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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02249949
Recruitment Status : Active, not recruiting
First Posted : September 26, 2014
Results First Posted : June 27, 2019
Last Update Posted : July 24, 2020
Sponsor:
Collaborators:
National Cancer Institute (NCI)
Daiichi Sankyo, Inc.
Information provided by (Responsible Party):
Alliance for Clinical Trials in Oncology

Tracking Information
First Submitted Date  ICMJE September 24, 2014
First Posted Date  ICMJE September 26, 2014
Results First Submitted Date  ICMJE June 10, 2019
Results First Posted Date  ICMJE June 27, 2019
Last Update Posted Date July 24, 2020
Actual Study Start Date  ICMJE October 2014
Actual Primary Completion Date October 4, 2018   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 10, 2019)
Confirmed Overall Response Rate Per the RECIST 1.1 Criteria [ Time Frame: 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.
Original Primary Outcome Measures  ICMJE
 (submitted: September 25, 2014)
Progression Free Survival [ Time Frame: 5 years post-study treatment ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: June 10, 2019)
  • 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 ]
    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 Frame: 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 [ Time Frame: 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
Original Secondary Outcome Measures  ICMJE
 (submitted: September 25, 2014)
  • Confirmed response rate [ Time Frame: Up to 5 years post-study treatment ]
  • Overall Survival [ Time Frame: Up to 5 years post-study treatment ]
  • Frequency and percentage of adverse events of grade 3+ according to Common Terminology Criteria for Adverse Events [ Time Frame: Up to 5 years post-study treatment ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Efatutazone Dihydrochloride in Treating Patients With Previously Treated Myxoid Liposarcoma That Cannot Be Removed by Surgery
Official Title  ICMJE A Phase II Study of the Peroxisome Proliferator-Activated Receptor Gamma Agonist, Efatutazone in Patients With Previously Treated, Unresectable Myxoid Liposarcoma
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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Liposarcoma
Intervention  ICMJE Drug: efatutazone
Given PO
Study Arms  ICMJE Experimental: efatutazone dihydrochloride
Patients receive efatutazone dihydrochloride PO BID continuously. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity.
Intervention: Drug: efatutazone
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Actual Enrollment  ICMJE
 (submitted: June 8, 2018)
15
Original Estimated Enrollment  ICMJE
 (submitted: September 25, 2014)
36
Study Completion Date  ICMJE Not Provided
Actual Primary Completion Date October 4, 2018   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE
  • 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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02249949
Other Study ID Numbers  ICMJE A091202
NCI-2014-01028 ( Registry Identifier: NCI Clinical Trials Reporting Program )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Alliance for Clinical Trials in Oncology
Study Sponsor  ICMJE Alliance for Clinical Trials in Oncology
Collaborators  ICMJE
  • National Cancer Institute (NCI)
  • Daiichi Sankyo, Inc.
Investigators  ICMJE
Study Chair: Michael Pishvaian, MD, PhD Georgetown University
PRS Account Alliance for Clinical Trials in Oncology
Verification Date July 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP