Trial Outcomes & Findings for Busulfan, Melphalan, and Thiotepa Followed By a Donor Stem Cell Transplant in Treating Patients With High-Risk Ewing's Tumors (NCT NCT00357396)
NCT ID: NCT00357396
Last Updated: 2015-11-25
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
10 participants
Primary outcome timeframe
2 years
Results posted on
2015-11-25
Participant Flow
Participant milestones
| Measure |
Patients With HIGH RISK EWING'S SARCOMA FAMILY TUMOR
MELPHALAN \& THIOTEPA TREATMENT OF HIGH RISK EWING'S SARCOMA FAMILY TUMOR
|
Related Donors
Any consenting healthy family donor who is HLA compatible with the recipient will be considered as a potential donor for transplant
|
|---|---|---|
|
Overall Study
STARTED
|
6
|
4
|
|
Overall Study
COMPLETED
|
5
|
4
|
|
Overall Study
NOT COMPLETED
|
1
|
0
|
Reasons for withdrawal
| Measure |
Patients With HIGH RISK EWING'S SARCOMA FAMILY TUMOR
MELPHALAN \& THIOTEPA TREATMENT OF HIGH RISK EWING'S SARCOMA FAMILY TUMOR
|
Related Donors
Any consenting healthy family donor who is HLA compatible with the recipient will be considered as a potential donor for transplant
|
|---|---|---|
|
Overall Study
Adverse Event
|
1
|
0
|
Baseline Characteristics
Busulfan, Melphalan, and Thiotepa Followed By a Donor Stem Cell Transplant in Treating Patients With High-Risk Ewing's Tumors
Baseline characteristics by cohort
| Measure |
Patients With HIGH RISK EWING'S SARCOMA FAMILY TUMOR
n=6 Participants
MELPHALAN \& THIOTEPA TREATMENT OF HIGH RISK EWING'S SARCOMA FAMILY TUMOR
|
Related Donors
n=4 Participants
Any consenting healthy family donor who is HLA compatible with the recipient will be considered as a potential donor for transplant
|
Total
n=10 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
3 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
3 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 2 yearsOutcome measures
| Measure |
Patients With HIGH RISK EWING'S SARCOMA FAMILY TUMOR
n=5 Participants
MELPHALAN \& THIOTEPA TREATMENT OF HIGH RISK EWING'S SARCOMA FAMILY TUMOR
|
|---|---|
|
Overall Objective Response
Very Good Partial Response (VGPR)
|
4 participants
|
|
Overall Objective Response
Complete Response (CR)
|
1 participants
|
Adverse Events
Patients With HIGH RISK EWING'S SARCOMA FAMILY TUMOR
Serious events: 4 serious events
Other events: 6 other events
Deaths: 0 deaths
Related Donors
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Patients With HIGH RISK EWING'S SARCOMA FAMILY TUMOR
n=6 participants at risk
MELPHALAN \& THIOTEPA TREATMENT OF HIGH RISK EWING'S SARCOMA FAMILY TUMOR
|
Related Donors
n=4 participants at risk
Any consenting healthy family donor who is HLA compatible with the recipient will be considered as a potential donor for transplant
|
|---|---|---|
|
General disorders
Constitutional Symptoms, other
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
|
General disorders
Death not assoc w CTCAE term-Disease prog NOS
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
50.0%
3/6 • Number of events 4
|
0.00%
0/4
|
|
Infections and infestations
Infection w/ Gr 3/4 neut, Abdomen NOS
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
|
Infections and infestations
Infection w/ Gr 3/4 neut, Blood
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
|
Gastrointestinal disorders
Liver dysfunction/failure
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
|
Renal and urinary disorders
Renal failure
|
33.3%
2/6 • Number of events 2
|
0.00%
0/4
|
Other adverse events
| Measure |
Patients With HIGH RISK EWING'S SARCOMA FAMILY TUMOR
n=6 participants at risk
MELPHALAN \& THIOTEPA TREATMENT OF HIGH RISK EWING'S SARCOMA FAMILY TUMOR
|
Related Donors
n=4 participants at risk
Any consenting healthy family donor who is HLA compatible with the recipient will be considered as a potential donor for transplant
|
|---|---|---|
|
Blood and lymphatic system disorders
ALT, SGPT
|
100.0%
6/6 • Number of events 6
|
0.00%
0/4
|
|
Blood and lymphatic system disorders
AST, SGOT
|
83.3%
5/6 • Number of events 5
|
0.00%
0/4
|
|
Metabolism and nutrition disorders
Albumin, low (hypoalbuminemia)
|
66.7%
4/6 • Number of events 4
|
0.00%
0/4
|
|
Metabolism and nutrition disorders
Alkaline phosphatase
|
83.3%
5/6 • Number of events 5
|
0.00%
0/4
|
|
Metabolism and nutrition disorders
Amylase
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
|
Metabolism and nutrition disorders
Bilirubin (hyperbilirubinemia)
|
50.0%
3/6 • Number of events 3
|
0.00%
0/4
|
|
Metabolism and nutrition disorders
Cholesterol,high(hypercholestremia)
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
|
Metabolism and nutrition disorders
Creatinine
|
50.0%
3/6 • Number of events 3
|
0.00%
0/4
|
|
Skin and subcutaneous tissue disorders
Dermatology/Skin
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
|
Gastrointestinal disorders
Diarrhea
|
50.0%
3/6 • Number of events 3
|
0.00%
0/4
|
|
Gastrointestinal disorders
Gastrointestinal, other
|
50.0%
3/6 • Number of events 3
|
0.00%
0/4
|
|
Metabolism and nutrition disorders
Glucose, high (hyperglycemia)
|
100.0%
6/6 • Number of events 6
|
0.00%
0/4
|
|
Blood and lymphatic system disorders
Hemoglobin
|
100.0%
6/6 • Number of events 6
|
0.00%
0/4
|
|
Renal and urinary disorders
Hemorrhage, Urinary NOS
|
33.3%
2/6 • Number of events 2
|
0.00%
0/4
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
50.0%
3/6 • Number of events 4
|
0.00%
0/4
|
|
Blood and lymphatic system disorders
Leukocytes (total WBC)
|
100.0%
6/6 • Number of events 6
|
0.00%
0/4
|
|
Metabolism and nutrition disorders
Lipase
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
|
Blood and lymphatic system disorders
Lymphopenia
|
100.0%
6/6 • Number of events 6
|
0.00%
0/4
|
|
Metabolism and nutrition disorders
Magnesium, high (hypermagnesemia)
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
|
Metabolism and nutrition disorders
Magnesium, low (hypomagnesemia)
|
50.0%
3/6 • Number of events 3
|
0.00%
0/4
|
|
General disorders
Mucositis (Clin exam)- Oral cavity
|
33.3%
2/6 • Number of events 2
|
0.00%
0/4
|
|
General disorders
Nausea
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
|
Blood and lymphatic system disorders
Neutrophils/granulocytes (ANC/AGC)
|
100.0%
6/6 • Number of events 6
|
0.00%
0/4
|
|
Blood and lymphatic system disorders
PTT
|
33.3%
2/6 • Number of events 2
|
0.00%
0/4
|
|
General disorders
Pain - Abdomen NOS
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
|
Metabolism and nutrition disorders
Phosphate, low (hypophosphatemia)
|
100.0%
6/6 • Number of events 6
|
0.00%
0/4
|
|
Blood and lymphatic system disorders
Platelets
|
100.0%
6/6 • Number of events 6
|
0.00%
0/4
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion (non-malignant)
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
|
Metabolism and nutrition disorders
Potassium, high (hyperkalemia)
|
66.7%
4/6 • Number of events 5
|
0.00%
0/4
|
|
Metabolism and nutrition disorders
Potassium, low (hypokalemia)
|
83.3%
5/6 • Number of events 5
|
0.00%
0/4
|
|
Metabolism and nutrition disorders
Sodium, high (hypernatremia)
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
|
Metabolism and nutrition disorders
Sodium, low (hyponatremia)
|
50.0%
3/6 • Number of events 3
|
0.00%
0/4
|
|
Metabolism and nutrition disorders
Trglycrde, high (hypertriglyceridemia)
|
50.0%
3/6 • Number of events 3
|
0.00%
0/4
|
|
General disorders
Vomiting
|
16.7%
1/6 • Number of events 1
|
0.00%
0/4
|
Additional Information
Dr. Susan Prockop
Memorial Sloan Kettering Cancer Center
Phone: 212-639-6715
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place