Trial · NCT02066181
Sorafenib Tosylate in Treating Patients With Desmoid Tumors or Aggressive Fibromatosis
NCT02066181, Phase 3, Completed, National Cancer Institute (NCI); results first posted 2019-09-20
3 sourcestrials-2026-11-12-0.5.0
Download JSON
Cite this record
ConductScience Library. Sorafenib Tosylate in Treating Patients With Desmoid Tumors or Aggressive Fibromatosis. Trials, record NCT02066181. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02066181
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Sorafenib Tosylate in Treating Patients With Desmoid Tumors or Aggressive Fibromatosis
- NCT number
- NCT02066181
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 87 participants, actual
- Lead sponsor
- National Cancer Institute (NCI)
- Conditions
- Desmoid Fibromatosis
- Interventions
- Laboratory Biomarker Analysis, Placebo Administration, Quality-of-Life Assessment, Sorafenib Tosylate
- First posted
- 2014-02-19
- Last updated
- 2026-06-24
- Results first posted
- 2019-09-20
Source ClinicalTrials.gov
Registered outcomes18
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Progression-free Survival(PFS) Rate | Time from randomization to the first occurrence of progression or death due to any cause, assessed up to 3 years | 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. |
| Secondary | Incidence of Adverse Events, Using the Patient Reported Outcomes-Common Terminology Criteria in Adverse Events Version 4.0 | Up to 3 years | 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. |
| Secondary | Time to Surgical Intervention During Treatment | Time between randomization to the patient undergoing therapeutic surgical resection for this disease, assessed up to 3 years | 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. |
| Secondary | Overall Survival | Time between the date of randomization to until death, assessed up to 3 years | 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. |
| Secondary | Best Objective Status Between the Two Treatment Arms According to Response Evaluation Criteria in Solid Tumors Version 1.1 | Up to 3 years | 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. |
| Secondary | Duration of Response | Time between first tumor response and progression, assessed up to 3 years | 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. |
| Other | 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 | Best response (ordinal variable) and percent T2 signal change (continuous) will be correlated by Spearman?s rho. |
| Other | Percent Changes in MRI T2 Signal (Correlative Companion Study-Imaging Study) | Baseline up to 3 years | 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). |
| Other | 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 | 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. |
| Other | Time to Pain Palliation 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 confirmed pain palliation is observed, assessed up to 12 weeks | 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. |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 registered outcomesOutcomes18
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Progression-free Survival(PFS) Rate | Time from randomization to the first occurrence of progression or death due to any cause, assessed up to 3 years | All patients that received treatment and were assessed for response. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Incidence of Adverse Events, Using the Patient Reported Outcomes-Common Terminology Criteria in Adverse Events Version 4.0 | Up to 3 years | Per protocol, crossover patients are excluded/censored from secondary analysis and endpoints. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Time to Surgical Intervention During Treatment | Time between randomization to the patient undergoing therapeutic surgical resection for this disease, assessed up to 3 years | Only the number of patients is presented due to the Protected Health Information as to the time to surgery for each of these individual patients. Crossover patients are per protocol, excluded/censored from secondary analysis and endpoints. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Overall Survival | Time between the date of randomization to until death, assessed up to 3 years | All patients that started treatment and were assessed for survival. Crossover patients were excluded/censored from secondary analysis and endpoints. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Best Objective Status Between the Two Treatment Arms According to Response Evaluation Criteria in Solid Tumors Version 1.1 | Up to 3 years | All patients that started treatment and were assessed for response. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Duration of Response | Time between first tumor response and progression, assessed up to 3 years | All patients that started treatment and were assessed for response. | Months | MEDIAN |
| OTHER_PRE_SPECIFIED | 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 | |||
| OTHER_PRE_SPECIFIED | Percent Changes in MRI T2 Signal (Correlative Companion Study-Imaging Study) | Baseline up to 3 years | |||
| OTHER_PRE_SPECIFIED | 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 | |||
| OTHER_PRE_SPECIFIED | Time to Pain Palliation 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 confirmed pain palliation is observed, assessed up to 12 weeks |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 outcomesResults by group27
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Progression-free Survival(PFS) Rate | Arm I (Sorafenib Tosylate) | Progressed or Died | 7 | Participants | |
| Progression-free Survival(PFS) Rate | Arm II (Placebo) | Progressed or Died | 22 | Participants | |
| Progression-free Survival(PFS) Rate | Arm I (Sorafenib Tosylate) | Alive and Progression Free | 42 | Participants | |
| Progression-free Survival(PFS) Rate | Arm II (Placebo) | Alive and Progression Free | 13 | Participants | |
| Incidence of Adverse Events, Using the Patient Reported Outcomes-Common Terminology Criteria in Adverse Events Version 4.0 | Arm I (Sorafenib Tosylate) | 49 | Participants | ||
| Incidence of Adverse Events, Using the Patient Reported Outcomes-Common Terminology Criteria in Adverse Events Version 4.0 | Arm II (Placebo) | 36 | Participants | ||
| Incidence of Adverse Events, Using the Patient Reported Outcomes-Common Terminology Criteria in Adverse Events Version 4.0 | Crossover Patients | 0 | Participants | ||
| Time to Surgical Intervention During Treatment | Arm I (Sorafenib Tosylate) | Had surgery during treatment | 1 | Participants | |
| Time to Surgical Intervention During Treatment | Arm II (Placebo) | Had surgery during treatment | 1 | Participants | |
| Time to Surgical Intervention During Treatment | Crossover Patients | Had surgery during treatment | 0 | Participants |
1 to 10 of 27Source ClinicalTrials.gov
See all 27 results by groupParticipant flow8
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Arm I (Sorafenib Tosylate) | 50 | |
| Overall Study | STARTED | Arm II (Placebo) | 37 | |
| Overall Study | Crossed Over | Arm I (Sorafenib Tosylate) | 0 | |
| Overall Study | Crossed Over | Arm II (Placebo) | 28 | |
| Overall Study | COMPLETED | Arm I (Sorafenib Tosylate) | 49 | |
| Overall Study | COMPLETED | Arm II (Placebo) | 36 | |
| Overall Study | NOT COMPLETED | Arm I (Sorafenib Tosylate) | 1 | |
| Overall Study | NOT COMPLETED | Arm II (Placebo) | 1 |
Source ClinicalTrials.gov
Adverse events552
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Anemia | Blood and lymphatic system disorders | Arm I (Sorafenib Tosylate) | 9 | 49 | 27 |
| other | Anemia | Blood and lymphatic system disorders | Arm II (Placebo) | 4 | 36 | 18 |
| other | Anemia | Blood and lymphatic system disorders | Crossover Group | 5 | 28 | 18 |
| other | Blood and lymphatic system disorders - Other, specify | Blood and lymphatic system disorders | Arm I (Sorafenib Tosylate) | 1 | 49 | 3 |
| other | Blood and lymphatic system disorders - Other, specify | Blood and lymphatic system disorders | Arm II (Placebo) | 0 | 36 | 0 |
| other | Blood and lymphatic system disorders - Other, specify | Blood and lymphatic system disorders | Crossover Group | 2 | 28 | 14 |
| other | Leukocytosis | Blood and lymphatic system disorders | Arm I (Sorafenib Tosylate) | 1 | 49 | 1 |
| other | Leukocytosis | Blood and lymphatic system disorders | Arm II (Placebo) | 0 | 36 | 0 |
| other | Leukocytosis | Blood and lymphatic system disorders | Crossover Group | 0 | 28 | 0 |
| other | Cardiac disorders - Other, specify | Cardiac disorders | Arm I (Sorafenib Tosylate) | 1 | 49 | 22 |
1 to 10 of 552Source ClinicalTrials.gov
See all 552 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Arm I (Sorafenib Tosylate) | 1 | 49 |
| Total, all-cause mortality | Arm II (Placebo) | 0 | 36 |
| Total, all-cause mortality | Crossover Group | 0 | 28 |
| Total, other adverse events | Arm I (Sorafenib Tosylate) | 49 | 49 |
| Total, other adverse events | Arm II (Placebo) | 34 | 36 |
| Total, other adverse events | Crossover Group | 27 | 28 |
| Total, serious adverse events | Arm I (Sorafenib Tosylate) | 11 | 49 |
| Total, serious adverse events | Arm II (Placebo) | 6 | 36 |
| Total, serious adverse events | Crossover Group | 5 | 28 |
Source ClinicalTrials.gov
Papers3
The papers the trial lists on ClinicalTrials.gov.
Grants3
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
| Project | Title | Principal investigators | Organization | Funder | Fiscal years | Source |
|---|---|---|---|---|---|---|
| U10CA180821 | Alliance for Clinical Trials in Oncology Operations Center | Galanis, Evanthia; Hahn, Olwen; Kozono, David | ALLIANCE NCTN FOUNDATION | NATIONAL CANCER INSTITUTE | 2014 to 2026 | NIH RePORTER |
| R01FD005105 | Phase III Randomized Blinded Trial of Sorafenib vs Placebo in Desmoid Tumor A091105/116279 12/2013 | GOUNDER, MRINAL | SLOAN-KETTERING INST CAN RESEARCH | FOOD AND DRUG ADMINISTRATION | 2015 to 2018 | NIH RePORTER |
| U10CA031946 | Cancer and Leukemia Group B | BERTAGNOLLI, MONICA M | BRIGHAM AND WOMEN'S HOSPITAL | NATIONAL CANCER INSTITUTE | 1985 to 2013 | NIH RePORTER |
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT02066181
GET https://conductscience.org/api/trials/v1/NCT02066181/results?page=2