Trial Outcomes & Findings for Treatment of Single or Double Umbilical Cord Trans + Graft-versus-host Disease (GVHD) Prophylaxis w/ Tacrolimus & Mycophenolate Mofetil (NCT NCT00608517)
NCT ID: NCT00608517
Last Updated: 2014-05-20
Results Overview
Evaluate the safety (as determined by the day 100 non-relapse mortality) and feasibility of single or double umbilical cord blood (UCB)stem cell transplant (SCT) in adult or pediatric patients with hematologic malignancies receiving graft-versus-host disease (GVHD) prophylaxis with tacrolimus and mycophenolate mofetil (MMF).
Recruitment status
TERMINATED
Study phase
NA
Target enrollment
6 participants
Primary outcome timeframe
100 days
Results posted on
2014-05-20
Participant Flow
Recruitment period = 9/23/2005 through 8/15/2008
A total of 7 people signed consent to take part in this study. Of those, 1 was determined to be ineligible.
Participant milestones
| Measure |
Pediatric Myeloablative Conditioning
Patients undergo total-body irradiation on days -7 to -4, and receive cyclophosphamide intravenous (IV) over 1 hour on days -3 and -2, methylprednisolone IV twice daily on days -3 to -1, and anti-thymocyte globulin IV over 4 hours on days -3 to -1.
|
Adult Myeloablative Conditioning
Patients receive fludarabine phosphate intravenous (IV) over 30 minutes on days -6 to -4, cyclophosphamide IV over 1 hour on days -5 and -4, and undergo total-body irradiation on days -3 to -1.
|
Reduced-intensity Conditioning
Patients receive fludarabine phosphate intravenous (IV) over 30 minutes on days -6 to -2 and cyclophosphamide IV over 1 hour on day -6 and undergo total-body irradiation on day -1.
|
|---|---|---|---|
|
Overall Study
STARTED
|
2
|
3
|
1
|
|
Overall Study
COMPLETED
|
1
|
3
|
1
|
|
Overall Study
NOT COMPLETED
|
1
|
0
|
0
|
Reasons for withdrawal
| Measure |
Pediatric Myeloablative Conditioning
Patients undergo total-body irradiation on days -7 to -4, and receive cyclophosphamide intravenous (IV) over 1 hour on days -3 and -2, methylprednisolone IV twice daily on days -3 to -1, and anti-thymocyte globulin IV over 4 hours on days -3 to -1.
|
Adult Myeloablative Conditioning
Patients receive fludarabine phosphate intravenous (IV) over 30 minutes on days -6 to -4, cyclophosphamide IV over 1 hour on days -5 and -4, and undergo total-body irradiation on days -3 to -1.
|
Reduced-intensity Conditioning
Patients receive fludarabine phosphate intravenous (IV) over 30 minutes on days -6 to -2 and cyclophosphamide IV over 1 hour on day -6 and undergo total-body irradiation on day -1.
|
|---|---|---|---|
|
Overall Study
Death
|
1
|
0
|
0
|
Baseline Characteristics
Treatment of Single or Double Umbilical Cord Trans + Graft-versus-host Disease (GVHD) Prophylaxis w/ Tacrolimus & Mycophenolate Mofetil
Baseline characteristics by cohort
| Measure |
Pediatric Myeloablative Conditioning
n=2 Participants
Patients undergo total-body irradiation on days -7 to -4, and receive cyclophosphamide intravenous (IV) over 1 hour on days -3 and -2, methylprednisolone IV twice daily on days -3 to -1, and anti-thymocyte globulin IV over 4 hours on days -3 to -1.
|
Adult Myeloablative Conditioning
n=3 Participants
Patients receive fludarabine phosphate intravenous (IV) over 30 minutes on days -6 to -4, cyclophosphamide IV over 1 hour on days -5 and -4, and undergo total-body irradiation on days -3 to -1.
|
Reduced-intensity Conditioning
n=1 Participants
Patients receive fludarabine phosphate intravenous (IV) over 30 minutes on days -6 to -2 and cyclophosphamide IV over 1 hour on day -6 and undergo total-body irradiation on day -1.
|
Total
n=6 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
4 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
|
|
Age, Continuous
|
8 years
STANDARD_DEVIATION 1 • n=5 Participants
|
31 years
STANDARD_DEVIATION 1 • n=7 Participants
|
42 years
STANDARD_DEVIATION 1 • n=5 Participants
|
31 years
STANDARD_DEVIATION 1 • n=4 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
3 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
3 Participants
n=4 Participants
|
|
Region of Enrollment
United States
|
2 participants
n=5 Participants
|
3 participants
n=7 Participants
|
1 participants
n=5 Participants
|
6 participants
n=4 Participants
|
PRIMARY outcome
Timeframe: 100 daysEvaluate the safety (as determined by the day 100 non-relapse mortality) and feasibility of single or double umbilical cord blood (UCB)stem cell transplant (SCT) in adult or pediatric patients with hematologic malignancies receiving graft-versus-host disease (GVHD) prophylaxis with tacrolimus and mycophenolate mofetil (MMF).
Outcome measures
| Measure |
Pediatric Myeloablative Conditioning
n=2 Participants
Patients undergo total-body irradiation on days -7 to -4, and receive cyclophosphamide intravenous (IV) over 1 hour on days -3 and -2, methylprednisolone IV twice daily on days -3 to -1, and anti-thymocyte globulin IV over 4 hours on days -3 to -1.
|
Adult Myeloablative Conditioning
n=3 Participants
Patients receive fludarabine phosphate intravenous (IV) over 30 minutes on days -6 to -4, cyclophosphamide IV over 1 hour on days -5 and -4, and undergo total-body irradiation on days -3 to -1.
|
Reduced-intensity Conditioning
n=1 Participants
Patients receive fludarabine phosphate intravenous (IV) over 30 minutes on days -6 to -2 and cyclophosphamide IV over 1 hour on day -6 and undergo total-body irradiation on day -1.
|
|---|---|---|---|
|
Number of Participants With 100-day Non-relapse Mortality
|
1 participants
|
0 participants
|
0 participants
|
SECONDARY outcome
Timeframe: 42 daysPopulation: Patients who received treatment and who did not die before day 42.
Recovery of the neutrophil portion of white blood cells and showing complete donor cells.
Outcome measures
| Measure |
Pediatric Myeloablative Conditioning
n=1 Participants
Patients undergo total-body irradiation on days -7 to -4, and receive cyclophosphamide intravenous (IV) over 1 hour on days -3 and -2, methylprednisolone IV twice daily on days -3 to -1, and anti-thymocyte globulin IV over 4 hours on days -3 to -1.
|
Adult Myeloablative Conditioning
n=2 Participants
Patients receive fludarabine phosphate intravenous (IV) over 30 minutes on days -6 to -4, cyclophosphamide IV over 1 hour on days -5 and -4, and undergo total-body irradiation on days -3 to -1.
|
Reduced-intensity Conditioning
Patients receive fludarabine phosphate intravenous (IV) over 30 minutes on days -6 to -2 and cyclophosphamide IV over 1 hour on day -6 and undergo total-body irradiation on day -1.
|
|---|---|---|---|
|
Number of Participants With Sustained Donor Engraftment of Umbilical Cord Blood Stem Cells
|
1 participants
|
1 participants
|
—
|
SECONDARY outcome
Timeframe: 100 daysParticipants who exhibit acute GVHD.
Outcome measures
| Measure |
Pediatric Myeloablative Conditioning
n=2 Participants
Patients undergo total-body irradiation on days -7 to -4, and receive cyclophosphamide intravenous (IV) over 1 hour on days -3 and -2, methylprednisolone IV twice daily on days -3 to -1, and anti-thymocyte globulin IV over 4 hours on days -3 to -1.
|
Adult Myeloablative Conditioning
n=3 Participants
Patients receive fludarabine phosphate intravenous (IV) over 30 minutes on days -6 to -4, cyclophosphamide IV over 1 hour on days -5 and -4, and undergo total-body irradiation on days -3 to -1.
|
Reduced-intensity Conditioning
n=1 Participants
Patients receive fludarabine phosphate intravenous (IV) over 30 minutes on days -6 to -2 and cyclophosphamide IV over 1 hour on day -6 and undergo total-body irradiation on day -1.
|
|---|---|---|---|
|
Number of Participants With Acute Graft-versus-host Disease (GVHD)
|
0 participants
|
1 participants
|
0 participants
|
SECONDARY outcome
Timeframe: 1 yearOutcome measures
| Measure |
Pediatric Myeloablative Conditioning
n=2 Participants
Patients undergo total-body irradiation on days -7 to -4, and receive cyclophosphamide intravenous (IV) over 1 hour on days -3 and -2, methylprednisolone IV twice daily on days -3 to -1, and anti-thymocyte globulin IV over 4 hours on days -3 to -1.
|
Adult Myeloablative Conditioning
n=3 Participants
Patients receive fludarabine phosphate intravenous (IV) over 30 minutes on days -6 to -4, cyclophosphamide IV over 1 hour on days -5 and -4, and undergo total-body irradiation on days -3 to -1.
|
Reduced-intensity Conditioning
n=1 Participants
Patients receive fludarabine phosphate intravenous (IV) over 30 minutes on days -6 to -2 and cyclophosphamide IV over 1 hour on day -6 and undergo total-body irradiation on day -1.
|
|---|---|---|---|
|
Number of Participants Who Relapsed at 1 Year
|
0 participants
|
0 participants
|
0 participants
|
SECONDARY outcome
Timeframe: at 100 daysDeath from any cause at 100 days
Outcome measures
| Measure |
Pediatric Myeloablative Conditioning
n=2 Participants
Patients undergo total-body irradiation on days -7 to -4, and receive cyclophosphamide intravenous (IV) over 1 hour on days -3 and -2, methylprednisolone IV twice daily on days -3 to -1, and anti-thymocyte globulin IV over 4 hours on days -3 to -1.
|
Adult Myeloablative Conditioning
n=3 Participants
Patients receive fludarabine phosphate intravenous (IV) over 30 minutes on days -6 to -4, cyclophosphamide IV over 1 hour on days -5 and -4, and undergo total-body irradiation on days -3 to -1.
|
Reduced-intensity Conditioning
n=1 Participants
Patients receive fludarabine phosphate intravenous (IV) over 30 minutes on days -6 to -2 and cyclophosphamide IV over 1 hour on day -6 and undergo total-body irradiation on day -1.
|
|---|---|---|---|
|
Number of Subjects With All-cause Mortality
|
1 participants
|
0 participants
|
0 participants
|
SECONDARY outcome
Timeframe: 1 yearOverall survival at 1 year
Outcome measures
| Measure |
Pediatric Myeloablative Conditioning
n=2 Participants
Patients undergo total-body irradiation on days -7 to -4, and receive cyclophosphamide intravenous (IV) over 1 hour on days -3 and -2, methylprednisolone IV twice daily on days -3 to -1, and anti-thymocyte globulin IV over 4 hours on days -3 to -1.
|
Adult Myeloablative Conditioning
n=3 Participants
Patients receive fludarabine phosphate intravenous (IV) over 30 minutes on days -6 to -4, cyclophosphamide IV over 1 hour on days -5 and -4, and undergo total-body irradiation on days -3 to -1.
|
Reduced-intensity Conditioning
n=1 Participants
Patients receive fludarabine phosphate intravenous (IV) over 30 minutes on days -6 to -2 and cyclophosphamide IV over 1 hour on day -6 and undergo total-body irradiation on day -1.
|
|---|---|---|---|
|
Overall Survival
|
1 participants
|
3 participants
|
1 participants
|
SECONDARY outcome
Timeframe: 100 daysAs opposed to acute GVHD, which is characterized by rash, cholestasis, and enteritis, chronic GVHD is characterized by nausea, anorexia, ocular and oral sicca, and other organ involvement
Outcome measures
| Measure |
Pediatric Myeloablative Conditioning
n=2 Participants
Patients undergo total-body irradiation on days -7 to -4, and receive cyclophosphamide intravenous (IV) over 1 hour on days -3 and -2, methylprednisolone IV twice daily on days -3 to -1, and anti-thymocyte globulin IV over 4 hours on days -3 to -1.
|
Adult Myeloablative Conditioning
n=3 Participants
Patients receive fludarabine phosphate intravenous (IV) over 30 minutes on days -6 to -4, cyclophosphamide IV over 1 hour on days -5 and -4, and undergo total-body irradiation on days -3 to -1.
|
Reduced-intensity Conditioning
n=1 Participants
Patients receive fludarabine phosphate intravenous (IV) over 30 minutes on days -6 to -2 and cyclophosphamide IV over 1 hour on day -6 and undergo total-body irradiation on day -1.
|
|---|---|---|---|
|
Number of Participants With Chronic Graft Versus Host Disease (GVHD)
|
0 participants
|
0 participants
|
0 participants
|
Adverse Events
Pediatric Myeloablative Conditioning
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Adult Myeloablative Conditioning
Serious events: 1 serious events
Other events: 2 other events
Deaths: 0 deaths
Reduced-intensity Conditioning
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Pediatric Myeloablative Conditioning
n=2 participants at risk
Patients undergo total-body irradiation on days -7 to -4, and receive cyclophosphamide intravenous (IV) over 1 hour on days -3 and -2, methylprednisolone IV twice daily on days -3 to -1, and anti-thymocyte globulin IV over 4 hours on days -3 to -1.
|
Adult Myeloablative Conditioning
n=3 participants at risk
Patients receive fludarabine phosphate intravenous (IV) over 30 minutes on days -6 to -4, cyclophosphamide IV over 1 hour on days -5 and -4, and undergo total-body irradiation on days -3 to -1.
|
Reduced-intensity Conditioning
n=1 participants at risk
Patients receive fludarabine phosphate intravenous (IV) over 30 minutes on days -6 to -2 and cyclophosphamide IV over 1 hour on day -6 and undergo total-body irradiation on day -1.
|
|---|---|---|---|
|
Infections and infestations
MRSE
|
0.00%
0/2
|
33.3%
1/3 • Number of events 1
|
0.00%
0/1
|
|
Infections and infestations
Gram negative rods septicemia
|
0.00%
0/2
|
33.3%
1/3 • Number of events 1
|
0.00%
0/1
|
Other adverse events
| Measure |
Pediatric Myeloablative Conditioning
n=2 participants at risk
Patients undergo total-body irradiation on days -7 to -4, and receive cyclophosphamide intravenous (IV) over 1 hour on days -3 and -2, methylprednisolone IV twice daily on days -3 to -1, and anti-thymocyte globulin IV over 4 hours on days -3 to -1.
|
Adult Myeloablative Conditioning
n=3 participants at risk
Patients receive fludarabine phosphate intravenous (IV) over 30 minutes on days -6 to -4, cyclophosphamide IV over 1 hour on days -5 and -4, and undergo total-body irradiation on days -3 to -1.
|
Reduced-intensity Conditioning
n=1 participants at risk
Patients receive fludarabine phosphate intravenous (IV) over 30 minutes on days -6 to -2 and cyclophosphamide IV over 1 hour on day -6 and undergo total-body irradiation on day -1.
|
|---|---|---|---|
|
Metabolism and nutrition disorders
Anorexia
|
0.00%
0/2
|
66.7%
2/3 • Number of events 2
|
0.00%
0/1
|
|
Gastrointestinal disorders
Mucositis (functional/symptomatic)
|
0.00%
0/2
|
66.7%
2/3 • Number of events 4
|
0.00%
0/1
|
|
Gastrointestinal disorders
Mucositis (clinical exam)
|
0.00%
0/2
|
66.7%
2/3 • Number of events 3
|
0.00%
0/1
|
|
Infections and infestations
Catheter-related infection Grade 3-4 neutrophils (ANC < 1.0 x 10e9/L)
|
0.00%
0/2
|
66.7%
2/3 • Number of events 2
|
0.00%
0/1
|
|
Infections and infestations
Infection with normal ANC or Grade 1-2 neutrophils
|
0.00%
0/2
|
66.7%
2/3 • Number of events 4
|
0.00%
0/1
|
|
Infections and infestations
Infection with grade 3-4 neutrophils (ANC< 1.0 x 10e9/L)
|
0.00%
0/2
|
33.3%
1/3 • Number of events 1
|
0.00%
0/1
|
|
Infections and infestations
Bladder infection
|
0.00%
0/2
|
33.3%
1/3 • Number of events 1
|
0.00%
0/1
|
|
Infections and infestations
Catheter-related infection grade 1-2 neutrophils
|
0.00%
0/2
|
33.3%
1/3 • Number of events 3
|
0.00%
0/1
|
|
Infections and infestations
Infection with nomral ANC or grade 1-2 joint
|
0.00%
0/2
|
33.3%
1/3 • Number of events 2
|
0.00%
0/1
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia
|
0.00%
0/2
|
33.3%
1/3 • Number of events 5
|
0.00%
0/1
|
|
Skin and subcutaneous tissue disorders
Cellulitis
|
0.00%
0/2
|
33.3%
1/3 • Number of events 1
|
0.00%
0/1
|
|
Infections and infestations
Urinary tract infection
|
0.00%
0/2
|
33.3%
1/3 • Number of events 2
|
0.00%
0/1
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
0.00%
0/2
|
66.7%
2/3 • Number of events 9
|
0.00%
0/1
|
|
Metabolism and nutrition disorders
Hyponatremia
|
0.00%
0/2
|
66.7%
2/3 • Number of events 3
|
0.00%
0/1
|
|
Investigations
Creatinine increased
|
0.00%
0/2
|
33.3%
1/3 • Number of events 1
|
0.00%
0/1
|
|
Metabolism and nutrition disorders
Hypophosphatemia
|
0.00%
0/2
|
33.3%
1/3 • Number of events 1
|
0.00%
0/1
|
|
Metabolism and nutrition disorders
Hyperkalemia
|
0.00%
0/2
|
33.3%
1/3 • Number of events 5
|
0.00%
0/1
|
|
Metabolism and nutrition disorders
Hyperuricemia
|
0.00%
0/2
|
33.3%
1/3 • Number of events 2
|
0.00%
0/1
|
|
Renal and urinary disorders
Cystitis
|
0.00%
0/2
|
66.7%
2/3 • Number of events 4
|
0.00%
0/1
|
|
Renal and urinary disorders
Bladder spasms
|
0.00%
0/2
|
33.3%
1/3 • Number of events 1
|
0.00%
0/1
|
|
Renal and urinary disorders
Renal failure
|
0.00%
0/2
|
33.3%
1/3 • Number of events 3
|
0.00%
0/1
|
|
Vascular disorders
Hypertension
|
0.00%
0/2
|
33.3%
1/3 • Number of events 2
|
0.00%
0/1
|
|
Cardiac disorders
Pericarditis
|
0.00%
0/2
|
33.3%
1/3 • Number of events 1
|
0.00%
0/1
|
|
Blood and lymphatic system disorders
Abnormal PTT
|
0.00%
0/2
|
33.3%
1/3 • Number of events 2
|
0.00%
0/1
|
|
Renal and urinary disorders
Urinary bleeding
|
0.00%
0/2
|
33.3%
1/3 • Number of events 1
|
0.00%
0/1
|
|
Musculoskeletal and connective tissue disorders
Osteonecrosis
|
0.00%
0/2
|
33.3%
1/3 • Number of events 3
|
0.00%
0/1
|
|
Musculoskeletal and connective tissue disorders
Limb pain
|
0.00%
0/2
|
33.3%
1/3 • Number of events 1
|
0.00%
0/1
|
|
Musculoskeletal and connective tissue disorders
Joint pain
|
0.00%
0/2
|
33.3%
1/3 • Number of events 3
|
0.00%
0/1
|
|
Reproductive system and breast disorders
Testical pain
|
0.00%
0/2
|
33.3%
1/3 • Number of events 1
|
0.00%
0/1
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
0.00%
0/2
|
33.3%
1/3 • Number of events 1
|
0.00%
0/1
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
0.00%
0/2
|
33.3%
1/3 • Number of events 4
|
0.00%
0/1
|
|
Reproductive system and breast disorders
Errectile dysfunction
|
0.00%
0/2
|
33.3%
1/3 • Number of events 1
|
0.00%
0/1
|
|
Reproductive system and breast disorders
Libido
|
0.00%
0/2
|
33.3%
1/3 • Number of events 1
|
0.00%
0/1
|
|
Vascular disorders
Thrombosis
|
0.00%
0/2
|
33.3%
1/3 • Number of events 1
|
0.00%
0/1
|
Additional Information
Brian Engelhardt, M.D.
Vanderbilt-Ingram Cancer Center
Phone: 615-936-1803
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place