Efatutazone Dihydrochloride in Treating Patients With Previously Treated Myxoid Liposarcoma That Cannot Be Removed by Surgery

Phase 2

Completed

Brief Summary: This phase II trial studies how well efatutazone dihydrochloride works in treating patients with previously treated myxoid liposarcoma that cannot be removed by surgery. Drugs used in chemotherapy, such as efatutazone dihydrochloride, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading.

Detailed Description: PRIMARY OBJECTIVES:

I. To determine the confirmed response rate for efatutazone dihydrochloride (efatutazone) in patients with advanced myxoid liposarcoma whose disease has progressed on at least one prior therapy.

SECONDARY OBJECTIVES:

I. To assess the progression free survival (PFS), overall survival (OS), and adverse event rates for efatutazone treated patients with advanced myxoid liposarcoma whose disease has progressed on at least one prior therapy.

TERTIARY OBJECTIVES:

I. To assess the predictive value of peroxisome proliferator-activated receptor (PPAR) and retinoid X receptors (RXR) tumor expression from archived patient tumor samples.

II. To assess the predictive value of the expression of PPARgamma-regulated markers of adipocytes differentiation.

III. To assess the predictive value of the expression of PPARgamma-regulated cell cycle proteins.

IV. To assess the effects of efatutazone treatment on serum adiponectin levels.

OUTLINE:

Patients receive efatutazone dihydrochloride orally (PO) twice daily (BID) continuously. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed up every 12 weeks for 2 years and then every 6 months for up to 5 years.

Recruitment & Eligibility

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Arm && Interventions

Group	Intervention	Description
efatutazone dihydrochloride	efatutazone	Patients receive efatutazone dihydrochloride PO BID continuously. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity.

Primary Outcome Measures

Name	Time	Method
Confirmed Overall Response Rate Per the RECIST 1.1 Criteria	Up to 24 weeks (8 cycles)	The response rate (percentage) is the percent of patients whose best response was Complete Response (CR) or Partial Response (PR) as defined by RECIST 1.1 criteria. Percentage of successes will be estimated by 100 times the number of successes divided by the total number of evaluable patients.

Secondary Outcome Measures

Name	Time	Method
Progression Free Survival (PFS) Determined Based on Response Evaluation Criteria in Solid Tumors (RECIST) 1.1	Time from study entry to the first of either disease progression or death from any cause, assessed up to 5 years	Progression free survival (PFS) is defined as the time from study entry to the date of disease progression or death resulting from any cause, whichever comes first. Progression is defined according to Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1. The median and 95% confidence intervals are estimated using the Kaplan-Meier estimator.
Overall Survival	Time from study entry to death from any cause, assessed up to 5 years	Overall survival time is defined as the time from study entry to death due to any cause. The median and 95% confidence intervals are estimated using the Kaplan-Meier estimator.
Incidence of Grade 3+ Adverse Events Summarized Using Common Terminology Criteria for Adverse Events Version 4.0	Up to 5 years	Incidence of grade 3+ adverse events summarized using Common Terminology Criteria for Adverse Events version 4.0: The frequency and percentage of grade 3+ adverse events will be estimated

Locations (127): University of Arkansas for Medical Sciences
🇺🇸
Little Rock, Arkansas, United States
University of Colorado Hospital
🇺🇸
Aurora, Colorado, United States
Yale University
🇺🇸
New Haven, Connecticut, United States
MedStar Georgetown University Hospital
🇺🇸
Washington, District of Columbia, United States
MedStar Washington Hospital Center
🇺🇸
Washington, District of Columbia, United States
Northside Hospital
🇺🇸
Atlanta, Georgia, United States
Northside Hospital-Forsyth
🇺🇸
Cumming, Georgia, United States
Hawaii Oncology Inc-Pali Momi
🇺🇸
'Aiea, Hawaii, United States
Pali Momi Medical Center
🇺🇸
'Aiea, Hawaii, United States
The Cancer Center of Hawaii-Pali Momi
🇺🇸
'Aiea, Hawaii, United States
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University of Arkansas for Medical Sciences
🇺🇸Little Rock, Arkansas, United States