Trial Outcomes & Findings for Umbilical Cord Blood Transplantation In Patients With Hematologic Malignancies Using A Myeloablative Preparative Regimen (NCT NCT01328496)

Last Updated: 2017-06-05

Results Overview

Estimate EFS for research participants at one-year post transplant by using single unit umbilical cord blood. The event is defined as relapse, graft failure, death due to any cause. The number of participants who did not experience any of those events (relapse, graft failure, death due to any cause) at year 1 post-transplant was given.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

14 participants

Primary outcome timeframe

1 year post-transplant

Results posted on

2017-06-05

Participant Flow

Fourteen patients were enrolled at St. Jude Children's Research Hospital between September 2011 and April 2014.

Participant milestones

Participant milestones
Measure	Research Arm Participant with high-risk hematologic malignancies undergoing Hematopoietic Cell Transplantation, who do not have a suitable Human Leukocyte Antigen -matched related/sibling donor, Matched Unrelated Donor or Killer immunoglobulin receptors ligand mismatched haploidentical donor identified, will receive a single UCB unit. Intervention: Preparative Regimen Preparative Regimen: Fludarabine (75 mg/m2), fractionated total body irradiation (TBI) (12.0 Gy), and cyclophosphamide (120mg/kg) with mesna. Fludarabine will be given once a day at 25 mg/m2 for three days on day -10 to day -8, TBI will be given twice a day at 150 cGy for four days on day -7 to day -4, and cyclophosphamide will be given once a day for at 60mg/kg for two days on day -3 and day -2. Post-transplantation immunosuppression with cyclosporine and MMF will begin on day -3. Cord Blood infusion will occur on day 0 and G-CSF will start on day +1.	Observational Arm Patients requiring two UCB units will be eligible for UCBT01 on the observational arm. Intervention: Preparative Regimen Preparative Regimen: Fludarabine (75 mg/m2), fractionated total body irradiation (TBI) (12.0 Gy), and cyclophosphamide (120mg/kg) with mesna. Fludarabine will be given once a day at 25 mg/m2 for three days on day -10 to day -8, TBI will be given twice a day at 150 cGy for four days on day -7 to day -4, and cyclophosphamide will be given once a day for at 60mg/kg for two days on day -3 and day -2. Post-transplantation immunosuppression with cyclosporine and MMF will begin on day -3. Cord Blood infusion will occur on day 0 and G-CSF will start on day +1.
Overall Study STARTED	10	4
Overall Study COMPLETED	9	4
Overall Study NOT COMPLETED	1	0

Reasons for withdrawal

Reasons for withdrawal
Measure	Research Arm Participant with high-risk hematologic malignancies undergoing Hematopoietic Cell Transplantation, who do not have a suitable Human Leukocyte Antigen -matched related/sibling donor, Matched Unrelated Donor or Killer immunoglobulin receptors ligand mismatched haploidentical donor identified, will receive a single UCB unit. Intervention: Preparative Regimen Preparative Regimen: Fludarabine (75 mg/m2), fractionated total body irradiation (TBI) (12.0 Gy), and cyclophosphamide (120mg/kg) with mesna. Fludarabine will be given once a day at 25 mg/m2 for three days on day -10 to day -8, TBI will be given twice a day at 150 cGy for four days on day -7 to day -4, and cyclophosphamide will be given once a day for at 60mg/kg for two days on day -3 and day -2. Post-transplantation immunosuppression with cyclosporine and MMF will begin on day -3. Cord Blood infusion will occur on day 0 and G-CSF will start on day +1.	Observational Arm Patients requiring two UCB units will be eligible for UCBT01 on the observational arm. Intervention: Preparative Regimen Preparative Regimen: Fludarabine (75 mg/m2), fractionated total body irradiation (TBI) (12.0 Gy), and cyclophosphamide (120mg/kg) with mesna. Fludarabine will be given once a day at 25 mg/m2 for three days on day -10 to day -8, TBI will be given twice a day at 150 cGy for four days on day -7 to day -4, and cyclophosphamide will be given once a day for at 60mg/kg for two days on day -3 and day -2. Post-transplantation immunosuppression with cyclosporine and MMF will begin on day -3. Cord Blood infusion will occur on day 0 and G-CSF will start on day +1.
Overall Study Significant Change in Health Status	1	0

Baseline Characteristics

Umbilical Cord Blood Transplantation In Patients With Hematologic Malignancies Using A Myeloablative Preparative Regimen

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Research Arm n=9 Participants Participant with high-risk hematologic malignancies undergoing Hematopoietic Cell Transplantation, who do not have a suitable Human Leukocyte Antigen -matched related/sibling donor, Matched Unrelated Donor or Killer immunoglobulin receptors ligand mismatched haploidentical donor identified, will receive a single UCB unit. Intervention: Preparative Regimen Preparative Regimen: Fludarabine (75 mg/m2), fractionated total body irradiation (TBI) (12.0 Gy), and cyclophosphamide (120mg/kg) with mesna. Fludarabine will be given once a day at 25 mg/m2 for three days on day -10 to day -8, TBI will be given twice a day at 150 cGy for four days on day -7 to day -4, and cyclophosphamide will be given once a day for at 60mg/kg for two days on day -3 and day -2. Post-transplantation immunosuppression with cyclosporine and MMF will begin on day -3. Cord Blood infusion will occur on day 0 and G-CSF will start on day +1.	Observational Arm n=4 Participants Patients requiring two UCB units will be eligible for UCBT01 on the observational arm. Intervention: Preparative Regimen Preparative Regimen: Fludarabine (75 mg/m2), fractionated total body irradiation (TBI) (12.0 Gy), and cyclophosphamide (120mg/kg) with mesna. Fludarabine will be given once a day at 25 mg/m2 for three days on day -10 to day -8, TBI will be given twice a day at 150 cGy for four days on day -7 to day -4, and cyclophosphamide will be given once a day for at 60mg/kg for two days on day -3 and day -2. Post-transplantation immunosuppression with cyclosporine and MMF will begin on day -3. Cord Blood infusion will occur on day 0 and G-CSF will start on day +1.	Total n=13 Participants Total of all reporting groups
Age, Continuous	3.40 years STANDARD_DEVIATION 2.54 • n=5 Participants	14.3 years STANDARD_DEVIATION 2.98 • n=7 Participants	6.76 years STANDARD_DEVIATION 5.83 • n=5 Participants
Sex: Female, Male Female	5 Participants n=5 Participants	2 Participants n=7 Participants	7 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants	2 Participants n=7 Participants	6 Participants n=5 Participants
Race/Ethnicity, Customized Mexican/Chicano	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race/Ethnicity, Customized NOS Spanish, Hispanic, latino	2 Participants n=5 Participants	2 Participants n=7 Participants	4 Participants n=5 Participants
Race/Ethnicity, Customized Non Spanish speaking, Non Hispanic	3 Participants n=5 Participants	2 Participants n=7 Participants	5 Participants n=5 Participants
Race/Ethnicity, Customized Puerto Rican	2 Participants n=5 Participants	0 Participants n=7 Participants	2 Participants n=5 Participants
Race/Ethnicity, Customized Declined to respond	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants

PRIMARY outcome

Timeframe: 1 year post-transplant

Outcome measures

Outcome measures
Measure	Research Arm n=9 Participants Participant with high-risk hematologic malignancies undergoing Hematopoietic Cell Transplantation, who do not have a suitable Human Leukocyte Antigen -matched related/sibling donor, Matched Unrelated Donor or Killer immunoglobulin receptors ligand mismatched haploidentical donor identified, will receive a single UCB unit. Intervention: Preparative Regimen Preparative Regimen: Fludarabine (75 mg/m2), fractionated total body irradiation (TBI) (12.0 Gy), and cyclophosphamide (120mg/kg) with mesna. Fludarabine will be given once a day at 25 mg/m2 for three days on day -10 to day -8, TBI will be given twice a day at 150 cGy for four days on day -7 to day -4, and cyclophosphamide will be given once a day for at 60mg/kg for two days on day -3 and day -2. Post-transplantation immunosuppression with cyclosporine and MMF will begin on day -3. Cord Blood infusion will occur on day 0 and G-CSF will start on day +1.	Observational Arm Patients requiring two UCB units will be eligible for UCBT01 on the observational arm. Intervention: Preparative Regimen Preparative Regimen: Fludarabine (75 mg/m2), fractionated total body irradiation (TBI) (12.0 Gy), and cyclophosphamide (120mg/kg) with mesna. Fludarabine will be given once a day at 25 mg/m2 for three days on day -10 to day -8, TBI will be given twice a day at 150 cGy for four days on day -7 to day -4, and cyclophosphamide will be given once a day for at 60mg/kg for two days on day -3 and day -2. Post-transplantation immunosuppression with cyclosporine and MMF will begin on day -3. Cord Blood infusion will occur on day 0 and G-CSF will start on day +1.
Event Free Survival (EFS) for Research Participants	4 Participants	—

SECONDARY outcome

Timeframe: 1 year

For patients enrolled in the observational arm (undergoing a double unit UCBT), the number of patients engrafted was given.

Outcome measures

Outcome measures
Measure	Research Arm n=4 Participants Participant with high-risk hematologic malignancies undergoing Hematopoietic Cell Transplantation, who do not have a suitable Human Leukocyte Antigen -matched related/sibling donor, Matched Unrelated Donor or Killer immunoglobulin receptors ligand mismatched haploidentical donor identified, will receive a single UCB unit. Intervention: Preparative Regimen Preparative Regimen: Fludarabine (75 mg/m2), fractionated total body irradiation (TBI) (12.0 Gy), and cyclophosphamide (120mg/kg) with mesna. Fludarabine will be given once a day at 25 mg/m2 for three days on day -10 to day -8, TBI will be given twice a day at 150 cGy for four days on day -7 to day -4, and cyclophosphamide will be given once a day for at 60mg/kg for two days on day -3 and day -2. Post-transplantation immunosuppression with cyclosporine and MMF will begin on day -3. Cord Blood infusion will occur on day 0 and G-CSF will start on day +1.	Observational Arm Patients requiring two UCB units will be eligible for UCBT01 on the observational arm. Intervention: Preparative Regimen Preparative Regimen: Fludarabine (75 mg/m2), fractionated total body irradiation (TBI) (12.0 Gy), and cyclophosphamide (120mg/kg) with mesna. Fludarabine will be given once a day at 25 mg/m2 for three days on day -10 to day -8, TBI will be given twice a day at 150 cGy for four days on day -7 to day -4, and cyclophosphamide will be given once a day for at 60mg/kg for two days on day -3 and day -2. Post-transplantation immunosuppression with cyclosporine and MMF will begin on day -3. Cord Blood infusion will occur on day 0 and G-CSF will start on day +1.
Number of Observational Arm Participants Engrafted	3 Participants	—

SECONDARY outcome

Timeframe: 1 year

The number of observational arm patients who relapsed was given.

Outcome measures

Outcome measures
Measure	Research Arm n=4 Participants Participant with high-risk hematologic malignancies undergoing Hematopoietic Cell Transplantation, who do not have a suitable Human Leukocyte Antigen -matched related/sibling donor, Matched Unrelated Donor or Killer immunoglobulin receptors ligand mismatched haploidentical donor identified, will receive a single UCB unit. Intervention: Preparative Regimen Preparative Regimen: Fludarabine (75 mg/m2), fractionated total body irradiation (TBI) (12.0 Gy), and cyclophosphamide (120mg/kg) with mesna. Fludarabine will be given once a day at 25 mg/m2 for three days on day -10 to day -8, TBI will be given twice a day at 150 cGy for four days on day -7 to day -4, and cyclophosphamide will be given once a day for at 60mg/kg for two days on day -3 and day -2. Post-transplantation immunosuppression with cyclosporine and MMF will begin on day -3. Cord Blood infusion will occur on day 0 and G-CSF will start on day +1.	Observational Arm Patients requiring two UCB units will be eligible for UCBT01 on the observational arm. Intervention: Preparative Regimen Preparative Regimen: Fludarabine (75 mg/m2), fractionated total body irradiation (TBI) (12.0 Gy), and cyclophosphamide (120mg/kg) with mesna. Fludarabine will be given once a day at 25 mg/m2 for three days on day -10 to day -8, TBI will be given twice a day at 150 cGy for four days on day -7 to day -4, and cyclophosphamide will be given once a day for at 60mg/kg for two days on day -3 and day -2. Post-transplantation immunosuppression with cyclosporine and MMF will begin on day -3. Cord Blood infusion will occur on day 0 and G-CSF will start on day +1.
Number of Observational Arm Patients Who Relapsed	1 Participants	—

SECONDARY outcome

Timeframe: 1 year

The number of observational arm patients who died was given.

Outcome measures

Outcome measures
Measure	Research Arm n=4 Participants Participant with high-risk hematologic malignancies undergoing Hematopoietic Cell Transplantation, who do not have a suitable Human Leukocyte Antigen -matched related/sibling donor, Matched Unrelated Donor or Killer immunoglobulin receptors ligand mismatched haploidentical donor identified, will receive a single UCB unit. Intervention: Preparative Regimen Preparative Regimen: Fludarabine (75 mg/m2), fractionated total body irradiation (TBI) (12.0 Gy), and cyclophosphamide (120mg/kg) with mesna. Fludarabine will be given once a day at 25 mg/m2 for three days on day -10 to day -8, TBI will be given twice a day at 150 cGy for four days on day -7 to day -4, and cyclophosphamide will be given once a day for at 60mg/kg for two days on day -3 and day -2. Post-transplantation immunosuppression with cyclosporine and MMF will begin on day -3. Cord Blood infusion will occur on day 0 and G-CSF will start on day +1.	Observational Arm Patients requiring two UCB units will be eligible for UCBT01 on the observational arm. Intervention: Preparative Regimen Preparative Regimen: Fludarabine (75 mg/m2), fractionated total body irradiation (TBI) (12.0 Gy), and cyclophosphamide (120mg/kg) with mesna. Fludarabine will be given once a day at 25 mg/m2 for three days on day -10 to day -8, TBI will be given twice a day at 150 cGy for four days on day -7 to day -4, and cyclophosphamide will be given once a day for at 60mg/kg for two days on day -3 and day -2. Post-transplantation immunosuppression with cyclosporine and MMF will begin on day -3. Cord Blood infusion will occur on day 0 and G-CSF will start on day +1.
Number of Deaths of Observational Arm Patients	2 Participants	—

SECONDARY outcome

Timeframe: First 100 days

The number of patients with TRM within the first 100 days post transplant was given.

Outcome measures

Outcome measures
Measure	Research Arm n=4 Participants Participant with high-risk hematologic malignancies undergoing Hematopoietic Cell Transplantation, who do not have a suitable Human Leukocyte Antigen -matched related/sibling donor, Matched Unrelated Donor or Killer immunoglobulin receptors ligand mismatched haploidentical donor identified, will receive a single UCB unit. Intervention: Preparative Regimen Preparative Regimen: Fludarabine (75 mg/m2), fractionated total body irradiation (TBI) (12.0 Gy), and cyclophosphamide (120mg/kg) with mesna. Fludarabine will be given once a day at 25 mg/m2 for three days on day -10 to day -8, TBI will be given twice a day at 150 cGy for four days on day -7 to day -4, and cyclophosphamide will be given once a day for at 60mg/kg for two days on day -3 and day -2. Post-transplantation immunosuppression with cyclosporine and MMF will begin on day -3. Cord Blood infusion will occur on day 0 and G-CSF will start on day +1.	Observational Arm Patients requiring two UCB units will be eligible for UCBT01 on the observational arm. Intervention: Preparative Regimen Preparative Regimen: Fludarabine (75 mg/m2), fractionated total body irradiation (TBI) (12.0 Gy), and cyclophosphamide (120mg/kg) with mesna. Fludarabine will be given once a day at 25 mg/m2 for three days on day -10 to day -8, TBI will be given twice a day at 150 cGy for four days on day -7 to day -4, and cyclophosphamide will be given once a day for at 60mg/kg for two days on day -3 and day -2. Post-transplantation immunosuppression with cyclosporine and MMF will begin on day -3. Cord Blood infusion will occur on day 0 and G-CSF will start on day +1.
Number of Observational Arm Patients With Transplant-related Mortality (TRM)	1 Participants	—

SECONDARY outcome

Timeframe: 1 year

The number of participants with incidence of acute GVHD by grade was given. Participants are graded on a scale from 1 to 4, with 1 being mild and 4 being severe.

Outcome measures

Outcome measures
Measure	Research Arm n=9 Participants Participant with high-risk hematologic malignancies undergoing Hematopoietic Cell Transplantation, who do not have a suitable Human Leukocyte Antigen -matched related/sibling donor, Matched Unrelated Donor or Killer immunoglobulin receptors ligand mismatched haploidentical donor identified, will receive a single UCB unit. Intervention: Preparative Regimen Preparative Regimen: Fludarabine (75 mg/m2), fractionated total body irradiation (TBI) (12.0 Gy), and cyclophosphamide (120mg/kg) with mesna. Fludarabine will be given once a day at 25 mg/m2 for three days on day -10 to day -8, TBI will be given twice a day at 150 cGy for four days on day -7 to day -4, and cyclophosphamide will be given once a day for at 60mg/kg for two days on day -3 and day -2. Post-transplantation immunosuppression with cyclosporine and MMF will begin on day -3. Cord Blood infusion will occur on day 0 and G-CSF will start on day +1.	Observational Arm n=4 Participants Patients requiring two UCB units will be eligible for UCBT01 on the observational arm. Intervention: Preparative Regimen Preparative Regimen: Fludarabine (75 mg/m2), fractionated total body irradiation (TBI) (12.0 Gy), and cyclophosphamide (120mg/kg) with mesna. Fludarabine will be given once a day at 25 mg/m2 for three days on day -10 to day -8, TBI will be given twice a day at 150 cGy for four days on day -7 to day -4, and cyclophosphamide will be given once a day for at 60mg/kg for two days on day -3 and day -2. Post-transplantation immunosuppression with cyclosporine and MMF will begin on day -3. Cord Blood infusion will occur on day 0 and G-CSF will start on day +1.
Number of Participants With Acute GVHD Grade 1	2 Participants	0 Participants
Number of Participants With Acute GVHD Grade 2	1 Participants	0 Participants
Number of Participants With Acute GVHD Grade 3	1 Participants	1 Participants
Number of Participants With Acute GVHD Grade 4	0 Participants	0 Participants
Number of Participants With Acute GVHD No Acute GVHD	5 Participants	3 Participants

SECONDARY outcome

Timeframe: 1 year

Due to the small sample size, cumulative incidence analysis was not done. The incidence of chronic GVHD was evaluated using NIH Consensus Global Severity Scoring. The number of patients with incidence of chronic GVHD by severity was provided.

Outcome measures

Outcome measures
Measure	Research Arm n=9 Participants Participant with high-risk hematologic malignancies undergoing Hematopoietic Cell Transplantation, who do not have a suitable Human Leukocyte Antigen -matched related/sibling donor, Matched Unrelated Donor or Killer immunoglobulin receptors ligand mismatched haploidentical donor identified, will receive a single UCB unit. Intervention: Preparative Regimen Preparative Regimen: Fludarabine (75 mg/m2), fractionated total body irradiation (TBI) (12.0 Gy), and cyclophosphamide (120mg/kg) with mesna. Fludarabine will be given once a day at 25 mg/m2 for three days on day -10 to day -8, TBI will be given twice a day at 150 cGy for four days on day -7 to day -4, and cyclophosphamide will be given once a day for at 60mg/kg for two days on day -3 and day -2. Post-transplantation immunosuppression with cyclosporine and MMF will begin on day -3. Cord Blood infusion will occur on day 0 and G-CSF will start on day +1.	Observational Arm n=4 Participants Patients requiring two UCB units will be eligible for UCBT01 on the observational arm. Intervention: Preparative Regimen Preparative Regimen: Fludarabine (75 mg/m2), fractionated total body irradiation (TBI) (12.0 Gy), and cyclophosphamide (120mg/kg) with mesna. Fludarabine will be given once a day at 25 mg/m2 for three days on day -10 to day -8, TBI will be given twice a day at 150 cGy for four days on day -7 to day -4, and cyclophosphamide will be given once a day for at 60mg/kg for two days on day -3 and day -2. Post-transplantation immunosuppression with cyclosporine and MMF will begin on day -3. Cord Blood infusion will occur on day 0 and G-CSF will start on day +1.
Number of Participants With Chronic GVHD Mild	0 Participants	0 Participants
Number of Participants With Chronic GVHD Moderate	0 Participants	0 Participants
Number of Participants With Chronic GVHD Severe	0 Participants	0 Participants
Number of Participants With Chronic GVHD No Chronic GVHD	9 Participants	4 Participants

SECONDARY outcome

Timeframe: first 100 days post transplant

Population: Those patients who did not reach engraftment are not included in the results provided below.

Platelet engraftment was defined as platelet count ≥20,000/mm\^3 for 3 consecutive tests performed on different days with no platelet transfusions in the preceding 7 days. Neutrophil engraftment will be defined as achieving ANC ≥ 500/mm3 for 3 consecutive tests performed on different days with evidence of donor cell engraftment. Descriptive statistics are provided.

Outcome measures

Outcome measures
Measure	Research Arm n=9 Participants Participant with high-risk hematologic malignancies undergoing Hematopoietic Cell Transplantation, who do not have a suitable Human Leukocyte Antigen -matched related/sibling donor, Matched Unrelated Donor or Killer immunoglobulin receptors ligand mismatched haploidentical donor identified, will receive a single UCB unit. Intervention: Preparative Regimen Preparative Regimen: Fludarabine (75 mg/m2), fractionated total body irradiation (TBI) (12.0 Gy), and cyclophosphamide (120mg/kg) with mesna. Fludarabine will be given once a day at 25 mg/m2 for three days on day -10 to day -8, TBI will be given twice a day at 150 cGy for four days on day -7 to day -4, and cyclophosphamide will be given once a day for at 60mg/kg for two days on day -3 and day -2. Post-transplantation immunosuppression with cyclosporine and MMF will begin on day -3. Cord Blood infusion will occur on day 0 and G-CSF will start on day +1.	Observational Arm Patients requiring two UCB units will be eligible for UCBT01 on the observational arm. Intervention: Preparative Regimen Preparative Regimen: Fludarabine (75 mg/m2), fractionated total body irradiation (TBI) (12.0 Gy), and cyclophosphamide (120mg/kg) with mesna. Fludarabine will be given once a day at 25 mg/m2 for three days on day -10 to day -8, TBI will be given twice a day at 150 cGy for four days on day -7 to day -4, and cyclophosphamide will be given once a day for at 60mg/kg for two days on day -3 and day -2. Post-transplantation immunosuppression with cyclosporine and MMF will begin on day -3. Cord Blood infusion will occur on day 0 and G-CSF will start on day +1.
Time to Engraftment of Research Arm Participants Days to Platelets ≥20,000	42.67 Days Standard Deviation 8.80	—
Time to Engraftment of Research Arm Participants Days to ANC ≥500	17.11 Days Standard Deviation 5.93	—

SECONDARY outcome

Timeframe: first 100 days post transplant

TRM is death occurring in patients in continuous complete remission. The numbers of patients with TRM was given.

Outcome measures

Outcome measures
Measure	Research Arm n=9 Participants Participant with high-risk hematologic malignancies undergoing Hematopoietic Cell Transplantation, who do not have a suitable Human Leukocyte Antigen -matched related/sibling donor, Matched Unrelated Donor or Killer immunoglobulin receptors ligand mismatched haploidentical donor identified, will receive a single UCB unit. Intervention: Preparative Regimen Preparative Regimen: Fludarabine (75 mg/m2), fractionated total body irradiation (TBI) (12.0 Gy), and cyclophosphamide (120mg/kg) with mesna. Fludarabine will be given once a day at 25 mg/m2 for three days on day -10 to day -8, TBI will be given twice a day at 150 cGy for four days on day -7 to day -4, and cyclophosphamide will be given once a day for at 60mg/kg for two days on day -3 and day -2. Post-transplantation immunosuppression with cyclosporine and MMF will begin on day -3. Cord Blood infusion will occur on day 0 and G-CSF will start on day +1.	Observational Arm n=4 Participants Patients requiring two UCB units will be eligible for UCBT01 on the observational arm. Intervention: Preparative Regimen Preparative Regimen: Fludarabine (75 mg/m2), fractionated total body irradiation (TBI) (12.0 Gy), and cyclophosphamide (120mg/kg) with mesna. Fludarabine will be given once a day at 25 mg/m2 for three days on day -10 to day -8, TBI will be given twice a day at 150 cGy for four days on day -7 to day -4, and cyclophosphamide will be given once a day for at 60mg/kg for two days on day -3 and day -2. Post-transplantation immunosuppression with cyclosporine and MMF will begin on day -3. Cord Blood infusion will occur on day 0 and G-CSF will start on day +1.
Incidence of Transplant-related Mortality (TRM)	0 Participants	1 Participants

SECONDARY outcome

Timeframe: first 100 days post transplant

Any patient who had adverse events listed either as probable or definite in the first 100 days post-transplant are counted as transplant related morbidity. The number of patients with transplant-related morbidity was given.

Outcome measures

Outcome measures
Measure	Research Arm n=9 Participants Participant with high-risk hematologic malignancies undergoing Hematopoietic Cell Transplantation, who do not have a suitable Human Leukocyte Antigen -matched related/sibling donor, Matched Unrelated Donor or Killer immunoglobulin receptors ligand mismatched haploidentical donor identified, will receive a single UCB unit. Intervention: Preparative Regimen Preparative Regimen: Fludarabine (75 mg/m2), fractionated total body irradiation (TBI) (12.0 Gy), and cyclophosphamide (120mg/kg) with mesna. Fludarabine will be given once a day at 25 mg/m2 for three days on day -10 to day -8, TBI will be given twice a day at 150 cGy for four days on day -7 to day -4, and cyclophosphamide will be given once a day for at 60mg/kg for two days on day -3 and day -2. Post-transplantation immunosuppression with cyclosporine and MMF will begin on day -3. Cord Blood infusion will occur on day 0 and G-CSF will start on day +1.	Observational Arm n=4 Participants Patients requiring two UCB units will be eligible for UCBT01 on the observational arm. Intervention: Preparative Regimen Preparative Regimen: Fludarabine (75 mg/m2), fractionated total body irradiation (TBI) (12.0 Gy), and cyclophosphamide (120mg/kg) with mesna. Fludarabine will be given once a day at 25 mg/m2 for three days on day -10 to day -8, TBI will be given twice a day at 150 cGy for four days on day -7 to day -4, and cyclophosphamide will be given once a day for at 60mg/kg for two days on day -3 and day -2. Post-transplantation immunosuppression with cyclosporine and MMF will begin on day -3. Cord Blood infusion will occur on day 0 and G-CSF will start on day +1.
The Number of Participants With Transplant-related Morbidity	8 Participants	3 Participants

Adverse Events

Research Arm

Serious events: 7 serious events

Other events: 9 other events

Deaths: 4 deaths

Observation Arm

Serious events: 3 serious events

Other events: 4 other events

Deaths: 2 deaths

Serious adverse events

Other adverse events

Additional Information

Amr Qudeimat, MD

St. Jude Children's Research Hospital

Phone: 901-595-8342

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	Research Arm n=9 participants at risk Participant with high-risk hematologic malignancies undergoing Hematopoietic Cell Transplantation, who do not have a suitable Human Leukocyte Antigen -matched related/sibling donor, Matched Unrelated Donor or Killer immunoglobulin receptors ligand mismatched haploidentical donor identified, will receive a single UCB unit. Intervention: Preparative Regimen Preparative Regimen: Fludarabine (75 mg/m2), fractionated total body irradiation (TBI) (12.0 Gy), and cyclophosphamide (120mg/kg) with mesna. Fludarabine will be given once a day at 25 mg/m2 for three days on day -10 to day -8, TBI will be given twice a day at 150 cGy for four days on day -7 to day -4, and cyclophosphamide will be given once a day for at 60mg/kg for two days on day -3 and day -2. Post-transplantation immunosuppression with cyclosporine and MMF will begin on day -3. Cord Blood infusion will occur on day 0 and G-CSF will start on day +1.	Observation Arm n=4 participants at risk Patients requiring two UCB units will be eligible for UCBT01 on the observational arm. Intervention: Preparative Regimen Preparative Regimen: Fludarabine (75 mg/m2), fractionated total body irradiation (TBI) (12.0 Gy), and cyclophosphamide (120mg/kg) with mesna. Fludarabine will be given once a day at 25 mg/m2 for three days on day -10 to day -8, TBI will be given twice a day at 150 cGy for four days on day -7 to day -4, and cyclophosphamide will be given once a day for at 60mg/kg for two days on day -3 and day -2. Post-transplantation immunosuppression with cyclosporine and MMF will begin on day -3. Cord Blood infusion will occur on day 0 and G-CSF will start on day +1.
Blood and lymphatic system disorders Anemia, Hemolytic	11.1% 1/9 • Number of events 1 • Adverse events were collected throughout from the start of initiation of conditioning to one year post transplant.	0.00% 0/4 • Adverse events were collected throughout from the start of initiation of conditioning to one year post transplant.
Cardiac disorders Effusion, Pericardial	11.1% 1/9 • Number of events 1 • Adverse events were collected throughout from the start of initiation of conditioning to one year post transplant.	0.00% 0/4 • Adverse events were collected throughout from the start of initiation of conditioning to one year post transplant.
Cardiac disorders Pericardial effusion (disorder)	11.1% 1/9 • Number of events 1 • Adverse events were collected throughout from the start of initiation of conditioning to one year post transplant.	0.00% 0/4 • Adverse events were collected throughout from the start of initiation of conditioning to one year post transplant.
General disorders Fever without neutropenia	55.6% 5/9 • Number of events 14 • Adverse events were collected throughout from the start of initiation of conditioning to one year post transplant.	75.0% 3/4 • Number of events 3 • Adverse events were collected throughout from the start of initiation of conditioning to one year post transplant.
Gastrointestinal disorders Dehydration (disorder	11.1% 1/9 • Number of events 1 • Adverse events were collected throughout from the start of initiation of conditioning to one year post transplant.	0.00% 0/4 • Adverse events were collected throughout from the start of initiation of conditioning to one year post transplant.
Gastrointestinal disorders Pneumatosis intestinalis	11.1% 1/9 • Number of events 1 • Adverse events were collected throughout from the start of initiation of conditioning to one year post transplant.	0.00% 0/4 • Adverse events were collected throughout from the start of initiation of conditioning to one year post transplant.
Blood and lymphatic system disorders Febrile neutropenia	22.2% 2/9 • Number of events 2 • Adverse events were collected throughout from the start of initiation of conditioning to one year post transplant.	0.00% 0/4 • Adverse events were collected throughout from the start of initiation of conditioning to one year post transplant.
Infections and infestations Infection, coagulase negative staph, blood	11.1% 1/9 • Number of events 1 • Adverse events were collected throughout from the start of initiation of conditioning to one year post transplant.	0.00% 0/4 • Adverse events were collected throughout from the start of initiation of conditioning to one year post transplant.
Infections and infestations Infection, enterococcus faecalis, blood	11.1% 1/9 • Number of events 1 • Adverse events were collected throughout from the start of initiation of conditioning to one year post transplant.	0.00% 0/4 • Adverse events were collected throughout from the start of initiation of conditioning to one year post transplant.
Infections and infestations Infection, escherichia coli, blood	11.1% 1/9 • Number of events 2 • Adverse events were collected throughout from the start of initiation of conditioning to one year post transplant.	0.00% 0/4 • Adverse events were collected throughout from the start of initiation of conditioning to one year post transplant.
Infections and infestations Infection, escherichia coli, urine	11.1% 1/9 • Number of events 1 • Adverse events were collected throughout from the start of initiation of conditioning to one year post transplant.	0.00% 0/4 • Adverse events were collected throughout from the start of initiation of conditioning to one year post transplant.
Metabolism and nutrition disorders Hyperkalemia (disorder)	11.1% 1/9 • Number of events 1 • Adverse events were collected throughout from the start of initiation of conditioning to one year post transplant.	0.00% 0/4 • Adverse events were collected throughout from the start of initiation of conditioning to one year post transplant.
Respiratory, thoracic and mediastinal disorders Acute respiratory distress (finding)	11.1% 1/9 • Number of events 1 • Adverse events were collected throughout from the start of initiation of conditioning to one year post transplant.	0.00% 0/4 • Adverse events were collected throughout from the start of initiation of conditioning to one year post transplant.
Respiratory, thoracic and mediastinal disorders Adult respiratory distress syndrome	11.1% 1/9 • Number of events 1 • Adverse events were collected throughout from the start of initiation of conditioning to one year post transplant.	0.00% 0/4 • Adverse events were collected throughout from the start of initiation of conditioning to one year post transplant.
Respiratory, thoracic and mediastinal disorders Left pneumothorax (disorder)	11.1% 1/9 • Number of events 1 • Adverse events were collected throughout from the start of initiation of conditioning to one year post transplant.	0.00% 0/4 • Adverse events were collected throughout from the start of initiation of conditioning to one year post transplant.
Respiratory, thoracic and mediastinal disorders Right pneumothorax (disorder)	11.1% 1/9 • Number of events 2 • Adverse events were collected throughout from the start of initiation of conditioning to one year post transplant.	0.00% 0/4 • Adverse events were collected throughout from the start of initiation of conditioning to one year post transplant.
Respiratory, thoracic and mediastinal disorders Upper airway obstruction	11.1% 1/9 • Number of events 1 • Adverse events were collected throughout from the start of initiation of conditioning to one year post transplant.	0.00% 0/4 • Adverse events were collected throughout from the start of initiation of conditioning to one year post transplant.
Respiratory, thoracic and mediastinal disorders Effusion, pericardial	0.00% 0/9 • Adverse events were collected throughout from the start of initiation of conditioning to one year post transplant.	25.0% 1/4 • Number of events 1 • Adverse events were collected throughout from the start of initiation of conditioning to one year post transplant.
Respiratory, thoracic and mediastinal disorders Pneumonia	0.00% 0/9 • Adverse events were collected throughout from the start of initiation of conditioning to one year post transplant.	25.0% 1/4 • Number of events 1 • Adverse events were collected throughout from the start of initiation of conditioning to one year post transplant.
Renal and urinary disorders Renal insufficiency	11.1% 1/9 • Number of events 1 • Adverse events were collected throughout from the start of initiation of conditioning to one year post transplant.	0.00% 0/4 • Adverse events were collected throughout from the start of initiation of conditioning to one year post transplant.
Nervous system disorders Posterior reversible encephalopathy syndrome (PRES)	11.1% 1/9 • Number of events 1 • Adverse events were collected throughout from the start of initiation of conditioning to one year post transplant.	0.00% 0/4 • Adverse events were collected throughout from the start of initiation of conditioning to one year post transplant.