Trial Outcomes & Findings for Hematopoietic Stem Cell Transplantation in the Treatment of Infant Leukemia (NCT NCT00357565)
NCT ID: NCT00357565
Last Updated: 2025-08-26
Results Overview
Defined as achieving donor derived neutrophil count \>500/uL by day 42 in young children with leukemia or myelodysplastic syndrome undergoing a partially matched single unit umbilical cord blood transplant (UCBT) after a myeloablative preparative regimen consisting of busulfan, melphalan and fludarabine.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
34 participants
Primary outcome timeframe
Day 42 After Transplant
Results posted on
2025-08-26
Participant Flow
Participant milestones
| Measure |
Double Unit UCB Transplantation
Patients that receive 2 units of umbilical cord blood transplantation (UCBT).
|
Single Unit UCB Transplantation
Patients that receive one unit of umbilical cord blood transplantation (only if 2 adequate size and matched units are not available).
|
BM Transplantation
Blood and Marrow transplantation
|
|---|---|---|---|
|
Overall Study
STARTED
|
15
|
15
|
4
|
|
Overall Study
COMPLETED
|
15
|
15
|
4
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Hematopoietic Stem Cell Transplantation in the Treatment of Infant Leukemia
Baseline characteristics by cohort
| Measure |
Double Unit UCB Transplantation
n=15 Participants
Patients that receive 2 units of umbilical cord blood transplantation (UCBT).
|
Single Unit UCB Transplantation
n=15 Participants
Patients that receive one unit of umbilical cord blood transplantation (only if 2 adequate size and matched units are not available).
|
BM Transplantation
n=4 Participants
Blood and Marrow transplantation
|
Total
n=34 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
15 Participants
n=5 Participants
|
15 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
34 Participants
n=4 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Sex: Female, Male
Female
|
8 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
17 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
7 Participants
n=5 Participants
|
10 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
17 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
12 Participants
n=5 Participants
|
14 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
30 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
2 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
|
3 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
5 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
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
White
|
10 Participants
n=5 Participants
|
13 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
26 Participants
n=4 Participants
|
|
Race (NIH/OMB)
More than one race
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
3 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Region of Enrollment
United States
|
15 participants
n=5 Participants
|
15 participants
n=7 Participants
|
4 participants
n=5 Participants
|
34 participants
n=4 Participants
|
PRIMARY outcome
Timeframe: Day 42 After TransplantDefined as achieving donor derived neutrophil count \>500/uL by day 42 in young children with leukemia or myelodysplastic syndrome undergoing a partially matched single unit umbilical cord blood transplant (UCBT) after a myeloablative preparative regimen consisting of busulfan, melphalan and fludarabine.
Outcome measures
| Measure |
Double Unit UCB Transplantation
n=15 Participants
Patients that receive 2 units of umbilical cord blood transplantation (UCBT).
|
Single Unit UCB Transplantation
n=15 Participants
Patients that receive one unit of umbilical cord blood transplantation (only if 2 adequate size and matched units are not available).
|
BM Transplantation
n=4 Participants
Blood and Marrow transplantation
|
|---|---|---|---|
|
Incidence of Engraftment
|
100 Percentage of participants
Interval 0.0 to 100.0
|
93 Percentage of participants
Interval 42.0 to 99.0
|
100 Percentage of participants
Interval 0.0 to 100.0
|
SECONDARY outcome
Timeframe: at 6 months after transplantdefined as death due to transplant
Outcome measures
| Measure |
Double Unit UCB Transplantation
n=15 Participants
Patients that receive 2 units of umbilical cord blood transplantation (UCBT).
|
Single Unit UCB Transplantation
n=15 Participants
Patients that receive one unit of umbilical cord blood transplantation (only if 2 adequate size and matched units are not available).
|
BM Transplantation
n=4 Participants
Blood and Marrow transplantation
|
|---|---|---|---|
|
Incidence of Transplant-related Mortality (TRM)
|
33 Percentage of participants
Interval 11.0 to 57.0
|
13 Percentage of participants
Interval 2.0 to 35.0
|
0 Percentage of participants
Interval 0.0 to 0.0
|
SECONDARY outcome
Timeframe: at 1 year after transplantdefined as platelet count \> 50,000
Outcome measures
| Measure |
Double Unit UCB Transplantation
n=15 Participants
Patients that receive 2 units of umbilical cord blood transplantation (UCBT).
|
Single Unit UCB Transplantation
n=15 Participants
Patients that receive one unit of umbilical cord blood transplantation (only if 2 adequate size and matched units are not available).
|
BM Transplantation
n=4 Participants
Blood and Marrow transplantation
|
|---|---|---|---|
|
Incidence of Platelet Engraftment
|
80 Percentage of participants
Interval 8.0 to 98.0
|
100 Percentage of participants
Interval 0.0 to 100.0
|
100 Percentage of participants
Interval 0.0 to 100.0
|
SECONDARY outcome
Timeframe: Day 100 After TransplantGraft-versus-host disease (GVHD) is a common complication of allogeneic bone marrow transplantation in which functional immune cells in the transplanted marrow recognize the recipient as "foreign" and mount an immunologic attack.
Outcome measures
| Measure |
Double Unit UCB Transplantation
n=15 Participants
Patients that receive 2 units of umbilical cord blood transplantation (UCBT).
|
Single Unit UCB Transplantation
n=15 Participants
Patients that receive one unit of umbilical cord blood transplantation (only if 2 adequate size and matched units are not available).
|
BM Transplantation
n=4 Participants
Blood and Marrow transplantation
|
|---|---|---|---|
|
Incidence of Acute Graft-versus-host Disease (GVHD) Grade II-IV and Grade III-IV
|
40 Percentage of participants
Interval 16.0 to 64.0
|
33 Percentage of participants
Interval 11.0 to 57.0
|
0 Percentage of participants
Interval 0.0 to 0.0
|
SECONDARY outcome
Timeframe: 1 Year After TransplantGraft-versus-host disease (GVHD) is a common complication of allogeneic bone marrow transplantation in which functional immune cells in the transplanted marrow recognize the recipient as "foreign" and mount an immunologic attack.
Outcome measures
| Measure |
Double Unit UCB Transplantation
n=15 Participants
Patients that receive 2 units of umbilical cord blood transplantation (UCBT).
|
Single Unit UCB Transplantation
n=15 Participants
Patients that receive one unit of umbilical cord blood transplantation (only if 2 adequate size and matched units are not available).
|
BM Transplantation
n=4 Participants
Blood and Marrow transplantation
|
|---|---|---|---|
|
Incidence of Chronic Graft-versus-host Disease (GVHD)
|
13 Percentage of participants
Interval 1.0 to 40.0
|
0 Percentage of participants
Interval 0.0 to 0.0
|
0 Percentage of participants
Interval 0.0 to 0.0
|
SECONDARY outcome
Timeframe: 1 and 2 years after transplantdefined using standard criteria (bone marrow blast count and cytogenetics).
Outcome measures
| Measure |
Double Unit UCB Transplantation
n=15 Participants
Patients that receive 2 units of umbilical cord blood transplantation (UCBT).
|
Single Unit UCB Transplantation
n=15 Participants
Patients that receive one unit of umbilical cord blood transplantation (only if 2 adequate size and matched units are not available).
|
BM Transplantation
n=4 Participants
Blood and Marrow transplantation
|
|---|---|---|---|
|
Incidence of Relapse
1 year
|
13 Percentage of participants
Interval 2.0 to 35.0
|
20 Percentage of participants
Interval 4.0 to 43.0
|
0 Percentage of participants
Interval 0.0 to 0.0
|
|
Incidence of Relapse
2 years
|
13 Percentage of participants
Interval 2.0 to 35.0
|
20 Percentage of participants
Interval 4.0 to 43.0
|
25 Percentage of participants
Interval 0.0 to 83.0
|
SECONDARY outcome
Timeframe: at 1 and 2 years after transplantAlive after transplant.
Outcome measures
| Measure |
Double Unit UCB Transplantation
n=15 Participants
Patients that receive 2 units of umbilical cord blood transplantation (UCBT).
|
Single Unit UCB Transplantation
n=15 Participants
Patients that receive one unit of umbilical cord blood transplantation (only if 2 adequate size and matched units are not available).
|
BM Transplantation
n=4 Participants
Blood and Marrow transplantation
|
|---|---|---|---|
|
Overall Survival
1 year
|
0.53 Probability of survival
Interval 0.33 to 0.86
|
0.67 Probability of survival
Interval 0.47 to 0.95
|
100 Probability of survival
Interval 100.0 to 100.0
|
|
Overall Survival
2 years
|
0.47 Probability of survival
Interval 0.27 to 0.8
|
0.67 Probability of survival
Interval 0.47 to 0.95
|
0.67 Probability of survival
Interval 0.3 to 1.0
|
SECONDARY outcome
Timeframe: at 1 and 2 years after transplantdefined as patients who are alive and in hematological remission.
Outcome measures
| Measure |
Double Unit UCB Transplantation
n=15 Participants
Patients that receive 2 units of umbilical cord blood transplantation (UCBT).
|
Single Unit UCB Transplantation
n=15 Participants
Patients that receive one unit of umbilical cord blood transplantation (only if 2 adequate size and matched units are not available).
|
BM Transplantation
n=4 Participants
Blood and Marrow transplantation
|
|---|---|---|---|
|
Disease-free Survival
1 year
|
0.13 Probability of survival
Interval 0.02 to 0.35
|
0.2 Probability of survival
Interval 0.04 to 0.43
|
0 Probability of survival
Interval 0.0 to 0.0
|
|
Disease-free Survival
2 years
|
0.13 Probability of survival
Interval 0.02 to 0.35
|
0.20 Probability of survival
Interval 0.04 to 0.43
|
0.33 Probability of survival
Interval 0.0 to 0.83
|
Adverse Events
Double Unit UCB Transplantation
Serious events: 8 serious events
Other events: 15 other events
Deaths: 8 deaths
Single Unit UCB Transplantation
Serious events: 5 serious events
Other events: 15 other events
Deaths: 5 deaths
BM Transplantation
Serious events: 1 serious events
Other events: 4 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
Double Unit UCB Transplantation
n=15 participants at risk
Patients that receive 2 units of umbilical cord blood transplantation (UCBT).
|
Single Unit UCB Transplantation
n=15 participants at risk
Patients that receive one unit of umbilical cord blood transplantation (only if 2 adequate size and matched units are not available).
|
BM Transplantation
n=4 participants at risk
Blood and Marrow transplantation
|
|---|---|---|---|
|
Infections and infestations
Bacteremia
|
6.7%
1/15 • Number of events 1 • 2 years
|
0.00%
0/15 • 2 years
|
0.00%
0/4 • 2 years
|
|
Investigations
Leukemia relapse
|
13.3%
2/15 • Number of events 2 • 2 years
|
20.0%
3/15 • Number of events 3 • 2 years
|
25.0%
1/4 • Number of events 1 • 2 years
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure (any cause)
|
13.3%
2/15 • Number of events 2 • 2 years
|
6.7%
1/15 • Number of events 1 • 2 years
|
0.00%
0/4 • 2 years
|
|
Investigations
Multi-organ Failure
|
13.3%
2/15 • Number of events 2 • 2 years
|
6.7%
1/15 • Number of events 1 • 2 years
|
0.00%
0/4 • 2 years
|
|
Investigations
Chronic Graft versus host disease
|
6.7%
1/15 • Number of events 1 • 2 years
|
0.00%
0/15 • 2 years
|
0.00%
0/4 • 2 years
|
Other adverse events
| Measure |
Double Unit UCB Transplantation
n=15 participants at risk
Patients that receive 2 units of umbilical cord blood transplantation (UCBT).
|
Single Unit UCB Transplantation
n=15 participants at risk
Patients that receive one unit of umbilical cord blood transplantation (only if 2 adequate size and matched units are not available).
|
BM Transplantation
n=4 participants at risk
Blood and Marrow transplantation
|
|---|---|---|---|
|
Cardiac disorders
PERICARDIAL EFFUSION
|
26.7%
4/15 • Number of events 4 • 2 years
|
13.3%
2/15 • Number of events 2 • 2 years
|
0.00%
0/4 • 2 years
|
|
Renal and urinary disorders
Renal replacement therapy / dialysis
|
20.0%
3/15 • Number of events 4 • 2 years
|
20.0%
3/15 • Number of events 3 • 2 years
|
0.00%
0/4 • 2 years
|
|
Hepatobiliary disorders
Veno-Occlusive Disease
|
26.7%
4/15 • Number of events 4 • 2 years
|
33.3%
5/15 • Number of events 6 • 2 years
|
0.00%
0/4 • 2 years
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure requiring intubation/ventilation
|
46.7%
7/15 • Number of events 7 • 2 years
|
26.7%
4/15 • Number of events 4 • 2 years
|
0.00%
0/4 • 2 years
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary Hemorrhage
|
20.0%
3/15 • Number of events 3 • 2 years
|
20.0%
3/15 • Number of events 3 • 2 years
|
0.00%
0/4 • 2 years
|
|
Cardiac disorders
Cardiopulmonary failure
|
0.00%
0/15 • 2 years
|
20.0%
3/15 • Number of events 3 • 2 years
|
0.00%
0/4 • 2 years
|
|
Infections and infestations
Viremia
|
40.0%
6/15 • Number of events 6 • 2 years
|
13.3%
2/15 • Number of events 2 • 2 years
|
50.0%
2/4 • Number of events 2 • 2 years
|
|
Infections and infestations
Gram positive bacteremia
|
40.0%
6/15 • Number of events 6 • 2 years
|
20.0%
3/15 • Number of events 3 • 2 years
|
25.0%
1/4 • Number of events 1 • 2 years
|
|
Infections and infestations
Gram negative bacteremia
|
6.7%
1/15 • Number of events 1 • 2 years
|
0.00%
0/15 • 2 years
|
75.0%
3/4 • Number of events 3 • 2 years
|
|
Infections and infestations
Pneumonia
|
46.7%
7/15 • Number of events 7 • 2 years
|
33.3%
5/15 • Number of events 5 • 2 years
|
0.00%
0/4 • 2 years
|
|
Investigations
Graft versus host disease grade 1-2
|
13.3%
2/15 • Number of events 2 • 2 years
|
20.0%
3/15 • Number of events 3 • 2 years
|
25.0%
1/4 • Number of events 1 • 2 years
|
|
Investigations
Chronic Graft versus host disease
|
13.3%
2/15 • Number of events 2 • 2 years
|
0.00%
0/15 • 2 years
|
0.00%
0/4 • 2 years
|
|
Investigations
Graft versus host disease grade 3-4
|
26.7%
4/15 • Number of events 4 • 2 years
|
20.0%
3/15 • Number of events 3 • 2 years
|
0.00%
0/4 • 2 years
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place