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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
Sponsor:
Collaborators:
National Cancer Institute (NCI)
Daiichi Sankyo, Inc.
Information provided by (Responsible Party):
Alliance for Clinical Trials in Oncology

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Liposarcoma
Intervention Drug: efatutazone
Enrollment 15
Recruitment Details  
Pre-assignment Details A protocol amendment (Update #02) removed the option for patients to be randomized to Placebo; the study went from being a randomized study to a single arm study. The discrepancy in the number of patients who 'Started' the study and the Protocol Enrollment number is due to there being 2 patients randomized to Placebo prior to Update #02.
Arm/Group Title Efatutazone Dihydrochloride
Hide Arm/Group Description Patients receive efatutazone dihydrochloride PO BID continuously. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity.
Period Title: Overall Study
Started 13
Completed 11
Not Completed 2
Reason Not Completed
Cancelled prior to treatment             2
Arm/Group Title Efatutazone Dihydrochloride
Hide Arm/Group Description Patients receive efatutazone dihydrochloride PO BID continuously. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity.
Overall Number of Baseline Participants 11
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 11 participants
51.4  (17.2)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 11 participants
Female
4
  36.4%
Male
7
  63.6%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 11 participants
Hispanic or Latino
2
  18.2%
Not Hispanic or Latino
9
  81.8%
Unknown or Not Reported
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 11 participants
American Indian or Alaska Native
0
   0.0%
Asian
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
1
   9.1%
White
8
  72.7%
More than one race
0
   0.0%
Unknown or Not Reported
2
  18.2%
ECOG Performance Status   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 11 participants
0
6
  54.5%
1
5
  45.5%
[1]
Measure Description: Eastern Cooperative Oncology Group PS Scale: 0)Fully active, able to carry on all pre-disease performance without restriction; 1)Restricted in physically strenuous activity but ambulatory and able to carry out work of a light or sedentary nature, e.g., light house work, office work; 2)Ambulatory and capable of all selfcare but unable to carry out any work activities. Up and about more than 50% of waking hours; 3)Capable of only limited selfcare, confined to bed or chair more than 50% of waking hours; 4)Completely disabled. Cannot carry on any selfcare. Totally confined to bed or chair.
1.Primary Outcome
Title Confirmed Overall Response Rate Per the RECIST 1.1 Criteria
Hide 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.
Time Frame Up to 24 weeks (8 cycles)
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Efatutazone Dihydrochloride
Hide Arm/Group Description:
Patients receive efatutazone dihydrochloride PO BID continuously. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity.
Overall Number of Participants Analyzed 11
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of patients
0
(0 to 28)
2.Secondary Outcome
Title Progression Free Survival (PFS) Determined Based on Response Evaluation Criteria in Solid Tumors (RECIST) 1.1
Hide 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.
Time Frame Time from study entry to the first of either disease progression or death from any cause, assessed up to 5 years
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Efatutazone Dihydrochloride
Hide Arm/Group Description:
Patients receive efatutazone dihydrochloride PO BID continuously. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity.
Overall Number of Participants Analyzed 11
Median (95% Confidence Interval)
Unit of Measure: months
1.4 [1] 
(1.2 to NA)
[1]
The 95% confidence interval upper limit was not obtained.
3.Secondary Outcome
Title Overall Survival
Hide 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.
Time Frame Time from study entry to death from any cause, assessed up to 5 years
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Efatutazone Dihydrochloride
Hide Arm/Group Description:
Patients receive efatutazone dihydrochloride PO BID continuously. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity.
Overall Number of Participants Analyzed 11
Median (95% Confidence Interval)
Unit of Measure: months
8.6 [1] 
(6.8 to NA)
[1]
The 95% confidence interval upper limit was not obtained.
4.Secondary Outcome
Title Incidence of Grade 3+ Adverse Events Summarized Using Common Terminology Criteria for Adverse Events Version 4.0
Hide 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
Time Frame Up to 5 years
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Efatutazone Dihydrochloride
Hide Arm/Group Description:
Patients receive efatutazone dihydrochloride PO BID continuously. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity.
Overall Number of Participants Analyzed 11
Measure Type: Count of Participants
Unit of Measure: Participants
at least one grade 3 or worse AE
6
  54.5%
at least one grade 4 or worse AE
3
  27.3%
Time Frame Adverse events are assessed weekly during cycle 1, and then on day 1 (+/- 4 days) for cycles 2-4; then day 1 (+/- 7 days) of every odd numbered cycle for Cycle 5 and beyond; up to 5 years.
Adverse Event Reporting Description Each CTCAE term is a representation of a specific event used for medical documentation & analysis & is a single MedDRA Lowest Level Term (LLT). All graded AEs are reported for completed patients. Serious AE (SAE) reports may include any secondary serious or non-serious events considered related to the primary event (the reason for filing an expedited report); collectively, these events are referred to as Expedited AEs, & appear in the SAE table.
 
Arm/Group Title Efatutazone Dihydrochloride
Hide Arm/Group Description Patients receive efatutazone dihydrochloride PO BID continuously. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity.
All-Cause Mortality
Efatutazone Dihydrochloride
Affected / at Risk (%)
Total   8/11 (72.73%)    
Hide Serious Adverse Events
Efatutazone Dihydrochloride
Affected / at Risk (%) # Events
Total   6/11 (54.55%)    
Blood and lymphatic system disorders   
Anemia  1  3/11 (27.27%)  4
Gastrointestinal disorders   
Abdominal pain  1  1/11 (9.09%)  1
Gastrointestinal disorders - Other, specify  1  1/11 (9.09%)  1
Small intestinal perforation  1  1/11 (9.09%)  1
General disorders   
Edema limbs  1  1/11 (9.09%)  1
Edema trunk  1  1/11 (9.09%)  1
Fever  1  1/11 (9.09%)  1
Infections and infestations   
Abdominal infection  1  1/11 (9.09%)  1
Bronchial infection  1  1/11 (9.09%)  1
Investigations   
Neutrophil count decreased  1  1/11 (9.09%)  2
Platelet count decreased  1  1/11 (9.09%)  2
Musculoskeletal and connective tissue disorders   
Back pain  1  2/11 (18.18%)  2
Neoplasms benign, malignant and unspecified (incl cysts and polyps)   
Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, specify  1  1/11 (9.09%)  1
Tumor pain  1  1/11 (9.09%)  1
1
Term from vocabulary, MedDRA 12
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Efatutazone Dihydrochloride
Affected / at Risk (%) # Events
Total   11/11 (100.00%)    
Blood and lymphatic system disorders   
Anemia  1  6/11 (54.55%)  19
Blood and lymphatic system disorders - Other, specify  1  1/11 (9.09%)  1
Gastrointestinal disorders   
Constipation  1  1/11 (9.09%)  1
Diarrhea  1  1/11 (9.09%)  1
Nausea  1  3/11 (27.27%)  4
Vomiting  1  1/11 (9.09%)  1
General disorders   
Edema face  1  1/11 (9.09%)  2
Edema limbs  1  8/11 (72.73%)  26
Edema trunk  1  1/11 (9.09%)  1
Fatigue  1  7/11 (63.64%)  21
Investigations   
Cholesterol high  1  1/11 (9.09%)  2
Creatinine increased  1  2/11 (18.18%)  2
Lymphocyte count decreased  1  2/11 (18.18%)  5
Neutrophil count decreased  1  1/11 (9.09%)  1
Weight gain  1  8/11 (72.73%)  23
White blood cell decreased  1  2/11 (18.18%)  2
Metabolism and nutrition disorders   
Anorexia  1  2/11 (18.18%)  3
Dehydration  1  1/11 (9.09%)  1
Hypertriglyceridemia  1  2/11 (18.18%)  4
Hyponatremia  1  1/11 (9.09%)  1
Musculoskeletal and connective tissue disorders   
Back pain  1  1/11 (9.09%)  1
Buttock pain  1  1/11 (9.09%)  1
Generalized muscle weakness  1  1/11 (9.09%)  1
Muscle weakness lower limb  1  1/11 (9.09%)  1
Pain in extremity  1  1/11 (9.09%)  1
Neoplasms benign, malignant and unspecified (incl cysts and polyps)   
Tumor pain  1  1/11 (9.09%)  1
Nervous system disorders   
Dizziness  1  1/11 (9.09%)  2
Dysgeusia  1  1/11 (9.09%)  2
Headache  1  1/11 (9.09%)  1
Psychiatric disorders   
Insomnia  1  1/11 (9.09%)  4
Reproductive system and breast disorders   
Genital edema  1  1/11 (9.09%)  1
Respiratory, thoracic and mediastinal disorders   
Cough  1  1/11 (9.09%)  1
Dyspnea  1  1/11 (9.09%)  1
Pleural effusion  1  2/11 (18.18%)  3
Skin and subcutaneous tissue disorders   
Periorbital edema  1  1/11 (9.09%)  1
Rash maculo-papular  1  1/11 (9.09%)  1
Skin and subcutaneous tissue disorders - Other, specify  1  1/11 (9.09%)  2
Vascular disorders   
Hypertension  1  2/11 (18.18%)  2
1
Term from vocabulary, MedDRA 12
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Michael J. Pishvaian, MD, PhD
Organization: Georgetown University
Phone: 202-444-2144
EMail: pishvaim@georgetown.edu
Layout table for additonal information
Responsible Party: Alliance for Clinical Trials in Oncology
ClinicalTrials.gov Identifier: NCT02249949    
Other Study ID Numbers: A091202
NCI-2014-01028 ( Registry Identifier: NCI Clinical Trials Reporting Program )
First Submitted: September 24, 2014
First Posted: September 26, 2014
Results First Submitted: June 10, 2019
Results First Posted: June 27, 2019
Last Update Posted: July 24, 2020