Trial Outcomes & Findings for Therapeutic Trial for Patients With Ewing Sarcoma Family of Tumor and Desmoplastic Small Round Cell Tumors (NCT NCT01946529)
NCT ID: NCT01946529
Last Updated: 2025-09-04
Results Overview
Response rate will be defined as the proportion of patients who achieved complete response or partial response (CR+PR) using the World Health Organization (WHO) criteria evaluated after two initial courses of temsirolimus, temozolomide and irinotecan in previously untreated patients with high risk Ewing Sarcoma Family of Tumor (ESFT). Participants who are treated in Group B with Desmoplastic Small Round Cell Tumor (DSRCT) or those who do not receive window therapy will not be included in this analysis.
Recruitment status
ACTIVE_NOT_RECRUITING
Study phase
PHASE2
Target enrollment
24 participants
Primary outcome timeframe
at 6 weeks after start of therapy (after 2 initial courses)
Results posted on
2025-09-04
Participant Flow
Participants were enrolled at St. Jude Children's Research Hospital between December 2013 and June 2015.
Participant milestones
| Measure |
Group A (Standard Risk)
Participants will receive vincristine, doxorubicin, cyclophosphamide, ifosfamide and etoposide. Doxorubicin will be omitted following a total cumulative dose of 375 mg/m\^2. Depending on the size and location of the participant's tumor, they will have surgery alone, radiation alone, or surgery followed by radiation. Local control measures (surgery and/or radiation therapy) will be instituted after 6 courses of chemotherapy. Total duration of treatment is approximately 29 weeks.
|
Group B (High Risk) - ESFT
Group B participants with a diagnosis of Ewing Sarcoma Family of Tumor (ESFT).
|
Group B (High Risk) - DSRCT
Group B participants with a diagnosis of Desmoplastic Small Round Cell Tumor (DSRCT).
|
|---|---|---|---|
|
End of Window Therapy (2 Courses)
STARTED
|
1
|
17
|
6
|
|
End of Window Therapy (2 Courses)
COMPLETED
|
1
|
16
|
6
|
|
End of Window Therapy (2 Courses)
NOT COMPLETED
|
0
|
1
|
0
|
|
Completion of All Therapy
STARTED
|
1
|
16
|
6
|
|
Completion of All Therapy
COMPLETED
|
0
|
0
|
0
|
|
Completion of All Therapy
NOT COMPLETED
|
1
|
16
|
6
|
Reasons for withdrawal
| Measure |
Group A (Standard Risk)
Participants will receive vincristine, doxorubicin, cyclophosphamide, ifosfamide and etoposide. Doxorubicin will be omitted following a total cumulative dose of 375 mg/m\^2. Depending on the size and location of the participant's tumor, they will have surgery alone, radiation alone, or surgery followed by radiation. Local control measures (surgery and/or radiation therapy) will be instituted after 6 courses of chemotherapy. Total duration of treatment is approximately 29 weeks.
|
Group B (High Risk) - ESFT
Group B participants with a diagnosis of Ewing Sarcoma Family of Tumor (ESFT).
|
Group B (High Risk) - DSRCT
Group B participants with a diagnosis of Desmoplastic Small Round Cell Tumor (DSRCT).
|
|---|---|---|---|
|
End of Window Therapy (2 Courses)
Physician Decision
|
0
|
1
|
0
|
|
Completion of All Therapy
Some pts. still receiving study therapy
|
1
|
16
|
6
|
Baseline Characteristics
Therapeutic Trial for Patients With Ewing Sarcoma Family of Tumor and Desmoplastic Small Round Cell Tumors
Baseline characteristics by cohort
| Measure |
Group A (Standard Risk)
n=1 Participants
Participants will receive vincristine, doxorubicin, cyclophosphamide, ifosfamide and etoposide. Doxorubicin will be omitted following a total cumulative dose of 375 mg/m\^2. Depending on the size and location of the participant's tumor, they will have surgery alone, radiation alone, or surgery followed by radiation. Local control measures (surgery and/or radiation therapy) will be instituted after 6 courses of chemotherapy. Total duration of treatment is approximately 29 weeks.
|
Group B (High Risk) - ESFT
n=16 Participants
Group B participants with a diagnosis of Ewing Sarcoma Family of Tumor (ESFT).
|
Group B (High Risk) - DSRCT
n=6 Participants
Group B participants with a diagnosis of Desmoplastic Small Round Cell Tumor (DSRCT).
|
Total
n=23 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
12.0 years
STANDARD_DEVIATION 0 • n=5 Participants
|
13.0 years
STANDARD_DEVIATION 5.7 • n=7 Participants
|
14.7 years
STANDARD_DEVIATION 4.1 • n=5 Participants
|
13.4 years
STANDARD_DEVIATION 5.2 • n=4 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
10 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
13 Participants
n=4 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
|
Race (NIH/OMB)
White
|
1 Participants
n=5 Participants
|
14 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
18 Participants
n=4 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
|
Primary disease site
Pelvis
|
0 participants
n=5 Participants
|
6 participants
n=7 Participants
|
5 participants
n=5 Participants
|
11 participants
n=4 Participants
|
|
Primary disease site
Other
|
1 participants
n=5 Participants
|
10 participants
n=7 Participants
|
1 participants
n=5 Participants
|
12 participants
n=4 Participants
|
|
Disease stage
Metastases
|
0 participants
n=5 Participants
|
11 participants
n=7 Participants
|
5 participants
n=5 Participants
|
16 participants
n=4 Participants
|
|
Disease stage
Local
|
1 participants
n=5 Participants
|
5 participants
n=7 Participants
|
1 participants
n=5 Participants
|
7 participants
n=4 Participants
|
PRIMARY outcome
Timeframe: at 6 weeks after start of therapy (after 2 initial courses)Population: Of the 17 Group B participants with high-risk ESFT, 12 received window therapy and are considered evaluable for this outcome.
Response rate will be defined as the proportion of patients who achieved complete response or partial response (CR+PR) using the World Health Organization (WHO) criteria evaluated after two initial courses of temsirolimus, temozolomide and irinotecan in previously untreated patients with high risk Ewing Sarcoma Family of Tumor (ESFT). Participants who are treated in Group B with Desmoplastic Small Round Cell Tumor (DSRCT) or those who do not receive window therapy will not be included in this analysis.
Outcome measures
| Measure |
Group B (High Risk) - ESFT
n=12 Participants
Participants received vincristine, doxorubicin, cyclophosphamide, ifosfamide, and etoposide. Patients with measurable disease were eligible to receive irinotecan, temozolomide, and temsirolimus. Additionally, all patients were eligible to receive a maintenance therapy at the end of treatment including bevacizumab and sorafenib. Depending on the size and location of the participant's tumor, they had surgery alone, radiation alone, or surgery followed by radiation.
|
|---|---|
|
Response to Window Therapy (2 Courses) for Group B (High-risk) - ESFT Participants
Partial Response (PR)
|
3 participants
|
|
Response to Window Therapy (2 Courses) for Group B (High-risk) - ESFT Participants
Stable disease (no response) (NR)
|
8 participants
|
|
Response to Window Therapy (2 Courses) for Group B (High-risk) - ESFT Participants
Progressive Disease (PD)
|
1 participants
|
SECONDARY outcome
Timeframe: Maximum of 11 years after the start of therapyOverall survival (OS) is defined as the time interval from the date on study to the date of death or the date of last follow-up. OS will be estimated using the method of Kaplan-Meier for group A and B participants, respectively.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Maximum of 11 years after the start of therapyProgression free survival (PFS) is defined as the time interval from the date on study to the date of disease progression or death or the date if last follow-up. PFS will be estimated using the method of Kaplan-Meier for group A and B participants, respectively.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Maximum of 11 years after the start of therapyMedian time to progression of group B patients will be estimated from the Kaplan-Meier curve.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Maximum of 11 years after the start of therapyLoco-regional failure is defined as the time interval from date of start of local therapy to date of loco-regional failure. Distant failure or death prior to loco-regional failure will be considered competing events in the analyses. The cumulative incidence of loco-regional failure will be estimated using methods described in Kalbfleisch and Prentice.
Outcome measures
Outcome data not reported
Adverse Events
Group A (Standard Risk)
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Group B (High Risk) - ESFT
Serious events: 1 serious events
Other events: 16 other events
Deaths: 0 deaths
Group B (High Risk) - DSRCT
Serious events: 0 serious events
Other events: 6 other events
Deaths: 0 deaths
Group B (High Risk) - Total
Serious events: 1 serious events
Other events: 22 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Group A (Standard Risk)
n=1 participants at risk
Participants will receive vincristine, doxorubicin, cyclophosphamide, ifosfamide and etoposide. Doxorubicin will be omitted following a total cumulative dose of 375 mg/m\^2. Depending on the size and location of the participant's tumor, they will have surgery alone, radiation alone, or surgery followed by radiation. Local control measures (surgery and/or radiation therapy) will be instituted after 6 courses of chemotherapy. Total duration of treatment is approximately 29 weeks.
|
Group B (High Risk) - ESFT
n=16 participants at risk
Group B participants with a diagnosis of Ewing Sarcoma Family of Tumor (ESFT).
|
Group B (High Risk) - DSRCT
n=6 participants at risk
Group B participants with a diagnosis of Desmoplastic Small Round Cell Tumor (DSRCT).
|
Group B (High Risk) - Total
n=22 participants at risk
All Group B High Risk participants with ESFT or DSRCT.
Participants received vincristine, doxorubicin, cyclophosphamide, ifosfamide, and etoposide. Patients with measurable disease were eligible to receive irinotecan, temozolomide, and temsirolimus. Additionally, all patients were eligible to receive a maintenance therapy at the end of treatment including bevacizumab and sorafenib. Depending on the size and location of the participant's tumor, they had surgery alone, radiation alone, or surgery followed by radiation.
|
|---|---|---|---|---|
|
Gastrointestinal disorders
Diarrhea
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
6.2%
1/16 • Number of events 2 • Adverse events were collected from the on-study date through 05/03/2016.
|
0.00%
0/6 • Adverse events were collected from the on-study date through 05/03/2016.
|
4.5%
1/22 • Number of events 2 • Adverse events were collected from the on-study date through 05/03/2016.
|
Other adverse events
| Measure |
Group A (Standard Risk)
n=1 participants at risk
Participants will receive vincristine, doxorubicin, cyclophosphamide, ifosfamide and etoposide. Doxorubicin will be omitted following a total cumulative dose of 375 mg/m\^2. Depending on the size and location of the participant's tumor, they will have surgery alone, radiation alone, or surgery followed by radiation. Local control measures (surgery and/or radiation therapy) will be instituted after 6 courses of chemotherapy. Total duration of treatment is approximately 29 weeks.
|
Group B (High Risk) - ESFT
n=16 participants at risk
Group B participants with a diagnosis of Ewing Sarcoma Family of Tumor (ESFT).
|
Group B (High Risk) - DSRCT
n=6 participants at risk
Group B participants with a diagnosis of Desmoplastic Small Round Cell Tumor (DSRCT).
|
Group B (High Risk) - Total
n=22 participants at risk
All Group B High Risk participants with ESFT or DSRCT.
Participants received vincristine, doxorubicin, cyclophosphamide, ifosfamide, and etoposide. Patients with measurable disease were eligible to receive irinotecan, temozolomide, and temsirolimus. Additionally, all patients were eligible to receive a maintenance therapy at the end of treatment including bevacizumab and sorafenib. Depending on the size and location of the participant's tumor, they had surgery alone, radiation alone, or surgery followed by radiation.
|
|---|---|---|---|---|
|
Gastrointestinal disorders
Diarrhea
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
93.8%
15/16 • Number of events 130 • Adverse events were collected from the on-study date through 05/03/2016.
|
100.0%
6/6 • Number of events 32 • Adverse events were collected from the on-study date through 05/03/2016.
|
95.5%
21/22 • Number of events 162 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Investigations
Weight loss
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
81.2%
13/16 • Number of events 33 • Adverse events were collected from the on-study date through 05/03/2016.
|
83.3%
5/6 • Number of events 12 • Adverse events were collected from the on-study date through 05/03/2016.
|
81.8%
18/22 • Number of events 45 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
75.0%
12/16 • Number of events 51 • Adverse events were collected from the on-study date through 05/03/2016.
|
83.3%
5/6 • Number of events 19 • Adverse events were collected from the on-study date through 05/03/2016.
|
77.3%
17/22 • Number of events 70 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Investigations
Platelet count decreased
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
75.0%
12/16 • Number of events 41 • Adverse events were collected from the on-study date through 05/03/2016.
|
83.3%
5/6 • Number of events 7 • Adverse events were collected from the on-study date through 05/03/2016.
|
77.3%
17/22 • Number of events 48 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Cardiac disorders
Sinus bradycardia
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
68.8%
11/16 • Number of events 31 • Adverse events were collected from the on-study date through 05/03/2016.
|
66.7%
4/6 • Number of events 10 • Adverse events were collected from the on-study date through 05/03/2016.
|
68.2%
15/22 • Number of events 41 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Cardiac disorders
Sinus tachycardia
|
100.0%
1/1 • Number of events 1 • Adverse events were collected from the on-study date through 05/03/2016.
|
93.8%
15/16 • Number of events 66 • Adverse events were collected from the on-study date through 05/03/2016.
|
100.0%
6/6 • Number of events 21 • Adverse events were collected from the on-study date through 05/03/2016.
|
95.5%
21/22 • Number of events 87 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Investigations
Lymphocyte count decreased
|
100.0%
1/1 • Number of events 7 • Adverse events were collected from the on-study date through 05/03/2016.
|
93.8%
15/16 • Number of events 215 • Adverse events were collected from the on-study date through 05/03/2016.
|
100.0%
6/6 • Number of events 48 • Adverse events were collected from the on-study date through 05/03/2016.
|
95.5%
21/22 • Number of events 263 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Investigations
Neutrophil count decreased
|
100.0%
1/1 • Number of events 6 • Adverse events were collected from the on-study date through 05/03/2016.
|
93.8%
15/16 • Number of events 133 • Adverse events were collected from the on-study date through 05/03/2016.
|
100.0%
6/6 • Number of events 36 • Adverse events were collected from the on-study date through 05/03/2016.
|
95.5%
21/22 • Number of events 169 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Investigations
White blood cell decreased
|
100.0%
1/1 • Number of events 4 • Adverse events were collected from the on-study date through 05/03/2016.
|
93.8%
15/16 • Number of events 150 • Adverse events were collected from the on-study date through 05/03/2016.
|
100.0%
6/6 • Number of events 36 • Adverse events were collected from the on-study date through 05/03/2016.
|
95.5%
21/22 • Number of events 186 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Vascular disorders
Hypertension
|
100.0%
1/1 • Number of events 6 • Adverse events were collected from the on-study date through 05/03/2016.
|
68.8%
11/16 • Number of events 60 • Adverse events were collected from the on-study date through 05/03/2016.
|
83.3%
5/6 • Number of events 22 • Adverse events were collected from the on-study date through 05/03/2016.
|
72.7%
16/22 • Number of events 82 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
100.0%
1/1 • Number of events 1 • Adverse events were collected from the on-study date through 05/03/2016.
|
56.2%
9/16 • Number of events 22 • Adverse events were collected from the on-study date through 05/03/2016.
|
50.0%
3/6 • Number of events 4 • Adverse events were collected from the on-study date through 05/03/2016.
|
54.5%
12/22 • Number of events 26 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Blood and lymphatic system disorders
Anemia
|
100.0%
1/1 • Number of events 3 • Adverse events were collected from the on-study date through 05/03/2016.
|
100.0%
16/16 • Number of events 96 • Adverse events were collected from the on-study date through 05/03/2016.
|
100.0%
6/6 • Number of events 34 • Adverse events were collected from the on-study date through 05/03/2016.
|
100.0%
22/22 • Number of events 130 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Infections and infestations
Wound infection
|
100.0%
1/1 • Number of events 1 • Adverse events were collected from the on-study date through 05/03/2016.
|
0.00%
0/16 • Adverse events were collected from the on-study date through 05/03/2016.
|
16.7%
1/6 • Number of events 1 • Adverse events were collected from the on-study date through 05/03/2016.
|
4.5%
1/22 • Number of events 1 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Metabolism and nutrition disorders
Hypophosphatemia
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
68.8%
11/16 • Number of events 33 • Adverse events were collected from the on-study date through 05/03/2016.
|
66.7%
4/6 • Number of events 6 • Adverse events were collected from the on-study date through 05/03/2016.
|
68.2%
15/22 • Number of events 39 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Gastrointestinal disorders
Mucositis oral
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
62.5%
10/16 • Number of events 19 • Adverse events were collected from the on-study date through 05/03/2016.
|
66.7%
4/6 • Number of events 10 • Adverse events were collected from the on-study date through 05/03/2016.
|
63.6%
14/22 • Number of events 29 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
62.5%
10/16 • Number of events 35 • Adverse events were collected from the on-study date through 05/03/2016.
|
66.7%
4/6 • Number of events 7 • Adverse events were collected from the on-study date through 05/03/2016.
|
63.6%
14/22 • Number of events 42 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Skin and subcutaneous tissue disorders
Palmar-plantar erythrodysesthesia syndrome
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
75.0%
12/16 • Number of events 32 • Adverse events were collected from the on-study date through 05/03/2016.
|
33.3%
2/6 • Number of events 3 • Adverse events were collected from the on-study date through 05/03/2016.
|
63.6%
14/22 • Number of events 35 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Renal and urinary disorders
Proteinuria
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
56.2%
9/16 • Number of events 23 • Adverse events were collected from the on-study date through 05/03/2016.
|
66.7%
4/6 • Number of events 8 • Adverse events were collected from the on-study date through 05/03/2016.
|
59.1%
13/22 • Number of events 31 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Cardiac disorders
Cardiac disorders - Other
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
62.5%
10/16 • Number of events 15 • Adverse events were collected from the on-study date through 05/03/2016.
|
33.3%
2/6 • Number of events 2 • Adverse events were collected from the on-study date through 05/03/2016.
|
54.5%
12/22 • Number of events 17 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Metabolism and nutrition disorders
Anorexia
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
62.5%
10/16 • Number of events 19 • Adverse events were collected from the on-study date through 05/03/2016.
|
33.3%
2/6 • Number of events 4 • Adverse events were collected from the on-study date through 05/03/2016.
|
54.5%
12/22 • Number of events 23 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Metabolism and nutrition disorders
Hypokalemia
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
56.2%
9/16 • Number of events 24 • Adverse events were collected from the on-study date through 05/03/2016.
|
50.0%
3/6 • Number of events 13 • Adverse events were collected from the on-study date through 05/03/2016.
|
54.5%
12/22 • Number of events 37 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Metabolism and nutrition disorders
Hyponatremia
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
56.2%
9/16 • Number of events 38 • Adverse events were collected from the on-study date through 05/03/2016.
|
33.3%
2/6 • Number of events 2 • Adverse events were collected from the on-study date through 05/03/2016.
|
50.0%
11/22 • Number of events 40 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
50.0%
8/16 • Number of events 10 • Adverse events were collected from the on-study date through 05/03/2016.
|
33.3%
2/6 • Number of events 4 • Adverse events were collected from the on-study date through 05/03/2016.
|
45.5%
10/22 • Number of events 14 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
General disorders
Fatigue
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
43.8%
7/16 • Number of events 7 • Adverse events were collected from the on-study date through 05/03/2016.
|
50.0%
3/6 • Number of events 4 • Adverse events were collected from the on-study date through 05/03/2016.
|
45.5%
10/22 • Number of events 11 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Metabolism and nutrition disorders
Hypoalbuminemia
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
50.0%
8/16 • Number of events 30 • Adverse events were collected from the on-study date through 05/03/2016.
|
33.3%
2/6 • Number of events 5 • Adverse events were collected from the on-study date through 05/03/2016.
|
45.5%
10/22 • Number of events 35 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
43.8%
7/16 • Number of events 27 • Adverse events were collected from the on-study date through 05/03/2016.
|
33.3%
2/6 • Number of events 4 • Adverse events were collected from the on-study date through 05/03/2016.
|
40.9%
9/22 • Number of events 31 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
General disorders
Fever
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
43.8%
7/16 • Number of events 12 • Adverse events were collected from the on-study date through 05/03/2016.
|
16.7%
1/6 • Number of events 3 • Adverse events were collected from the on-study date through 05/03/2016.
|
36.4%
8/22 • Number of events 15 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Immune system disorders
Allergic reaction
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
37.5%
6/16 • Number of events 6 • Adverse events were collected from the on-study date through 05/03/2016.
|
33.3%
2/6 • Number of events 3 • Adverse events were collected from the on-study date through 05/03/2016.
|
36.4%
8/22 • Number of events 9 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
43.8%
7/16 • Number of events 8 • Adverse events were collected from the on-study date through 05/03/2016.
|
0.00%
0/6 • Adverse events were collected from the on-study date through 05/03/2016.
|
31.8%
7/22 • Number of events 8 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Investigations
Alanine aminotransferase increased
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
31.2%
5/16 • Number of events 13 • Adverse events were collected from the on-study date through 05/03/2016.
|
33.3%
2/6 • Number of events 3 • Adverse events were collected from the on-study date through 05/03/2016.
|
31.8%
7/22 • Number of events 16 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Gastrointestinal disorders
Colitis
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
31.2%
5/16 • Number of events 6 • Adverse events were collected from the on-study date through 05/03/2016.
|
16.7%
1/6 • Number of events 1 • Adverse events were collected from the on-study date through 05/03/2016.
|
27.3%
6/22 • Number of events 7 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Infections and infestations
Mucosa infection
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
31.2%
5/16 • Number of events 6 • Adverse events were collected from the on-study date through 05/03/2016.
|
16.7%
1/6 • Number of events 1 • Adverse events were collected from the on-study date through 05/03/2016.
|
27.3%
6/22 • Number of events 7 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Investigations
Aspartate aminotransferase increased
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
25.0%
4/16 • Number of events 8 • Adverse events were collected from the on-study date through 05/03/2016.
|
33.3%
2/6 • Number of events 3 • Adverse events were collected from the on-study date through 05/03/2016.
|
27.3%
6/22 • Number of events 11 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Metabolism and nutrition disorders
Hypocalcemia
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
31.2%
5/16 • Number of events 40 • Adverse events were collected from the on-study date through 05/03/2016.
|
16.7%
1/6 • Number of events 5 • Adverse events were collected from the on-study date through 05/03/2016.
|
27.3%
6/22 • Number of events 45 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
31.2%
5/16 • Number of events 6 • Adverse events were collected from the on-study date through 05/03/2016.
|
16.7%
1/6 • Number of events 1 • Adverse events were collected from the on-study date through 05/03/2016.
|
27.3%
6/22 • Number of events 7 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Metabolism and nutrition disorders
Hypertriglyceridemia
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
18.8%
3/16 • Number of events 4 • Adverse events were collected from the on-study date through 05/03/2016.
|
33.3%
2/6 • Number of events 2 • Adverse events were collected from the on-study date through 05/03/2016.
|
22.7%
5/22 • Number of events 6 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Renal and urinary disorders
Hematuria
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
25.0%
4/16 • Number of events 7 • Adverse events were collected from the on-study date through 05/03/2016.
|
16.7%
1/6 • Number of events 1 • Adverse events were collected from the on-study date through 05/03/2016.
|
22.7%
5/22 • Number of events 8 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Metabolism and nutrition disorders
Dehydration
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
18.8%
3/16 • Number of events 4 • Adverse events were collected from the on-study date through 05/03/2016.
|
16.7%
1/6 • Number of events 1 • Adverse events were collected from the on-study date through 05/03/2016.
|
18.2%
4/22 • Number of events 5 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Nervous system disorders
Headache
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
12.5%
2/16 • Number of events 2 • Adverse events were collected from the on-study date through 05/03/2016.
|
33.3%
2/6 • Number of events 2 • Adverse events were collected from the on-study date through 05/03/2016.
|
18.2%
4/22 • Number of events 4 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Renal and urinary disorders
Urinary tract pain
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
25.0%
4/16 • Number of events 8 • Adverse events were collected from the on-study date through 05/03/2016.
|
0.00%
0/6 • Adverse events were collected from the on-study date through 05/03/2016.
|
18.2%
4/22 • Number of events 8 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
18.8%
3/16 • Number of events 7 • Adverse events were collected from the on-study date through 05/03/2016.
|
16.7%
1/6 • Number of events 1 • Adverse events were collected from the on-study date through 05/03/2016.
|
18.2%
4/22 • Number of events 8 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
General disorders
Malaise
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
12.5%
2/16 • Number of events 2 • Adverse events were collected from the on-study date through 05/03/2016.
|
16.7%
1/6 • Number of events 1 • Adverse events were collected from the on-study date through 05/03/2016.
|
13.6%
3/22 • Number of events 3 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Infections and infestations
Enterocolitis infectious
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
18.8%
3/16 • Number of events 5 • Adverse events were collected from the on-study date through 05/03/2016.
|
0.00%
0/6 • Adverse events were collected from the on-study date through 05/03/2016.
|
13.6%
3/22 • Number of events 5 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Infections and infestations
Lung infection
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
18.8%
3/16 • Number of events 3 • Adverse events were collected from the on-study date through 05/03/2016.
|
0.00%
0/6 • Adverse events were collected from the on-study date through 05/03/2016.
|
13.6%
3/22 • Number of events 3 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Investigations
GGT increased
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
12.5%
2/16 • Number of events 2 • Adverse events were collected from the on-study date through 05/03/2016.
|
16.7%
1/6 • Number of events 1 • Adverse events were collected from the on-study date through 05/03/2016.
|
13.6%
3/22 • Number of events 3 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Metabolism and nutrition disorders
Hypercalcemia
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
12.5%
2/16 • Number of events 2 • Adverse events were collected from the on-study date through 05/03/2016.
|
16.7%
1/6 • Number of events 1 • Adverse events were collected from the on-study date through 05/03/2016.
|
13.6%
3/22 • Number of events 3 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Nervous system disorders
Dizziness
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
18.8%
3/16 • Number of events 3 • Adverse events were collected from the on-study date through 05/03/2016.
|
0.00%
0/6 • Adverse events were collected from the on-study date through 05/03/2016.
|
13.6%
3/22 • Number of events 3 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Nervous system disorders
Dysgeusia
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
6.2%
1/16 • Number of events 1 • Adverse events were collected from the on-study date through 05/03/2016.
|
33.3%
2/6 • Number of events 2 • Adverse events were collected from the on-study date through 05/03/2016.
|
13.6%
3/22 • Number of events 3 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Cardiac disorders
Pericardial effusion
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
6.2%
1/16 • Number of events 1 • Adverse events were collected from the on-study date through 05/03/2016.
|
16.7%
1/6 • Number of events 1 • Adverse events were collected from the on-study date through 05/03/2016.
|
9.1%
2/22 • Number of events 2 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Gastrointestinal disorders
Esophagitis
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
6.2%
1/16 • Number of events 1 • Adverse events were collected from the on-study date through 05/03/2016.
|
16.7%
1/6 • Number of events 1 • Adverse events were collected from the on-study date through 05/03/2016.
|
9.1%
2/22 • Number of events 2 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Gastrointestinal disorders
Oral pain
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
12.5%
2/16 • Number of events 2 • Adverse events were collected from the on-study date through 05/03/2016.
|
0.00%
0/6 • Adverse events were collected from the on-study date through 05/03/2016.
|
9.1%
2/22 • Number of events 2 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Infections and infestations
Anorectal infection
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
6.2%
1/16 • Number of events 1 • Adverse events were collected from the on-study date through 05/03/2016.
|
16.7%
1/6 • Number of events 1 • Adverse events were collected from the on-study date through 05/03/2016.
|
9.1%
2/22 • Number of events 2 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Infections and infestations
Esophageal infection
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
12.5%
2/16 • Number of events 3 • Adverse events were collected from the on-study date through 05/03/2016.
|
0.00%
0/6 • Adverse events were collected from the on-study date through 05/03/2016.
|
9.1%
2/22 • Number of events 3 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Investigations
Alkaline phosphatase increased
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
12.5%
2/16 • Number of events 2 • Adverse events were collected from the on-study date through 05/03/2016.
|
0.00%
0/6 • Adverse events were collected from the on-study date through 05/03/2016.
|
9.1%
2/22 • Number of events 2 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Investigations
Blood bilirubin increased
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
6.2%
1/16 • Number of events 2 • Adverse events were collected from the on-study date through 05/03/2016.
|
16.7%
1/6 • Number of events 1 • Adverse events were collected from the on-study date through 05/03/2016.
|
9.1%
2/22 • Number of events 3 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Investigations
Cardiac troponin T increased
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
12.5%
2/16 • Number of events 3 • Adverse events were collected from the on-study date through 05/03/2016.
|
0.00%
0/6 • Adverse events were collected from the on-study date through 05/03/2016.
|
9.1%
2/22 • Number of events 3 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Investigations
Creatinine increased
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
6.2%
1/16 • Number of events 1 • Adverse events were collected from the on-study date through 05/03/2016.
|
16.7%
1/6 • Number of events 1 • Adverse events were collected from the on-study date through 05/03/2016.
|
9.1%
2/22 • Number of events 2 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Metabolism and nutrition disorders
Hyperkalemia
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
12.5%
2/16 • Number of events 2 • Adverse events were collected from the on-study date through 05/03/2016.
|
0.00%
0/6 • Adverse events were collected from the on-study date through 05/03/2016.
|
9.1%
2/22 • Number of events 2 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Metabolism and nutrition disorders
Hyperuricemia
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
6.2%
1/16 • Number of events 1 • Adverse events were collected from the on-study date through 05/03/2016.
|
16.7%
1/6 • Number of events 1 • Adverse events were collected from the on-study date through 05/03/2016.
|
9.1%
2/22 • Number of events 2 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Metabolism and nutrition disorders
Hypoglycemia
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
12.5%
2/16 • Number of events 2 • Adverse events were collected from the on-study date through 05/03/2016.
|
0.00%
0/6 • Adverse events were collected from the on-study date through 05/03/2016.
|
9.1%
2/22 • Number of events 2 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Metabolism and nutrition disorders
Hypomagnesemia
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
12.5%
2/16 • Number of events 4 • Adverse events were collected from the on-study date through 05/03/2016.
|
0.00%
0/6 • Adverse events were collected from the on-study date through 05/03/2016.
|
9.1%
2/22 • Number of events 4 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Renal and urinary disorders
Cystitis noninfective
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
12.5%
2/16 • Number of events 4 • Adverse events were collected from the on-study date through 05/03/2016.
|
0.00%
0/6 • Adverse events were collected from the on-study date through 05/03/2016.
|
9.1%
2/22 • Number of events 4 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Renal and urinary disorders
Urinary frequency
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
12.5%
2/16 • Number of events 2 • Adverse events were collected from the on-study date through 05/03/2016.
|
0.00%
0/6 • Adverse events were collected from the on-study date through 05/03/2016.
|
9.1%
2/22 • Number of events 2 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Renal and urinary disorders
Urinary retention
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
12.5%
2/16 • Number of events 2 • Adverse events were collected from the on-study date through 05/03/2016.
|
0.00%
0/6 • Adverse events were collected from the on-study date through 05/03/2016.
|
9.1%
2/22 • Number of events 2 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Respiratory, thoracic and mediastinal disorders
Allergic rhinitis
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
12.5%
2/16 • Number of events 3 • Adverse events were collected from the on-study date through 05/03/2016.
|
0.00%
0/6 • Adverse events were collected from the on-study date through 05/03/2016.
|
9.1%
2/22 • Number of events 3 • Adverse events were collected from the on-study date through 05/03/2016.
|
|
Respiratory, thoracic and mediastinal disorders
Pneumothorax
|
0.00%
0/1 • Adverse events were collected from the on-study date through 05/03/2016.
|
6.2%
1/16 • Number of events 3 • Adverse events were collected from the on-study date through 05/03/2016.
|
16.7%
1/6 • Number of events 1 • Adverse events were collected from the on-study date through 05/03/2016.
|
9.1%
2/22 • Number of events 4 • Adverse events were collected from the on-study date through 05/03/2016.
|
Additional Information
Sara Federico, MD
St. Jude Children's Research Hospital
Phone: 901-595-2220
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place