Trial Outcomes & Findings for Evaluating the Safety and Effectiveness of an Umbilical Cord Blood Stem Cell Transplant (BMT CTN 0604) (NCT NCT00864227)

NCT ID: NCT00864227

Last Updated: 2022-12-14

Results Overview

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 54 participants
• Primary outcome timeframe: Measured at Month 6 and Year 1
• Results posted on: 2022-12-14

Participant Flow

Participant milestones

Measure	dUCB Transplant Hematopoietic Umbilical Cord Blood Stem Cell Transplantation using a non-myeloablative preparative regimen.
Overall Study STARTED	54
Overall Study COMPLETED	50
Overall Study NOT COMPLETED	4

Reasons for withdrawal

Measure	dUCB Transplant Hematopoietic Umbilical Cord Blood Stem Cell Transplantation using a non-myeloablative preparative regimen.
Overall Study Withdrawal by Subject	1
Overall Study Disease Relapse/Progression	3

Baseline Characteristics

Evaluating the Safety and Effectiveness of an Umbilical Cord Blood Stem Cell Transplant (BMT CTN 0604)

Baseline characteristics by cohort

Measure	dUCB Transplant n=50 Participants Hematopoietic Umbilical Cord Blood Stem Cell Transplantation using a non-myeloablative preparative regimen.
Age, Continuous	53.4 years STANDARD_DEVIATION 13.6 • n=113 Participants
Sex: Female, Male Female	24 Participants n=113 Participants
Sex: Female, Male Male	26 Participants n=113 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=113 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	46 Participants n=113 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	2 Participants n=113 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=113 Participants
Race (NIH/OMB) Asian	4 Participants n=113 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=113 Participants
Race (NIH/OMB) Black or African American	1 Participants n=113 Participants
Race (NIH/OMB) White	45 Participants n=113 Participants
Race (NIH/OMB) More than one race	0 Participants n=113 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=113 Participants
Karnofsky Performance-status Score 100%	10 participants n=113 Participants
Karnofsky Performance-status Score 90%	30 participants n=113 Participants
Karnofsky Performance-status Score 80%	7 participants n=113 Participants
Karnofsky Performance-status Score 70%	2 participants n=113 Participants
Karnofsky Performance-status Score 60%	1 participants n=113 Participants
Primary Disease Acute Lymphoblastic Leukemia	6 participants n=113 Participants
Primary Disease Acute Myelogeneous Leukemia	29 participants n=113 Participants
Primary Disease Biphenotypic/Undifferentiated Leukemia	1 participants n=113 Participants
Primary Disease Burkitt's Lymphoma	1 participants n=113 Participants
Primary Disease Hodgkins Lymphoma	5 participants n=113 Participants
Primary Disease Large Cell Lymphoma	3 participants n=113 Participants
Primary Disease Marginal Zone B-cell Lymphoma	1 participants n=113 Participants
Primary Disease Follicular Non-Hodgkins Lymphoma	4 participants n=113 Participants
HLA Typing Match Score - 1st Cord 4/6	24 participants n=113 Participants
HLA Typing Match Score - 1st Cord 5/6	21 participants n=113 Participants
HLA Typing Match Score - 1st Cord 6/6	5 participants n=113 Participants
HLA Typing Match Score - 2nd Cord 4/6	30 participants n=113 Participants
HLA Typing Match Score - 2nd Cord 5/6	17 participants n=113 Participants
HLA Typing Match Score - 2nd Cord 6/6	3 participants n=113 Participants
HLA Typing Match Score - 1st Cord to 2nd Cord 4/6	35 participants n=113 Participants
HLA Typing Match Score - 1st Cord to 2nd Cord 5/6	11 participants n=113 Participants
HLA Typing Match Score - 1st Cord to 2nd Cord 6/6	4 participants n=113 Participants

PRIMARY outcome

Timeframe: Measured at Month 6 and Year 1

Outcome measures

Measure	dUCB Transplant n=50 Participants Hematopoietic Umbilical Cord Blood Stem Cell Transplantation using a non-myeloablative preparative regimen.
Overall Survival at 180 Days From the Time of Transplant 6 months	73.5 percentage of participants Interval 58.8 to 83.7
Overall Survival at 180 Days From the Time of Transplant 1 year	54.0 percentage of participants Interval 38.3 to 67.3

SECONDARY outcome

Timeframe: Measured at Days 28, 56, 90, and 100

Neutrophil recovery is defined as achieving an absolute neutrophil count ≥ 500/mm3 for three consecutive measurements on different days.

Outcome measures

Measure	dUCB Transplant n=50 Participants Hematopoietic Umbilical Cord Blood Stem Cell Transplantation using a non-myeloablative preparative regimen.
Neutrophil Recovery Day 100	94.0 percentage of participants Interval 86.6 to 100.0
Neutrophil Recovery Day 28	86.0 percentage of participants Interval 76.0 to 96.0
Neutrophil Recovery Day 56	94.0 percentage of participants Interval 86.6 to 100.0
Neutrophil Recovery Day 90	94.0 percentage of participants Interval 86.6 to 100.0

SECONDARY outcome

Timeframe: Measured at Day 100

Primary graft failure is defined as < 5% donor chimerism on all measurements prior to and day-100.

Outcome measures

Measure	dUCB Transplant n=50 Participants Hematopoietic Umbilical Cord Blood Stem Cell Transplantation using a non-myeloablative preparative regimen.
Primary Graft Failure	5 participants

SECONDARY outcome

Timeframe: Measured at Day 100

Secondary graft failure is defined initial recovery followed by neutropenia with < 5% donor chimerism.

Outcome measures

Measure	dUCB Transplant n=50 Participants Hematopoietic Umbilical Cord Blood Stem Cell Transplantation using a non-myeloablative preparative regimen.
Secondary Graft Failure	1 participants

SECONDARY outcome

Timeframe: Measured at Days 56, 90, and 100

Platelet recovery is defined as the first day of a minimum of three consecutive measurements on different days such that the patient has achieved a platelet count >20,000/mm3 with no platelet transfusions in the preceding seven days.

Outcome measures

Measure	dUCB Transplant n=50 Participants Hematopoietic Umbilical Cord Blood Stem Cell Transplantation using a non-myeloablative preparative regimen.
Platelet Recovery to 20K Day 56	70.0 percentage of participants Interval 57.1 to 82.9
Platelet Recovery to 20K Day 90	82.0 percentage of participants Interval 71.0 to 93.0
Platelet Recovery to 20K Day 100	82.0 percentage of participants Interval 71.0 to 93.0

SECONDARY outcome

Timeframe: Measured at Day 56

Marrow or Blood Sample. Donor cell engraftment is defined as donor chimerism ≥ 5% on Day ≥ 56 after transplantation. Chimerism should be evaluated on Days ~28, ~56, ~180, and ~365 after transplantation. Chimerism may be evaluated in whole blood or mononuclear fraction.

Outcome measures

Measure	dUCB Transplant n=50 Participants Hematopoietic Umbilical Cord Blood Stem Cell Transplantation using a non-myeloablative preparative regimen.
Donor Cell Engraftment Chimerism Performed	36 participants
Donor Cell Engraftment Donor Percentage >=95%	30 participants
Donor Cell Engraftment Donor Percentage 5%-95%	6 participants
Donor Cell Engraftment Donor Percentage <5%	0 participants

SECONDARY outcome

Timeframe: Measured at Day 100

Outcome measures

Measure	dUCB Transplant n=50 Participants Hematopoietic Umbilical Cord Blood Stem Cell Transplantation using a non-myeloablative preparative regimen.
Acute Graft-versus-host Disease (GVHD) Grade II-IV Acute GVHD	40.0 percentage of participants Interval 26.3 to 53.7
Acute Graft-versus-host Disease (GVHD) Grade III-IV Acute GVHD	21.4 percentage of participants Interval 5.9 to 36.9

SECONDARY outcome

Timeframe: Measured at Year 1

Outcome measures

Measure	dUCB Transplant n=50 Participants Hematopoietic Umbilical Cord Blood Stem Cell Transplantation using a non-myeloablative preparative regimen.
Chronic GVHD	25.3 percentage of participants Interval 11.8 to 38.8

SECONDARY outcome

Timeframe: Measured at Year 1

Progression-free survival is defined as the minimum time interval to relapse/ recurrence/progression, to death or to last follow-up.

Outcome measures

Measure	dUCB Transplant n=50 Participants Hematopoietic Umbilical Cord Blood Stem Cell Transplantation using a non-myeloablative preparative regimen.
Progression-free Survival	45.7 percentage of participants Interval 30.7 to 59.6

SECONDARY outcome

Timeframe: Measured at 6 months and 1 year

Outcome measures

Measure	dUCB Transplant n=50 Participants Hematopoietic Umbilical Cord Blood Stem Cell Transplantation using a non-myeloablative preparative regimen.
Treatment-related Mortality (TRM) 6 months	16.3 percentage of participants Interval 5.9 to 26.7
Treatment-related Mortality (TRM) 1 year	23.8 percentage of participants Interval 11.3 to 36.3

SECONDARY outcome

Timeframe: Measured at Year 1

Number of participants that experienced at least one infection.

Outcome measures

Measure	dUCB Transplant n=50 Participants Hematopoietic Umbilical Cord Blood Stem Cell Transplantation using a non-myeloablative preparative regimen.
Incidence of Infections 1 Infection	11 Infections
Incidence of Infections 2 Infections	11 Infections
Incidence of Infections 4 Infections	4 Infections
Incidence of Infections 5 Infections	3 Infections
Incidence of Infections 6-10 Infections	8 Infections
Incidence of Infections >10 Infections	1 Infections
Incidence of Infections 3 Infections	3 Infections

SECONDARY outcome

Timeframe: Measured at Days 56, 90, and 100

Platelet recovery is defined as the first day of a minimum of three consecutive measurements on different days such that the patient has achieved a platelet count >50,000/mm3 with no platelet transfusions in the preceding seven days.

Outcome measures

Measure	dUCB Transplant n=50 Participants Hematopoietic Umbilical Cord Blood Stem Cell Transplantation using a non-myeloablative preparative regimen.
Platelet Recovery to 50K Day 56	46.0 percentage of participants Interval 31.9 to 60.1
Platelet Recovery to 50K Day 90	58.5 percentage of participants Interval 44.4 to 72.6
Platelet Recovery to 50K Day 100	58.5 percentage of participants Interval 44.4 to 72.6

Adverse Events

dUCB Transplant

Serious events: 9 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Measure	dUCB Transplant n=50 participants at risk Hematopoietic Umbilical Cord Blood Stem Cell Transplantation using a non-myeloablative preparative regimen.
Cardiac disorders Cardiac failure congestive	2.0% 1/50 • Number of events 1 • 1-year post-transplant Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.
Cardiac disorders Left ventricular dysfunction	4.0% 2/50 • Number of events 2 • 1-year post-transplant Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.
General disorders Chest pain	2.0% 1/50 • Number of events 1 • 1-year post-transplant Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.
Hepatobiliary disorders Gallbladder obstruction	2.0% 1/50 • Number of events 1 • 1-year post-transplant Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.
Infections and infestations Infection	2.0% 1/50 • Number of events 1 • 1-year post-transplant Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.
Metabolism and nutrition disorders Hypercalcaemia	2.0% 1/50 • Number of events 1 • 1-year post-transplant Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.
Metabolism and nutrition disorders Hypokalaemia	2.0% 1/50 • Number of events 1 • 1-year post-transplant Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Bladder cancer	2.0% 1/50 • Number of events 1 • 1-year post-transplant Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.
Nervous system disorders Toxic encephalopathy	2.0% 1/50 • Number of events 1 • 1-year post-transplant Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.
Psychiatric disorders Delirium	2.0% 1/50 • Number of events 1 • 1-year post-transplant Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.
Renal and urinary disorders Renal failure	4.0% 2/50 • Number of events 2 • 1-year post-transplant Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.
Surgical and medical procedures Mechanical ventilation	2.0% 1/50 • Number of events 1 • 1-year post-transplant Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.
Vascular disorders Hypertension	2.0% 1/50 • Number of events 1 • 1-year post-transplant Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.

Other adverse events

Measure	dUCB Transplant n=50 participants at risk Hematopoietic Umbilical Cord Blood Stem Cell Transplantation using a non-myeloablative preparative regimen.
Nervous system disorders Headache	2.0% 1/50 • Number of events 1 • 1-year post-transplant Serious adverse events are defined as events associated with death, life-threatening event, disability, congenital anomaly, required intervention to prevent permanent impairment or damage, hospitalization or other serious adverse event.

Additional Information

Adam Mendizabal

The EMMES Corporation

Phone: 301-251-1161

Email: amendizabal@EMMES.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place