Trial Outcomes & Findings for Donor Umbilical Cord Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies (NCT NCT01093586)
NCT ID: NCT01093586
Last Updated: 2019-01-23
Results Overview
Number of participants alive at 180 days post engraftment.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
14 participants
Primary outcome timeframe
On day +180
Results posted on
2019-01-23
Participant Flow
Participants recruited from medical hospital from 9/2007 thru 2/2013.
Participant milestones
| Measure |
Arm I
PREPARATIVE REGIMEN: Patients receive oral busulfan on days -8 to -5, cyclophosphamide IV on days -4 to -3, and anti-thymocyte globulin or methylprednisolone IV on days -3 to -1. TRANSPLANTATION: Patients undergo a double-unit umbilical cord blood allogeneic stem cell transplantation on day 0. GRAFT-VS-HOST DISEASE PROPHYLAXIS: Beginning on day -2, patients receive cyclosporine IV and taper beginning on day 100. Patients also receive mycophenolate mofetil IV or orally on days -3 to 45.
|
|---|---|
|
Overall Study
STARTED
|
14
|
|
Overall Study
COMPLETED
|
11
|
|
Overall Study
NOT COMPLETED
|
3
|
Reasons for withdrawal
| Measure |
Arm I
PREPARATIVE REGIMEN: Patients receive oral busulfan on days -8 to -5, cyclophosphamide IV on days -4 to -3, and anti-thymocyte globulin or methylprednisolone IV on days -3 to -1. TRANSPLANTATION: Patients undergo a double-unit umbilical cord blood allogeneic stem cell transplantation on day 0. GRAFT-VS-HOST DISEASE PROPHYLAXIS: Beginning on day -2, patients receive cyclosporine IV and taper beginning on day 100. Patients also receive mycophenolate mofetil IV or orally on days -3 to 45.
|
|---|---|
|
Overall Study
Failed to engraft
|
3
|
Baseline Characteristics
Donor Umbilical Cord Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies
Baseline characteristics by cohort
| Measure |
Arm I
n=14 Participants
PREPARATIVE REGIMEN: Patients receive oral busulfan on days -8 to -5, cyclophosphamide IV on days -4 to -3, and anti-thymocyte globulin or methylprednisolone IV on days -3 to -1. TRANSPLANTATION: Patients undergo a double-unit umbilical cord blood allogeneic stem cell transplantation on day 0. GRAFT-VS-HOST DISEASE PROPHYLAXIS: Beginning on day -2, patients receive cyclosporine IV and taper beginning on day 100. Patients also receive mycophenolate mofetil IV or orally on days -3 to 45.
|
|---|---|
|
Age, Customized
10-19 yrs
|
2 Participants
n=5 Participants
|
|
Age, Customized
20-29 yrs
|
2 Participants
n=5 Participants
|
|
Age, Customized
30-39 yrs
|
3 Participants
n=5 Participants
|
|
Age, Customized
40-49 yrs
|
5 Participants
n=5 Participants
|
|
Age, Customized
50-59 yrs
|
2 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
8 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
6 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
13 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
11 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
14 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: On day +180Population: All participants that went on study, whether completing engraftment or not.
Number of participants alive at 180 days post engraftment.
Outcome measures
| Measure |
Arm I
n=14 Participants
PREPARATIVE REGIMEN: Patients receive oral busulfan on days -8 to -5, cyclophosphamide IV on days -4 to -3, and anti-thymocyte globulin or methylprednisolone IV on days -3 to -1. TRANSPLANTATION: Patients undergo a double-unit umbilical cord blood allogeneic stem cell transplantation on day 0. GRAFT-VS-HOST DISEASE PROPHYLAXIS: Beginning on day -2, patients receive cyclosporine IV and taper beginning on day 100. Patients also receive mycophenolate mofetil IV or orally on days -3 to 45.
|
|---|---|
|
Overall Survival
|
8 Participants
|
SECONDARY outcome
Timeframe: On day +42Population: Participants that went on study.
Number of participants that were able to complete engraftment by day 42.
Outcome measures
| Measure |
Arm I
n=14 Participants
PREPARATIVE REGIMEN: Patients receive oral busulfan on days -8 to -5, cyclophosphamide IV on days -4 to -3, and anti-thymocyte globulin or methylprednisolone IV on days -3 to -1. TRANSPLANTATION: Patients undergo a double-unit umbilical cord blood allogeneic stem cell transplantation on day 0. GRAFT-VS-HOST DISEASE PROPHYLAXIS: Beginning on day -2, patients receive cyclosporine IV and taper beginning on day 100. Patients also receive mycophenolate mofetil IV or orally on days -3 to 45.
|
|---|---|
|
Hematologic Engraftment
|
10 Participants
|
SECONDARY outcome
Timeframe: At 1 yearPopulation: Participants that completed engraftment. 10 participants completed engraftment by day 42 and 1 participant completed engraftment by day 46.
Number of participants that were alive.
Outcome measures
| Measure |
Arm I
n=11 Participants
PREPARATIVE REGIMEN: Patients receive oral busulfan on days -8 to -5, cyclophosphamide IV on days -4 to -3, and anti-thymocyte globulin or methylprednisolone IV on days -3 to -1. TRANSPLANTATION: Patients undergo a double-unit umbilical cord blood allogeneic stem cell transplantation on day 0. GRAFT-VS-HOST DISEASE PROPHYLAXIS: Beginning on day -2, patients receive cyclosporine IV and taper beginning on day 100. Patients also receive mycophenolate mofetil IV or orally on days -3 to 45.
|
|---|---|
|
Overall Survival
|
6 Participants
|
SECONDARY outcome
Timeframe: At 2 yearsPopulation: Participants that completed engraftment. 10 participants completed engraftment by day 42 and 1 participant completed engraftment by day 46.
Number of participants that were alive.
Outcome measures
| Measure |
Arm I
n=11 Participants
PREPARATIVE REGIMEN: Patients receive oral busulfan on days -8 to -5, cyclophosphamide IV on days -4 to -3, and anti-thymocyte globulin or methylprednisolone IV on days -3 to -1. TRANSPLANTATION: Patients undergo a double-unit umbilical cord blood allogeneic stem cell transplantation on day 0. GRAFT-VS-HOST DISEASE PROPHYLAXIS: Beginning on day -2, patients receive cyclosporine IV and taper beginning on day 100. Patients also receive mycophenolate mofetil IV or orally on days -3 to 45.
|
|---|---|
|
Overall Survival
|
4 Participants
|
SECONDARY outcome
Timeframe: At 1 yearPopulation: Participants that completed engraftment. 10 participants completed engraftment by day 42 and 1 participant completed engraftment by day 46.
Number of participants that were disease free
Outcome measures
| Measure |
Arm I
n=11 Participants
PREPARATIVE REGIMEN: Patients receive oral busulfan on days -8 to -5, cyclophosphamide IV on days -4 to -3, and anti-thymocyte globulin or methylprednisolone IV on days -3 to -1. TRANSPLANTATION: Patients undergo a double-unit umbilical cord blood allogeneic stem cell transplantation on day 0. GRAFT-VS-HOST DISEASE PROPHYLAXIS: Beginning on day -2, patients receive cyclosporine IV and taper beginning on day 100. Patients also receive mycophenolate mofetil IV or orally on days -3 to 45.
|
|---|---|
|
Disease Free
|
4 Participants
|
SECONDARY outcome
Timeframe: At 2 yearsPopulation: Participants that completed engraftment. 10 participants completed engraftment by day 42 and 1 participant completed engraftment by day 46.
Number of participants that were disease free
Outcome measures
| Measure |
Arm I
n=11 Participants
PREPARATIVE REGIMEN: Patients receive oral busulfan on days -8 to -5, cyclophosphamide IV on days -4 to -3, and anti-thymocyte globulin or methylprednisolone IV on days -3 to -1. TRANSPLANTATION: Patients undergo a double-unit umbilical cord blood allogeneic stem cell transplantation on day 0. GRAFT-VS-HOST DISEASE PROPHYLAXIS: Beginning on day -2, patients receive cyclosporine IV and taper beginning on day 100. Patients also receive mycophenolate mofetil IV or orally on days -3 to 45.
|
|---|---|
|
Disease Free
|
4 Participants
|
SECONDARY outcome
Timeframe: one year in patients post UCBTPopulation: Participants that completed engraftment. 10 participants completed engraftment by day 42 and 1 participant completed engraftment by day 46.
Number of subjects that had disease recurrence
Outcome measures
| Measure |
Arm I
n=11 Participants
PREPARATIVE REGIMEN: Patients receive oral busulfan on days -8 to -5, cyclophosphamide IV on days -4 to -3, and anti-thymocyte globulin or methylprednisolone IV on days -3 to -1. TRANSPLANTATION: Patients undergo a double-unit umbilical cord blood allogeneic stem cell transplantation on day 0. GRAFT-VS-HOST DISEASE PROPHYLAXIS: Beginning on day -2, patients receive cyclosporine IV and taper beginning on day 100. Patients also receive mycophenolate mofetil IV or orally on days -3 to 45.
|
|---|---|
|
Recurrence or Relapse
|
2 Participants
|
SECONDARY outcome
Timeframe: two years post transplantPopulation: Subjects who had completed engraftment
Number of subjects that had disease recurrence
Outcome measures
| Measure |
Arm I
n=11 Participants
PREPARATIVE REGIMEN: Patients receive oral busulfan on days -8 to -5, cyclophosphamide IV on days -4 to -3, and anti-thymocyte globulin or methylprednisolone IV on days -3 to -1. TRANSPLANTATION: Patients undergo a double-unit umbilical cord blood allogeneic stem cell transplantation on day 0. GRAFT-VS-HOST DISEASE PROPHYLAXIS: Beginning on day -2, patients receive cyclosporine IV and taper beginning on day 100. Patients also receive mycophenolate mofetil IV or orally on days -3 to 45.
|
|---|---|
|
Recurrence or Relapse
|
2 Participants
|
SECONDARY outcome
Timeframe: On day 100 post transplantPopulation: Participants that went on study
Number of subjects that died because of transplant
Outcome measures
| Measure |
Arm I
n=14 Participants
PREPARATIVE REGIMEN: Patients receive oral busulfan on days -8 to -5, cyclophosphamide IV on days -4 to -3, and anti-thymocyte globulin or methylprednisolone IV on days -3 to -1. TRANSPLANTATION: Patients undergo a double-unit umbilical cord blood allogeneic stem cell transplantation on day 0. GRAFT-VS-HOST DISEASE PROPHYLAXIS: Beginning on day -2, patients receive cyclosporine IV and taper beginning on day 100. Patients also receive mycophenolate mofetil IV or orally on days -3 to 45.
|
|---|---|
|
Transplant Related Mortality
|
2 Participants
|
SECONDARY outcome
Timeframe: On day 180 post transplantPopulation: All participants that went on study
Number of subjects that died because of transplant
Outcome measures
| Measure |
Arm I
n=14 Participants
PREPARATIVE REGIMEN: Patients receive oral busulfan on days -8 to -5, cyclophosphamide IV on days -4 to -3, and anti-thymocyte globulin or methylprednisolone IV on days -3 to -1. TRANSPLANTATION: Patients undergo a double-unit umbilical cord blood allogeneic stem cell transplantation on day 0. GRAFT-VS-HOST DISEASE PROPHYLAXIS: Beginning on day -2, patients receive cyclosporine IV and taper beginning on day 100. Patients also receive mycophenolate mofetil IV or orally on days -3 to 45.
|
|---|---|
|
Transplant Related Mortality
|
6 Participants
|
SECONDARY outcome
Timeframe: 1 yearPopulation: All participants that went on study
Number of participants that had infections
Outcome measures
| Measure |
Arm I
n=14 Participants
PREPARATIVE REGIMEN: Patients receive oral busulfan on days -8 to -5, cyclophosphamide IV on days -4 to -3, and anti-thymocyte globulin or methylprednisolone IV on days -3 to -1. TRANSPLANTATION: Patients undergo a double-unit umbilical cord blood allogeneic stem cell transplantation on day 0. GRAFT-VS-HOST DISEASE PROPHYLAXIS: Beginning on day -2, patients receive cyclosporine IV and taper beginning on day 100. Patients also receive mycophenolate mofetil IV or orally on days -3 to 45.
|
|---|---|
|
Occurrence of Serious Infections
|
13 Participants
|
SECONDARY outcome
Timeframe: Periodically for 2 yearsPopulation: Data not collected, panel not done because of funding.
Immunodificency panel to see recovery of immune system. Number of participants that recovered.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: by day +42Population: Patients that received transplant
Number of participants that experienced toxicity related to the transplant
Outcome measures
| Measure |
Arm I
n=11 Participants
PREPARATIVE REGIMEN: Patients receive oral busulfan on days -8 to -5, cyclophosphamide IV on days -4 to -3, and anti-thymocyte globulin or methylprednisolone IV on days -3 to -1. TRANSPLANTATION: Patients undergo a double-unit umbilical cord blood allogeneic stem cell transplantation on day 0. GRAFT-VS-HOST DISEASE PROPHYLAXIS: Beginning on day -2, patients receive cyclosporine IV and taper beginning on day 100. Patients also receive mycophenolate mofetil IV or orally on days -3 to 45.
|
|---|---|
|
Toxicity Related to UCB Transplantation and Cytoreduction as Assessed by CTC v3.0
|
8 Participants
|
SECONDARY outcome
Timeframe: At 100 daysPopulation: All participants who went on study
Number of participants that had acute GVHD
Outcome measures
| Measure |
Arm I
n=14 Participants
PREPARATIVE REGIMEN: Patients receive oral busulfan on days -8 to -5, cyclophosphamide IV on days -4 to -3, and anti-thymocyte globulin or methylprednisolone IV on days -3 to -1. TRANSPLANTATION: Patients undergo a double-unit umbilical cord blood allogeneic stem cell transplantation on day 0. GRAFT-VS-HOST DISEASE PROPHYLAXIS: Beginning on day -2, patients receive cyclosporine IV and taper beginning on day 100. Patients also receive mycophenolate mofetil IV or orally on days -3 to 45.
|
|---|---|
|
Incidence of Acute Graft-versus-host Disease (GVHD)
|
11 Participants
|
SECONDARY outcome
Timeframe: At 1 yearPopulation: All participants that went on study
Number of participants that have chronic GVHD. Chronic GVHD will be diagnosed and graded on clinical and histological criteria from the Center for International Blood and Marrow Transplant Research (CIBMTR)
Outcome measures
| Measure |
Arm I
n=14 Participants
PREPARATIVE REGIMEN: Patients receive oral busulfan on days -8 to -5, cyclophosphamide IV on days -4 to -3, and anti-thymocyte globulin or methylprednisolone IV on days -3 to -1. TRANSPLANTATION: Patients undergo a double-unit umbilical cord blood allogeneic stem cell transplantation on day 0. GRAFT-VS-HOST DISEASE PROPHYLAXIS: Beginning on day -2, patients receive cyclosporine IV and taper beginning on day 100. Patients also receive mycophenolate mofetil IV or orally on days -3 to 45.
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|---|---|
|
Incidence of Chronic GVHD
|
1 Participants
|
Adverse Events
Arm I
Serious events: 9 serious events
Other events: 0 other events
Deaths: 11 deaths
Serious adverse events
| Measure |
Arm I
n=14 participants at risk
PREPARATIVE REGIMEN: Patients receive oral busulfan on days -8 to -5, cyclophosphamide IV on days -4 to -3, and anti-thymocyte globulin or methylprednisolone IV on days -3 to -1. TRANSPLANTATION: Patients undergo a double-unit umbilical cord blood allogeneic stem cell transplantation on day 0. GRAFT-VS-HOST DISEASE PROPHYLAXIS: Beginning on day -2, patients receive cyclosporine IV and taper beginning on day 100. Patients also receive mycophenolate mofetil IV or orally on days -3 to 45.
|
|---|---|
|
Gastrointestinal disorders
Diarrhea
|
21.4%
3/14 • Number of events 4 • Adverse Events were collected from participant start on study to going off study up to a period of 2 years.
Non serious events were not collected. The protocol was assessing efficacy of cord blood in transplants, therefore the research was only gathering SAEs of grade 3 or 4.
|
|
Cardiac disorders
Supraventricular and nodal arrhythmia - Sinus tachycardia
|
7.1%
1/14 • Number of events 1 • Adverse Events were collected from participant start on study to going off study up to a period of 2 years.
Non serious events were not collected. The protocol was assessing efficacy of cord blood in transplants, therefore the research was only gathering SAEs of grade 3 or 4.
|
|
General disorders
Death not associated with CTCAE term - Multi-organ failure
|
21.4%
3/14 • Number of events 3 • Adverse Events were collected from participant start on study to going off study up to a period of 2 years.
Non serious events were not collected. The protocol was assessing efficacy of cord blood in transplants, therefore the research was only gathering SAEs of grade 3 or 4.
|
|
Respiratory, thoracic and mediastinal disorders
Hemorrhage, pulmonary/upper respiratory - Nose
|
7.1%
1/14 • Number of events 1 • Adverse Events were collected from participant start on study to going off study up to a period of 2 years.
Non serious events were not collected. The protocol was assessing efficacy of cord blood in transplants, therefore the research was only gathering SAEs of grade 3 or 4.
|
|
Hepatobiliary disorders
Bilirubin (hyperbilirubinemia)
|
7.1%
1/14 • Number of events 1 • Adverse Events were collected from participant start on study to going off study up to a period of 2 years.
Non serious events were not collected. The protocol was assessing efficacy of cord blood in transplants, therefore the research was only gathering SAEs of grade 3 or 4.
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
28.6%
4/14 • Number of events 5 • Adverse Events were collected from participant start on study to going off study up to a period of 2 years.
Non serious events were not collected. The protocol was assessing efficacy of cord blood in transplants, therefore the research was only gathering SAEs of grade 3 or 4.
|
|
General disorders
Death not associated with CTCAE term - Death NOS
|
14.3%
2/14 • Number of events 2 • Adverse Events were collected from participant start on study to going off study up to a period of 2 years.
Non serious events were not collected. The protocol was assessing efficacy of cord blood in transplants, therefore the research was only gathering SAEs of grade 3 or 4.
|
|
Cardiac disorders
Hypotension
|
7.1%
1/14 • Number of events 1 • Adverse Events were collected from participant start on study to going off study up to a period of 2 years.
Non serious events were not collected. The protocol was assessing efficacy of cord blood in transplants, therefore the research was only gathering SAEs of grade 3 or 4.
|
|
Blood and lymphatic system disorders
Bone marrow cellularity - Primary graft failure
|
7.1%
1/14 • Number of events 1 • Adverse Events were collected from participant start on study to going off study up to a period of 2 years.
Non serious events were not collected. The protocol was assessing efficacy of cord blood in transplants, therefore the research was only gathering SAEs of grade 3 or 4.
|
|
Product Issues
Infection with normal ANC or Grade 1 or 2 neutrophils
|
14.3%
2/14 • Number of events 2 • Adverse Events were collected from participant start on study to going off study up to a period of 2 years.
Non serious events were not collected. The protocol was assessing efficacy of cord blood in transplants, therefore the research was only gathering SAEs of grade 3 or 4.
|
|
General disorders
Fever (in the absence of neutropenia, where neutropenia is defined as ANC <1.0 x 10e9/L)
|
7.1%
1/14 • Number of events 1 • Adverse Events were collected from participant start on study to going off study up to a period of 2 years.
Non serious events were not collected. The protocol was assessing efficacy of cord blood in transplants, therefore the research was only gathering SAEs of grade 3 or 4.
|
|
Nervous system disorders
Infection with normal ANC or Grade 1 or 2 neutrophils - Brain (encephalitis, infectious)
|
7.1%
1/14 • Number of events 1 • Adverse Events were collected from participant start on study to going off study up to a period of 2 years.
Non serious events were not collected. The protocol was assessing efficacy of cord blood in transplants, therefore the research was only gathering SAEs of grade 3 or 4.
|
|
Respiratory, thoracic and mediastinal disorders
Infection with normal ANC or Grade 1 or 2 neutrophils - Lung (pneumonia)
|
7.1%
1/14 • Number of events 1 • Adverse Events were collected from participant start on study to going off study up to a period of 2 years.
Non serious events were not collected. The protocol was assessing efficacy of cord blood in transplants, therefore the research was only gathering SAEs of grade 3 or 4.
|
|
Gastrointestinal disorders
Colitis, infectious (e.g., Clostridium difficile)
|
7.1%
1/14 • Number of events 1 • Adverse Events were collected from participant start on study to going off study up to a period of 2 years.
Non serious events were not collected. The protocol was assessing efficacy of cord blood in transplants, therefore the research was only gathering SAEs of grade 3 or 4.
|
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place