Description:
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.
Title
- Brief Title: Efatutazone Dihydrochloride in Treating Patients With Previously Treated Myxoid Liposarcoma That Cannot Be Removed by Surgery
- Official Title: A Phase II Study of the Peroxisome Proliferator-Activated Receptor Gamma Agonist, Efatutazone in Patients With Previously Treated, Unresectable Myxoid Liposarcoma
Clinical Trial IDs
- ORG STUDY ID:
A091202
- SECONDARY ID:
NCI-2014-01028
- NCT ID:
NCT02249949
Conditions
Interventions
| Drug | Synonyms | Arms |
|---|
| efatutazone | | efatutazone dihydrochloride |
Purpose
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.
Trial Arms
| Name | Type | Description | Interventions |
|---|
| efatutazone dihydrochloride | Experimental | Patients receive efatutazone dihydrochloride PO BID continuously. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity. | |
Eligibility Criteria
- Patients must have a formalin-fixed, paraffin-embedded (FFPE) tumor block OR 1
representative hematoxylin and eosin (H&E) and 20 unstained myxoid liposarcoma tissue
slides available for submission to central pathology review; this review is mandatory
prior to registration to confirm eligibility
- Measurable disease
- Progression on at least one prior systemic chemotherapy for advanced, unresectable or
metastatic disease; prior adjuvant or neoadjuvant therapy is not included as prior
systemic chemotherapy unless treatment occurred within the 6 months prior to study
enrollment
- There is no limit to the number of prior lines of treatment a patient has
received
- No treatment with biologic therapy, immunotherapy, chemotherapy, investigational
agent for malignancy, or radiation =< 28 days before study registration; no
treatment with nitrosourea or mitomycin =< 42 days before study registration
- Patients should have resolution of any toxic effects of prior therapy (except
alopecia) to National Cancer Institute (NCI) Common Terminology Criteria for
Adverse Events (CTCAE), version 4.0, grade 1 or less
- No history of the following:
- Class III or IV congestive heart failure (CHF)
- Pericardial effusion =< 12 months (grade 3 or 4)
- Pericardial involvement with tumor
- Grade 2 or higher pleural effusion =< 6 months
- No symptomatic, untreated, or uncontrolled brain metastases present
- Not pregnant and not nursing; for women of childbearing potential only, a negative
pregnancy test done =< 7 days prior to registration is required; a female of
childbearing potential is a sexually mature female who:
- Has not undergone a hysterectomy or bilateral oophorectomy; or
- Has not been naturally postmenopausal for at least 12 consecutive months (i.e.,
has had menses at any time in the preceding 12 consecutive months)
- Patients with diabetes mellitus requiring concurrent treatment with insulin or
thiazolidinedione (TZD) oral agents are not eligible
- Patients with known hypersensitivity to any TZD oral agents are not eligible
- Eastern Cooperative Oncology Group (ECOG) performance status 0-2
- Absolute neutrophil count (ANC) >= 1,000/mm^3
- Platelet count >= 75,000/mm^3
- Creatinine =< 1.5 mg/dL x upper limits of normal (ULN) OR calculated (calc.)
creatinine clearance >= 30 mL/min
- Bilirubin =< 1.5 x ULN; for subjects with liver metastases =< 3 x ULN is allowed
- Serum glutamic oxaloacetic transaminase (SGOT) (aspartate aminotransferase [AST]) and
serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) =< 2.5 x
ULN; for subjects with liver metastases, SGOT (AST) and SGPT (ALT) < 5 x the upper
normal limit of institution's normal range is allowed
- Eligible patients must have histopathologically confirmed myxoid liposarcoma with
confirmation of DDIT3 rearrangement
| Maximum Eligible Age: | N/A |
| Minimum Eligible Age: | 18 Years |
| Eligible Gender: | All |
| Healthy Volunteers: | No |
Primary Outcome Measures
| Measure: | Confirmed Overall Response Rate Per the RECIST 1.1 Criteria |
| Time Frame: | Up to 24 weeks (8 cycles) |
| Safety Issue: | |
| Description: | 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
| Measure: | Progression Free Survival (PFS) Determined Based on Response Evaluation Criteria in Solid Tumors (RECIST) 1.1 |
| Time Frame: | Time from study entry to the first of either disease progression or death from any cause, assessed up to 5 years |
| Safety Issue: | |
| Description: | 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. |
| Measure: | Overall Survival |
| Time Frame: | Time from study entry to death from any cause, assessed up to 5 years |
| Safety Issue: | |
| Description: | 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. |
| Measure: | Incidence of Grade 3+ Adverse Events Summarized Using Common Terminology Criteria for Adverse Events Version 4.0 |
| Time Frame: | Up to 5 years |
| Safety Issue: | |
| Description: | 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 |
Details
| Phase: | Phase 2 |
| Primary Purpose: | Interventional |
| Overall Status: | Active, not recruiting |
| Lead Sponsor: | Alliance for Clinical Trials in Oncology |
Last Updated
August 18, 2021
