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

• Sponsor: Alliance for Clinical Trials in Oncology (Other)
• Overall Status: Active, not recruiting
• CT.gov ID: NCT02249949
• Collaborator: National Cancer Institute (NCI) (NIH), Daiichi Sankyo, Inc. (Industry)
• Enrollment: 15
• Locations: 127
• Arm: 1
• Patients Per Site: 0.1

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Liposarcoma	Drug: efatutazone	Phase 2

Detailed Description

PRIMARY OBJECTIVES:

1. 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:

1. 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:

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

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

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

4. 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 Design

• Study Type: Interventional
• Actual Enrollment: 15 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• 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: Oct 1, 2014
• Actual Primary Completion Date: Oct 4, 2018

Arms and Interventions

Arm	Intervention/Treatment
Experimental: efatutazone dihydrochloride Patients receive efatutazone dihydrochloride PO BID continuously. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity.	Drug: efatutazone; Given PO

Outcome Measures

Primary Outcome Measures

1. 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

1. 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.

2. 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.

3. 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

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

• 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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	University of Arkansas for Medical Sciences	Little Rock	Arkansas	United States	72205
2	University of Colorado Hospital	Aurora	Colorado	United States	80045
3	Yale University	New Haven	Connecticut	United States	06520
4	MedStar Georgetown University Hospital	Washington	District of Columbia	United States	20007
5	MedStar Washington Hospital Center	Washington	District of Columbia	United States	20010
6	Northside Hospital	Atlanta	Georgia	United States	30342
7	Northside Hospital-Forsyth	Cumming	Georgia	United States	30041
8	Hawaii Oncology Inc-Pali Momi	'Aiea	Hawaii	United States	96701
9	Pali Momi Medical Center	'Aiea	Hawaii	United States	96701
10	The Cancer Center of Hawaii-Pali Momi	'Aiea	Hawaii	United States	96701
11	Hawaii Cancer Care Inc-POB II	Honolulu	Hawaii	United States	96813
12	Hawaii Oncology Inc-POB I	Honolulu	Hawaii	United States	96813
13	Island Urology	Honolulu	Hawaii	United States	96813
14	Queen's Medical Center	Honolulu	Hawaii	United States	96813
15	Straub Clinic and Hospital	Honolulu	Hawaii	United States	96813
16	University of Hawaii Cancer Center	Honolulu	Hawaii	United States	96813
17	Hawaii Cancer Care Inc-Liliha	Honolulu	Hawaii	United States	96817
18	Hawaii Oncology Inc-Kuakini	Honolulu	Hawaii	United States	96817
19	Kuakini Medical Center	Honolulu	Hawaii	United States	96817
20	The Cancer Center of Hawaii-Liliha	Honolulu	Hawaii	United States	96817
21	Kapiolani Medical Center for Women and Children	Honolulu	Hawaii	United States	96826
22	Wilcox Memorial Hospital and Kauai Medical Clinic	Lihue	Hawaii	United States	96766
23	Rush - Copley Medical Center	Aurora	Illinois	United States	60504
24	Saint Joseph Medical Center	Bloomington	Illinois	United States	61701
25	Illinois CancerCare-Bloomington	Bloomington	Illinois	United States	61704
26	Illinois CancerCare-Canton	Canton	Illinois	United States	61520
27	Memorial Hospital of Carbondale	Carbondale	Illinois	United States	62902
28	SIH Cancer Institute	Carterville	Illinois	United States	62918
29	Illinois CancerCare-Carthage	Carthage	Illinois	United States	62321
30	Centralia Oncology Clinic	Centralia	Illinois	United States	62801
31	Northwestern University	Chicago	Illinois	United States	60611
32	Carle on Vermilion	Danville	Illinois	United States	61832
33	Cancer Care Specialists of Central Illinois	Decatur	Illinois	United States	62526
34	Decatur Memorial Hospital	Decatur	Illinois	United States	62526
35	Carle Physician Group-Effingham	Effingham	Illinois	United States	62401
36	Crossroads Cancer Center	Effingham	Illinois	United States	62401
37	Illinois CancerCare-Eureka	Eureka	Illinois	United States	61530
38	Illinois CancerCare-Galesburg	Galesburg	Illinois	United States	61401
39	Western Illinois Cancer Treatment Center	Galesburg	Illinois	United States	61401
40	Illinois CancerCare-Kewanee Clinic	Kewanee	Illinois	United States	61443
41	Illinois CancerCare-Macomb	Macomb	Illinois	United States	61455
42	Carle Physician Group-Mattoon/Charleston	Mattoon	Illinois	United States	61938
43	Loyola University Medical Center	Maywood	Illinois	United States	60153
44	Good Samaritan Regional Health Center	Mount Vernon	Illinois	United States	62864
45	Illinois CancerCare-Ottawa Clinic	Ottawa	Illinois	United States	61350
46	Radiation Oncology of Northern Illinois	Ottawa	Illinois	United States	61350
47	Illinois CancerCare-Pekin	Pekin	Illinois	United States	61554
48	OSF Saint Francis Radiation Oncology at Pekin Cancer Treatment Center	Pekin	Illinois	United States	61554
49	Illinois CancerCare-Peoria	Peoria	Illinois	United States	61615
50	OSF Saint Francis Radiation Oncology at Peoria Cancer Center	Peoria	Illinois	United States	61615
51	Methodist Medical Center of Illinois	Peoria	Illinois	United States	61636
52	OSF Saint Francis Medical Center	Peoria	Illinois	United States	61637
53	Illinois CancerCare-Peru	Peru	Illinois	United States	61354
54	Valley Radiation Oncology	Peru	Illinois	United States	61354
55	Illinois CancerCare-Princeton	Princeton	Illinois	United States	61356
56	Central Illinois Hematology Oncology Center	Springfield	Illinois	United States	62702
57	Southern Illinois University School of Medicine	Springfield	Illinois	United States	62702
58	Springfield Clinic	Springfield	Illinois	United States	62702
59	Memorial Medical Center	Springfield	Illinois	United States	62781
60	Cancer Care Specialists of Illinois-Swansea	Swansea	Illinois	United States	62226
61	Memorial and Saint Elizabeth's Health Care Services LLP	Swansea	Illinois	United States	62226
62	Carle Cancer Center	Urbana	Illinois	United States	61801
63	The Carle Foundation Hospital	Urbana	Illinois	United States	61801
64	Rush-Copley Healthcare Center	Yorkville	Illinois	United States	60560
65	Deaconess Clinic Downtown	Evansville	Indiana	United States	47713
66	Franciscan Saint Anthony Health-Michigan City	Michigan City	Indiana	United States	46360
67	Woodland Cancer Care Center	Michigan City	Indiana	United States	46360
68	Chancellor Center for Oncology	Newburgh	Indiana	United States	47630
69	Kansas Institute of Medicine Cancer and Blood Center	Lenexa	Kansas	United States	66219
70	Minimally Invasive Surgery Hospital	Lenexa	Kansas	United States	66219
71	Menorah Medical Center	Overland Park	Kansas	United States	66209
72	Saint Luke's South Hospital	Overland Park	Kansas	United States	66213
73	Abbott-Northwestern Hospital	Minneapolis	Minnesota	United States	55407
74	Saint Louis Cancer and Breast Institute-Ballwin	Ballwin	Missouri	United States	63011
75	Central Care Cancer Center - Bolivar	Bolivar	Missouri	United States	65613
76	Parkland Health Center-Bonne Terre	Bonne Terre	Missouri	United States	63628
77	Cox Cancer Center Branson	Branson	Missouri	United States	65616
78	Saint Francis Medical Center	Cape Girardeau	Missouri	United States	63703
79	Southeast Cancer Center	Cape Girardeau	Missouri	United States	63703
80	Siteman Cancer Center at West County Hospital	Creve Coeur	Missouri	United States	63141
81	Centerpoint Medical Center LLC	Independence	Missouri	United States	64057
82	Capital Region Southwest Campus	Jefferson City	Missouri	United States	65109
83	Freeman Health System	Joplin	Missouri	United States	64804
84	Mercy Hospital Joplin	Joplin	Missouri	United States	64804
85	Saint Luke's Hospital of Kansas City	Kansas City	Missouri	United States	64111
86	Research Medical Center	Kansas City	Missouri	United States	64132
87	Saint Luke's East - Lee's Summit	Lee's Summit	Missouri	United States	64086
88	Delbert Day Cancer Institute at PCRMC	Rolla	Missouri	United States	65401
89	Mercy Clinic-Rolla-Cancer and Hematology	Rolla	Missouri	United States	65401
90	Heartland Regional Medical Center	Saint Joseph	Missouri	United States	64507
91	Saint Louis Cancer and Breast Institute-South City	Saint Louis	Missouri	United States	63109
92	Washington University School of Medicine	Saint Louis	Missouri	United States	63110
93	Siteman Cancer Center-South County	Saint Louis	Missouri	United States	63129
94	Missouri Baptist Medical Center	Saint Louis	Missouri	United States	63131
95	Mercy Hospital Saint Louis	Saint Louis	Missouri	United States	63141
96	Sainte Genevieve County Memorial Hospital	Sainte Genevieve	Missouri	United States	63670
97	Mercy Hospital Springfield	Springfield	Missouri	United States	65804
98	CoxHealth South Hospital	Springfield	Missouri	United States	65807
99	Missouri Baptist Sullivan Hospital	Sullivan	Missouri	United States	63080
100	Missouri Baptist Outpatient Center-Sunset Hills	Sunset Hills	Missouri	United States	63127
101	Mercy Hospital Washington	Washington	Missouri	United States	63090
102	University of Nebraska Medical Center	Omaha	Nebraska	United States	68198
103	Dartmouth Hitchcock Medical Center	Lebanon	New Hampshire	United States	03756
104	Norris Cotton Cancer Center-Manchester	Manchester	New Hampshire	United States	03102
105	Norris Cotton Cancer Center-Nashua	Nashua	New Hampshire	United States	03063
106	Hackensack University Medical Center	Hackensack	New Jersey	United States	07601
107	University of New Mexico Cancer Center	Albuquerque	New Mexico	United States	87102
108	Roswell Park Cancer Institute	Buffalo	New York	United States	14263
109	Columbia University/Herbert Irving Cancer Center	New York	New York	United States	10032
110	Memorial Sloan Kettering Cancer Center	New York	New York	United States	10065
111	State University of New York Upstate Medical University	Syracuse	New York	United States	13210
112	Integris Southwest Medical Center	Oklahoma City	Oklahoma	United States	73109
113	Mercy Hospital Oklahoma City	Oklahoma City	Oklahoma	United States	73120
114	Integris Cancer Institute of Oklahoma	Oklahoma City	Oklahoma	United States	73142
115	Greenville Health System Cancer Institute-Easley	Easley	South Carolina	United States	29640
116	Greenville Health System Cancer Institute-Andrews	Greenville	South Carolina	United States	29601
117	Greenville Health System Cancer Institute-Butternut	Greenville	South Carolina	United States	29605
118	Greenville Health System Cancer Institute-Faris	Greenville	South Carolina	United States	29605
119	Greenville Memorial Hospital	Greenville	South Carolina	United States	29605
120	Greenville Health System Cancer Institute-Eastside	Greenville	South Carolina	United States	29615
121	Greenville Health System Cancer Institute-Greer	Greer	South Carolina	United States	29650
122	Greenville Health System Cancer Institute-Seneca	Seneca	South Carolina	United States	29672
123	Greenville Health System Cancer Institute-Spartanburg	Spartanburg	South Carolina	United States	29307
124	Norris Cotton Cancer Center-North	Saint Johnsbury	Vermont	United States	05819
125	Fred Hutchinson Cancer Research Center	Seattle	Washington	United States	98109
126	Seattle Cancer Care Alliance	Seattle	Washington	United States	98109
127	University of Washington Medical Center	Seattle	Washington	United States	98195

Sponsors and Collaborators

• Alliance for Clinical Trials in Oncology
• National Cancer Institute (NCI)
• Daiichi Sankyo, Inc.

Investigators

• Study Chair: Michael Pishvaian, MD, PhD, Georgetown University

Study Documents (Full-Text)

• Study Protocol and Statistical Analysis Plan - Jun 26, 2018

More Information

Publications

None provided.

• Responsible Party: Alliance for Clinical Trials in Oncology
• ClinicalTrials.gov Identifier: NCT02249949
• Other Study ID Numbers: A091202; NCI-2014-01028
• First Posted: Sep 26, 2014
• Last Update Posted: Aug 18, 2021
• Last Verified: Aug 1, 2021

• Additional relevant MeSH terms: Liposarcoma; Liposarcoma, Myxoid; Efatutazone

Study Results

Participant Flow

Pre-assignment Detail	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
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

Baseline Characteristics

Arm/Group Title	Efatutazone Dihydrochloride
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 Participants	11
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	51.4 (17.2)
Sex: Female, Male (Count of Participants)
Female	4 36.4%
Male	7 63.6%
Ethnicity (NIH/OMB) (Count of Participants)
Hispanic or Latino	2 18.2%
Not Hispanic or Latino	9 81.8%
Unknown or Not Reported	0 0%
Race (NIH/OMB) (Count of Participants)
American Indian or Alaska Native	0 0%
Asian	0 0%
Native Hawaiian or Other Pacific Islander	0 0%
Black or African American	1 9.1%
White	8 72.7%
More than one race	0 0%
Unknown or Not Reported	2 18.2%
ECOG Performance Status (Count of Participants)
0	6 54.5%
1	5 45.5%

Outcome Measures

1. Primary Outcome

Title	Confirmed Overall Response Rate Per the RECIST 1.1 Criteria
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)

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Efatutazone Dihydrochloride
Arm/Group Description	Patients receive efatutazone dihydrochloride PO BID continuously. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity.
Measure Participants	11
Number (95% Confidence Interval) [percentage of patients]	0

2. Secondary Outcome

Title	Progression Free Survival (PFS) Determined Based on Response Evaluation Criteria in Solid Tumors (RECIST) 1.1
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

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Efatutazone Dihydrochloride
Arm/Group Description	Patients receive efatutazone dihydrochloride PO BID continuously. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity.
Measure Participants	11
Median (95% Confidence Interval) [months]	1.4

3. Secondary Outcome

Title	Overall Survival
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

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Efatutazone Dihydrochloride
Arm/Group Description	Patients receive efatutazone dihydrochloride PO BID continuously. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity.
Measure Participants	11
Median (95% Confidence Interval) [months]	8.6

4. Secondary Outcome

Title	Incidence of Grade 3+ Adverse Events Summarized Using Common Terminology Criteria for Adverse Events Version 4.0
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

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Efatutazone Dihydrochloride
Arm/Group Description	Patients receive efatutazone dihydrochloride PO BID continuously. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity.
Measure Participants	11
at least one grade 3 or worse AE	6 54.5%
at least one grade 4 or worse AE	3 27.3%

Adverse Events

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
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 (%)	# Events
Total	8/11 (72.7%)
Serious Adverse Events
	Efatutazone Dihydrochloride
	Affected / at Risk (%)	# Events
Total	6/11 (54.5%)
Blood and lymphatic system disorders
Anemia	3/11 (27.3%)	4
Gastrointestinal disorders
Abdominal pain	1/11 (9.1%)	1
Gastrointestinal disorders - Other, specify	1/11 (9.1%)	1
Small intestinal perforation	1/11 (9.1%)	1
General disorders
Edema limbs	1/11 (9.1%)	1
Edema trunk	1/11 (9.1%)	1
Fever	1/11 (9.1%)	1
Infections and infestations
Abdominal infection	1/11 (9.1%)	1
Bronchial infection	1/11 (9.1%)	1
Investigations
Neutrophil count decreased	1/11 (9.1%)	2
Platelet count decreased	1/11 (9.1%)	2
Musculoskeletal and connective tissue disorders
Back pain	2/11 (18.2%)	2
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, specify	1/11 (9.1%)	1
Tumor pain	1/11 (9.1%)	1
Other (Not Including Serious) Adverse Events
	Efatutazone Dihydrochloride
	Affected / at Risk (%)	# Events
Total	11/11 (100%)
Blood and lymphatic system disorders
Anemia	6/11 (54.5%)	19
Blood and lymphatic system disorders - Other, specify	1/11 (9.1%)	1
Gastrointestinal disorders
Constipation	1/11 (9.1%)	1
Diarrhea	1/11 (9.1%)	1
Nausea	3/11 (27.3%)	4
Vomiting	1/11 (9.1%)	1
General disorders
Edema face	1/11 (9.1%)	2
Edema limbs	8/11 (72.7%)	26
Edema trunk	1/11 (9.1%)	1
Fatigue	7/11 (63.6%)	21
Investigations
Cholesterol high	1/11 (9.1%)	2
Creatinine increased	2/11 (18.2%)	2
Lymphocyte count decreased	2/11 (18.2%)	5
Neutrophil count decreased	1/11 (9.1%)	1
Weight gain	8/11 (72.7%)	23
White blood cell decreased	2/11 (18.2%)	2
Metabolism and nutrition disorders
Anorexia	2/11 (18.2%)	3
Dehydration	1/11 (9.1%)	1
Hypertriglyceridemia	2/11 (18.2%)	4
Hyponatremia	1/11 (9.1%)	1
Musculoskeletal and connective tissue disorders
Back pain	1/11 (9.1%)	1
Buttock pain	1/11 (9.1%)	1
Generalized muscle weakness	1/11 (9.1%)	1
Muscle weakness lower limb	1/11 (9.1%)	1
Pain in extremity	1/11 (9.1%)	1
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Tumor pain	1/11 (9.1%)	1
Nervous system disorders
Dizziness	1/11 (9.1%)	2
Dysgeusia	1/11 (9.1%)	2
Headache	1/11 (9.1%)	1
Psychiatric disorders
Insomnia	1/11 (9.1%)	4
Reproductive system and breast disorders
Genital edema	1/11 (9.1%)	1
Respiratory, thoracic and mediastinal disorders
Cough	1/11 (9.1%)	1
Dyspnea	1/11 (9.1%)	1
Pleural effusion	2/11 (18.2%)	3
Skin and subcutaneous tissue disorders
Periorbital edema	1/11 (9.1%)	1
Rash maculo-papular	1/11 (9.1%)	1
Skin and subcutaneous tissue disorders - Other, specify	1/11 (9.1%)	2
Vascular disorders
Hypertension	2/11 (18.2%)	2

Limitations/Caveats

[Not Specified]

More Information

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.