Trial Outcomes & Findings for Conditioning Regimen and the Transplantation of Unrelated Donor Umbilical Cord Blood in Patients With Hematologic Malignancies. (NCT NCT00739141)

Last Updated: 2022-12-08

Results Overview

To obtain a preliminary estimate of progression free survival at 1 year post UCBT. Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

86 participants

Primary outcome timeframe

1 year post UCBT

Results posted on

2022-12-08

Participant Flow

Participant milestones

Participant milestones
Measure	Participants With Hematologic Malignancies There are three chemotherapy drugs involved. They are called fludarabine (5 doses), cyclophosphamide (1 dose), and thiotepa (2 doses). Also two days of radiation therapy. This is called Total Body Irradiation or TBI. The TBI if given for two days before, the transplant. On transplant day, the cord blood cells will be given through a catheter. The immune suppressing drugs given are called cyclosporine-A (CSA) and mycophenolate mofetil (MMF). These will be started 3 days before the transplant and will be given through the catheter. Later they can be given as tablets.
Overall Study STARTED	86
Overall Study COMPLETED	81
Overall Study NOT COMPLETED	5

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Conditioning Regimen and the Transplantation of Unrelated Donor Umbilical Cord Blood in Patients With Hematologic Malignancies.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Participants With Hematologic Malignancies n=86 Participants There are three chemotherapy drugs involved. They are called fludarabine (5 doses), cyclophosphamide (1 dose), and thiotepa (2 doses). Also two days of radiation therapy. This is called Total Body Irradiation or TBI. The TBI if given for two days before, the transplant. On transplant day, the cord blood cells will be given through a catheter. The immune suppressing drugs given are called cyclosporine-A (CSA) and mycophenolate mofetil (MMF). These will be started 3 days before the transplant and will be given through the catheter. Later they can be given as tablets.
Age, Continuous	51 years n=5 Participants
Sex: Female, Male Female	38 Participants n=5 Participants
Sex: Female, Male Male	48 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	6 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	80 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	13 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	13 Participants n=5 Participants
Race (NIH/OMB) White	53 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	7 Participants n=5 Participants
Region of Enrollment United States	86 Participants n=5 Participants

PRIMARY outcome

Timeframe: 1 year post UCBT

Outcome measures

Outcome measures
Measure	Participants With Hematologic Malignancies n=86 Participants There are three chemotherapy drugs involved. They are called fludarabine (5 doses), cyclophosphamide (1 dose), and thiotepa (2 doses). Also two days of radiation therapy. This is called Total Body Irradiation or TBI. The TBI if given for two days before, the transplant. On transplant day, the cord blood cells will be given through a catheter. The immune suppressing drugs given are called cyclosporine-A (CSA) and mycophenolate mofetil (MMF). These will be started 3 days before the transplant and will be given through the catheter. Later they can be given as tablets.
Progression Free Survival/PFS at 1 Year Post UCBT.	84 percentage of participants Interval 77.0 to 92.0

SECONDARY outcome

Timeframe: up to 13 days from engraftment

Population: 43 haplo-dCBT recipients in this analysis 43 dCBT recipient

Successful primary donor engraftment = neutrophill recovery within the first 45 days after transplant and partial/complete donor chimerism (\>/= 10%)

Outcome measures

Outcome measures
Measure	Participants With Hematologic Malignancies n=86 Participants There are three chemotherapy drugs involved. They are called fludarabine (5 doses), cyclophosphamide (1 dose), and thiotepa (2 doses). Also two days of radiation therapy. This is called Total Body Irradiation or TBI. The TBI if given for two days before, the transplant. On transplant day, the cord blood cells will be given through a catheter. The immune suppressing drugs given are called cyclosporine-A (CSA) and mycophenolate mofetil (MMF). These will be started 3 days before the transplant and will be given through the catheter. Later they can be given as tablets.
Number of Participants With Neutrophil Recovery Post Allograft for Haplo-dCBT Recipients Remaining dCBT recipient	43 participants
Number of Participants With Neutrophil Recovery Post Allograft for Haplo-dCBT Recipients Haplo-dCBT with early haplo-derived myeloid bridge	17 participants
Number of Participants With Neutrophil Recovery Post Allograft for Haplo-dCBT Recipients Haplo-dCBT with transient haplo-derived bridge with second neutrophil nadir	13 participants
Number of Participants With Neutrophil Recovery Post Allograft for Haplo-dCBT Recipients Haplo-dCBT with no bridge	13 participants

SECONDARY outcome

Timeframe: 100 days

The day 100 cumulative incidence of sustained CB-derived neutrophil engraftment.

Outcome measures

Outcome measures
Measure	Participants With Hematologic Malignancies n=86 Participants There are three chemotherapy drugs involved. They are called fludarabine (5 doses), cyclophosphamide (1 dose), and thiotepa (2 doses). Also two days of radiation therapy. This is called Total Body Irradiation or TBI. The TBI if given for two days before, the transplant. On transplant day, the cord blood cells will be given through a catheter. The immune suppressing drugs given are called cyclosporine-A (CSA) and mycophenolate mofetil (MMF). These will be started 3 days before the transplant and will be given through the catheter. Later they can be given as tablets.
Percentage of Participants With Sustained CB-derived Neutrophil Engraftment	99 Percentage of participants Interval 87.0 to 99.0

SECONDARY outcome

Timeframe: 100 days

The day 100 cumulative incidence of sustained CB-derived platelet engraftment to \>/= 20 x 10\^9/L

Outcome measures

Outcome measures
Measure	Participants With Hematologic Malignancies n=86 Participants There are three chemotherapy drugs involved. They are called fludarabine (5 doses), cyclophosphamide (1 dose), and thiotepa (2 doses). Also two days of radiation therapy. This is called Total Body Irradiation or TBI. The TBI if given for two days before, the transplant. On transplant day, the cord blood cells will be given through a catheter. The immune suppressing drugs given are called cyclosporine-A (CSA) and mycophenolate mofetil (MMF). These will be started 3 days before the transplant and will be given through the catheter. Later they can be given as tablets.
Percentage of Participants With Sustained CB-derived Platelet Engraftment	93 Percentage of participants Interval 85.0 to 97.0

Adverse Events

Participants With Hematologic Malignancies

Serious events: 41 serious events

Other events: 47 other events

Deaths: 36 deaths

Serious adverse events

Serious adverse events
Measure	Participants With Hematologic Malignancies n=86 participants at risk There are three chemotherapy drugs involved. They are called fludarabine (5 doses), cyclophosphamide (1 dose), and thiotepa (2 doses). Also two days of radiation therapy. This is called Total Body Irradiation or TBI. The TBI if given for two days before, the transplant. On transplant day, the cord blood cells will be given through a catheter. The immune suppressing drugs given are called cyclosporine-A (CSA) and mycophenolate mofetil (MMF). These will be started 3 days before the transplant and will be given through the catheter. Later they can be given as tablets.
Metabolism and nutrition disorders Anorexia	2.3% 2/86 • Up to 3 years
Respiratory, thoracic and mediastinal disorders Aspiration	1.2% 1/86 • Up to 3 years
Blood and lymphatic system disorders Blood/Bone Marrow, other	1.2% 1/86 • Up to 3 years
Investigations Creatinine	1.2% 1/86 • Up to 3 years
General disorders Death not assoc w CTCAE term- Death NOS	30.2% 26/86 • Up to 3 years
General disorders Death not assoc w CTCAE term- Multi-organ failure	3.5% 3/86 • Up to 3 years
General disorders Death not assoc w CTCAE term- Sudden death	1.2% 1/86 • Up to 3 years
General disorders Death not assoc w CTCAE term-Disease prog NOS	4.7% 4/86 • Up to 3 years
Skin and subcutaneous tissue disorders Dermatology/Skin, other	1.2% 1/86 • Up to 3 years
Gastrointestinal disorders Diarrhea	7.0% 6/86 • Up to 3 years
Respiratory, thoracic and mediastinal disorders Dyspnea (shortness of breath)	1.2% 1/86 • Up to 3 years
Nervous system disorders Encephalopathy	1.2% 1/86 • Up to 3 years
Musculoskeletal and connective tissue disorders Extremity-upper (function)	1.2% 1/86 • Up to 3 years
General disorders Fever (in the absence of neutropenia)	2.3% 2/86 • Up to 3 years
Gastrointestinal disorders Gastrointestinal, other	1.2% 1/86 • Up to 3 years
Investigations Hemoglobin	1.2% 1/86 • Up to 3 years
Blood and lymphatic system disorders Hemolysis	2.3% 2/86 • Up to 3 years
Vascular disorders Hypertension	1.2% 1/86 • Up to 3 years
Vascular disorders Hypotension	2.3% 2/86 • Up to 3 years
Respiratory, thoracic and mediastinal disorders Hypoxia	7.0% 6/86 • Up to 3 years
Infections and infestations Infection w/ Gr 3/4 neut, Blood	1.2% 1/86 • Up to 3 years
Infections and infestations Infection, other	11.6% 10/86 • Up to 3 years
Musculoskeletal and connective tissue disorders Muscle weakness - Whole body/general	1.2% 1/86 • Up to 3 years
Gastrointestinal disorders Nausea	2.3% 2/86 • Up to 3 years
Nervous system disorders Neurology - Other (specify)	1.2% 1/86 • Up to 3 years
Nervous system disorders Neuropathy: motor	1.2% 1/86 • Up to 3 years
Gastrointestinal disorders Pain - Abdomen NOS	1.2% 1/86 • Up to 3 years
Cardiac disorders Pain - Cardiac/heart	1.2% 1/86 • Up to 3 years
Nervous system disorders Pain - Head/headache	1.2% 1/86 • Up to 3 years
Renal and urinary disorders Pain - Urethra	1.2% 1/86 • Up to 3 years
Respiratory, thoracic and mediastinal disorders Pulm/upp respiratory - Other (spec)	3.5% 3/86 • Up to 3 years
Skin and subcutaneous tissue disorders Rash/desquamation	1.2% 1/86 • Up to 3 years
Renal and urinary disorders Renal/Genitourinary-Other Specify	2.3% 2/86 • Up to 3 years
Vascular disorders Thrombosis/thrombus/embolism	2.3% 2/86 • Up to 3 years
Gastrointestinal disorders Vomiting	3.5% 3/86 • Up to 3 years
Investigations Weight loss	1.2% 1/86 • Up to 3 years

Other adverse events

Other adverse events
Measure	Participants With Hematologic Malignancies n=86 participants at risk There are three chemotherapy drugs involved. They are called fludarabine (5 doses), cyclophosphamide (1 dose), and thiotepa (2 doses). Also two days of radiation therapy. This is called Total Body Irradiation or TBI. The TBI if given for two days before, the transplant. On transplant day, the cord blood cells will be given through a catheter. The immune suppressing drugs given are called cyclosporine-A (CSA) and mycophenolate mofetil (MMF). These will be started 3 days before the transplant and will be given through the catheter. Later they can be given as tablets.
Investigations ALT, SGPT	23.3% 20/86 • Up to 3 years
Investigations AST, SGOT	10.5% 9/86 • Up to 3 years
Investigations Alkaline phosphatase	7.0% 6/86 • Up to 3 years
Investigations Bilirubin (hyperbilirubinemia)	43.0% 37/86 • Up to 3 years
Investigations Creatinine	8.1% 7/86 • Up to 3 years
Metabolism and nutrition disorders Glucose, high (hyperglycemia)	54.7% 47/86 • Up to 3 years
Metabolism and nutrition disorders Magnesium, high (hypermagnesemia)	30.2% 26/86 • Up to 3 years

Additional Information

Juliet Barker, MBBS

Memorial Sloan Kettering Cancer Center

Phone: 646-608-3756

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place