Trial Outcomes & Findings for Efatutazone Dihydrochloride in Treating Patients With Previously Treated Myxoid Liposarcoma That Cannot Be Removed by Surgery (NCT NCT02249949)

Last Updated: 2025-01-27

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

15 participants

Primary outcome timeframe

Up to 24 weeks (8 cycles)

Results posted on

2025-01-27

Participant Flow

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.

Participant milestones

Participant milestones
Measure	Efatutazone Dihydrochloride Patients receive efatutazone dihydrochloride PO BID continuously. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity.
Overall Study STARTED	13
Overall Study COMPLETED	11
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Reasons for withdrawal
Measure	Efatutazone Dihydrochloride Patients receive efatutazone dihydrochloride PO BID continuously. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity.
Overall Study Cancelled prior to treatment	2

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Efatutazone Dihydrochloride n=11 Participants Patients receive efatutazone dihydrochloride PO BID continuously. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity.
Age, Continuous	51.4 years STANDARD_DEVIATION 17.2 • n=5 Participants
Sex: Female, Male Female	4 Participants n=5 Participants
Sex: Female, Male Male	7 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	9 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants
Race (NIH/OMB) White	8 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	2 Participants n=5 Participants
ECOG Performance Status 0	6 Participants n=5 Participants
ECOG Performance Status 1	5 Participants n=5 Participants

PRIMARY outcome

Timeframe: Up to 24 weeks (8 cycles)

Outcome measures

Outcome measures
Measure	Efatutazone Dihydrochloride n=11 Participants 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	0 percentage of patients Interval 0.0 to 28.0

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Efatutazone Dihydrochloride n=11 Participants Patients receive efatutazone dihydrochloride PO BID continuously. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity.
Progression Free Survival (PFS) Determined Based on Response Evaluation Criteria in Solid Tumors (RECIST) 1.1	1.4 months Interval 1.2 to The 95% confidence interval upper limit was not obtained.

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Efatutazone Dihydrochloride n=11 Participants Patients receive efatutazone dihydrochloride PO BID continuously. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity.
Overall Survival	8.6 months Interval 6.8 to The 95% confidence interval upper limit was not obtained.

SECONDARY outcome

Timeframe: 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

Outcome measures

Outcome measures
Measure	Efatutazone Dihydrochloride n=11 Participants Patients receive efatutazone dihydrochloride PO BID continuously. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity.
Incidence of Grade 3+ Adverse Events Summarized Using Common Terminology Criteria for Adverse Events Version 4.0 at least one grade 3 or worse AE	6 Participants
Incidence of Grade 3+ Adverse Events Summarized Using Common Terminology Criteria for Adverse Events Version 4.0 at least one grade 4 or worse AE	3 Participants

Adverse Events

Efatutazone Dihydrochloride

Serious events: 6 serious events

Other events: 11 other events

Deaths: 8 deaths

Serious adverse events

Serious adverse events
Measure	Efatutazone Dihydrochloride n=11 participants at risk Patients receive efatutazone dihydrochloride PO BID continuously. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity.
Blood and lymphatic system disorders Anemia	27.3% 3/11 • Number of events 4 • 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. 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.
Gastrointestinal disorders Abdominal pain	9.1% 1/11 • Number of events 1 • 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. 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.
Gastrointestinal disorders Gastrointestinal disorders - Other, specify	9.1% 1/11 • Number of events 1 • 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. 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.
Gastrointestinal disorders Small intestinal perforation	9.1% 1/11 • Number of events 1 • 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. 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.
General disorders Edema limbs	9.1% 1/11 • Number of events 1 • 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. 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.
General disorders Edema trunk	9.1% 1/11 • Number of events 1 • 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. 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.
General disorders Fever	9.1% 1/11 • Number of events 1 • 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. 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.
Infections and infestations Abdominal infection	9.1% 1/11 • Number of events 1 • 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. 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.
Infections and infestations Bronchial infection	9.1% 1/11 • Number of events 1 • 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. 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.
Investigations Neutrophil count decreased	9.1% 1/11 • Number of events 2 • 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. 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.
Investigations Platelet count decreased	9.1% 1/11 • Number of events 2 • 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. 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.
Musculoskeletal and connective tissue disorders Back pain	18.2% 2/11 • Number of events 2 • 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. 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.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, specify	9.1% 1/11 • Number of events 1 • 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. 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.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Tumor pain	9.1% 1/11 • Number of events 1 • 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. 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.

Other adverse events

Other adverse events
Measure	Efatutazone Dihydrochloride n=11 participants at risk Patients receive efatutazone dihydrochloride PO BID continuously. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity.
Blood and lymphatic system disorders Anemia	54.5% 6/11 • Number of events 19 • 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. 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.
Blood and lymphatic system disorders Blood and lymphatic system disorders - Other, specify	9.1% 1/11 • Number of events 1 • 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. 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.
Gastrointestinal disorders Constipation	9.1% 1/11 • Number of events 1 • 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. 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.
Gastrointestinal disorders Diarrhea	9.1% 1/11 • Number of events 1 • 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. 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.
Gastrointestinal disorders Nausea	27.3% 3/11 • Number of events 4 • 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. 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.
Gastrointestinal disorders Vomiting	9.1% 1/11 • Number of events 1 • 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. 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.
General disorders Edema face	9.1% 1/11 • Number of events 2 • 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. 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.
General disorders Edema limbs	72.7% 8/11 • Number of events 26 • 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. 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.
General disorders Edema trunk	9.1% 1/11 • Number of events 1 • 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. 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.
General disorders Fatigue	63.6% 7/11 • Number of events 21 • 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. 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.
Investigations Cholesterol high	9.1% 1/11 • Number of events 2 • 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. 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.
Investigations Creatinine increased	18.2% 2/11 • Number of events 2 • 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. 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.
Investigations Lymphocyte count decreased	18.2% 2/11 • Number of events 5 • 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. 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.
Investigations Neutrophil count decreased	9.1% 1/11 • Number of events 1 • 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. 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.
Investigations Weight gain	72.7% 8/11 • Number of events 23 • 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. 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.
Investigations White blood cell decreased	18.2% 2/11 • Number of events 2 • 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. 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.
Metabolism and nutrition disorders Anorexia	18.2% 2/11 • Number of events 3 • 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. 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.
Metabolism and nutrition disorders Dehydration	9.1% 1/11 • Number of events 1 • 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. 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.
Metabolism and nutrition disorders Hypertriglyceridemia	18.2% 2/11 • Number of events 4 • 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. 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.
Metabolism and nutrition disorders Hyponatremia	9.1% 1/11 • Number of events 1 • 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. 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.
Musculoskeletal and connective tissue disorders Back pain	9.1% 1/11 • Number of events 1 • 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. 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.
Musculoskeletal and connective tissue disorders Buttock pain	9.1% 1/11 • Number of events 1 • 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. 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.
Musculoskeletal and connective tissue disorders Generalized muscle weakness	9.1% 1/11 • Number of events 1 • 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. 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.
Musculoskeletal and connective tissue disorders Muscle weakness lower limb	9.1% 1/11 • Number of events 1 • 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. 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.
Musculoskeletal and connective tissue disorders Pain in extremity	9.1% 1/11 • Number of events 1 • 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. 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.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Tumor pain	9.1% 1/11 • Number of events 1 • 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. 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.
Nervous system disorders Dizziness	9.1% 1/11 • Number of events 2 • 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. 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.
Nervous system disorders Dysgeusia	9.1% 1/11 • Number of events 2 • 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. 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.
Nervous system disorders Headache	9.1% 1/11 • Number of events 1 • 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. 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.
Psychiatric disorders Insomnia	9.1% 1/11 • Number of events 4 • 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. 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.
Reproductive system and breast disorders Genital edema	9.1% 1/11 • Number of events 1 • 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. 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.
Respiratory, thoracic and mediastinal disorders Cough	9.1% 1/11 • Number of events 1 • 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. 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.
Respiratory, thoracic and mediastinal disorders Dyspnea	9.1% 1/11 • Number of events 1 • 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. 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.
Respiratory, thoracic and mediastinal disorders Pleural effusion	18.2% 2/11 • Number of events 3 • 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. 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.
Skin and subcutaneous tissue disorders Periorbital edema	9.1% 1/11 • Number of events 1 • 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. 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.
Skin and subcutaneous tissue disorders Rash maculo-papular	9.1% 1/11 • Number of events 1 • 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. 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.
Skin and subcutaneous tissue disorders Skin and subcutaneous tissue disorders - Other, specify	9.1% 1/11 • Number of events 2 • 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. 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.
Vascular disorders Hypertension	18.2% 2/11 • Number of events 2 • 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. 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.

Additional Information

Michael J. Pishvaian, MD, PhD

Georgetown University

Phone: 202-444-2144

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place