Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| NCI-2012-01572 | Registry Identifier | CTRP (Clinical Trial Reporting Program) | |
| 2603 | |||
| 2603.00 | Other Identifier | Fred Hutch/University of Washington Cancer Consortium | |
| P30CA015704 | U.S. NIH Grant/Contract | View source | |
| P50HL110787 | U.S. NIH Grant/Contract | View source |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Class |
|---|---|
| National Cancer Institute (NCI) | NIH |
| National Heart, Lung, and Blood Institute (NHLBI) | NIH |
Not provided
Not provided
Not provided
Not provided
This randomized phase II trial studies how well donor umbilical cord blood transplant with or without ex-vivo expanded cord blood progenitor cells works in treating patients with acute myeloid leukemia, acute lymphoblastic leukemia, chronic myelogenous leukemia, or myelodysplastic syndromes. Giving chemotherapy and total-body irradiation before a donor umbilical cord blood transplant helps stop the growth of cancer cells. It may also stop the patient's immune system from rejecting the donor's cells. When the healthy stem cells and ex-vivo expanded cord blood progenitor cells are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. It is not yet known whether giving donor umbilical cord blood transplant plus ex-vivo expanded cord blood progenitor cells is more effective than giving a donor umbilical cord blood transplant alone.
PRIMARY OBJECTIVES:
I. Compare the time to neutrophil engraftment (absolute neutrophil count [ANC] >= 500) in patients receiving a standard of care myeloablative cord blood transplant (CBT) augmented with an off-the-shelf pre-expanded and cryopreserved cord blood product to those who do not receive the product.
SECONDARY OBJECTIVES:
I. Provide initial data on clinical and economic benefit, such as time to platelet engraftment, duration of initial hospitalization, transplant-related mortality (TRM), death without engraftment, and incidence of severe infections in the first 100 days post-transplant.
II. The kinetics of immune system recovery will also be evaluated in both arms.
OUTLINE: Patients are randomized to 1 of 2 treatment arms.
Standard of Care Arm:
CONDITIONING REGIMEN: One of two possible conditioning regimens is chosen by the attending physician: Patients receive fludarabine phosphate intravenously (IV) over 30 minutes on days -8 to -6, cyclophosphamide IV on days -7 to -6., and undergo high dose total-body irradiation (TBI) twice daily (BID) on days -4 to -1 OR, patients receive fludarabine phosphate IV over 30-60 minutes on days -6 to -2, cyclophosphamide IV on day -6, thiotepa IV over 4 hours on days -5 to -4, and undergo middle intensity TBI once daily (QD) on days -2 to -1.
TRANSPLANT: Patients undergo single-unit or double-unit unmanipulated umbilical cord blood (UCB) transplant on day 0.
GRAFT-VERSUS-HOST DISEASE (GVHD) PROPHYLAXIS: Patients receive cyclosporine IV over 1 hour BID (adults) or thrice daily (TID) (children) or orally (PO) on days -3 to 100 with taper beginning on day 101. Patients also receive mycophenolate mofetil (MMF) IV TID on days 0-7 then may receive MMF PO TID. Patients remain on MMF TID for a minimum of 30 days, and then may begin taper if there is no evidence of GVHD and are well-engrafted from one donor unit.
Experimental Arm:
CONDITIONING REGIMEN: Patients receive the conditioning regimen chosen by the attending physician as in Standard of Care Arm.
TRANSPLANT: Patients undergo single-unit or double-unit unmanipulated UCB transplant on day 0. Patients also receive an infusion of ex vivo-expanded cord blood progenitors at least 4 hours after completion of UCB transplant.
GVHD PROPHYLAXIS: Patients receive cyclosporine IV or PO and mycophenolate mofetil IV or PO as in Standard of Care Arm.
After completion of study treatment, patients are followed up periodically for 2 years.
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Arm I (standard of care) | Active Comparator | CONDITIONING REGIMEN: One of two possible conditioning regimens is chosen by the attending physician: Patients receive fludarabine phosphate IV over 30 minutes on days -8 to -6, cyclophosphamide IV on days -7 to -6., and undergo high dose TBI BID on days -4 to -1 OR Patients receive fludarabine phosphate IV over 30-60 minutes on days -6 to -2, cyclophosphamide IV on day -6, thiotepa IV over 4 hours on days -5 to -4, and undergo middle intensity TBI QD on days -2 to -1. TRANSPLANT: Patients undergo single-unit or double-unit unmanipulated UCB transplant on day 0. GVHD PROPHYLAXIS: Patients receive cyclosporine IV over 1 hour BID (adults) or TID (children) or PO on days -3 to 100 with taper beginning on day 101. Patients also receive MMF IV TID a day on days 0-7 then may receive MMF PO TID. Patients remain on MMF TID for a minimum of 30 days, and then may begin a taper if there is no evidence of GVHD and are well-engrafted from one donor unit. |
|
| Arm II (experimental) | Experimental | CONDITIONING REGIMEN: Patients receive the conditioning regimen chosen by the attending physician as in Standard of Care Arm. TRANSPLANT: Patients undergo single-unit or double-unit unmanipulated UCB transplant on day 0. Patients also undergo infusion of ex vivo-expanded cord blood progenitor cell infusion at least 4 hours after completion of UCB transplant. GVHD PROPHYLAXIS: Patients receive cyclosporine IV or PO and mycophenolate mofetil IV or PO as in Standard of Care Arm. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Cyclophosphamide | Drug | Given IV |
| |
| Measure | Description | Time Frame |
|---|---|---|
| Time to Neutrophil Engraftment | First of two consecutive days with absolute neutrophil count (ANC) equal to or greater than 500 cell/microliter measured from day of transplant. | Up to 55 days post-transplant |
| Measure | Description | Time Frame |
|---|---|---|
| Time to Platelet Engraftment (20k) | First day of seven consecutive days with platelet count equal to or greater than 20,000 cells/microliter without platelet transfusions measured from day of transplant. | Up to 100 days post-transplant |
| Overall Survival |
Not provided
Inclusion Criteria:
Age criteria:
Acute myeloid leukemia, including biphenotypic acute leukemia or mixed-lineage leukemia
Acute lymphoblastic leukemia, including biphenotypic acute leukemia or mixed-lineage leukemia
Chronic myelogenous leukemia excluding refractory blast crisis; to be eligible in first chronic phase (CP1) patient must have failed or be intolerant to tyrosine kinase inhibitor therapy
Myelodysplasia (MDS) International Prognostic Scoring System (IPSS) intermediate (Int)-2 or high risk (i.e., refractory anemia with excess blasts [RAEB], refractory anemia with excess blasts in transformation [RAEBt]) or refractory anemia with severe pancytopenia or high risk cytogenetics; blasts must be < 10% by a representative bone marrow aspirate morphology
Karnofsky (>= 16 years old) >= 70 or Eastern Cooperative Oncology Group (ECOG) 0-1
Lansky (< 16 years old) >= 60
Adults: calculated creatinine clearance must be > 60 mL and serum creatinine =< 2 mg/dL
Children (< 18 years old): calculated creatinine clearance must be > 60 mL/min
Total serum bilirubin must be < 3 mg/dL unless the elevation is thought to be due to Gilbert's disease or hemolysis
Transaminases must be < 3 x the upper limit of normal per reference values of referring institution
Diffusing capacity of the lung for carbon monoxide (DLCO) corrected > 60% normal
For pediatric patients unable to perform pulmonary function tests, oxygen (O2) saturation > 92% on room air
May not be on supplemental oxygen
Left ventricular ejection fraction > 45% OR
Shortening fraction > 26%
Ability to understand and the willingness to sign a written informed consent document
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Filippo Milano | Fred Hutch/University of Washington Cancer Consortium | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| City of Hope Comprehensive Cancer Center | Duarte | California | 91010 | United States | ||
| Stanford Cancer Institute Palo Alto |
Not provided
The Full Analysis Set includes all randomized subjects (78 in Arm I SOC and 82 in Arm II Expanded Arm). The modified intent to treat (mITT) analysis set requires subjects in the FAS to undergo hematopoietic cell transplant (HCT) and receive study drug. Two subjects in Arm II withdrew prior to receiving study drug and are therefore not included in the mITT analysis set (78 in SOC and 80 in Expanded Arm).
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Arm I (Standard of Care) | CONDITIONING REGIMEN: One of two possible conditioning regimens is chosen by the attending physician: Patients receive fludarabine phosphate IV over 30 minutes on days -8 to -6, cyclophosphamide IV on days -7 to -6., and undergo high dose TBI BID on days -4 to -1 OR Patients receive fludarabine phosphate IV over 30-60 minutes on days -6 to -2, cyclophosphamide IV on day -6, thiotepa IV over 4 hours on days -5 to -4, and undergo middle intensity TBI QD on days -2 to -1. TRANSPLANT: Patients undergo single-unit or double-unit unmanipulated UCB transplant on day 0. GVHD PROPHYLAXIS: Patients receive cyclosporine IV over 1 hour BID (adults) or TID (children) or PO on days -3 to 100 with taper beginning on day 101. Patients also receive MMF IV TID a day on days 0-7 then may receive MMF PO TID. Patients remain on MMF TID for a minimum of 30 days, and then may begin a taper if there is no evidence of GVHD and are well-engrafted from one donor unit. Cyclophosphamide: Given IV Cyclosporine: Given IV or PO Fludarabine Phosphate: Given IV Mycophenolate Mofetil: Given IV or PO Thiotepa: Given IV Total-Body Irradiation: Undergo high dose or middle intensity TBI Umbilical Cord Blood Transplantation: Undergo single-unit or double-unit unmanipulated umbilical cord blood transplant |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
Not provided
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Mar 27, 2019 | Oct 10, 2019 |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Cyclosporine |
| Drug |
Given IV or PO |
|
| Ex Vivo-Expanded Cord Blood Progenitor Cell Infusion | Biological | Given IV |
|
|
| Fludarabine Phosphate | Drug | Given IV |
|
| Mycophenolate Mofetil | Drug | Given IV or PO |
|
| Thiotepa | Drug | Given IV |
|
| Total-Body Irradiation | Radiation | Undergo high dose or middle intensity TBI |
|
| Umbilical Cord Blood Transplantation | Procedure | Undergo single-unit or double-unit unmanipulated umbilical cord blood transplant |
|
|
| Up to 2 years |
| Non-relapse Mortality | Up to 2 years |
| Proportion of Patients With Severe Acute Graft Versus Host Disease | Up to 100 days post-transplant |
| Proportion of Participants With Chronic Graft Versus Host Disease | Up to 2 years |
| Palo Alto |
| California |
| 94304 |
| United States |
| University of Colorado | Denver | Colorado | 80217-3364 | United States |
| Dana-Farber Cancer Institute | Boston | Massachusetts | 02215 | United States |
| Mount Sinai Hospital | New York | New York | 10029 | United States |
| Duke University Medical Center | Durham | North Carolina | 27710 | United States |
| Cleveland Clinic Foundation | Cleveland | Ohio | 44195 | United States |
| Fred Hutch/University of Washington Cancer Consortium | Seattle | Washington | 98109 | United States |
| FG001 | Arm II (Experimental) | CONDITIONING REGIMEN: Patients receive the conditioning regimen chosen by the attending physician as in Standard of Care Arm. TRANSPLANT: Patients undergo single-unit or double-unit unmanipulated UCB transplant on day 0. Patients also undergo infusion of ex vivo-expanded cord blood progenitor cell infusion at least 4 hours after completion of UCB transplant. GVHD PROPHYLAXIS: Patients receive cyclosporine IV or PO and mycophenolate mofetil IV or PO as in Standard of Care Arm. Cyclophosphamide: Given IV Cyclosporine: Given IV or PO Ex Vivo-Expanded Cord Blood Progenitor Cell Infusion: Given IV Fludarabine Phosphate: Given IV Mycophenolate Mofetil: Given IV or PO Thiotepa: Given IV Total-Body Irradiation: Undergo high dose or middle intensity TBI Umbilical Cord Blood Transplantation: Undergo single-unit or double-unit unmanipulated umbilical cord blood transplant |
| Modified Intent to Treat (mITT) |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Arm I (Standard of Care) | CONDITIONING REGIMEN: One of two possible conditioning regimens is chosen by the attending physician: Patients receive fludarabine phosphate IV over 30 minutes on days -8 to -6, cyclophosphamide IV on days -7 to -6., and undergo high dose TBI BID on days -4 to -1 OR Patients receive fludarabine phosphate IV over 30-60 minutes on days -6 to -2, cyclophosphamide IV on day -6, thiotepa IV over 4 hours on days -5 to -4, and undergo middle intensity TBI QD on days -2 to -1. TRANSPLANT: Patients undergo single-unit or double-unit unmanipulated UCB transplant on day 0. GVHD PROPHYLAXIS: Patients receive cyclosporine IV over 1 hour BID (adults) or TID (children) or PO on days -3 to 100 with taper beginning on day 101. Patients also receive MMF IV TID a day on days 0-7 then may receive MMF PO TID. Patients remain on MMF TID for a minimum of 30 days, and then may begin a taper if there is no evidence of GVHD and are well-engrafted from one donor unit. Cyclophosphamide: Given IV Cyclosporine: Given IV or PO Fludarabine Phosphate: Given IV Mycophenolate Mofetil: Given IV or PO Thiotepa: Given IV Total-Body Irradiation: Undergo high dose or middle intensity TBI Umbilical Cord Blood Transplantation: Undergo single-unit or double-unit unmanipulated umbilical cord blood transplant |
| BG001 | Arm II (Experimental) | CONDITIONING REGIMEN: Patients receive the conditioning regimen chosen by the attending physician as in Standard of Care Arm. TRANSPLANT: Patients undergo single-unit or double-unit unmanipulated UCB transplant on day 0. Patients also undergo infusion of ex vivo-expanded cord blood progenitor cell infusion at least 4 hours after completion of UCB transplant. GVHD PROPHYLAXIS: Patients receive cyclosporine IV or PO and mycophenolate mofetil IV or PO as in Standard of Care Arm. Cyclophosphamide: Given IV Cyclosporine: Given IV or PO Ex Vivo-Expanded Cord Blood Progenitor Cell Infusion: Given IV Fludarabine Phosphate: Given IV Mycophenolate Mofetil: Given IV or PO Thiotepa: Given IV Total-Body Irradiation: Undergo high dose or middle intensity TBI Umbilical Cord Blood Transplantation: Undergo single-unit or double-unit unmanipulated umbilical cord blood transplant |
| 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 | Mean | Standard Deviation | Years |
| |||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Region of Enrollment | Number | participants |
| ||||||||||||||||||
| Height (cm) | Mean | Standard Deviation | centimeters |
| |||||||||||||||||
| Weight (kg) | Mean | Standard Deviation | kilograms |
| |||||||||||||||||
| Cord Blood Units Received | 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 | Time to Neutrophil Engraftment | First of two consecutive days with absolute neutrophil count (ANC) equal to or greater than 500 cell/microliter measured from day of transplant. | The primary analysis set for the analysis of Neutrophil Enrgraftment and Platelet Engraftment was the modified intent to treat (mITT) analysis set. The difference between the full analysis set (FAS) and the mITT is the requirement to undergo hematopoietic cell transplant (HCT) and receive study drug. Two subjects in Arm II withdrew prior to receiving study drug, and are therefore not included in the mITT analysis set, nor the primary efficacy analyses. | Posted | Median | 95% Confidence Interval | days | Up to 55 days post-transplant |
|
|
| ||||||||||||||||||||||||||||
| Secondary | Time to Platelet Engraftment (20k) | First day of seven consecutive days with platelet count equal to or greater than 20,000 cells/microliter without platelet transfusions measured from day of transplant. | The primary analysis set for the analysis of Neutrophil Enrgraftment and Platelet Engraftment was the modified intent to treat (mITT) analysis set. The difference between the full analysis set (FAS) and the mITT is the requirement to undergo hematopoietic cell transplant (HCT) and receive study drug. Two subjects in Arm II withdrew prior to receiving study drug, and are therefore not included in the mITT analysis set, nor the primary efficacy analyses. | Posted | Median | 95% Confidence Interval | days | Up to 100 days post-transplant |
| ||||||||||||||||||||||||||||||
| Secondary | Overall Survival | Posted | Count of Participants | Participants | Up to 2 years |
| |||||||||||||||||||||||||||||||||
| Secondary | Non-relapse Mortality | Posted | Count of Participants | Participants | Up to 2 years |
| |||||||||||||||||||||||||||||||||
| Secondary | Proportion of Patients With Severe Acute Graft Versus Host Disease | Posted | Number | 95% Confidence Interval | proportion of participants | Up to 100 days post-transplant |
| ||||||||||||||||||||||||||||||||
| Secondary | Proportion of Participants With Chronic Graft Versus Host Disease | Posted | Count of Participants | Participants | Up to 2 years |
|
84 days
Not provided
Not provided
| 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 (Standard of Care) | CONDITIONING REGIMEN: One of two possible conditioning regimens is chosen by the attending physician: Patients receive fludarabine phosphate IV over 30 minutes on days -8 to -6, cyclophosphamide IV on days -7 to -6., and undergo high dose TBI BID on days -4 to -1 OR Patients receive fludarabine phosphate IV over 30-60 minutes on days -6 to -2, cyclophosphamide IV on day -6, thiotepa IV over 4 hours on days -5 to -4, and undergo middle intensity TBI QD on days -2 to -1. TRANSPLANT: Patients undergo single-unit or double-unit unmanipulated UCB transplant on day 0. GVHD PROPHYLAXIS: Patients receive cyclosporine IV over 1 hour BID (adults) or TID (children) or PO on days -3 to 100 with taper beginning on day 101. Patients also receive MMF IV TID a day on days 0-7 then may receive MMF PO TID. Patients remain on MMF TID for a minimum of 30 days, and then may begin a taper if there is no evidence of GVHD and are well-engrafted from one donor unit. Cyclophosphamide: Given IV Cyclosporine: Given IV or PO Fludarabine Phosphate: Given IV Mycophenolate Mofetil: Given IV or PO Thiotepa: Given IV Total-Body Irradiation: Undergo high dose or middle intensity TBI Umbilical Cord Blood Transplantation: Undergo single-unit or double-unit unmanipulated umbilical cord blood transplant | 25 | 78 | 48 | 78 | 71 | 78 |
| EG001 | Arm II (Experimental) | CONDITIONING REGIMEN: Patients receive the conditioning regimen chosen by the attending physician as in Standard of Care Arm. TRANSPLANT: Patients undergo single-unit or double-unit unmanipulated UCB transplant on day 0. Patients also undergo infusion of ex vivo-expanded cord blood progenitor cell infusion at least 4 hours after completion of UCB transplant. GVHD PROPHYLAXIS: Patients receive cyclosporine IV or PO and mycophenolate mofetil IV or PO as in Standard of Care Arm. Cyclophosphamide: Given IV Cyclosporine: Given IV or PO Ex Vivo-Expanded Cord Blood Progenitor Cell Infusion: Given IV Fludarabine Phosphate: Given IV Mycophenolate Mofetil: Given IV or PO Thiotepa: Given IV Total-Body Irradiation: Undergo high dose or middle intensity TBI Umbilical Cord Blood Transplantation: Undergo single-unit or double-unit unmanipulated umbilical cord blood transplant | 21 | 82 | 55 | 82 | 73 | 82 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Pyrexia | General disorders | Systematic Assessment |
| ||
| Multiple organ dysfunction syndrome | General disorders | Systematic Assessment |
| ||
| Chest pain | General disorders | Systematic Assessment |
| ||
| Chills | General disorders | Systematic Assessment |
| ||
| Complication associated with device | General disorders | Systematic Assessment |
| ||
| Death | General disorders | Systematic Assessment |
| ||
| Non-cardiac chest pain | General disorders | Systematic Assessment |
| ||
| Clostridium difficile infection | Infections and infestations | Systematic Assessment |
| ||
| Cytomegalovirus gastritis | Infections and infestations | Systematic Assessment |
| ||
| Cytomegalovirus infection | Infections and infestations | Systematic Assessment |
| ||
| Eye infection | Infections and infestations | Systematic Assessment |
| ||
| Herpes virus infection | Infections and infestations | Systematic Assessment |
| ||
| Klebsiella infection | Infections and infestations | Systematic Assessment |
| ||
| Oral infection | Infections and infestations | Systematic Assessment |
| ||
| Pneumonia klebsiella | Infections and infestations | Systematic Assessment |
| ||
| Pseudomonas infection | Infections and infestations | Systematic Assessment |
| ||
| Rhinovirus infection | Infections and infestations | Systematic Assessment |
| ||
| Sinusitis | Infections and infestations | Systematic Assessment |
| ||
| Skin infection | Infections and infestations | Systematic Assessment |
| ||
| Systemic candida | Infections and infestations | Systematic Assessment |
| ||
| Urinary tract infection | Infections and infestations | Systematic Assessment |
| ||
| Urinary tract infection enterococcal | Infections and infestations | Systematic Assessment |
| ||
| Sepsis | Infections and infestations | Systematic Assessment |
| ||
| Staphylococcal infection | Infections and infestations | Systematic Assessment |
| ||
| BK virus infection | Infections and infestations | Systematic Assessment |
| ||
| Cytomegalovirus viraemia | Infections and infestations | Systematic Assessment |
| ||
| Lung infection | Infections and infestations | Systematic Assessment |
| ||
| Pneumonia fungal | Infections and infestations | Systematic Assessment |
| ||
| Staphylococcal bacteraemia | Infections and infestations | Systematic Assessment |
| ||
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Acute respiratory distress syndrome | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Cough | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Organising pneumonia | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Pulmonary alveolar haemorrhage | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Pulmonary infarction | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Pulmonary mass | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Pulmonary oedema | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Hypoxia | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Abdominal pain | Gastrointestinal disorders | Systematic Assessment |
| ||
| Jejunal perforation | Gastrointestinal disorders | Systematic Assessment |
| ||
| Lower gastrointestinal haemorrhage | Gastrointestinal disorders | Systematic Assessment |
| ||
| Pancreatitis | Gastrointestinal disorders | Systematic Assessment |
| ||
| Pneumatosis intestinalis | Gastrointestinal disorders | Systematic Assessment |
| ||
| Diarrhoea | Gastrointestinal disorders | Systematic Assessment |
| ||
| Colitis | Gastrointestinal disorders | Systematic Assessment |
| ||
| Nausea | Gastrointestinal disorders | Systematic Assessment |
| ||
| Vomiting | Gastrointestinal disorders | Systematic Assessment |
| ||
| Acute graft versus host disease in intestine | Immune system disorders | Systematic Assessment |
| ||
| Graft versus host disease in gastrointestinal tract | Immune system disorders | Systematic Assessment |
| ||
| Hypersensitivity | Immune system disorders | Systematic Assessment |
| ||
| Acute graft versus host disease in liver | Immune system disorders | Systematic Assessment |
| ||
| Graft versus host disease | Immune system disorders | Systematic Assessment |
| ||
| Haematuria | Renal and urinary disorders | Systematic Assessment |
| ||
| Acute kidney injury | Renal and urinary disorders | Systematic Assessment |
| ||
| Hypertension | Vascular disorders | Systematic Assessment |
| ||
| Hypotension | Vascular disorders | Systematic Assessment |
| ||
| Infusion related reaction | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Transplant failure | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Subdural haematoma | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Encephalopathy | Nervous system disorders | Systematic Assessment |
| ||
| Haemorrhage intracranial | Nervous system disorders | Systematic Assessment |
| ||
| Headache | Nervous system disorders | Systematic Assessment |
| ||
| Neuropathy peripheral | Nervous system disorders | Systematic Assessment |
| ||
| Posterior reversible encephalopathy syndrome | Nervous system disorders | Systematic Assessment |
| ||
| Seizure | Nervous system disorders | Systematic Assessment |
| ||
| Atrial flutter | Cardiac disorders | Systematic Assessment |
| ||
| Cardiac arrest | Cardiac disorders | Systematic Assessment |
| ||
| Cardiac failure | Cardiac disorders | Systematic Assessment |
| ||
| Pericardial effusion | Cardiac disorders | Systematic Assessment |
| ||
| Sinus bradycardia | Cardiac disorders | Systematic Assessment |
| ||
| Tachycardia | Cardiac disorders | Systematic Assessment |
| ||
| Disseminated intravascular coagulation | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Lymphatic disorder | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Febrile neutropenia | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Alanine aminotransferase increased | Investigations | Systematic Assessment |
| ||
| Aspartate aminotransferase increased | Investigations | Systematic Assessment |
| ||
| Blood bilirubin increased | Investigations | Systematic Assessment |
| ||
| Platelet count decreased | Investigations | Systematic Assessment |
| ||
| Hepatic failure | Hepatobiliary disorders | Systematic Assessment |
| ||
| Hyperuricaemia | Metabolism and nutrition disorders | Systematic Assessment |
| ||
| Hypokalemia | Metabolism and nutrition disorders | Systematic Assessment |
| ||
| Hypophosphataemia | Metabolism and nutrition disorders | Systematic Assessment |
| ||
| Delirium | Psychiatric disorders | Systematic Assessment |
| ||
| Skin ulcer | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Decreased appetite | Metabolism and nutrition disorders | Systematic Assessment |
| ||
| Hypokalemia | Metabolism and nutrition disorders | Systematic Assessment |
| ||
| Hyperglycaemia | Metabolism and nutrition disorders | Systematic Assessment |
| ||
| Hyponatraemia | Metabolism and nutrition disorders | Systematic Assessment |
| ||
| Hypophosphataemia | Metabolism and nutrition disorders | Systematic Assessment |
| ||
| Hypertriglyceridaemia | Metabolism and nutrition disorders | Systematic Assessment |
| ||
| Hyperkalaemia | Metabolism and nutrition disorders | Systematic Assessment |
| ||
| Hypocalcaemia | Metabolism and nutrition disorders | Systematic Assessment |
| ||
| Acidosis | Metabolism and nutrition disorders | Systematic Assessment |
| ||
| Hypermagnesaemia | Metabolism and nutrition disorders | Systematic Assessment |
| ||
| Hypernatraemia | Metabolism and nutrition disorders | Systematic Assessment |
| ||
| Iron overload | Metabolism and nutrition disorders | Systematic Assessment |
| ||
| Dehydration | Metabolism and nutrition disorders | Systematic Assessment |
| ||
| Hyperuricaemia | Metabolism and nutrition disorders | Systematic Assessment |
| ||
| Hypoalbuminaemia | Metabolism and nutrition disorders | Systematic Assessment |
| ||
| Hypomagnesaemia | Metabolism and nutrition disorders | Systematic Assessment |
| ||
| Colitis | Gastrointestinal disorders | Systematic Assessment |
| ||
| Enterocolitis | Gastrointestinal disorders | Systematic Assessment |
| ||
| Gastritis | Gastrointestinal disorders | Systematic Assessment |
| ||
| Gastrointestinal pain | Gastrointestinal disorders | Systematic Assessment |
| ||
| Ileus | Gastrointestinal disorders | Systematic Assessment |
| ||
| Large intestinal haemorrhage | Gastrointestinal disorders | Systematic Assessment |
| ||
| Gastrointestinal haemorrhage | Gastrointestinal disorders | Systematic Assessment |
| ||
| Impaired gastric emptying | Gastrointestinal disorders | Systematic Assessment |
| ||
| Lower gastrointestinal haemorrhage | Gastrointestinal disorders | Systematic Assessment |
| ||
| Oesophagitis | Gastrointestinal disorders | Systematic Assessment |
| ||
| Periodontal disease | Gastrointestinal disorders | Systematic Assessment |
| ||
| Proctitis | Gastrointestinal disorders | Systematic Assessment |
| ||
| Stomatitis | Gastrointestinal disorders | Systematic Assessment |
| ||
| Nausea | Gastrointestinal disorders | Systematic Assessment |
| ||
| Diarrhoea | Gastrointestinal disorders | Systematic Assessment |
| ||
| Vomiting | Gastrointestinal disorders | Systematic Assessment |
| ||
| Abdominal pain | Gastrointestinal disorders | Systematic Assessment |
| ||
| Embolism | Vascular disorders | Systematic Assessment |
| ||
| Hypertension | Vascular disorders | Systematic Assessment |
| ||
| Hypotension | Vascular disorders | Systematic Assessment |
| ||
| Rash maculo-papular | Skin and subcutaneous tissue disorders | Systematic Assessment |
| ||
| Rash | Skin and subcutaneous tissue disorders | Systematic Assessment |
| ||
| Pruritis | Skin and subcutaneous tissue disorders | Systematic Assessment |
| ||
| Rash macular | Skin and subcutaneous tissue disorders | Systematic Assessment |
| ||
| Rash pruritic | Skin and subcutaneous tissue disorders | Systematic Assessment |
| ||
| Skin ulcer | Skin and subcutaneous tissue disorders | Systematic Assessment |
| ||
| Enterococcal infection | Infections and infestations | Systematic Assessment |
| ||
| Adenovirus infection | Infections and infestations | Systematic Assessment |
| ||
| Alpha haemolytic streptococcal infection | Infections and infestations | Systematic Assessment |
| ||
| Cystitis | Infections and infestations | Systematic Assessment |
| ||
| Urinary tract infection | Infections and infestations | Systematic Assessment |
| ||
| BK virus infection | Infections and infestations | Systematic Assessment |
| ||
| Device related infection | Infections and infestations | Systematic Assessment |
| ||
| Sinusitis | Infections and infestations | Systematic Assessment |
| ||
| Skin infection | Infections and infestations | Systematic Assessment |
| ||
| Candida infection | Infections and infestations | Systematic Assessment |
| ||
| Encephalitis | Infections and infestations | Systematic Assessment |
| ||
| Escherichia infection | Infections and infestations | Systematic Assessment |
| ||
| Human polyomavirus infection | Infections and infestations | Systematic Assessment |
| ||
| Pseudomonas infection | Infections and infestations | Systematic Assessment |
| ||
| Sepsis | Infections and infestations | Systematic Assessment |
| ||
| Stomatococcal infection | Infections and infestations | Systematic Assessment |
| ||
| Upper respiratory tract infection | Infections and infestations | Systematic Assessment |
| ||
| Actinomycotic sepsis | Infections and infestations | Systematic Assessment |
| ||
| Administration site cellulitis | Infections and infestations | Systematic Assessment |
| ||
| Bacillus infection | Infections and infestations | Systematic Assessment |
| ||
| Bacteraemia | Infections and infestations | Systematic Assessment |
| ||
| Coronavirus infection | Infections and infestations | Systematic Assessment |
| ||
| Corynebacterium infection | Infections and infestations | Systematic Assessment |
| ||
| Cytomegalovirus colitis | Infections and infestations | Systematic Assessment |
| ||
| Enterobacter infection | Infections and infestations | Systematic Assessment |
| ||
| Enterocolitis infection | Infections and infestations | Systematic Assessment |
| ||
| Escherichia bacteraemia | Infections and infestations | Systematic Assessment |
| ||
| Fusobacterium infection | Infections and infestations | Systematic Assessment |
| ||
| Gastric infection | Infections and infestations | Systematic Assessment |
| ||
| Influenza | Infections and infestations | Systematic Assessment |
| ||
| Leuconostoc infection | Infections and infestations | Systematic Assessment |
| ||
| Lip infection | Infections and infestations | Systematic Assessment |
| ||
| Osteomyelitis | Infections and infestations | Systematic Assessment |
| ||
| Wound infection | Infections and infestations | Systematic Assessment |
| ||
| Staphylococcal infection | Infections and infestations | Systematic Assessment |
| ||
| Lung infection | Infections and infestations | Systematic Assessment |
| ||
| Human herpesvirus 6 infection | Infections and infestations | Systematic Assessment |
| ||
| Cytomegalovirus infection | Infections and infestations | Systematic Assessment |
| ||
| Streptococcal infection | Infections and infestations | Systematic Assessment |
| ||
| Bacterial infection | Infections and infestations | Systematic Assessment |
| ||
| Clostridium difficile infection | Infections and infestations | Systematic Assessment |
| ||
| Blood bilirubin increased | Investigations | Systematic Assessment |
| ||
| Alanine aminotransferase increased | Investigations | Systematic Assessment |
| ||
| Aspartate aminotransferase increased | Investigations | Systematic Assessment |
| ||
| Blood aluminium increased | Investigations | Systematic Assessment |
| ||
| Blood alkaline phosphatase increased | Investigations | Systematic Assessment |
| ||
| Weight decreased | Investigations | Systematic Assessment |
| ||
| White blood cell count increased | Investigations | Systematic Assessment |
| ||
| Electrocardiogram QT prolonged | Investigations | Systematic Assessment |
| ||
| Urine output decreased | Investigations | Systematic Assessment |
| ||
| Weight increased | Investigations | Systematic Assessment |
| ||
| Platelet count decreased | Investigations | Systematic Assessment |
| ||
| Neutrophil count decreased | Investigations | Systematic Assessment |
| ||
| White blood cell count decreased | Investigations | Systematic Assessment |
| ||
| Blood creatinine increased | Investigations | Systematic Assessment |
| ||
| Pharyngeal inflammation | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Pneumonitis | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Acute respiratory distress syndrome | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Cough | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Organising pneumonia | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Pneumothorax | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Pulmonary oedema | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Stridor | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Epistaxis | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Hypoxia | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Seizure | Nervous system disorders | Systematic Assessment |
| ||
| Tremor | Nervous system disorders | Systematic Assessment |
| ||
| Cognitive disorder | Nervous system disorders | Systematic Assessment |
| ||
| Dysaesthesia | Nervous system disorders | Systematic Assessment |
| ||
| Myoclonus | Nervous system disorders | Systematic Assessment |
| ||
| Somnolence | Nervous system disorders | Systematic Assessment |
| ||
| Headache | Nervous system disorders | Systematic Assessment |
| ||
| Peripheral motor neuropathy | Nervous system disorders | Systematic Assessment |
| ||
| Syncope | Nervous system disorders | Systematic Assessment |
| ||
| Fatigue | General disorders | Systematic Assessment |
| ||
| Oedema peripheral | General disorders | Systematic Assessment |
| ||
| Chills | General disorders | Systematic Assessment |
| ||
| Mucosal inflammation | General disorders | Systematic Assessment |
| ||
| Multiple organ dysfunction syndrome | General disorders | Systematic Assessment |
| ||
| Pneumatosis | General disorders | Systematic Assessment |
| ||
| Pain | Gastrointestinal disorders | Systematic Assessment |
| ||
| Pyrexia | Gastrointestinal disorders | Systematic Assessment |
| ||
| Haemolytic uraemic syndrome | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Febrile neutropenia | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Thrombotic microangiopathy | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Thrombotic thrombocytopenic purpura | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Haemolysis | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Anaemia | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Acute kidney injury | Renal and urinary disorders | Systematic Assessment |
| ||
| Chronic kidney disease | Renal and urinary disorders | Systematic Assessment |
| ||
| Urinary tract pain | Renal and urinary disorders | Systematic Assessment |
| ||
| Haematuria | Renal and urinary disorders | Systematic Assessment |
| ||
| Depression | Psychiatric disorders | Systematic Assessment |
| ||
| Insomnia | Psychiatric disorders | Systematic Assessment |
| ||
| Anxiety | Psychiatric disorders | Systematic Assessment |
| ||
| Confusional state | Psychiatric disorders | Systematic Assessment |
| ||
| Delirium | Psychiatric disorders | Systematic Assessment |
| ||
| Bone pain | Musculoskeletal and connective tissue disorders | Systematic Assessment |
| ||
| Back pain | Musculoskeletal and connective tissue disorders | Systematic Assessment |
| ||
| Muscular weakness | Musculoskeletal and connective tissue disorders | Systematic Assessment |
| ||
| Myalgia | Musculoskeletal and connective tissue disorders | Systematic Assessment |
| ||
| Pain in extremity | Musculoskeletal and connective tissue disorders | Systematic Assessment |
| ||
| Pericardial effusion | Cardiac disorders | Systematic Assessment |
| ||
| Cardiac failure | Cardiac disorders | Systematic Assessment |
| ||
| Bradycardia | Cardiac disorders | Systematic Assessment |
| ||
| Myocardial infarction | Cardiac disorders | Systematic Assessment |
| ||
| Tachycardia | Cardiac disorders | Systematic Assessment |
| ||
| Menorrhagia | Reproductive system and breast disorders | Systematic Assessment |
| ||
| Vulvovaginal inflammation | Reproductive system and breast disorders | Systematic Assessment |
| ||
| Vaginal haemorrhage | Reproductive system and breast disorders | Systematic Assessment |
| ||
| Infusion related reaction | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Post procedural haemorrhage | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Radiation skin injury | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Tracheal haemorrhage | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Adrenal insufficiency | Endocrine disorders | Systematic Assessment |
| ||
| Anaphylactic reaction | Immune system disorders | Systematic Assessment |
| ||
| Graft versus host disease | Immune system disorders | Systematic Assessment |
|
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Chief Scientific Officer | Deverra Therapeutics | 206-519-5304 | cdelaney@deverratx.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Feb 26, 2019 | Oct 10, 2019 | SAP_001.pdf |
| ID | Term |
|---|---|
| D015456 | Leukemia, Biphenotypic, Acute |
| D015464 | Leukemia, Myelogenous, Chronic, BCR-ABL Positive |
| 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 |
|---|---|
| D003520 | Cyclophosphamide |
| D016572 | Cyclosporine |
| C042382 | fludarabine phosphate |
| D009173 | Mycophenolic Acid |
| D013852 | Thiotepa |
| D014916 | Whole-Body Irradiation |
| D036101 | Cord Blood Stem Cell Transplantation |
| ID | Term |
|---|---|
| D010752 | Phosphoramide Mustards |
| D009588 | Nitrogen Mustard Compounds |
| D009150 | Mustard Compounds |
| D006846 | Hydrocarbons, Halogenated |
| D006838 | Hydrocarbons |
| D009930 | Organic Chemicals |
| D063088 | Phosphoramides |
| D009943 | Organophosphorus Compounds |
| D003524 | Cyclosporins |
| 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 |
| D005227 | Fatty Acids |
| D008055 | Lipids |
| D013721 | Triethylenephosphoramide |
| D001388 | Aziridines |
| D001389 | Azirines |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
| 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 |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| 2 Cord Blood Units |
|
| OG001 | Arm II (Experimental) | CONDITIONING REGIMEN: Patients receive the conditioning regimen chosen by the attending physician as in Standard of Care Arm. TRANSPLANT: Patients undergo single-unit or double-unit unmanipulated UCB transplant on day 0. Patients also undergo infusion of ex vivo-expanded cord blood progenitor cell infusion at least 4 hours after completion of UCB transplant. GVHD PROPHYLAXIS: Patients receive cyclosporine IV or PO and mycophenolate mofetil IV or PO as in Standard of Care Arm. Cyclophosphamide: Given IV Cyclosporine: Given IV or PO Ex Vivo-Expanded Cord Blood Progenitor Cell Infusion: Given IV Fludarabine Phosphate: Given IV Mycophenolate Mofetil: Given IV or PO Thiotepa: Given IV Total-Body Irradiation: Undergo high dose or middle intensity TBI Umbilical Cord Blood Transplantation: Undergo single-unit or double-unit unmanipulated umbilical cord blood transplant |
|
|
|
|
|
|
|
|
|
|