NCT02066181

Sorafenib Tosylate in Treating Patients With Desmoid Tumors or Aggressive Fibromatosis

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

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 87 (actual)
• Sponsor: National Cancer Institute (NCI) · NIH
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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. \[Funding Source - FDA OOPD\]

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.

Conditions

• Desmoid Fibromatosis

Interventions

Type	Name	Description
OTHER	Laboratory Biomarker Analysis	Optional correlative studies
OTHER	Placebo Administration	Given PO
OTHER	Quality-of-Life Assessment	Optional ancillary studies
DRUG	Sorafenib Tosylate	Given PO

Timeline

• Start date: 2014-03-21
• Primary completion: 2019-07-03
• Completion: 2022-12-01
• First posted: 2014-02-19
• Last updated: 2023-06-07
• Results posted: 2019-09-20

Locations

150 sites across 2 countries: United States, Canada