Hospital
    Burnsville, Minnesota 55337, United States
  • Mercy Hospital
    Coon Rapids, Minnesota 55433, United States
  • Fairview Southdale Hospital
    Edina, Minnesota 55435, United States
  • Unity Hospital
    Fridley, Minnesota 55432, United States
  • Hutchinson Area Health Care
    Hutchinson, Minnesota 55350, United States
  • Minnesota Oncology Hematology PA-Maplewood
    Maplewood, Minnesota 55109, United States
  • Saint John's Hospital - Healtheast
    Maplewood, Minnesota 55109, United States
  • Abbott-Northwestern Hospital
    Minneapolis, Minnesota 55407, United States
  • Hennepin County Medical Center
    Minneapolis, Minnesota 55415, United States
  • Health Partners Inc
    Minneapolis, Minnesota 55454, United States
  • New Ulm Medical Center
    New Ulm, Minnesota 56073, United States
  • North Memorial Medical Health Center
    Robbinsdale, Minnesota 55422, United States
  • Mayo Clinic in Rochester
    Rochester, Minnesota 55905, United States
  • Metro Minnesota Community Oncology Research Consortium
    Saint Louis Park, Minnesota 55416, United States
  • Park Nicollet Clinic - Saint Louis Park
    Saint Louis Park, Minnesota 55416, United States
  • Regions Hospital
    Saint Paul, Minnesota 55101, United States
  • United Hospital
    Saint Paul, Minnesota 55102, United States
  • Saint Francis Regional Medical Center
    Shakopee, Minnesota 55379, United States
  • Lakeview Hospital
    Stillwater, Minnesota 55082, United States
  • Ridgeview Medical Center
    Waconia, Minnesota 55387, United States
  • Rice Memorial Hospital
    Willmar, Minnesota 56201, United States
  • Minnesota Oncology Hematology PA-Woodbury
    Woodbury, Minnesota 55125, United States
  • Central Care Cancer Center - Bolivar
    Bolivar, Missouri 65613, United States
  • Cox Cancer Center Branson
    Branson, Missouri 65616, 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
  • Freeman Health System
    Joplin, Missouri 64804, United States
  • Mercy Hospital Joplin
    Joplin, Missouri 64804, United States
  • Delbert Day Cancer Institute at PCRMC
    Rolla, Missouri 65401, United States
  • Mercy Clinic-Rolla-Cancer and Hematology
    Rolla, Missouri 65401, United States
  • Mercy Hospital Springfield
    Springfield, Missouri 65804, United States
  • CoxHealth South Hospital
    Springfield, Missouri 65807, United States
  • Saint Louis Cancer and Breast Institute-South City
    St Louis, Missouri 63109, United States
  • Washington University School of Medicine
    St Louis, Missouri 63110, United States
  • Siteman Cancer Center-South County
    St Louis, Missouri 63129, United States
  • Mercy Hospital Saint Louis
    St Louis, Missouri 63141, United States
  • Nebraska Hematology and Oncology
    Lincoln, Nebraska 68506, United States
  • Nebraska Cancer Research Center
    Lincoln, Nebraska 68510, United States
  • Southeast Nebraska Cancer Center - 68th Street Place
    Lincoln, Nebraska 68516, United States
  • Faith Regional Health Services Carson Cancer Center
    Norfolk, Nebraska 68701, United States
  • Great Plains Health Callahan Cancer Center
    North Platte, Nebraska 69101, United States
  • Missouri Valley Cancer Consortium
    Omaha, Nebraska 68106, United States
  • Alegent Health Immanuel Medical Center
    Omaha, Nebraska 68122, United States
  • Alegent Health Bergan Mercy Medical Center
    Omaha, Nebraska 68124, United States
  • Nebraska Cancer Specialists - Omaha
    Omaha, Nebraska 68124, United States
  • Alegent Health Lakeside Hospital
    Omaha, Nebraska 68130, United States
  • Oncology Hematology West PC
    Omaha, Nebraska 68130, United States
  • Creighton University Medical Center
    Omaha, Nebraska 68131, United States
  • Regional West Medical Center Cancer Center
    Scottsbluff, Nebraska 69361, United States
  • Cancer and Blood Specialists-Henderson
    Henderson, Nevada 89052, United States
  • Comprehensive Cancer Centers of Nevada - Henderson
    Henderson, Nevada 89052, United States
  • Las Vegas Cancer Center-Henderson
    Henderson, Nevada 89052, United States
  • Comprehensive Cancer Centers of Nevada-Southeast Henderson
    Henderson, Nevada 89074, United States
  • GenesisCare USA - Henderson
    Henderson, Nevada 89074, United States
  • Cancer and Blood Specialists-Shadow
    Las Vegas, Nevada 89106, United States
  • Radiation Oncology Centers of Nevada Central
    Las Vegas, Nevada 89106, United States
  • GenesisCare USA - Las Vegas
    Las Vegas, Nevada 89109, United States
  • HealthCare Partners Medical Group Oncology/Hematology-Maryland Parkway
    Las Vegas, Nevada 89109, United States

Showing the first 100 of 150 sites across 2 countries.

09

References and documents

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 ↗

Study documents

  • Protocol and statistical analysis plan · Dec 18, 2018

Documents are hosted by the registry — open the source record to download them.

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Jun 24, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT02066181
Lead sponsor
National Cancer Institute (NCI)
Responsible party
Sponsor
First posted
Feb 19, 2014
Start date
Mar 21, 2014
Primary completion
Jul 3, 2019
Completion
Dec 1, 2022
Results posted
Sep 20, 2019
Last update
Jun 24, 2026

Study contacts

Mrinal M Gounder
principal investigator · Alliance for Clinical Trials in Oncology

Oversight

Data monitoring committee
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in May 2026. You cannot join it, but the record below documents what was studied.

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