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SARC023: Ganetespib and Sirolimus in Patients With MPNST (Malignant Peripheral Nerve Sheath Tumors)

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: NCT02008877
Recruitment Status : Completed
First Posted : December 11, 2013
Results First Posted : May 2, 2019
Last Update Posted : May 15, 2019
Sponsor:
Collaborators:
Synta Pharmaceuticals Corp.
United States Department of Defense
Information provided by (Responsible Party):
Sarcoma Alliance for Research through Collaboration

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Malignant Peripheral Nerve Sheath Tumors (MPNST)
Sarcoma
Interventions Drug: ganetespib
Drug: Sirolimus
Enrollment 20
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Phase 1 Dose 1 Phase 1 Dose 2 Phase 2
Hide Arm/Group Description

For each 28-day cycle, subjects took doses of both ganetespib + sirolimus:

ganetespib: 150 mg/m² IV on days 1, 8, and 15 intravenously over 1 hour

Sirolimus: 4mg taken orally once daily on a continuous dosing schedule; Loading dose 12 mg on cycle 1 day 1 only.

For each 28-day cycle, subjects took doses of both ganetespib + sirolimus:

ganetespib: 200 mg/m² IV on days 1, 8, and 15 intravenously over 1 hour

Sirolimus: 4mg taken orally once daily on a continuous dosing schedule; Loading dose 12 mg on cycle 1 day 1 only.

For each 28-day cycle, subjects took doses of both ganetespib + sirolimus:

ganetespib: 200 mg/m² IV on days 1, 8, and 15 intravenously over 1 hour

Sirolimus: 4mg taken orally once daily on a continuous dosing schedule; Loading dose 12 mg on cycle 1 day 1 only.

Period Title: Overall Study
Started 3 7 10
Completed 3 6 10
Not Completed 0 1 0
Reason Not Completed
Inevaluable             0             1             0
Arm/Group Title Ganetespib / Sirolimus
Hide Arm/Group Description

28-day cycles of ganetespib + sirolimus

ganetespib: 200 mg/m² IV on days 1, 8, and 15 intravenously over 1 hour

Sirolimus: 2mg taken orally once daily on a continuous dosing schedule; Loading dose 12 mg on cycle 1 day 1 only.

Overall Number of Baseline Participants 20
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous   [1] 
Median (Full Range)
Unit of measure:  Years
Phase I Number Analyzed 10 participants
26
(16 to 89)
Phase 2 Number Analyzed 10 participants
38
(24 to 61)
[1]
Measure Analysis Population Description: 10 patients were enrolled in Phase 1 and 10 patients were enrolled in Phase 2 of the study. Overall number of patients enrolled was 20.
Sex: Female, Male   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Phase I Number Analyzed 10 participants
Female
2
  20.0%
Male
8
  80.0%
Phase 2 Number Analyzed 10 participants
Female
4
  40.0%
Male
6
  60.0%
[1]
Measure Analysis Population Description: 10 patients were enrolled in Phase 1 and 10 patients were enrolled in Phase 2 of the study. Overall number of patients enrolled was 20.
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 20 participants
Hispanic or Latino
4
  20.0%
Not Hispanic or Latino
16
  80.0%
Unknown or Not Reported
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 20 participants
American Indian or Alaska Native
0
   0.0%
Asian
2
  10.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
2
  10.0%
White
14
  70.0%
More than one race
0
   0.0%
Unknown or Not Reported
2
  10.0%
Sarcoma subtype   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Phase I Number Analyzed 10 participants
Alveolar soft part sarcoma
1
  10.0%
Ewing sarcoma
1
  10.0%
Leiomyosarcoma
2
  20.0%
Liposarcoma
3
  30.0%
MPNST NF1 Associated
3
  30.0%
MPNST Sporadic
0
   0.0%
Phase 2 Number Analyzed 10 participants
Alveolar soft part sarcoma
0
   0.0%
Ewing sarcoma
0
   0.0%
Leiomyosarcoma
0
   0.0%
Liposarcoma
0
   0.0%
MPNST NF1 Associated
5
  50.0%
MPNST Sporadic
5
  50.0%
[1]
Measure Analysis Population Description: 10 patients were enrolled in Phase 1 and 10 patients were enrolled in Phase 2 of the study. Overall number of patients enrolled was 20.
Tumor location at diagnosis   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Phase I Number Analyzed 10 participants
Abdomen
1
  10.0%
Extremity
1
  10.0%
Head
1
  10.0%
Lung
0
   0.0%
Mediastinum
1
  10.0%
Peritoneum
1
  10.0%
Skin
1
  10.0%
Spine
1
  10.0%
Other
3
  30.0%
Phase 2 Number Analyzed 10 participants
Abdomen
1
  10.0%
Extremity
4
  40.0%
Head
0
   0.0%
Lung
1
  10.0%
Mediastinum
0
   0.0%
Peritoneum
0
   0.0%
Skin
0
   0.0%
Spine
2
  20.0%
Other
2
  20.0%
[1]
Measure Analysis Population Description: 10 patients were enrolled in Phase 1 and 10 patients were enrolled in Phase 2 of the study. Overall number of patients enrolled was 20.
History of metastatic disease   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Phase 1 Number Analyzed 10 participants
9
  90.0%
Phase 2 Number Analyzed 10 participants
9
  90.0%
[1]
Measure Analysis Population Description: 10 patients were enrolled in Phase 1 and 10 patients were enrolled in Phase 2 of the study. Overall number of patients enrolled was 20.
Prior chemotherapy regimen   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Phase 1 Number Analyzed 10 participants
10
 100.0%
Phase 2 Number Analyzed 10 participants
8
  80.0%
[1]
Measure Analysis Population Description: 10 patients were enrolled in Phase 1 and 10 patients were enrolled in Phase 2 of the study. Overall number of patients enrolled was 20.
Prior radiation   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Phase 1 Number Analyzed 10 participants
7
  70.0%
Phase 2 Number Analyzed 10 participants
6
  60.0%
[1]
Measure Analysis Population Description: 10 patients were enrolled in Phase 1 and 10 patients were enrolled in Phase 2 of the study. Overall number of patients enrolled was 20.
Prior surgery   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Phase 1 Number Analyzed 10 participants
10
 100.0%
Phase 2 Number Analyzed 10 participants
9
  90.0%
[1]
Measure Analysis Population Description: 10 patients were enrolled in Phase 1 and 10 patients were enrolled in Phase 2 of the study. Overall number of patients enrolled was 20.
1.Primary Outcome
Title Number of Dose Limiting Toxicities of Ganetespib When Administered in Combination With Sirolimus.
Hide Description To assess the safety, tolerability, and maximum tolerated/ recommended dose of ganetespib when administered in combination with sirolimus in patients with refractory sarcomas or unresectable or metastatic sporadic or neurofibromatosis type 1 (NF1) associated MPNST. Toxicities observed during the first cycle will be used to define the MTD/Recommended dose. Toxicity will be graded using the NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.0. DLT will be defined as any of the following events that are possibly, probably, or definitely attributable to ganetespib or sirolimus. The DLT observation period for the purposes of dose escalation will be the first cycle of therapy.
Time Frame Toxicities will be evaluated over the first treatment cycle (each cycle=28 days)
Hide Outcome Measure Data
Hide Analysis Population Description
10 patients were enrolled in Phase 1 of the study to determine MTD/RD. Three patients were enrolled in the first dose level. Six patients were enrolled at dose level 2. 1 patient was inevaluable.
Arm/Group Title Ganetespib / Sirolimus
Hide Arm/Group Description:

28-day cycles of ganetespib + sirolimus

ganetespib: 200 mg/m² IV on days 1, 8, and 15 intravenously over 1 hour

Sirolimus: 2mg taken orally once daily on a continuous dosing schedule; Loading dose 12 mg on cycle 1 day 1 only.

Overall Number of Participants Analyzed 9
Measure Type: Number
Unit of Measure: DLTs
Dose Level 1 Number Analyzed 3 participants
0
Dose Level 2 Number Analyzed 6 participants
1
2.Primary Outcome
Title Clinical Benefit of Ganetespib in Combination With Sirolimus
Hide Description Assessed using the World Health Organization (WHO) criteria. Tumor assessments will be obtained every 2 cycles. Clinical benefit is defined as stable disease, Partial Response (PR), Complete Response (CR). For patients who experience progression by WHO but in the opinion of the treating investigator are deriving benefit from therapy and have not otherwise met off treatment or off study criteria, may continue on treatment as long as patient has not met progression by RECIST 1.1.
Time Frame Response evaluations will be performed after every 2 treatment cycles (each cycle=28 days)
Hide Outcome Measure Data
Hide Analysis Population Description
10 patients were enrolled in Phase 1 and 10 patients were enrolled in Phase 2 of the study. Overall number of patients enrolled was 20.
Arm/Group Title Ganetespib / Sirolimus
Hide Arm/Group Description:

28-day cycles of ganetespib + sirolimus

ganetespib: 200 mg/m² IV on days 1, 8, and 15 intravenously over 1 hour

Sirolimus: 2mg taken orally once daily on a continuous dosing schedule; Loading dose 12 mg on cycle 1 day 1 only.

Overall Number of Participants Analyzed 20
Measure Type: Count of Participants
Unit of Measure: Participants
Phase 1 Number Analyzed 10 participants
1
  10.0%
Phase 2 Number Analyzed 10 participants
0
   0.0%
3.Secondary Outcome
Title Change in Plasma Pharmacokinetic Profile of Ganetespib and Sirolimus When Administered in Combination-Observed Maximum Plasma Concentration (Cmax)
Hide Description To describe the plasma pharmacokinetic profile of ganetespib and sirolimus when administered in combination therapy.
Time Frame Pre-therapy levels drawn at baseline and pharmacokinetic analysis occurs on Cycle 1 Day 15
Hide Outcome Measure Data
Hide Analysis Population Description
10 patients were enrolled in Phase 1 of the study. Three patients were enrolled in the first dose level. Six patients were enrolled at dose level 2. 1 patient was inevaluable.
Arm/Group Title Ganetespib / Sirolimus
Hide Arm/Group Description:

28-day cycles of ganetespib + sirolimus

ganetespib: 200 mg/m² IV on days 1, 8, and 15 intravenously over 1 hour

Sirolimus: 2mg taken orally once daily on a continuous dosing schedule; Loading dose 12 mg on cycle 1 day 1 only.

Overall Number of Participants Analyzed 9
Mean (Standard Deviation)
Unit of Measure: ng/mL
Dose Level 1- Sirolimus trough Number Analyzed 3 participants
12.1  (3.6)
Dose Level 2- Sirolimus trough Number Analyzed 6 participants
12.5  (8.9)
4.Secondary Outcome
Title Changes in Pharmacodynamic Parameters in Peripheral Blood Mononuclear Cells
Hide Description To explore changes in pharmacodynamic parameters in peripheral blood mononuclear cells performed on day 1 prior to ganetespib and sirolimus administration, and on day 15, 6 hours post drug administration. Hsp inhibition (Hsp70), mTOR inhibition (phospho-S6 and Akt Phosphorylation), UPR activation (EIF2alpha phosphorylation) will be explored. Western blot analyses were performed for phospho (p)-Akt, p-eIF2α, p-S6, and Hsp70. The absorbance of each phosphoprotein lane was recorded and protein levels were determined after normalizing for levels of corresponding total protein.
Time Frame Baseline and Cycle 1 Day 15
Hide Outcome Measure Data
Hide Analysis Population Description
11 subjects provided consent and had adequate specimens for analysis.
Arm/Group Title Ganetespib / Sirolimus
Hide Arm/Group Description:

28-day cycles of ganetespib + sirolimus

ganetespib: 200 mg/m² IV on days 1, 8, and 15 intravenously over 1 hour

Sirolimus: 2mg taken orally once daily on a continuous dosing schedule; Loading dose 12 mg on cycle 1 day 1 only.

Overall Number of Participants Analyzed 11
Mean (Standard Deviation)
Unit of Measure: ratio
Hsp70 at Baseline 0.81  (0.39)
Hsp70 at Cycle 1 Day 15 0.84  (0.56)
pAKT at Baseline 1.20  (0.70)
pAKT at Cycle 1 Day 15 1.44  (1.00)
p-S6 at Baseline 1.25  (0.40)
p-S6 at Cycle 1 Day 15 0.54  (0.24)
p-eiF2alpha at Baseline 1.02  (0.26)
p-eiF2alpha at Cycle 1 Day 15 0.49  (0.22)
5.Secondary Outcome
Title Patient-reported Pain Severity and the Impact of Pain on Daily Activities
Hide Description To assess patient-reported pain severity and the impact of pain on daily activities before and during treatment with ganetespib and sirolimus. The Numerical Rating Scale-11 (NRS-11) will be used to assess pain severity. The NRS-11 is a self-report segmented 11-point numeric scale that assesses pain severity. It consists of a horizontal line with 0 representing "no pain" at the right end of the line and 10 representing "worst pain you can imagine" at the left end.The Brief Pain Inventory is a 7-item self-report questionnaire that measures the extent to which pain interferes with daily functioning. Patients are asked to indicate how much pain interfered with various activities in the past week, with scores ranging from 0 (does not interfere) to 10 (completely interferes). A total score is obtained by taking the mean of the scores for all 7 items; thus, the total pain interference score can range from 0 to 10.
Time Frame Baseline and prior to Cycle 3
Hide Outcome Measure Data
Hide Analysis Population Description
Thirteen subjects in phase 1 and phase 2 cohorts combined had MPNST and completed the pain evaluations. 4/13 subjects completed both baseline and pre-cycle 3 evaluations for pain.
Arm/Group Title Ganetespib / Sirolimus
Hide Arm/Group Description:

28-day cycles of ganetespib + sirolimus

ganetespib: 200 mg/m² IV on days 1, 8, and 15 intravenously over 1 hour

Sirolimus: 2mg taken orally once daily on a continuous dosing schedule; Loading dose 12 mg on cycle 1 day 1 only.

Overall Number of Participants Analyzed 4
Mean (Full Range)
Unit of Measure: units on a scale
Baseline- Overall pain intensity
7.5
(0 to 10)
Baseline- Tumor pain intensity
7.5
(0 to 10)
Baseline- Pain interference
6.49
(0 to 10)
Prior to Cycle 3- Overall pain intensity
5
(0 to 10)
Prior to Cycle 3- Tumor pain intensity
4.75
(0 to 10)
Prior to Cycle 3- Pain interference
3.14
(0 to 10)
6.Secondary Outcome
Title Utility of Three-dimensional MRI (3D-MRI) Analysis in Comparison to 1-dimensional and 2-dimensional Measurements
Hide Description To evaluate the utility of three-dimensional MRI (3D-MRI) analysis in comparison to 1-dimensional and 2-dimensional measurements as a method to more sensitively monitor response.
Time Frame 4 months
Hide Outcome Measure Data
Hide Analysis Population Description
The 3D-MRI were not measurable with current modalities. Due to their nature and incomplete imaging, volumetric analysis was not feasible.
Arm/Group Title Ganetespib / Sirolimus
Hide Arm/Group Description:

28-day cycles of ganetespib + sirolimus

ganetespib: 200 mg/m² IV on days 1, 8, and 15 intravenously over 1 hour

Sirolimus: 2mg taken orally once daily on a continuous dosing schedule; Loading dose 12 mg on cycle 1 day 1 only.

Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
7.Secondary Outcome
Title Plasma Pharmacokinetic Profile of Ganetespib When Administered in Combination With Sirolimus
Hide Description To describe the plasma pharmacokinetic profile of ganetespib in terms of half life.
Time Frame Cycle 1 Day 15
Hide Outcome Measure Data
Hide Analysis Population Description
10 patients were enrolled in Phase 1 of the study. 1 patient was inevaluable.
Arm/Group Title Ganetespib / Sirolimus
Hide Arm/Group Description:

28-day cycles of ganetespib + sirolimus

ganetespib: 200 mg/m² IV on days 1, 8, and 15 intravenously over 1 hour

Sirolimus: 2mg taken orally once daily on a continuous dosing schedule; Loading dose 12 mg on cycle 1 day 1 only.

Overall Number of Participants Analyzed 9
Mean (Standard Deviation)
Unit of Measure: hours
Ganetespib half life for dose level 1 Number Analyzed 3 participants
6.4  (2.1)
Ganetespib half life for dose level 2 Number Analyzed 6 participants
6.0  (0.9)
8.Secondary Outcome
Title Determine Maximum Tolerated Dose (MTD)/Recommended Dose (RD) of Ganetespib
Hide Description A conventional 3+3 dose escalation design was used for phase 1. All patients in phase 2 were treated with the recommended dose.
Time Frame Phase 1 of study
Hide Outcome Measure Data
Hide Analysis Population Description
1 patient was inevaluable in Phase 1 of study. The recommended dose of ganetespib was determined to be 200 mg/m2 intravenously on days 1, 8, 15 with sirolimus 4mg orally once daily with a cycle 1 day 1 loading dose of 12mg. All patients in phase 2 were treated with the recommended dose.
Arm/Group Title Phase 1 Dose Level 1 Phase 1 Dose Level 2 Phase 2
Hide Arm/Group Description:

For each 28-day cycle, subjects took doses of both ganetespib + sirolimus:

ganetespib: 150 mg/m² IV on days 1, 8, and 15 intravenously over 1 hour

Sirolimus: 4mg taken orally once daily on a continuous dosing schedule; Loading dose 12 mg on cycle 1 day 1 only.

For each 28-day cycle, subjects took doses of both ganetespib + sirolimus:

ganetespib: 200 mg/m² IV on days 1, 8, and 15 intravenously over 1 hour

Sirolimus: 4mg taken orally once daily on a continuous dosing schedule; Loading dose 12 mg on cycle 1 day 1 only.

For each 28-day cycle, subjects took doses of both ganetespib + sirolimus:

ganetespib: 200 mg/m² IV on days 1, 8, and 15 intravenously over 1 hour

Sirolimus: 4mg taken orally once daily on a continuous dosing schedule; Loading dose 12 mg on cycle 1 day 1 only.

Overall Number of Participants Analyzed 3 6 10
Measure Type: Count of Participants
Unit of Measure: Participants
3
 100.0%
6
 100.0%
10
 100.0%
Time Frame 3 years
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Phase 1 Dose 1 Phase 1 Dose 2 Phase 2
Hide Arm/Group Description

For each 28-day cycle, subjects took doses of both ganetespib + sirolimus:

ganetespib: 150 mg/m² IV on days 1, 8, and 15 intravenously over 1 hour

Sirolimus: 4mg taken orally once daily on a continuous dosing schedule; Loading dose 12 mg on cycle 1 day 1 only.

For each 28-day cycle, subjects took doses of both ganetespib + sirolimus:

ganetespib: 200 mg/m² IV on days 1, 8, and 15 intravenously over 1 hour

Sirolimus: 4mg taken orally once daily on a continuous dosing schedule; Loading dose 12 mg on cycle 1 day 1 only.

For each 28-day cycle, subjects took doses of both ganetespib + sirolimus:

ganetespib: 200 mg/m² IV on days 1, 8, and 15 intravenously over 1 hour

Sirolimus: 4mg taken orally once daily on a continuous dosing schedule; Loading dose 12 mg on cycle 1 day 1 only.

All-Cause Mortality
Phase 1 Dose 1 Phase 1 Dose 2 Phase 2
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/3 (0.00%)      1/7 (14.29%)      1/10 (10.00%)    
Hide Serious Adverse Events
Phase 1 Dose 1 Phase 1 Dose 2 Phase 2
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   1/3 (33.33%)      2/7 (28.57%)      6/10 (60.00%)    
Gastrointestinal disorders       
Gastrointestinal disorders- other   0/3 (0.00%)  0 0/7 (0.00%)  0 1/10 (10.00%)  1
Diarrhea   0/3 (0.00%)  0 0/7 (0.00%)  0 1/10 (10.00%)  1
Gastric obstruction   0/3 (0.00%)  0 0/7 (0.00%)  0 1/10 (10.00%)  1
General disorders       
Fever   0/3 (0.00%)  0 1/7 (14.29%)  1 0/10 (0.00%)  0
Infusion related reaction   0/3 (0.00%)  0 1/7 (14.29%)  1 0/10 (0.00%)  0
Hepatobiliary disorders       
Cholecystitis   0/3 (0.00%)  0 1/7 (14.29%)  1 1/10 (10.00%)  1
Infections and infestations       
Lung infection   0/3 (0.00%)  0 0/7 (0.00%)  0 1/10 (10.00%)  1
Investigations       
Alanine Aminotransferase increased   0/3 (0.00%)  0 0/7 (0.00%)  0 1/10 (10.00%)  1
Aspartate Aminotransferase increased   0/3 (0.00%)  0 0/7 (0.00%)  0 1/10 (10.00%)  1
Lymphocyte count decreased   0/3 (0.00%)  0 0/7 (0.00%)  0 1/10 (10.00%)  1
Metabolism and nutrition disorders       
Dehydration   0/3 (0.00%)  0 0/7 (0.00%)  0 1/10 (10.00%)  1
Hypoglycemia   1/3 (33.33%)  1 0/7 (0.00%)  0 0/10 (0.00%)  0
Neoplasms benign, malignant and unspecified (incl cysts and polyps)       
Tumor pain   0/3 (0.00%)  0 0/7 (0.00%)  0 1/10 (10.00%)  1
Renal and urinary disorders       
Urinary retention   0/3 (0.00%)  0 0/7 (0.00%)  0 1/10 (10.00%)  1
Respiratory, thoracic and mediastinal disorders       
Dyspnea   1/3 (33.33%)  1 0/7 (0.00%)  0 0/10 (0.00%)  0
Vascular disorders       
Thromboembolic event   0/3 (0.00%)  0 0/7 (0.00%)  0 1/10 (10.00%)  1
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Phase 1 Dose 1 Phase 1 Dose 2 Phase 2
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   3/3 (100.00%)      7/7 (100.00%)      10/10 (100.00%)    
Blood and lymphatic system disorders       
Anemia   1/3 (33.33%)  1 2/7 (28.57%)  4 3/10 (30.00%)  3
Cardiac disorders       
Sinus tachycardia   0/3 (0.00%)  0 0/7 (0.00%)  0 2/10 (20.00%)  2
Eye disorders       
Dry eye   0/3 (0.00%)  0 1/7 (14.29%)  1 0/10 (0.00%)  0
Gastrointestinal disorders       
Abdominal pain   0/3 (0.00%)  0 0/7 (0.00%)  0 2/10 (20.00%)  2
Cheilitis   1/3 (33.33%)  1 0/7 (0.00%)  0 0/10 (0.00%)  0
Constipation   0/3 (0.00%)  0 0/7 (0.00%)  0 1/10 (10.00%)  1
Dental caries   0/3 (0.00%)  0 1/7 (14.29%)  1 0/10 (0.00%)  0
Diarrhea   2/3 (66.67%)  5 7/7 (100.00%)  11 8/10 (80.00%)  14
Dry mouth   0/3 (0.00%)  0 2/7 (28.57%)  2 2/10 (20.00%)  2
Dyspepsia   0/3 (0.00%)  0 0/7 (0.00%)  0 1/10 (10.00%)  1
Dysphagia   0/3 (0.00%)  0 1/7 (14.29%)  1 0/10 (0.00%)  0
Gastroesophageal reflux disease   0/3 (0.00%)  0 0/7 (0.00%)  0 1/10 (10.00%)  1
Gastrointestinal disorders-other   0/3 (0.00%)  0 1/7 (14.29%)  1 2/10 (20.00%)  2
Gastrointestinal pain   0/3 (0.00%)  0 1/7 (14.29%)  1 0/10 (0.00%)  0
Hemorrhoids   0/3 (0.00%)  0 1/7 (14.29%)  1 0/10 (0.00%)  0
Mucositis oral   2/3 (66.67%)  2 2/7 (28.57%)  4 1/10 (10.00%)  1
Nausea   1/3 (33.33%)  2 3/7 (42.86%)  3 7/10 (70.00%)  9
Oral pain   0/3 (0.00%)  0 1/7 (14.29%)  1 0/10 (0.00%)  0
Vomiting   1/3 (33.33%)  1 1/7 (14.29%)  1 5/10 (50.00%)  6
General disorders       
Chills   0/3 (0.00%)  0 1/7 (14.29%)  1 0/10 (0.00%)  0
Edema limbs   0/3 (0.00%)  0 2/7 (28.57%)  3 1/10 (10.00%)  1
Edema trunk   0/3 (0.00%)  0 1/7 (14.29%)  1 0/10 (0.00%)  0
Fatigue   2/3 (66.67%)  3 1/7 (14.29%)  1 6/10 (60.00%)  6
Fever   0/3 (0.00%)  0 1/7 (14.29%)  1 2/10 (20.00%)  2
General disorders and admin site conditions-other   1/3 (33.33%)  3 0/7 (0.00%)  0 1/10 (10.00%)  2
Infusion related reaction   0/3 (0.00%)  0 2/7 (28.57%)  2 1/10 (10.00%)  1
Irritability   0/3 (0.00%)  0 1/7 (14.29%)  2 0/10 (0.00%)  0
Non-cardiac chest pain   0/3 (0.00%)  0 2/7 (28.57%)  2 0/10 (0.00%)  0
Pain   0/3 (0.00%)  0 1/7 (14.29%)  1 0/10 (0.00%)  0
Hepatobiliary disorders       
Gallbladder pain   0/3 (0.00%)  0 1/7 (14.29%)  1 0/10 (0.00%)  0
Infections and infestations       
Infections and infestations-other   0/3 (0.00%)  0 0/7 (0.00%)  0 1/10 (10.00%)  1
Lung infection   1/3 (33.33%)  1 0/7 (0.00%)  0 0/10 (0.00%)  0
Injury, poisoning and procedural complications       
Bruising   1/3 (33.33%)  1 0/7 (0.00%)  0 0/10 (0.00%)  0
Fall   0/3 (0.00%)  0 0/7 (0.00%)  0 1/10 (10.00%)  1
Investigations       
Activated Partial Thromboplastin Time Prolonged   0/3 (0.00%)  0 2/7 (28.57%)  2 3/10 (30.00%)  3
Alanine Aminotransferase increased   1/3 (33.33%)  3 1/7 (14.29%)  2 2/10 (20.00%)  8
Alkaline Phosphatase increased   1/3 (33.33%)  1 1/7 (14.29%)  2 5/10 (50.00%)  9
Aspartate Aminotransferase increased   2/3 (66.67%)  2 1/7 (14.29%)  2 3/10 (30.00%)  6
Blood bilirubin increased   0/3 (0.00%)  0 1/7 (14.29%)  1 1/10 (10.00%)  2
High cholesterol   0/3 (0.00%)  0 2/7 (28.57%)  3 0/10 (0.00%)  0
CPK increased   0/3 (0.00%)  0 1/7 (14.29%)  2 0/10 (0.00%)  0
Creatinine increased   0/3 (0.00%)  0 2/7 (28.57%)  2 2/10 (20.00%)  2
Investigations-other   1/3 (33.33%)  1 0/7 (0.00%)  0 0/10 (0.00%)  0
Lymphocyte count decreased   2/3 (66.67%)  3 3/7 (42.86%)  8 4/10 (40.00%)  8
Neutrophil count decreased   1/3 (33.33%)  3 0/7 (0.00%)  0 1/10 (10.00%)  1
Platelet count decreased   2/3 (66.67%)  2 3/7 (42.86%)  7 2/10 (20.00%)  2
Weight loss   0/3 (0.00%)  0 1/7 (14.29%)  1 0/10 (0.00%)  0
White blood cell decreased   2/3 (66.67%)  3 0/7 (0.00%)  0 2/10 (20.00%)  2
Metabolism and nutrition disorders       
Anorexia   2/3 (66.67%)  3 0/7 (0.00%)  0 1/10 (10.00%)  2
Dehydration   0/3 (0.00%)  0 0/7 (0.00%)  0 1/10 (10.00%)  1
Hypercalcemia   0/3 (0.00%)  0 0/7 (0.00%)  0 1/10 (10.00%)  1
Hyperglycemia   2/3 (66.67%)  3 2/7 (28.57%)  3 2/10 (20.00%)  2
Hyperkalemia   0/3 (0.00%)  0 1/7 (14.29%)  1 0/10 (0.00%)  0
Hypertriglyceridemia   0/3 (0.00%)  0 2/7 (28.57%)  5 0/10 (0.00%)  0
Hypoalbuminemia   0/3 (0.00%)  0 1/7 (14.29%)  3 3/10 (30.00%)  4
Hypocalcemia   0/3 (0.00%)  0 0/7 (0.00%)  0 1/10 (10.00%)  1
Hypokalemia   1/3 (33.33%)  2 0/7 (0.00%)  0 1/10 (10.00%)  1
Hyponatremia   0/3 (0.00%)  0 2/7 (28.57%)  3 4/10 (40.00%)  4
Hypophosphatemia   2/3 (66.67%)  2 1/7 (14.29%)  1 1/10 (10.00%)  1
Musculoskeletal and connective tissue disorders       
Arthralgia   1/3 (33.33%)  1 0/7 (0.00%)  0 0/10 (0.00%)  0
Back pain   1/3 (33.33%)  1 1/7 (14.29%)  1 1/10 (10.00%)  1
Chest wall pain   0/3 (0.00%)  0 2/7 (28.57%)  2 0/10 (0.00%)  0
Muscle weakness lower limb   0/3 (0.00%)  0 0/7 (0.00%)  0 1/10 (10.00%)  1
Musculoskeletal and connective tissue disorder-other   0/3 (0.00%)  0 1/7 (14.29%)  1 1/10 (10.00%)  1
Pain in extremity   0/3 (0.00%)  0 1/7 (14.29%)  1 0/10 (0.00%)  0
Neoplasms benign, malignant and unspecified (incl cysts and polyps)       
Tumor pain   0/3 (0.00%)  0 1/7 (14.29%)  1 0/10 (0.00%)  0
Nervous system disorders       
Ataxia   0/3 (0.00%)  0 1/7 (14.29%)  1 0/10 (0.00%)  0
Headache   2/3 (66.67%)  4 3/7 (42.86%)  3 0/10 (0.00%)  0
Psychiatric disorders       
Insomnia   1/3 (33.33%)  1 2/7 (28.57%)  2 1/10 (10.00%)  1
Libido decreased   0/3 (0.00%)  0 1/7 (14.29%)  1 0/10 (0.00%)  0
Renal and urinary disorders       
Proteinuria   0/3 (0.00%)  0 1/7 (14.29%)  1 1/10 (10.00%)  1
Urinary frequency   1/3 (33.33%)  1 0/7 (0.00%)  0 0/10 (0.00%)  0
Reproductive system and breast disorders       
Erectile Dysfunction   0/3 (0.00%)  0 1/7 (14.29%)  1 0/10 (0.00%)  0
Respiratory, thoracic and mediastinal disorders       
Allergic Rhinitis   0/3 (0.00%)  0 0/7 (0.00%)  0 2/10 (20.00%)  2
Bronchopulmonary hemorrhage   0/3 (0.00%)  0 0/7 (0.00%)  0 1/10 (10.00%)  1
Cough   2/3 (66.67%)  2 0/7 (0.00%)  0 1/10 (10.00%)  1
Dyspnea   1/3 (33.33%)  1 0/7 (0.00%)  0 1/10 (10.00%)  1
Nasal congestion   1/3 (33.33%)  1 1/7 (14.29%)  1 1/10 (10.00%)  1
Pleural effusion   0/3 (0.00%)  0 0/7 (0.00%)  0 1/10 (10.00%)  1
Postnasal drip   0/3 (0.00%)  0 1/7 (14.29%)  1 0/10 (0.00%)  0
Respiratory, thoracic and mediastinal disorders-other   1/3 (33.33%)  1 0/7 (0.00%)  0 0/10 (0.00%)  0
Sore throat   0/3 (0.00%)  0 1/7 (14.29%)  1 1/10 (10.00%)  1
Skin and subcutaneous tissue disorders       
Dry skin   0/3 (0.00%)  0 1/7 (14.29%)  1 0/10 (0.00%)  0
Hyperhidrosis   0/3 (0.00%)  0 1/7 (14.29%)  2 0/10 (0.00%)  0
Pruritus   0/3 (0.00%)  0 2/7 (28.57%)  2 0/10 (0.00%)  0
Rash acneiform   0/3 (0.00%)  0 1/7 (14.29%)  1 0/10 (0.00%)  0
Rash maculo-papular   1/3 (33.33%)  1 2/7 (28.57%)  2 1/10 (10.00%)  1
Skin and subcutaneous tissue disorders-other   0/3 (0.00%)  0 1/7 (14.29%)  1 1/10 (10.00%)  1
Surgical and medical procedures       
Surgical and medical procedures-other   0/3 (0.00%)  0 1/7 (14.29%)  1 0/10 (0.00%)  0
Vascular disorders       
Hypertension   0/3 (0.00%)  0 0/7 (0.00%)  0 2/10 (20.00%)  4
Thromboembolic event   1/3 (33.33%)  1 1/7 (14.29%)  1 0/10 (0.00%)  0
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT 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: SARC
Organization: SARC
Phone: (734) 930-7600
EMail: sarc@sarctrials.org
Layout table for additonal information
Responsible Party: Sarcoma Alliance for Research through Collaboration
ClinicalTrials.gov Identifier: NCT02008877    
Other Study ID Numbers: SARC023
CDMRP-NF120087 ( Other Grant/Funding Number: Department of Defense )
First Submitted: November 25, 2013
First Posted: December 11, 2013
Results First Submitted: September 6, 2018
Results First Posted: May 2, 2019
Last Update Posted: May 15, 2019