Trial Outcomes & Findings for Clofarabine, Melphalan, and Thiotepa Followed By a Donor Stem Cell Transplant in Treating Patients With High-Risk and/or Advanced Hematologic Cancer or Other Disease (NCT NCT00423514)
NCT ID: NCT00423514
Last Updated: 2022-12-27
Results Overview
* A complete response (CR) will be defined as less than 5% bone marrow blasts in the setting of a neutrophil count of \>/= 1.0 K/ul and a platelet count of \>/= 75,000/ul * A complete response except platelets (CRp) will be defined as less than 5% bone marrow blasts in the setting of a neutrophil count of \>/= 1.0 K/ul and a platelet count of \>/= 75,000/ul * A partial response (PR) will be defined as 5%-25% bone marrow blasts in the setting of a neutrophil count of \>/= 1.0 K/ul and a platelet count of \>/= 75,000/ul * A partial response except platelets (PRp) will be defined as 5%-25% bone marrow blasts in the setting of a neutrophil count of \>/= 1.0 K/ul and a platelet count of \>/= 75,000/ul
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
38 participants
Primary outcome timeframe
1 year
Results posted on
2022-12-27
Participant Flow
Participant milestones
| Measure |
Dose Level 1: Clofarabine at 20 mg/m^2/Dose x 5 + THIO-MEL
This is a single arm phase I/II clinical trial to assess efficacy (the antileukemic potential and relapse rate), and safety (peri-transplant morbidity and mortality) of a novel cytoreduction regimen in preparation for allogeneic hematopoietic stem cell transplantation (HSCT).
|
Dose Level 2: Clofarabine at 30 mg/m^2/Dose x 5 + THIO-MEL
This is a single arm phase I/II clinical trial to assess efficacy (the antileukemic potential and relapse rate), and safety (peri-transplant morbidity and mortality) of a novel cytoreduction regimen in preparation for allogeneic hematopoietic stem cell transplantation (HSCT).
|
|---|---|---|
|
Overall Study
STARTED
|
31
|
7
|
|
Overall Study
COMPLETED
|
30
|
7
|
|
Overall Study
NOT COMPLETED
|
1
|
0
|
Reasons for withdrawal
| Measure |
Dose Level 1: Clofarabine at 20 mg/m^2/Dose x 5 + THIO-MEL
This is a single arm phase I/II clinical trial to assess efficacy (the antileukemic potential and relapse rate), and safety (peri-transplant morbidity and mortality) of a novel cytoreduction regimen in preparation for allogeneic hematopoietic stem cell transplantation (HSCT).
|
Dose Level 2: Clofarabine at 30 mg/m^2/Dose x 5 + THIO-MEL
This is a single arm phase I/II clinical trial to assess efficacy (the antileukemic potential and relapse rate), and safety (peri-transplant morbidity and mortality) of a novel cytoreduction regimen in preparation for allogeneic hematopoietic stem cell transplantation (HSCT).
|
|---|---|---|
|
Overall Study
Ineligible
|
1
|
0
|
Baseline Characteristics
Clofarabine, Melphalan, and Thiotepa Followed By a Donor Stem Cell Transplant in Treating Patients With High-Risk and/or Advanced Hematologic Cancer or Other Disease
Baseline characteristics by cohort
| Measure |
Dose Level 1: Clofarabine at 20 mg/m^2/Dose x 5 + THIO-MEL
n=31 Participants
This is a single arm phase I/II clinical trial to assess efficacy (the antileukemic potential and relapse rate), and safety (peri-transplant morbidity and mortality) of a novel cytoreduction regimen in preparation for allogeneic hematopoietic stem cell transplantation (HSCT).
|
Dose Level 2: Clofarabine at 30 mg/m^2/Dose x 5 + THIO-MEL
n=7 Participants
This is a single arm phase I/II clinical trial to assess efficacy (the antileukemic potential and relapse rate), and safety (peri-transplant morbidity and mortality) of a novel cytoreduction regimen in preparation for allogeneic hematopoietic stem cell transplantation (HSCT).
|
Total
n=38 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
17 years
n=5 Participants
|
26 years
n=7 Participants
|
18 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
12 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
15 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
19 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
23 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
4 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
27 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
33 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=5 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
|
|
Race (NIH/OMB)
Black or African American
|
5 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
24 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
30 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States · USA
|
29 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
36 Participants
n=5 Participants
|
|
Region of Enrollment
United States · England
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Region of Enrollment
United States · Canada
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 1 year* A complete response (CR) will be defined as less than 5% bone marrow blasts in the setting of a neutrophil count of \>/= 1.0 K/ul and a platelet count of \>/= 75,000/ul * A complete response except platelets (CRp) will be defined as less than 5% bone marrow blasts in the setting of a neutrophil count of \>/= 1.0 K/ul and a platelet count of \>/= 75,000/ul * A partial response (PR) will be defined as 5%-25% bone marrow blasts in the setting of a neutrophil count of \>/= 1.0 K/ul and a platelet count of \>/= 75,000/ul * A partial response except platelets (PRp) will be defined as 5%-25% bone marrow blasts in the setting of a neutrophil count of \>/= 1.0 K/ul and a platelet count of \>/= 75,000/ul
Outcome measures
| Measure |
Dose Level 1: Clofarabine at 20 mg/m^2/Dose x 5 + THIO-MEL
n=31 Participants
This is a single arm phase I/II clinical trial to assess efficacy (the antileukemic potential and relapse rate), and safety (peri-transplant morbidity and mortality) of a novel cytoreduction regimen in preparation for allogeneic hematopoietic stem cell transplantation (HSCT).
|
Dose Level 2: Clofarabine at 30 mg/m^2/Dose x 5 + THIO-MEL
n=7 Participants
This is a single arm phase I/II clinical trial to assess efficacy (the antileukemic potential and relapse rate), and safety (peri-transplant morbidity and mortality) of a novel cytoreduction regimen in preparation for allogeneic hematopoietic stem cell transplantation (HSCT).
|
|---|---|---|
|
Response to Therapy
Relapse
|
1 Participants
|
0 Participants
|
|
Response to Therapy
Complete Response
|
27 Participants
|
4 Participants
|
|
Response to Therapy
Not evaluable
|
3 Participants
|
3 Participants
|
PRIMARY outcome
Timeframe: 1 yearOutcome measures
| Measure |
Dose Level 1: Clofarabine at 20 mg/m^2/Dose x 5 + THIO-MEL
n=31 Participants
This is a single arm phase I/II clinical trial to assess efficacy (the antileukemic potential and relapse rate), and safety (peri-transplant morbidity and mortality) of a novel cytoreduction regimen in preparation for allogeneic hematopoietic stem cell transplantation (HSCT).
|
Dose Level 2: Clofarabine at 30 mg/m^2/Dose x 5 + THIO-MEL
n=7 Participants
This is a single arm phase I/II clinical trial to assess efficacy (the antileukemic potential and relapse rate), and safety (peri-transplant morbidity and mortality) of a novel cytoreduction regimen in preparation for allogeneic hematopoietic stem cell transplantation (HSCT).
|
|---|---|---|
|
Overall Survival
Alive
|
19 Participants
|
4 Participants
|
|
Overall Survival
Dead
|
12 Participants
|
3 Participants
|
SECONDARY outcome
Timeframe: 1 yearOutcome measures
| Measure |
Dose Level 1: Clofarabine at 20 mg/m^2/Dose x 5 + THIO-MEL
n=31 Participants
This is a single arm phase I/II clinical trial to assess efficacy (the antileukemic potential and relapse rate), and safety (peri-transplant morbidity and mortality) of a novel cytoreduction regimen in preparation for allogeneic hematopoietic stem cell transplantation (HSCT).
|
Dose Level 2: Clofarabine at 30 mg/m^2/Dose x 5 + THIO-MEL
n=7 Participants
This is a single arm phase I/II clinical trial to assess efficacy (the antileukemic potential and relapse rate), and safety (peri-transplant morbidity and mortality) of a novel cytoreduction regimen in preparation for allogeneic hematopoietic stem cell transplantation (HSCT).
|
|---|---|---|
|
Participants Evaluated for Early Post-transplant Regimen-related Severe Morbidity (Grade III to IV Nonhematologic Toxicity) and Mortality as Measured by the NCI Cancer Therapy Evaluation Program CTCAE v 3.0
|
31 Participants
|
7 Participants
|
Adverse Events
Dose Level 1: Clofarabine at 20 mg/m^2/Dose x 5 + THIO-MEL
Serious events: 8 serious events
Other events: 31 other events
Deaths: 12 deaths
Dose Level 2: Clofarabine at 30 mg/m^2/Dose x 5 + THIO-MEL
Serious events: 3 serious events
Other events: 7 other events
Deaths: 3 deaths
Serious adverse events
| Measure |
Dose Level 1: Clofarabine at 20 mg/m^2/Dose x 5 + THIO-MEL
n=31 participants at risk
This is a single arm phase I/II clinical trial to assess efficacy (the antileukemic potential and relapse rate), and safety (peri-transplant morbidity and mortality) of a novel cytoreduction regimen in preparation for allogeneic hematopoietic stem cell transplantation (HSCT).
|
Dose Level 2: Clofarabine at 30 mg/m^2/Dose x 5 + THIO-MEL
n=7 participants at risk
This is a single arm phase I/II clinical trial to assess efficacy (the antileukemic potential and relapse rate), and safety (peri-transplant morbidity and mortality) of a novel cytoreduction regimen in preparation for allogeneic hematopoietic stem cell transplantation (HSCT).
|
|---|---|---|
|
Nervous system disorders
CNS cerebrovascular ischemia
|
3.2%
1/31 • 1 year
|
0.00%
0/7 • 1 year
|
|
General disorders
Death not assoc w CTCAE term - Disease prog NOS
|
3.2%
1/31 • 1 year
|
0.00%
0/7 • 1 year
|
|
Gastrointestinal disorders
Diarrhea
|
3.2%
1/31 • 1 year
|
0.00%
0/7 • 1 year
|
|
Nervous system disorders
Encephalopathy
|
0.00%
0/31 • 1 year
|
14.3%
1/7 • 1 year
|
|
Vascular disorders
Hypertension
|
3.2%
1/31 • 1 year
|
0.00%
0/7 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
9.7%
3/31 • 1 year
|
0.00%
0/7 • 1 year
|
|
Gastrointestinal disorders
Ileus, GI (func obstruction of bowel)
|
3.2%
1/31 • 1 year
|
0.00%
0/7 • 1 year
|
|
Infections and infestations
Infection, other
|
3.2%
1/31 • 1 year
|
14.3%
1/7 • 1 year
|
|
Hepatobiliary disorders
Liver dysfunction/failure
|
3.2%
1/31 • 1 year
|
14.3%
1/7 • 1 year
|
|
Metabolism and nutrition disorders
Metabolic/Lab - Other (specify)
|
3.2%
1/31 • 1 year
|
0.00%
0/7 • 1 year
|
|
Gastrointestinal disorders
Mucositis (Clin exam) - Oral cavity
|
3.2%
1/31 • 1 year
|
14.3%
1/7 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis/pulm infiltrates
|
3.2%
1/31 • 1 year
|
0.00%
0/7 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Pulm/upp respiratory - Other (spec)
|
3.2%
1/31 • 1 year
|
14.3%
1/7 • 1 year
|
|
Skin and subcutaneous tissue disorders
Rash; erythema multiforme
|
3.2%
1/31 • 1 year
|
0.00%
0/7 • 1 year
|
|
Renal and urinary disorders
Renal failure
|
3.2%
1/31 • 1 year
|
28.6%
2/7 • 1 year
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Secondary malig - poss related to ca txt specify
|
3.2%
1/31 • 1 year
|
0.00%
0/7 • 1 year
|
|
Vascular disorders
Thrombosis/thrombus/embolism
|
3.2%
1/31 • 1 year
|
0.00%
0/7 • 1 year
|
Other adverse events
| Measure |
Dose Level 1: Clofarabine at 20 mg/m^2/Dose x 5 + THIO-MEL
n=31 participants at risk
This is a single arm phase I/II clinical trial to assess efficacy (the antileukemic potential and relapse rate), and safety (peri-transplant morbidity and mortality) of a novel cytoreduction regimen in preparation for allogeneic hematopoietic stem cell transplantation (HSCT).
|
Dose Level 2: Clofarabine at 30 mg/m^2/Dose x 5 + THIO-MEL
n=7 participants at risk
This is a single arm phase I/II clinical trial to assess efficacy (the antileukemic potential and relapse rate), and safety (peri-transplant morbidity and mortality) of a novel cytoreduction regimen in preparation for allogeneic hematopoietic stem cell transplantation (HSCT).
|
|---|---|---|
|
Investigations
ALT, SGPT
|
64.5%
20/31 • 1 year
|
71.4%
5/7 • 1 year
|
|
Investigations
AST, SGOT
|
51.6%
16/31 • 1 year
|
71.4%
5/7 • 1 year
|
|
Investigations
Alkaline phosphatase
|
3.2%
1/31 • 1 year
|
14.3%
1/7 • 1 year
|
|
Investigations
Bilirubin (hyperbilirubinemia)
|
25.8%
8/31 • 1 year
|
28.6%
2/7 • 1 year
|
|
Investigations
Creatinine
|
3.2%
1/31 • 1 year
|
28.6%
2/7 • 1 year
|
|
Investigations
Fibrinogen
|
6.5%
2/31 • 1 year
|
0.00%
0/7 • 1 year
|
|
Metabolism and nutrition disorders
Glucose, high (hyperglycemia)
|
64.5%
20/31 • 1 year
|
42.9%
3/7 • 1 year
|
|
Blood and lymphatic system disorders
Hemoglobin
|
96.8%
30/31 • 1 year
|
100.0%
7/7 • 1 year
|
|
Investigations
Leukocytes (total WBC)
|
96.8%
30/31 • 1 year
|
100.0%
7/7 • 1 year
|
|
Investigations
Lymphopenia
|
96.8%
30/31 • 1 year
|
100.0%
7/7 • 1 year
|
|
Metabolism and nutrition disorders
Magnesium, high (hypermagnesemia)
|
41.9%
13/31 • 1 year
|
14.3%
1/7 • 1 year
|
|
Investigations
Neutrophils/granulocytes (ANC/AGC)
|
96.8%
30/31 • 1 year
|
100.0%
7/7 • 1 year
|
|
Investigations
PTT
|
9.7%
3/31 • 1 year
|
0.00%
0/7 • 1 year
|
|
Metabolism and nutrition disorders
Phosphate, low (hypophosphatemia)
|
16.1%
5/31 • 1 year
|
28.6%
2/7 • 1 year
|
|
Investigations
Platelets
|
96.8%
30/31 • 1 year
|
100.0%
7/7 • 1 year
|
|
Metabolism and nutrition disorders
Potassium, high (hyperkalemia)
|
12.9%
4/31 • 1 year
|
0.00%
0/7 • 1 year
|
|
Metabolism and nutrition disorders
Potassium, low (hypokalemia)
|
51.6%
16/31 • 1 year
|
14.3%
1/7 • 1 year
|
|
Metabolism and nutrition disorders
Sodium, low (hyponatremia)
|
35.5%
11/31 • 1 year
|
28.6%
2/7 • 1 year
|
|
Investigations
INR
|
0.00%
0/31 • 1 year
|
14.3%
1/7 • 1 year
|
Additional Information
Dr. Farid Boulad, MD
Memorial Sloan Kettering Cancer Center
Phone: 1-833-MSK-KIDS
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place