Trial Outcomes & Findings for A Phase II Study of Umbilical Cord Blood Transplantation (NCT NCT00676806)
NCT ID: NCT00676806
Last Updated: 2017-06-01
Results Overview
Number of participants with neutrophil engraftment receiving umbilical cord blood for hematopoietic rescue following myeloablative or non-myeloablative conditioning
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
7 participants
Primary outcome timeframe
+45 and 90 days
Results posted on
2017-06-01
Participant Flow
One subject signed consent but did not receive a transplant, so was dropped from the study and was not assigned to either arm.
Participant milestones
| Measure |
Myeloablative Conditioning
Patients receiving umbilical cord blood for hematopoietic rescue following myeloablative conditioning
Umbilical Cord Blood Transplantation After Myeloablative Conditioning: Fully-myeloablative Conditioning Regimen: cyclophosphamide (60mg/m2 days -6 \& -5), fludarabine (25 mg/m2 days -7, -6, \& -5) and total body irradiation (days -3, -2, \& -1, total 1200 cGy) followed by cord blood infusion on day 0.
|
Reduced Intensity Conditioning
Patients receiving umbilical cord blood for hematopoietic rescue following non-myeloablative conditioning
Umbilical Cord Blood Transplantation After Reduced-Intensity Conditioning: Reduced Intensity Conditioning Regimen: Extracorporeal Photopheresis (days -8 \& -7), cyclophosphamide 50 mg/kg (day -6) pentostatin 4 mg/kg/d (continuous infusion days -5 \& -4), total body irradiation (days -3 \& -2, total 600cGy) followed by Umbilical Cord Blood Infusion day 0.
|
|---|---|---|
|
Overall Study
STARTED
|
3
|
3
|
|
Overall Study
COMPLETED
|
3
|
3
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Phase II Study of Umbilical Cord Blood Transplantation
Baseline characteristics by cohort
| Measure |
Myeloablative Conditioning
n=3 Participants
Patients receiving umbilical cord blood for hematopoietic rescue following myeloablative conditioning
Umbilical Cord Blood Transplantation After Myeloablative Conditioning: Fully-myeloablative Conditioning Regimen: cyclophosphamide (60mg/m2 days -6 \& -5), fludarabine (25 mg/m2 days -7, -6, \& -5) and total body irradiation (days -3, -2, \& -1, total 1200 cGy) followed by cord blood infusion on day 0.
|
Reduced Intensity Conditioning
n=3 Participants
Patients receiving umbilical cord blood for hematopoietic rescue following non-myeloablative conditioning
Umbilical Cord Blood Transplantation After Reduced-Intensity Conditioning: Reduced Intensity Conditioning Regimen: Extracorporeal Photopheresis (days -8 \& -7), cyclophosphamide 50 mg/kg (day -6) pentostatin 4 mg/kg/d (continuous infusion days -5 \& -4), total body irradiation (days -3 \& -2, total 600cGy) followed by Umbilical Cord Blood Infusion day 0.
|
Total
n=6 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 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
|
|
Age, Continuous
|
43 years
STANDARD_DEVIATION 8.1 • n=5 Participants
|
62.6 years
STANDARD_DEVIATION 2.3 • n=7 Participants
|
52.8 years
STANDARD_DEVIATION 12 • n=5 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
3 participants
n=5 Participants
|
3 participants
n=7 Participants
|
6 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: +45 and 90 daysPopulation: Only 2 subjects were evaluable from each group at the +45 day mark. The same number were evaluable at the +90 day mark.
Number of participants with neutrophil engraftment receiving umbilical cord blood for hematopoietic rescue following myeloablative or non-myeloablative conditioning
Outcome measures
| Measure |
Myeloablative Conditioning
n=2 Participants
Patients receiving umbilical cord blood for hematopoietic rescue following myeloablative conditioning.
2/2 evaluable subjects engrafted at +45 and +90 days.
|
Reduced Intensity Conditioning
n=2 Participants
Patients receiving umbilical cord blood for hematopoietic rescue following non-myeloablative conditioning
1/2 evaluable subjects engrafted at +45 and +90 days.
|
|---|---|---|
|
Number of Participants With Neutrophil Engraftment
|
2 participants
|
1 participants
|
SECONDARY outcome
Timeframe: +45, 90, and 180 daysProportion of patients engrafting by days +45, +90, and +180.
Outcome measures
| Measure |
Myeloablative Conditioning
n=2 Participants
Patients receiving umbilical cord blood for hematopoietic rescue following myeloablative conditioning.
2/2 evaluable subjects engrafted at +45 and +90 days.
|
Reduced Intensity Conditioning
n=2 Participants
Patients receiving umbilical cord blood for hematopoietic rescue following non-myeloablative conditioning
1/2 evaluable subjects engrafted at +45 and +90 days.
|
|---|---|---|
|
Proportion of Subjects With Platelet Engraftment
Platelet Engraftment +45 days
|
0 participants
|
2 participants
|
|
Proportion of Subjects With Platelet Engraftment
Platelet Engraftment +90 days
|
0 participants
|
0 participants
|
|
Proportion of Subjects With Platelet Engraftment
Platelet Engraftment +180 days
|
1 participants
|
0 participants
|
SECONDARY outcome
Timeframe: Day +100Outcome measures
| Measure |
Myeloablative Conditioning
n=3 Participants
Patients receiving umbilical cord blood for hematopoietic rescue following myeloablative conditioning.
2/2 evaluable subjects engrafted at +45 and +90 days.
|
Reduced Intensity Conditioning
n=3 Participants
Patients receiving umbilical cord blood for hematopoietic rescue following non-myeloablative conditioning
1/2 evaluable subjects engrafted at +45 and +90 days.
|
|---|---|---|
|
Incidence of Acute GVHD
|
1 participants
|
0 participants
|
SECONDARY outcome
Timeframe: Day +100Outcome measures
| Measure |
Myeloablative Conditioning
n=3 Participants
Patients receiving umbilical cord blood for hematopoietic rescue following myeloablative conditioning.
2/2 evaluable subjects engrafted at +45 and +90 days.
|
Reduced Intensity Conditioning
n=3 Participants
Patients receiving umbilical cord blood for hematopoietic rescue following non-myeloablative conditioning
1/2 evaluable subjects engrafted at +45 and +90 days.
|
|---|---|---|
|
Infectious Complications in UCB Recipients.
|
3 participants
|
3 participants
|
SECONDARY outcome
Timeframe: After Day +100Population: No subject receiving reduced intensity conditioning was evaluable for the event of chronic GVHD (0/3 subjects survived to day +100)
Outcome measures
| Measure |
Myeloablative Conditioning
n=2 Participants
Patients receiving umbilical cord blood for hematopoietic rescue following myeloablative conditioning.
2/2 evaluable subjects engrafted at +45 and +90 days.
|
Reduced Intensity Conditioning
Patients receiving umbilical cord blood for hematopoietic rescue following non-myeloablative conditioning
1/2 evaluable subjects engrafted at +45 and +90 days.
|
|---|---|---|
|
Incidence of Chronic GVHD
|
1 participants
|
—
|
SECONDARY outcome
Timeframe: +180 daysComposite endpoint of GVH or infection. Too few events to compare between arms.
Outcome measures
| Measure |
Myeloablative Conditioning
n=3 Participants
Patients receiving umbilical cord blood for hematopoietic rescue following myeloablative conditioning.
2/2 evaluable subjects engrafted at +45 and +90 days.
|
Reduced Intensity Conditioning
n=3 Participants
Patients receiving umbilical cord blood for hematopoietic rescue following non-myeloablative conditioning
1/2 evaluable subjects engrafted at +45 and +90 days.
|
|---|---|---|
|
Compare Rates of Complications Between Patients Receiving Ablative vs. Non-myeloablative Conditioning Prior to UCB Transplantation
|
5 events
|
3 events
|
Adverse Events
Myeloablative Conditioning
Serious events: 3 serious events
Other events: 1 other events
Deaths: 0 deaths
Reduced Intensity Conditioning
Serious events: 3 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Myeloablative Conditioning
n=3 participants at risk
Patients receiving umbilical cord blood for hematopoietic rescue following myeloablative conditioning
Umbilical Cord Blood Transplantation After Myeloablative Conditioning: Fully-myeloablative Conditioning Regimen: cyclophosphamide (60mg/m2 days -6 \& -5), fludarabine (25 mg/m2 days -7, -6, \& -5) and total body irradiation (days -3, -2, \& -1, total 1200 cGy) followed by cord blood infusion on day 0.
|
Reduced Intensity Conditioning
n=3 participants at risk
Patients receiving umbilical cord blood for hematopoietic rescue following non-myeloablative conditioning
Umbilical Cord Blood Transplantation After Reduced-Intensity Conditioning: Reduced Intensity Conditioning Regimen: Extracorporeal Photopheresis (days -8 \& -7), cyclophosphamide 50 mg/kg (day -6) pentostatin 4 mg/kg/d (continuous infusion days -5 \& -4), total body irradiation (days -3 \& -2, total 600cGy) followed by Umbilical Cord Blood Infusion day 0.
|
|---|---|---|
|
Infections and infestations
Sepsis
|
100.0%
3/3 • Number of events 3
|
66.7%
2/3 • Number of events 2
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory Failure
|
33.3%
1/3 • Number of events 1
|
66.7%
2/3 • Number of events 2
|
|
Infections and infestations
Lung Infection
|
66.7%
2/3 • Number of events 2
|
33.3%
1/3 • Number of events 1
|
|
Gastrointestinal disorders
Typhlitis
|
33.3%
1/3 • Number of events 1
|
66.7%
2/3 • Number of events 2
|
|
Blood and lymphatic system disorders
Febrile Neutropenia
|
100.0%
3/3 • Number of events 3
|
0.00%
0/3
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary Hemorrhage
|
33.3%
1/3 • Number of events 1
|
0.00%
0/3
|
|
Renal and urinary disorders
Renal Failure
|
33.3%
1/3 • Number of events 1
|
0.00%
0/3
|
|
Immune system disorders
Gastrointestinal GVHD
|
66.7%
2/3 • Number of events 2
|
0.00%
0/3
|
|
Vascular disorders
Thrombotic microangiopathy
|
0.00%
0/3
|
33.3%
1/3 • Number of events 1
|
|
Cardiac disorders
Atrial fibrillation
|
0.00%
0/3
|
33.3%
1/3 • Number of events 1
|
|
Vascular disorders
Thromboembolic event
|
0.00%
0/3
|
33.3%
1/3 • Number of events 1
|
|
Blood and lymphatic system disorders
Bone marrow hypocellular
|
0.00%
0/3
|
33.3%
1/3 • Number of events 1
|
Other adverse events
| Measure |
Myeloablative Conditioning
n=3 participants at risk
Patients receiving umbilical cord blood for hematopoietic rescue following myeloablative conditioning
Umbilical Cord Blood Transplantation After Myeloablative Conditioning: Fully-myeloablative Conditioning Regimen: cyclophosphamide (60mg/m2 days -6 \& -5), fludarabine (25 mg/m2 days -7, -6, \& -5) and total body irradiation (days -3, -2, \& -1, total 1200 cGy) followed by cord blood infusion on day 0.
|
Reduced Intensity Conditioning
n=3 participants at risk
Patients receiving umbilical cord blood for hematopoietic rescue following non-myeloablative conditioning
Umbilical Cord Blood Transplantation After Reduced-Intensity Conditioning: Reduced Intensity Conditioning Regimen: Extracorporeal Photopheresis (days -8 \& -7), cyclophosphamide 50 mg/kg (day -6) pentostatin 4 mg/kg/d (continuous infusion days -5 \& -4), total body irradiation (days -3 \& -2, total 600cGy) followed by Umbilical Cord Blood Infusion day 0.
|
|---|---|---|
|
Skin and subcutaneous tissue disorders
Skin Nodules
|
33.3%
1/3 • Number of events 1
|
0.00%
0/3
|
|
Psychiatric disorders
Confusion
|
33.3%
1/3 • Number of events 1
|
0.00%
0/3
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place