Efatutazone Dihydrochloride in Treating Patients With Previously Treated Myxoid Liposarcoma That Cannot Be Removed by Surgery
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|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
|Condition or disease||Intervention/treatment||Phase|
|Liposarcoma||Drug: efatutazone||Phase 2|
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.
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.
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.
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 :||Interventional (Clinical Trial)|
|Actual Enrollment :||15 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase II Study of the Peroxisome Proliferator-Activated Receptor Gamma Agonist, Efatutazone in Patients With Previously Treated, Unresectable Myxoid Liposarcoma|
|Actual Study Start Date :||October 2014|
|Actual Primary Completion Date :||October 4, 2018|
Experimental: efatutazone dihydrochloride
Patients receive efatutazone dihydrochloride PO BID continuously. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity.
- 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.
- 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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02249949
|Study Chair:||Michael Pishvaian, MD, PhD||Georgetown University|