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| ID | Type | Description | Link |
|---|---|---|---|
| NCI-2010-00299 | Registry Identifier | CTRP (Clinical Trial Reporting Program) | |
| 2275 | |||
| 2275.00 | Other Identifier | Fred Hutch/University of Washington Cancer Consortium | |
| RG2808000 | Other Identifier | Fred Hutch/University of Washington Cancer Consortium |
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This phase II trial studies how well giving treosulfan together with fludarabine phosphate and total-body irradiation (TBI) works in treating patients with hematological cancer who are undergoing umbilical cord blood transplant (UCBT). Giving chemotherapy, such as treosulfan and fludarabine phosphate, and TBI before a donor UCBT helps stop the growth of cancer cells and helps stop the patient's immune system from rejecting the donor's stem cells. When the stem cells from a related or unrelated donor, that do not exactly match the patient's blood, are infused into the patient, they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving cyclosporine (CsA) and mycophenolate mofetil (MMF) after the transplant may stop this from happening.
PRIMARY OBJECTIVES:
I. Graft failure/rejection and secondary graft failure.
II. Day -200 non-relapse mortality.
SECONDARY OBJECTIVES:
I. Platelet engraftment by six months.
II. Grade II-IV and III-IV acute graft-versus-host disease (GVHD) at day 100 and one year.
III. Chronic GVHD.
IV. Clinically significant infections.
V. Overall survival.
VI. Relapse or disease progression.
VII. Immune reconstitution (Fred Hutchinson Cancer Research Center [FHCRC] only).
VIII. Emergence of a dominant unit (FHCRC only).
OUTLINE: Patients are assigned to 1 of 2 arms.
ARM I (low risk for graft failure): Patients receive a conditioning regimen comprising fludarabine phosphate intravenously (IV) over 1 hour once daily (QD) on days -6 to -2 and treosulfan IV over 120 minutes on days - 6 to -4. Patients undergo TBI on day -1. Patients then undergo donor UCBT on day 0. Patients receive GVHD prophylaxis comprising cyclosporine IV over 1 hour or orally (PO) 2-3 times daily on days -3 to 100, followed by a taper in the absence of GVHD. Patients also receive mycophenolate mofetil IV 3 times daily on days 0 to 40, followed by a taper in the absence of GVHD.
ARM II (high risk for graft failure): Patients receive a conditioning regimen, TBI, donor UCBT, GVHD prophylaxis, and mycophenolate mofetil as in Arm I.
After completion of the study treatment, patients are followed up at 6 months and 1 and 2 years.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Arm I (low risk for graft failure) | Experimental | Patients receive a conditioning regimen comprising fludarabine phosphate IV over 1 hour QD on days -6 to -2 and treosulfan IV over 120 minutes on days - 6 to -4. Patients undergo TBI on day -1. Patients then undergo donor UCBT on day 0. Patients receive GVHD prophylaxis comprising cyclosporine IV over 1 hour or PO 2-3 times daily on days -3 to 100, followed by a taper in the absence of GVHD. Patients also receive mycophenolate mofetil IV 3 times daily on days 0 to 40, followed by a taper in the absence of GVHD. |
|
| Arm II (high risk for graft failure) | Experimental | Patients receive a conditioning regimen, TBI, donor UCBT, GVHD prophylaxis, and mycophenolate mofetil as in Arm I. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Cyclosporine | Drug | Given IV or PO |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Graft Failure/Rejection | Patients will be considered primary graft failure provided they meet any criteria listed below, in the absence of documented disease persistence/recurrence or active bone marrow infection (ex. Cytomegalovirus (CMV)): i. Absence of 3 consecutive days with neutrophils >500/u1 combined with host CD3+ peripheral blood chimerism ≥50% at day 42 ii. Absence of 3 consecutive days with neutrophils >500/u1 under any circumstances at day 55 iii. Death after day 28 with neutrophil count <100/u1 without any evidence of engraftment (< 5% donor CD3+) iv. Primary autologous count recovery with < 5% donor CD3+ peripheral blood chimerism at count recovery and without relapse | Up to 100 days |
| Number of Participants With Secondary Graft Failure | Secondary graft failure is defined as decline of neutrophil count to <500/ul with loss of donor chimerism after day 55 | Up to 2 years |
| Number of Patients With Non-relapse Mortality (NRM) | Defined as death from any cause without sign of disease progression or relapse. Loss to follow up are censored, whereas disease relapse or progression are considered competing risks. | At day -200 |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants Surviving by 1 Year | Overall survival was measured from the first day of CBT infusion until death from any cause. | At 1 year |
| The Number of Participants Alive at Two-years Follow up. |
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Inclusion Criteria:
Acute myeloid leukemia/acute lymphoblastic leukemia, including biphenotypic acute leukemia or mixed-lineage leukemia: Must have < 5% morphologic marrow blasts in an evaluable marrow sample (> 25% of normal cellularity for age collected less than one month prior to start of conditioning; patients in which adequate marrow/biopsy specimens cannot be obtained to determine remission status by morphologic assessment, but have fulfilled criteria of remission by flow cytometry, recovery of peripheral blood counts with no circulating blasts, and/or normal cytogenetics (if applicable) may still be eligible; reasonable attempts must be made to obtain an adequate specimen for morphologic assessment, including possible repeat procedures; these patients must be discussed with the principal investigator prior to enrollment; patients persistently aplastic for greater than one month since completing last chemotherapy are also eligible with principal investigator (PI) approval
Myelodysplastic syndrome (MDS): Any 2001 World Health Organization (WHO) classification subtype; refractory anemia with excess blasts (RAEB)-2 patients may proceed directly to transplant, but may also be considered for induction chemotherapy before transplant; patients with >= 20% morphologic marrow blasts require induction therapy to reduce morphologic marrow blasts below 5% before transplant
Chronic myelogenous leukemia: All types, except refractory blast crisis; chronic phase patients must have failed or been intolerant to Gleevec or other tyrosine kinase inhibitors
Patients =< 50 must have performance status score: Karnofsky (for adults) >= 70 or Eastern Cooperative Oncology Group (ECOG) 0-1; Lansky (for children) score >= 50
Patients > 50 must have Karnofsky performance score >= 70 or ECOG 0-1 and comorbidity index < 5
Adequate cardiac function defined as absence of decompensated congestive heart failure or uncontrolled arrhythmia AND left ventricular ejection fraction >= 35% OR fractional shortening > 22%
Adequate pulmonary function defined as absence of oxygen (O2) requirements and one of the following:
Adequate hepatic function; patients with clinical or laboratory evidence of liver disease will be evaluated for the cause of liver disease, its clinical severity in terms of liver function, histology, and the degree of portal hypertension; patients with fulminant liver failure, cirrhosis with evidence of portal hypertension or bridging fibrosis, alcoholic hepatitis, esophageal varices, a history of bleeding esophageal varices, hepatic encephalopathy, or correctable hepatic synthetic dysfunction evidenced by prolongation of the prothrombin time, ascites related to portal hypertension, bacterial or fungal abscess, biliary obstruction, chronic viral hepatitis with total serum bilirubin > 3 mg/dL, and symptomatic biliary disease will be excluded
Adequate renal function defined as creatinine =< 2.0 mg/dl (adults) or estimated creatinine clearance > 40 ml/min (pediatrics); all adults with a creatinine > 1.2 or a history of renal dysfunction must have estimated creatinine clearance > 40 ml/min
If recent mold infection, e.g., Aspergillus, must be cleared by infectious disease to proceed
Prior hematopoietic cell transplant: must be >= 3 months after previous transplant
DONOR: Human leukocyte antigen (HLA) matching:
DONOR: Selection of two CB units is mandatory when a single cord blood unit does not meet the following criteria in the table below
Match grade
6/6
5/6, 4/6
If two CB units are used, the total cell dose of the combined units must be at least 3.0 x 10^7 TNC per kilogram recipient weight based on pre-cryopreservation numbers, with each CB unit containing a MINIMUM of 1.5 x 10^7 TNC/kg
DONOR: The minimum recommended CD34/kg cell dose should be 2 x 10^5 CD34/kg, total dose from a single or combined double
DONOR: The unmanipulated CB unit(s) will be Food and Drug Administration (FDA) licensed or will be obtained under a separate investigational new drug (IND), such as the National Marrow Donor Program (NMDP) Protocol 10-CBA conducted under BB IND-7555 or another IND sponsored by (1) a participating institution or (2) an investigator at FHCRC or one of the participating institutions
DONOR (FHCRC only): Up to 5% of the unmanipulated cord blood product (s), when ready for infusion, may be withheld for research purposes as long as thresholds for infused TNC dose are met; these products will be used to conduct studies involving the kinetics of engraftment and immunobiology of double cord transplantation
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Filippo Milano | Fred Hutch/University of Washington Cancer Consortium | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Colorado Hospital | Aurora | Colorado | 80045 | United States | ||
| Oregon Health and Science University |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 32706891 | Derived | Milano F, Gutman JA, Deeg HJ, Nemecek ER, Baumgart J, Thur L, Dahlberg A, Salit RB, Summers C, Appelbaum FR, Delaney C. Treosulfan-based conditioning is feasible and effective for cord blood recipients: a phase 2 multicenter study. Blood Adv. 2020 Jul 28;4(14):3302-3310. doi: 10.1182/bloodadvances.2020002222. |
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All participants who signed consent were assigned to one of two arms and all completed enrollment.
Between February 2009 and October 2019, 130 cord blood transplant (CBT) recipients were enrolled in this prospective multi-center phase II study.
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| ID | Title | Description |
|---|---|---|
| FG000 | Arm I (Low Risk for Graft Failure) | Patients receive a conditioning regimen comprising fludarabine phosphate intravenously (IV) over 1 hour every day (QD) on days -6 to -2 and treosulfan IV over 120 minutes on days - 6 to -4. Patients undergo total body irradiation (TBI) on day -1. Patients then undergo donor cord blood transplant (CBT) on day 0. Patients receive graft versus host disease (GVHD) prophylaxis comprising cyclosporine IV over 1 hour or by mouth (PO) 2-3 times daily on days -3 to 100, followed by a taper in the absence of GVHD. Patients also receive mycophenolate mofetil IV 3 times daily on days 0 to 40, followed by a taper in the absence of GVHD. Cyclosporine: Given IV or PO Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given IV Total-Body Irradiation: TBI administered day -1: 200 centigray (cGy) (or escalated to 300 cGy, 400 cGy, or 450 cGy per protocol statistical section) Treosulfan: Given IV Umbilical Cord Blood Transplantation (UCBT): Undergo single or double unit UCBT |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Apr 14, 2022 |
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| Fludarabine Phosphate | Drug | Given IV |
|
|
| Laboratory Biomarker Analysis | Other | Correlative studies |
|
| Mycophenolate Mofetil | Drug | Given IV |
|
|
| Total-Body Irradiation | Radiation | TBI administered day -1: 200 cGy (or escalated to 300 cGy, 400 cGy, or 450 cGy per protocol statistical section) |
|
|
| Treosulfan | Drug | Given IV |
|
|
| Umbilical Cord Blood Transplantation | Procedure | Undergo single or double unit UCBT |
|
|
Overall survival of participants after two-years of follow up.
| Up to 2 years |
| Non-relapse Mortality | Death of any cause other than relapse or disease progression was considered. | Up to 2 years |
| Duration (Days) Until Participants Obtained Platelet Engraftment | Summarized using a range and median of measure in days until participants reached platelet engraftment. Platelet engraftment was defined as the first of 7 consecutive days when the platelet count exceeded 20 x 109/L (untransfused). | At 6 months |
| Number of Participants With Acute Graft-versus-host Disease (GVHD) Grade II-IV by Day 100 | Grade I GVHD is characterized as mild disease, grade II GVHD as moderate, grade III as severe, and grade IV life-threatening. Diagnosing and grading acute GVHD is based on clinical findings (the organ stage, response to treatment and whether GVHD was a major cause of death) and frequently varies between transplant centers and independent reviewers. | Day 100 |
| Incidence of Acute or Chronic Graft-versus-host Disease (GVHD) | Overall GVHD is determined based on the organ stage, response to treatment and whether GVHD was a major cause of death. Chronic GVHD will be defined according to the National Institutes of Health Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-versus-Host Disease. Described as mild, moderate, or severe as graded according to the attached Organ Scoring Sheet. Symptoms consistent with both chronic and acute GVHD occurring after day 100 will be considered overlap chronic GVHD syndrome. | At 1 year |
| Incidence of Relapse or Disease Progression | Cumulative incidence estimates using non-relapse mortality as competitive event. | Up to 2 years |
| Number of Participants Surviving up to 2 Years Without Disease Progression | Progression-free survival is the time interval from start of treatment to documented evidence of disease progression. Disease progression was defined by European LeukemiaNet 2017 criteria and International Working Group (IWG) within 3 years of transplant. | Up to 2 years |
| Incidence of Clinically Significant Infections | Collected and graded according to the modified National Cancer Institute Common Toxicity Criteria. | At 6 months |
| Portland |
| Oregon |
| 97239 |
| United States |
| Fred Hutch/University of Washington Cancer Consortium | Seattle | Washington | 98109 | United States |
| FG001 | Arm II (High Risk for Graft Failure) | Patients receive a conditioning regimen, total body irradiation (TBI), donor cord blood transplant (CBT), graft versus host disease (GVHD) prophylaxis, and mycophenolate mofetil as in Arm I. Cyclosporine: Given intravenously (IV) or by mouth (PO) Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given IV Total-Body Irradiation: TBI administered day -1: 200 centigray (cGy) (or escalated to 300 cGy, 400 cGy, or 450 cGy per protocol statistical section) Treosulfan: Given IV Umbilical Cord Blood Transplantation (UCBT): Undergo single or double unit UCBT |
| COMPLETED |
|
| NOT COMPLETED |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Arm I (Low Risk for Graft Failure) | Patients receive a conditioning regimen comprising fludarabine phosphate IV over 1 hour every day (QD) on days -6 to -2 and treosulfan IV over 120 minutes on days - 6 to -4. Patients undergo total body irradiation (TBI) on day -1. Patients then undergo donor umbilical cord blood transplantation (UCBT) on day 0. Patients receive graft-versus-host disease (GVHD) prophylaxis comprising cyclosporine IV over 1 hour or by mouth (PO) 2-3 times daily on days -3 to 100, followed by a taper in the absence of GVHD. Patients also receive mycophenolate mofetil IV 3 times daily on days 0 to 40, followed by a taper in the absence of GVHD. Cyclosporine: Given IV or PO Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil (MMF): Given IV Total-Body Irradiation: TBI administered day -1: 200 cGy (or escalated to 300 cGy, 400 cGy, or 450 cGy per protocol statistical section) Treosulfan: Given IV Umbilical Cord Blood Transplantation: Undergo single or double unit UCBT |
| BG001 | Arm II (High Risk for Graft Failure) | Patients receive a conditioning regimen, TBI, donor UCBT, GVHD prophylaxis, and mycophenolate mofetil as in Arm I. Cyclosporine: Given IV or PO Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given IV Total-Body Irradiation: TBI administered day -1: 200 cGy (or escalated to 300 cGy, 400 cGy, or 450 cGy per protocol statistical section) Treosulfan: Given IV Umbilical Cord Blood Transplantation: Undergo single or double unit UCBT |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
| ||||||||||||||||||
| Age, Continuous | Median | Full Range | years |
| |||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||
| Race (NIH/OMB) | Racial and ethnicity categories based on the Office of Management and Budget (OMB) and the National Institutes of Health (NIH) minimum categories for race and ethnicity. | Count of Participants | Participants |
| |||||||||||||||||
| Region of Enrollment | Count of Participants | Participants |
| ||||||||||||||||||
| Hematopoietic Cell Transplantation-Comorbidity Index (HCT-CI) | Hematopoietic Cell Transplantation-Comorbidity Index (HCT-CI). The HCT-CI is a comorbidity index that comprises 17 different categories of organ dysfunction. Positive findings are summated into a total score. The HCT-CI provides information with regard to the overall as well as non-relapse mortality risk a patient is likely to experience after hematopoietic cell transplantation. The HCT-CI scores were further collapsed into 3 risk groups: . A score of 0 indicates low risk, a score of 1-2 indicates intermediate risk and any score ≥3 indicates a high risk of non-relapse mortality. | Median | Full Range | units on a scale |
| ||||||||||||||||
| Diagnosis | Count of Participants | Participants |
| ||||||||||||||||||
| Human leukocyte antigens (HLA) matching to recipients | A close match between a donor's and a patient's HLA markers is essential for a successful transplant outcome. HLA matching promotes the growth and development of new healthy blood cells (called engraftment) and reduces the risk of a post-transplant complication called graft-versus-host (GVHD) disease. | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Number of Participants With Graft Failure/Rejection | Patients will be considered primary graft failure provided they meet any criteria listed below, in the absence of documented disease persistence/recurrence or active bone marrow infection (ex. Cytomegalovirus (CMV)): i. Absence of 3 consecutive days with neutrophils >500/u1 combined with host CD3+ peripheral blood chimerism ≥50% at day 42 ii. Absence of 3 consecutive days with neutrophils >500/u1 under any circumstances at day 55 iii. Death after day 28 with neutrophil count <100/u1 without any evidence of engraftment (< 5% donor CD3+) iv. Primary autologous count recovery with < 5% donor CD3+ peripheral blood chimerism at count recovery and without relapse | Posted | Count of Participants | Participants | Up to 100 days |
|
|
| ||||||||||||||||||||||||||||||
| Primary | Number of Participants With Secondary Graft Failure | Secondary graft failure is defined as decline of neutrophil count to <500/ul with loss of donor chimerism after day 55 | Posted | Count of Participants | Participants | Up to 2 years |
| ||||||||||||||||||||||||||||||||
| Primary | Number of Patients With Non-relapse Mortality (NRM) | Defined as death from any cause without sign of disease progression or relapse. Loss to follow up are censored, whereas disease relapse or progression are considered competing risks. | Posted | Count of Participants | Participants | At day -200 |
| ||||||||||||||||||||||||||||||||
| Secondary | Number of Participants Surviving by 1 Year | Overall survival was measured from the first day of CBT infusion until death from any cause. | Posted | Count of Participants | Participants | At 1 year |
| ||||||||||||||||||||||||||||||||
| Secondary | The Number of Participants Alive at Two-years Follow up. | Overall survival of participants after two-years of follow up. | Posted | Count of Participants | Participants | Up to 2 years |
| ||||||||||||||||||||||||||||||||
| Secondary | Non-relapse Mortality | Death of any cause other than relapse or disease progression was considered. | Posted | Count of Participants | Participants | Up to 2 years |
| ||||||||||||||||||||||||||||||||
| Secondary | Duration (Days) Until Participants Obtained Platelet Engraftment | Summarized using a range and median of measure in days until participants reached platelet engraftment. Platelet engraftment was defined as the first of 7 consecutive days when the platelet count exceeded 20 x 109/L (untransfused). | Posted | Median | Full Range | days | At 6 months |
| |||||||||||||||||||||||||||||||
| Secondary | Number of Participants With Acute Graft-versus-host Disease (GVHD) Grade II-IV by Day 100 | Grade I GVHD is characterized as mild disease, grade II GVHD as moderate, grade III as severe, and grade IV life-threatening. Diagnosing and grading acute GVHD is based on clinical findings (the organ stage, response to treatment and whether GVHD was a major cause of death) and frequently varies between transplant centers and independent reviewers. | Posted | Count of Participants | Participants | Day 100 |
| ||||||||||||||||||||||||||||||||
| Secondary | Incidence of Acute or Chronic Graft-versus-host Disease (GVHD) | Overall GVHD is determined based on the organ stage, response to treatment and whether GVHD was a major cause of death. Chronic GVHD will be defined according to the National Institutes of Health Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-versus-Host Disease. Described as mild, moderate, or severe as graded according to the attached Organ Scoring Sheet. Symptoms consistent with both chronic and acute GVHD occurring after day 100 will be considered overlap chronic GVHD syndrome. | Posted | Count of Participants | Participants | At 1 year |
| ||||||||||||||||||||||||||||||||
| Secondary | Incidence of Relapse or Disease Progression | Cumulative incidence estimates using non-relapse mortality as competitive event. | Posted | Count of Participants | Participants | Up to 2 years |
| ||||||||||||||||||||||||||||||||
| Secondary | Number of Participants Surviving up to 2 Years Without Disease Progression | Progression-free survival is the time interval from start of treatment to documented evidence of disease progression. Disease progression was defined by European LeukemiaNet 2017 criteria and International Working Group (IWG) within 3 years of transplant. | Posted | Count of Participants | Participants | Up to 2 years |
| ||||||||||||||||||||||||||||||||
| Secondary | Incidence of Clinically Significant Infections | Collected and graded according to the modified National Cancer Institute Common Toxicity Criteria. | Posted | Count of Participants | Participants | At 6 months |
|
Adverse events (AE) will be monitored and recorded in study-specific case report forms (CRFs) from the time of first exposure to an investigational product in this study through day +100 post-transplant. The start of the adverse event reporting for a study subject will coincide with signing of the informed consent. All-cause mortality data was censored at two-year follow-up.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Arm I (Low Risk for Graft Failure) | Patients receive a conditioning regimen comprising fludarabine phosphate IV over 1 hour every day (QD) on days -6 to -2 and treosulfan IV over 120 minutes on days - 6 to -4. Patients undergo (total body irradiation (TBI) on day -1. Patients then undergo donor cord blood transplantation (CBT) on day 0. Patients receive graft-versus-host disease (GVHD) prophylaxis comprising cyclosporine IV over 1 hour or by mouth (PO) 2-3 times daily on days -3 to 100, followed by a taper in the absence of GVHD. Patients also receive mycophenolate mofetil IV 3 times daily on days 0 to 40, followed by a taper in the absence of GVHD. Cyclosporine: Given IV or PO Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given IV Total-Body Irradiation: TBI administered day -1: 200 centigray (cGy) (or escalated to 300 cGy, 400 cGy, or 450 cGy per protocol statistical section) Treosulfan: Given IV Umbilical Cord Blood Transplantation (UCBT): Undergo single or double unit UCBT | 16 | 66 | 27 | 66 | 63 | 66 |
| EG001 | Arm II (High Risk for Graft Failure) | Patients receive a conditioning regimen, TBI, donor UCBT, GVHD prophylaxis, and mycophenolate mofetil as in Arm I. Cyclosporine: Given IV or PO Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given IV Total-Body Irradiation: TBI administered day -1: 200 cGy (or escalated to 300 cGy, 400 cGy, or 450 cGy per protocol statistical section) Treosulfan: Given IV Umbilical Cord Blood Transplantation: Undergo single or double unit UCBT | 20 | 64 | 23 | 64 | 59 | 64 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Allergic Reaction | Immune system disorders | Systematic Assessment |
| ||
| Acute cardiomyopathy | Cardiac disorders | Systematic Assessment |
| ||
| Left Ventricular Systolic dysfunction | Cardiac disorders | Systematic Assessment |
| ||
| Cardiac troponin I increased | Investigations | Systematic Assessment |
| ||
| Hypertension | Vascular disorders | Systematic Assessment |
| ||
| Hypotension | Vascular disorders | Systematic Assessment |
| ||
| Pericarditis | Cardiac disorders | Systematic Assessment |
| ||
| Multi-organ failure | General disorders | Systematic Assessment |
| ||
| Infusional Toxicity/Stunned Heart | Cardiac disorders | Systematic Assessment |
| ||
| Heart Failure | Cardiac disorders | Systematic Assessment |
| ||
| Sinusoidal obstruction syndrome | Hepatobiliary disorders | Systematic Assessment |
| ||
| Gastrointestinal hemorrhage | Gastrointestinal disorders | Systematic Assessment |
| ||
| Anal hemorrhage | Gastrointestinal disorders | Systematic Assessment |
| ||
| Bronchopulmonary hemorrhage | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Hypoxia | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Renal failure | Renal and urinary disorders | Systematic Assessment |
| ||
| Seizure | Nervous system disorders | Systematic Assessment |
| ||
| Psychosis | Psychiatric disorders | Systematic Assessment |
| ||
| Myelitis | Infections and infestations | Systematic Assessment |
| ||
| Encephalopathy | Nervous system disorders | Systematic Assessment |
| ||
| Cognitive disturbance | Nervous system disorders | Systematic Assessment |
| ||
| Other - autonomic dysreflexia exacerbation | Nervous system disorders | Systematic Assessment |
| ||
| Proteinuria | Renal and urinary disorders | Systematic Assessment |
| ||
| Sepsis | Infections and infestations | Systematic Assessment |
| ||
| Upper respiratory infection | Infections and infestations | Systematic Assessment |
| ||
| Anorectal infection | Infections and infestations | Systematic Assessment |
| ||
| Encephalitis infection | Infections and infestations | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Allergic Reaction | Immune system disorders | Systematic Assessment |
| ||
| Hemolysis | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Atrial Fibrillation | Cardiac disorders | Systematic Assessment |
| ||
| Atrial Flutter | Cardiac disorders | Systematic Assessment |
| ||
| Sinus tachycardia | Cardiac disorders | Systematic Assessment |
| ||
| Heart Failure | Cardiac disorders | Systematic Assessment |
| ||
| Hypotension | Vascular disorders | Systematic Assessment |
| ||
| Portal vein thrombosis | Hepatobiliary disorders | Systematic Assessment |
| ||
| Disemminated vascular coagulation | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Sinusoidal obstruction syndrome | Hepatobiliary disorders | Systematic Assessment |
| ||
| Rash maculopapular | Skin and subcutaneous tissue disorders | Systematic Assessment |
| ||
| Colitis | Gastrointestinal disorders | Systematic Assessment |
| ||
| Diarrhea | Gastrointestinal disorders | Systematic Assessment |
| ||
| Gastric Ulcer | Gastrointestinal disorders | Systematic Assessment |
| ||
| Mucositis | Gastrointestinal disorders | Systematic Assessment |
| ||
| Pancreatitis | Gastrointestinal disorders | Systematic Assessment |
| ||
| Bronchopulmonary hemorrhage | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Gastric hemorrhage | Gastrointestinal disorders | Systematic Assessment |
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| Blood bilirubin increased | Investigations | Systematic Assessment |
| ||
| Febrile Neutropenia | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Sepsis | Infections and infestations | Systematic Assessment |
| ||
| Lung infection | Infections and infestations | Systematic Assessment |
| ||
| Urinary tract infection | Infections and infestations | Systematic Assessment |
| ||
| Skin infection | Infections and infestations | Systematic Assessment |
| ||
| Encephalitis infection | Infections and infestations | Systematic Assessment |
| ||
| Enterocolitis infectious | Infections and infestations | Systematic Assessment |
| ||
| Eye infection | Infections and infestations | Systematic Assessment |
| ||
| Upper respiratory infection | Infections and infestations | Systematic Assessment |
| ||
| Syncope | Nervous system disorders | Systematic Assessment |
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| Apnea | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
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| Hypoxia | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
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| Creatinine increased | Investigations | Systematic Assessment |
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| Renal failure | Renal and urinary disorders | Systematic Assessment |
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| Left Ventricular Systolic dysfunction | Cardiac disorders | Systematic Assessment |
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| Pericarditis | Cardiac disorders | Systematic Assessment |
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| Hypertension | Cardiac disorders | Systematic Assessment |
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| Pericardial effusion | Cardiac disorders | Systematic Assessment |
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| Gatritis | Gastrointestinal disorders | Systematic Assessment |
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| Seizure | Nervous system disorders | Systematic Assessment |
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| Otitis Externa | Infections and infestations | Systematic Assessment |
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| Bacteremia | Infections and infestations | Systematic Assessment |
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| Duodenal infection | Infections and infestations | Systematic Assessment |
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| Thromboembolic event | Vascular disorders | Systematic Assessment |
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| Epistaxis | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
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| Oral cavity | Infections and infestations | Systematic Assessment |
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| Other: genitourinary | Infections and infestations | Systematic Assessment |
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| Sinusitis | Infections and infestations | Systematic Assessment |
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| Anorectal infection | Infections and infestations | Systematic Assessment |
| ||
| Other: pulmonary insufficiency | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
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| Other: Thrombosis | Vascular disorders | Systematic Assessment |
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| Other: Coagulopathy | Blood and lymphatic system disorders | Systematic Assessment |
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| Other: Thrombotic microangiopathy | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Other: Genitalia infection | Infections and infestations | Systematic Assessment |
| ||
| Other: Melena | Gastrointestinal disorders | Systematic Assessment |
|
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Filippo Milano | Fred Hutchinson Cancer Center | 206-667-5925 | fmilano@fredhutch.org |
| Jan 23, 2023 |
| Prot_SAP_000.pdf |
| ID | Term |
|---|---|
| D015456 | Leukemia, Biphenotypic, Acute |
| D015464 | Leukemia, Myelogenous, Chronic, BCR-ABL Positive |
| D015466 | Leukemia, Myeloid, Chronic-Phase |
| D009190 | Myelodysplastic Syndromes |
| ID | Term |
|---|---|
| D007945 | Leukemia, Lymphoid |
| D007938 | Leukemia |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D008232 | Lymphoproliferative Disorders |
| D008206 | Lymphatic Diseases |
| D007160 | Immunoproliferative Disorders |
| D007154 | Immune System Diseases |
| D007951 | Leukemia, Myeloid |
| D009196 | Myeloproliferative Disorders |
| D001855 | Bone Marrow Diseases |
| D002908 | Chronic Disease |
| D020969 | Disease Attributes |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
Not provided
Not provided
| ID | Term |
|---|---|
| D016572 | Cyclosporine |
| D003524 | Cyclosporins |
| C042382 | fludarabine phosphate |
| D009173 | Mycophenolic Acid |
| D014916 | Whole-Body Irradiation |
| C018404 | treosulfan |
| D036101 | Cord Blood Stem Cell Transplantation |
| ID | Term |
|---|---|
| D010456 | Peptides, Cyclic |
| D047028 | Macrocyclic Compounds |
| D011083 | Polycyclic Compounds |
| D010455 | Peptides |
| D000602 | Amino Acids, Peptides, and Proteins |
| D002208 | Caproates |
| D000144 | Acids, Acyclic |
| D002264 | Carboxylic Acids |
| D009930 | Organic Chemicals |
| D005227 | Fatty Acids |
| D008055 | Lipids |
| D011878 | Radiotherapy |
| D013812 | Therapeutics |
| D008919 | Investigative Techniques |
| D033581 | Stem Cell Transplantation |
| D017690 | Cell Transplantation |
| D064987 | Cell- and Tissue-Based Therapy |
| D001691 | Biological Therapy |
| D014180 | Transplantation |
| D013514 | Surgical Procedures, Operative |
Not provided
Not provided
| >=65 years |
|
| Male |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| Acute lymphoblastic leukemia (ALL) |
|
| Biphenotypic |
|
| Myelodysplastic Syndrome (MDS) |
|
| Myeloproliferative |
|
| 5/6 |
|
| 4/6 |
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Patients receive a conditioning regimen, TBI, donor UCBT, GVHD prophylaxis, and mycophenolate mofetil as in Arm I. Cyclosporine: Given IV or PO Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given IV Total-Body Irradiation: TBI administered day -1: 200 cGy (or escalated to 300 cGy, 400 cGy, or 450 cGy per protocol statistical section) Treosulfan: Given IV Umbilical Cord Blood Transplantation: Undergo single or double unit UCBT |
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