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| ID | Type | Description | Link |
|---|---|---|---|
| NCI-2010-01277 | Registry Identifier | CTRP (Clinical Trial Reporting Program) | |
| 2256.00 | Other Identifier | Fred Hutch/University of Washington Cancer Consortium | |
| P30CA015704 | U.S. NIH Grant/Contract | View source | |
| RG2809001 | Other Identifier | Fred Hutch/University of Washington Cancer Consortium |
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| Name | Class |
|---|---|
| medac GmbH | INDUSTRY |
| National Cancer Institute (NCI) | NIH |
| National Heart, Lung, and Blood Institute (NHLBI) | NIH |
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This phase II clinical trial studies how well treosulfan and fludarabine phosphate with or without low dose radiation before donor stem cell transplantation works in treating patients with nonmalignant (noncancerous) diseases. Hematopoietic cell transplantation has been shown to be curative for many patients with nonmalignant (noncancerous) diseases such as primary immunodeficiency disorders, bone marrow failure syndromes, hemoglobinopathies, and inborn errors of metabolism (metabolic disorders). Powerful chemotherapy drugs and/or radiation are often used to condition the patient before infusion of the new healthy donor cells. The purpose of the conditioning therapy is to destroy the patient's abnormal bone marrow which doesn't work properly in order to make way for the new healthy donor cells which functions normally. Although effective in curing the patient's disease, many hematopoietic cell transplantation regimens use intensive chemotherapy and/or radiation which can be quite toxic, have significant side effects, and can potentially be life-threatening. Investigators are investigating whether a new conditioning regimen that uses less intensive drugs (treosulfan and fludarabine phosphate) with or without low dose radiation results in new blood-forming cells (engraftment) of the new donor cells without increased toxicities in patients with nonmalignant (noncancerous) diseases.
OUTLINE:
CONDITIONING REGIMEN: Patients receive treosulfan intravenously (IV) over 2 hours on days -6 to -4 and fludarabine phosphate IV over 1 hour on days -6 to -2. Patients receive anti-thymocyte globulin IV over 4-6 hours on days -4 to -2. Patients undergoing umbilical cord blood transplantation will also receive low dose total-body irradiation on day -1.
TRANSPLANTATION: Patients receive either bone marrow, peripheral blood stem cells (PBSC), or umbilical cord blood (UCB) from the donor on day 0. The use of either bone marrow, PBSC, or umbilical cord blood will depend on the donor status.
IMMUNOSUPPRESSION: Patients receive a combination of immunosuppressive medications to try and prevent graft-versus-host disease. There are 2 regimens depending on the donor.
Regimen A: Patients undergoing bone marrow or PBSC transplantation receive tacrolimus daily from day -1 to 50 followed by a taper until day 180 in the absence of GVHD. Patients also receive methotrexate IV on days 1, 3, 6, and 11.
Regimen B: Patients undergoing UCB transplantation receive cyclosporine on days -3 to 100 followed by a taper until day 180 in the absence of GVHD. Patients also receive mycophenolate mofetil on days 0 to 40 followed by a taper until day 96 in the absence of GVHD.
After completion of study treatment, patients are followed up periodically for 5 years.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Regimen A (PBSCT and BMT) | Experimental | CONDITIONING REGIMEN A : Patients receive treosulfan IV on days -6 to -4 and fludarabine phosphate IV on days -6 to -2. Patients receive anti-thymocyte globulin IV on days -4 to -2. Patients undergoing umbilical cord blood transplantation will also receive low dose total-body irradiation on day -1. TRANSPLANTATION: Patients receive either bone marrow, peripheral blood stem cells (PBSC), or umbilical cord blood from the donor on day 0. The use of either bone marrow, PBSC, or umbilical cord blood will depend on the donor status. Patients undergoing bone marrow or PBSC transplantation receive tacrolimus IV continuously or PO twice daily on days -1 to 50 followed by a taper until day 180 in the absence of GVHD. Patients also receive methotrexate IV on days 1, 3, 6, and 11. |
|
| Regimen B (UBCT) | Experimental | CONDITIONING REGIMEN B: Patients receive treosulfan IV on days -6 to -4 and fludarabine phosphate IV on days -6 to -2. Patients receive anti-thymocyte globulin IV on days -4 to -2. Patients undergoing umbilical cord blood transplantation will also receive low dose total-body irradiation on day -1 . TRANSPLANTATION: Patients receive either bone marrow, peripheral blood stem cells (PBSC), or umbilical cord blood from the donor on day 0. The use of either bone marrow, PBSC, or umbilical cord blood will depend on the donor status. Patients undergoing UCB transplantation receive cyclosporine IV over 1 hour every 8-12 hours on days -3 to 100 followed by a taper until day 180 in the absence of GVHD. Patients also receive mycophenolate mofetil IV or PO every 8 hours on days 0 to 40 followed by a taper until day 96 in the absence of GVHD. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Allogeneic Bone Marrow Transplantation | Procedure | Infused IV |
|
| Measure | Description | Time Frame |
|---|---|---|
| Preliminary Efficacy | Number of patients engrafted (>5% donor CD3+ peripheral blood chimerisms) at 1 year following transplant | 1 year following transplant |
| Measure | Description | Time Frame |
|---|---|---|
| Non-relapse Mortality | Number of patients who experienced non-relapse mortality by 1 year following transplant | 1 year following transplant |
| Number of Patients With Grade II-IV Acute Graft-versus-host Disease |
Not provided
Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Lauri M. Burroughs | Fred Hutch/University of Washington Cancer Consortium | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's Hospital Colorado | Aurora | Colorado | 80045 | United States | ||
| Oregon Health and Science University |
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| ID | Title | Description |
|---|---|---|
| FG000 | Regimen A (PBSCT and BMT) | CONDITIONING REGIMEN A : Patients receive treosulfan IV on days -6 to -4 and fludarabine phosphate IV on days -6 to -2. Patients receive anti-thymocyte globulin IV on days -4 to -2. Patients undergoing umbilical cord blood transplantation will also receive low dose total-body irradiation on day -1. TRANSPLANTATION: Patients receive either bone marrow, peripheral blood stem cells (PBSC), or umbilical cord blood from the donor on day 0. The use of either bone marrow, PBSC, or umbilical cord blood will depend on the donor status. Patients undergoing bone marrow or PBSC transplantation receive tacrolimus IV continuously or PO twice daily on days -1 to 50 followed by a taper until day 180 in the absence of GVHD. Patients also receive methotrexate IV on days 1, 3, 6, and 11. Allogeneic Bone Marrow Transplantation: Infused IV Anti-Thymocyte Globulin: Given IV Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Methotrexate: Given IV Peripheral Blood Stem Cell Transplantation: Infused IV Tacrolimus: Given IV or PO Treosulfan: Given IV |
| 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 | May 21, 2020 |
Not provided
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| Anti-Thymocyte Globulin | Biological | Given IV |
|
|
| Cyclosporine | Drug | Given IV or PO |
|
|
| Fludarabine Phosphate | Drug | Given IV |
|
|
| Laboratory Biomarker Analysis | Other | Correlative studies |
|
| Methotrexate | Drug | Given IV |
|
|
| Mycophenolate Mofetil | Drug | Given IV or PO |
|
|
| Peripheral Blood Stem Cell Transplantation | Procedure | Infused IV |
|
|
| Tacrolimus | Drug | Given IV or PO |
|
|
| Total-Body Irradiation | Radiation | Undergo total body irradiation |
|
|
| Treosulfan | Drug | Given IV |
|
|
| Umbilical Cord Blood Transplantation | Procedure | Single or double unit umbilical cord blood transplant, infused IV |
|
|
Number of patients diagnosed with overall grade II-IV acute GVHD by Day 100 post transplant
| Day 100 post transplant |
| Number of Patients With of Chronic Graft-versus-host Disease | Number of patients diagnosed with chronic GVHD and requiring systemic immunosuppression within 1 year following transplant | 1 year following transplant |
| Donor Chimerism CD3 at 100 Days Post Transplant | Number of patients with peripheral blood donor chimerism for CD3 less than 5%, 5-49%, 50-94% and greater than or equal to 95% at 100 days post transplant. | Day 100 post transplant |
| Disease Response at One Year Following Hematopoietic Cell Transplantation | Number of patients with no evidence of disease at one year following transplant | 1 year following transplant |
| Immune Reconstitution Following Hematopoietic Cell Transplantation | Number of patients with immune reconstitution (defined by a normal range CD3) at 1 year post transplant | 1 year following transplant |
| Number of Participants With Infections | Number of participants with clinically significant infections (bacterial, fungal, viral) requiring treatment within 100 days following transplant | 100 days post transplant |
| Overall Survival | Number of patients alive at 1 year following transplant | 1 year following transplant |
| Donor Chimerism CD33 at Day 100 Post Transplant | Number of patients with peripheral blood donor chimerism for CD33 less than 5%, 5-49%, 50-94% and greater than or equal to 95% at 100 days post transplant. | 100 days post transplant |
| Portland |
| Oregon |
| 97239 |
| United States |
| Vanderbilt University/Ingram Cancer Center | Nashville | Tennessee | 37232 | United States |
| Seattle Children's Hospital | Seattle | Washington | 98105 | United States |
| Fred Hutch/University of Washington Cancer Consortium | Seattle | Washington | 98109 | United States |
| Children's Hospital of Wisconsin | Milwaukee | Wisconsin | 53226 | United States |
| FG001 | Regimen B (UBCT) | CONDITIONING REGIMEN B: Patients receive treosulfan IV on days -6 to -4 and fludarabine phosphate IV on days -6 to -2. Patients receive anti-thymocyte globulin IV on days -4 to -2. Patients undergoing umbilical cord blood transplantation will also receive low dose total-body irradiation on day -1 . TRANSPLANTATION: Patients receive either bone marrow, peripheral blood stem cells (PBSC), or umbilical cord blood from the donor on day 0. The use of either bone marrow, PBSC, or umbilical cord blood will depend on the donor status. Patients undergoing UCB transplantation receive cyclosporine IV over 1 hour every 8-12 hours on days -3 to 100 followed by a taper until day 180 in the absence of GVHD. Patients also receive mycophenolate mofetil IV or PO every 8 hours on days 0 to 40 followed by a taper until day 96 in the absence of GVHD. Anti-Thymocyte Globulin: Given IV Cyclosporine: Given IV or PO Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given IV or PO Total-Body Irradiation: Undergo total body irradiation Treosulfan: Given IV Umbilical Cord Blood Transplantation: Single or double unit umbilical cord blood transplant, infused IV |
| COMPLETED |
|
| NOT COMPLETED |
|
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Regimen A (PBSCT and BMT) | CONDITIONING REGIMEN A : Patients receive treosulfan IV on days -6 to -4 and fludarabine phosphate IV on days -6 to -2. Patients receive anti-thymocyte globulin IV on days -4 to -2. Patients undergoing umbilical cord blood transplantation will also receive low dose total-body irradiation on day -1. TRANSPLANTATION: Patients receive either bone marrow, peripheral blood stem cells (PBSC), or umbilical cord blood from the donor on day 0. The use of either bone marrow, PBSC, or umbilical cord blood will depend on the donor status. Patients undergoing bone marrow or PBSC transplantation receive tacrolimus IV continuously or PO twice daily on days -1 to 50 followed by a taper until day 180 in the absence of GVHD. Patients also receive methotrexate IV on days 1, 3, 6, and 11. Allogeneic Bone Marrow Transplantation: Infused IV Anti-Thymocyte Globulin: Given IV Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Methotrexate: Given IV Peripheral Blood Stem Cell Transplantation: Infused IV Tacrolimus: Given IV or PO Treosulfan: Given IV |
| BG001 | Regimen B (UBCT) | CONDITIONING REGIMEN B: Patients receive treosulfan IV on days -6 to -4 and fludarabine phosphate IV on days -6 to -2. Patients receive anti-thymocyte globulin IV on days -4 to -2. Patients undergoing umbilical cord blood transplantation will also receive low dose total-body irradiation on day -1 . TRANSPLANTATION: Patients receive either bone marrow, peripheral blood stem cells (PBSC), or umbilical cord blood from the donor on day 0. The use of either bone marrow, PBSC, or umbilical cord blood will depend on the donor status. Patients undergoing UCB transplantation receive cyclosporine IV over 1 hour every 8-12 hours on days -3 to 100 followed by a taper until day 180 in the absence of GVHD. Patients also receive mycophenolate mofetil IV or PO every 8 hours on days 0 to 40 followed by a taper until day 96 in the absence of GVHD. Anti-Thymocyte Globulin: Given IV Cyclosporine: Given IV or PO Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given IV or PO Total-Body Irradiation: Undergo total body irradiation Treosulfan: Given IV Umbilical Cord Blood Transplantation: Single or double unit umbilical cord blood transplant, infused IV |
| 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 |
| ||||||||||||||||||
| 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 |
|
| 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 | Preliminary Efficacy | Number of patients engrafted (>5% donor CD3+ peripheral blood chimerisms) at 1 year following transplant | Regimen B: Three patients expired without CD3+ chimerisms being performed, therefore could not be analyzed for primary efficacy | Posted | Count of Participants | Participants | 1 year following transplant |
|
|
| |||||||||||||||||||||||||||||
| Secondary | Non-relapse Mortality | Number of patients who experienced non-relapse mortality by 1 year following transplant | Posted | Count of Participants | Participants | 1 year following transplant |
| ||||||||||||||||||||||||||||||||
| Secondary | Number of Patients With Grade II-IV Acute Graft-versus-host Disease | Number of patients diagnosed with overall grade II-IV acute GVHD by Day 100 post transplant | Regimen B: 2 patients expired too early for evaluation for acute GVHD and could not be evaluated for this outcome | Posted | Count of Participants | Participants | Day 100 post transplant |
| |||||||||||||||||||||||||||||||
| Secondary | Number of Patients With of Chronic Graft-versus-host Disease | Number of patients diagnosed with chronic GVHD and requiring systemic immunosuppression within 1 year following transplant | Regimen B: 3 patients expired too early for evaluation for chronic GVHD and could not be evaluated for this outcome | Posted | Count of Participants | Participants | 1 year following transplant |
| |||||||||||||||||||||||||||||||
| Secondary | Donor Chimerism CD3 at 100 Days Post Transplant | Number of patients with peripheral blood donor chimerism for CD3 less than 5%, 5-49%, 50-94% and greater than or equal to 95% at 100 days post transplant. | Regimen B: 3 patients expired without CD3+ chimerisms being performed and could not be evaluated for this outcome | Posted | Count of Participants | Participants | Day 100 post transplant |
| |||||||||||||||||||||||||||||||
| Secondary | Disease Response at One Year Following Hematopoietic Cell Transplantation | Number of patients with no evidence of disease at one year following transplant | Regimen B: 3 patients expired prior to disease response being evaluated and could not be evaluated for this outcome | Posted | Count of Participants | Participants | 1 year following transplant |
| |||||||||||||||||||||||||||||||
| Secondary | Immune Reconstitution Following Hematopoietic Cell Transplantation | Number of patients with immune reconstitution (defined by a normal range CD3) at 1 year post transplant | Regimen A: 11 patients did not have lymphocyte subsets drawn post-transplant and could not be evaluated for outcome; Regimen B: 3 patients expired prior to lymphocyte subsets being evaluated and 2 patients did not have lymphocyte subsets drawn post-transplant and could not be evaluated for outcome | Posted | Count of Participants | Participants | 1 year following transplant |
| |||||||||||||||||||||||||||||||
| Secondary | Number of Participants With Infections | Number of participants with clinically significant infections (bacterial, fungal, viral) requiring treatment within 100 days following transplant | Posted | Count of Participants | Participants | 100 days post transplant |
| ||||||||||||||||||||||||||||||||
| Secondary | Overall Survival | Number of patients alive at 1 year following transplant | Posted | Count of Participants | Participants | 1 year following transplant |
| ||||||||||||||||||||||||||||||||
| Secondary | Donor Chimerism CD33 at Day 100 Post Transplant | Number of patients with peripheral blood donor chimerism for CD33 less than 5%, 5-49%, 50-94% and greater than or equal to 95% at 100 days post transplant. | Regimen B: 2 patients expired without CD33+ chimerisms being performed and could not be evaluated for this outcome | Posted | Count of Participants | Participants | 100 days post transplant |
|
Day 100 post initiation of conditioning therapy
Adverse Events were collected 100 days post initiation of conditioning therapy. All-Cause Mortality was assessed through 10.6 years post transplant (through study completion date 6/10/2020).
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 | Regimen A (PBSCT and BMT) | CONDITIONING REGIMEN A : Patients receive treosulfan IV on days -6 to -4 and fludarabine phosphate IV on days -6 to -2. Patients receive anti-thymocyte globulin IV on days -4 to -2. Patients undergoing umbilical cord blood transplantation will also receive low dose total-body irradiation on day -1. TRANSPLANTATION: Patients receive either bone marrow, peripheral blood stem cells (PBSC), or umbilical cord blood from the donor on day 0. The use of either bone marrow, PBSC, or umbilical cord blood will depend on the donor status. Patients undergoing bone marrow or PBSC transplantation receive tacrolimus IV continuously or PO twice daily on days -1 to 50 followed by a taper until day 180 in the absence of GVHD. Patients also receive methotrexate IV on days 1, 3, 6, and 11. Allogeneic Bone Marrow Transplantation: Infused IV Anti-Thymocyte Globulin: Given IV Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Methotrexate: Given IV Peripheral Blood Stem Cell Transplantation: Infused IV Tacrolimus: Given IV or PO Treosulfan: Given IV | 7 | 84 | 17 | 84 | 53 | 84 |
| EG001 | Regimen B (UBCT) | CONDITIONING REGIMEN B: Patients receive treosulfan IV on days -6 to -4 and fludarabine phosphate IV on days -6 to -2. Patients receive anti-thymocyte globulin IV on days -4 to -2. Patients undergoing umbilical cord blood transplantation will also receive low dose total-body irradiation on day -1 . TRANSPLANTATION: Patients receive either bone marrow, peripheral blood stem cells (PBSC), or umbilical cord blood from the donor on day 0. The use of either bone marrow, PBSC, or umbilical cord blood will depend on the donor status. Patients undergoing UCB transplantation receive cyclosporine IV over 1 hour every 8-12 hours on days -3 to 100 followed by a taper until day 180 in the absence of GVHD. Patients also receive mycophenolate mofetil IV or PO every 8 hours on days 0 to 40 followed by a taper until day 96 in the absence of GVHD. Anti-Thymocyte Globulin: Given IV Cyclosporine: Given IV or PO Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given IV or PO Total-Body Irradiation: Undergo total body irradiation Treosulfan: Given IV Umbilical Cord Blood Transplantation: Single or double unit umbilical cord blood transplant, infused IV | 6 | 14 | 5 | 14 | 10 | 14 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Gastrointestinal Other: Pneumatosis | Gastrointestinal disorders | Systematic Assessment | grade 3 |
| |
| Pneumonitis | Respiratory, thoracic and mediastinal disorders | Systematic Assessment | grade 4 |
| |
| Sepsis with multiorgan failure | Infections and infestations | Systematic Assessment | grade 5 |
| |
| Apnea: Requiring intubation following BAL; attributed to underlying infection | Respiratory, thoracic and mediastinal disorders | Systematic Assessment | grade 4 |
| |
| Anaphylaxis reaction (rATG) | Immune system disorders | Systematic Assessment | grade 4 |
| |
| Cardiac Asystole following central venous catheter placement | Cardiac disorders | Systematic Assessment | grade 5 |
| |
| Hypotension: requiring intervention | Vascular disorders | Systematic Assessment | grade 3 |
| |
| Hypotension: shock, pressors | Vascular disorders | Systematic Assessment | grade 4 |
| |
| Abdominal Pain | Gastrointestinal disorders | Systematic Assessment | grade 3 |
| |
| Nausea | Gastrointestinal disorders | Systematic Assessment | grade 3 |
| |
| Diarrhea (associated with GVHD): >15ml/kg per day with abdominal pain | Gastrointestinal disorders | Systematic Assessment | grade 4 |
| |
| Febrile neutropenia | Blood and lymphatic system disorders | Systematic Assessment | grade 3 |
| |
| Fever (not neutropenic): attributed to rATG | General disorders | Systematic Assessment | grade 3 |
| |
| Fever (not neutropenic) | General disorders | Systematic Assessment | grade 3 |
| |
| Febrile neutropenia: attributed to rATG | Blood and lymphatic system disorders | Systematic Assessment | grade 3 |
| |
| Febrile Neutropenia: with life- threatening sepsis | Blood and lymphatic system disorders | Systematic Assessment | grade 4 |
| |
| Infections and infestations -Other, specify: parainfluenza or sinusitis | Infections and infestations | Systematic Assessment |
| ||
| Infections and infestations -Other, specify: CMV meningitis/encephalo | Infections and infestations | Systematic Assessment | grade 3 |
| |
| Infections and infestations -Other, specify: Clostridium septicum sepsis | Infections and infestations | Systematic Assessment | grade 3 |
| |
| Adult respiratory distress syndrome (ARDS) | Respiratory, thoracic and mediastinal disorders | Systematic Assessment | grade 4 |
| |
| Acute kidney injury (reversible, requiring CRRT/dialysis) | Renal and urinary disorders | Systematic Assessment | grade 3 |
| |
| Dyspnea | Respiratory, thoracic and mediastinal disorders | Systematic Assessment | grade 3 |
| |
| Hypoxia: Decreased O2 sat requiring support | Respiratory, thoracic and mediastinal disorders | Systematic Assessment | grade 4 |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaphylaxis reaction: Possible reaction to rATG | Immune system disorders | Systematic Assessment | grade 3 |
| |
| Pericardial effusion: Tamponade | Cardiac disorders | Systematic Assessment | grade 4 |
| |
| Thrombotic microangiopathy (requiring intervention) with acute renal fail: Hemolytic uremic syndrome | Blood and lymphatic system disorders | Systematic Assessment | grade 4 |
| |
| Fluid retention - evidence of ascites | Gastrointestinal disorders | Systematic Assessment | grade 3 |
| |
| Diarrhea (associated with GVHD): >15ml/kg per day | Gastrointestinal disorders | Systematic Assessment | grade 3 |
| |
| Gastrointestinal disorders -Other, specify: pneumatosis | Gastrointestinal disorders | Systematic Assessment | grade 3 |
| |
| Oral mucositis: Requiring intervention | Gastrointestinal disorders | Systematic Assessment | grade 3 |
| |
| Esophageal ulcers: Presumed viral esophagitis | Gastrointestinal disorders | Systematic Assessment | grade 3 |
| |
| Pulmonary hemorrhage | Respiratory, thoracic and mediastinal disorders | Systematic Assessment | grade 3 |
| |
| Hematuria | Renal and urinary disorders | Systematic Assessment | grade 3 |
| |
| Elevated bilirubin (>3-10x ULN) | Investigations | Systematic Assessment | grade 3 |
| |
| Febrile neutropenia | Blood and lymphatic system disorders | Systematic Assessment | grade 3 |
| |
| Fever (not neutropenic): attributed to rATG | General disorders | Systematic Assessment | grade 3 |
| |
| Fever (not neutropenic) | General disorders | Systematic Assessment | grade 3 |
| |
| Infections and infestations -Other, specify: life-threatening disseminated Adenovirus | Infections and infestations | Systematic Assessment | grade 4 |
| |
| sleep apnea: Obstructive | Respiratory, thoracic and mediastinal disorders | Systematic Assessment | grade 3 |
| |
| Hypoxia: decreased O2 sat | Respiratory, thoracic and mediastinal disorders | Systematic Assessment | grade 3 |
| |
| Hypoxia: requiring intubation | Respiratory, thoracic and mediastinal disorders | Systematic Assessment | grade 4 |
| |
| Adult respiratory distress syndrome (ARDS) | Respiratory, thoracic and mediastinal disorders | Systematic Assessment | grade 4 |
| |
| Acute kidney injury (reversible, requiring CRRT/dialysis) | Renal and urinary disorders | Systematic Assessment | grade 3 |
| |
| Hemolysis (AIHA): Requiring transfusion and sterioids | Blood and lymphatic system disorders | Systematic Assessment | grade 3 |
| |
| Hypotension: requiring intervention | Vascular disorders | Systematic Assessment | grade 3 |
| |
| Cardiopulmonary arrest: Requiring intubation | Cardiac disorders | Systematic Assessment | grade 4 |
| |
| Thrombotic microangiopathy (not requiring intervention) | Blood and lymphatic system disorders | Systematic Assessment | grade 3 |
| |
| Rash (>50%) | Skin and subcutaneous tissue disorders | Systematic Assessment | grade 3 |
| |
| Colitis: Abdominal pain | Gastrointestinal disorders | Systematic Assessment | grade 3 |
| |
| Pancreatitis | Gastrointestinal disorders | Systematic Assessment | grade 3 |
| |
| Febrile neutropenia: attributed to rATG | Blood and lymphatic system disorders | Systematic Assessment | grade 3 |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | Systematic Assessment | grade 3 |
| |
| Dysfunctional urerine (menstrual) bleeding: requiring transfusion | Reproductive system and breast disorders | Systematic Assessment | grade 3 |
| |
| Oral hemorrhage: Bleeding (requiring transfusion) | Gastrointestinal disorders | Systematic Assessment | grade 3 |
| |
| nfections and infestations -Other, specify: MSSA septic shock | Infections and infestations | Systematic Assessment | grade 4 |
| |
| Infections and infestations -Other, specify: Strep viridans, enterococcus bacteremia | Infections and infestations | Systematic Assessment | grade 3 |
| |
| Reversible posterior leukoencephalopathy syndrome (PRES) | Nervous system disorders | Systematic Assessment | grade 3 |
| |
| Seizure (conciousness altered) | Nervous system disorders | Systematic Assessment | grade 3 |
| |
| Carbon monoxide diffusing capacity decreased (DLCO) | Investigations | Systematic Assessment | grade 3 |
|
Not provided
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Lauri Burroughs | Fred Hutchinson Cancer Research Center | 206-667-2396 | lburroug@fredhutch.org |
| May 12, 2021 |
| Prot_SAP_000.pdf |
| ID | Term |
|---|---|
| D000081207 | Primary Immunodeficiency Diseases |
| D004194 | Disease |
| C580192 | Immune Dysregulation, Polyendocrinopathy, Enteropathy, X-Linked Syndrome |
| D051359 | Lymphohistiocytosis, Hemophagocytic |
| D014923 | Wiskott-Aldrich Syndrome |
| D000080983 | Bone Marrow Failure Disorders |
| D000081003 | Shwachman-Diamond Syndrome |
| D019871 | Dyskeratosis Congenita |
| D029503 | Anemia, Diamond-Blackfan |
| D008661 | Metabolism, Inborn Errors |
| D008659 | Metabolic Diseases |
| D006453 | Hemoglobinopathies |
| D000755 | Anemia, Sickle Cell |
| D013789 | Thalassemia |
| ID | Term |
|---|---|
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007153 | Immunologic Deficiency Syndromes |
| D007154 | Immune System Diseases |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D015616 | Histiocytosis, Non-Langerhans-Cell |
| D015614 | Histiocytosis |
| D008206 | Lymphatic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D008231 | Lymphopenia |
| D007970 | Leukopenia |
| D000095542 | Cytopenia |
| D006474 | Hemorrhagic Disorders |
| D007960 | Leukocyte Disorders |
| D040181 | Genetic Diseases, X-Linked |
| D001855 | Bone Marrow Diseases |
| D010188 | Exocrine Pancreatic Insufficiency |
| D010182 | Pancreatic Diseases |
| D004066 | Digestive System Diseases |
| D000080984 | Congenital Bone Marrow Failure Syndromes |
| D008052 | Lipid Metabolism, Inborn Errors |
| D052439 | Lipid Metabolism Disorders |
| D009750 | Nutritional and Metabolic Diseases |
| D008068 | Lipomatosis |
| D012868 | Skin Abnormalities |
| D000013 | Congenital Abnormalities |
| D012873 | Skin Diseases, Genetic |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D029502 | Anemia, Hypoplastic, Congenital |
| D000741 | Anemia, Aplastic |
| D000740 | Anemia |
| D012010 | Red-Cell Aplasia, Pure |
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
Not provided
Not provided
| ID | Term |
|---|---|
| D000961 | Antilymphocyte Serum |
| C512542 | thymoglobulin |
| D016572 | Cyclosporine |
| D003524 | Cyclosporins |
| C042382 | fludarabine phosphate |
| D008727 | Methotrexate |
| C015342 | merphos |
| D009173 | Mycophenolic Acid |
| D036102 | Peripheral Blood Stem Cell Transplantation |
| D016559 | Tacrolimus |
| D014916 | Whole-Body Irradiation |
| C018404 | treosulfan |
| D036101 | Cord Blood Stem Cell Transplantation |
| ID | Term |
|---|---|
| D007106 | Immune Sera |
| D000906 | Antibodies |
| D007136 | Immunoglobulins |
| D007162 | Immunoproteins |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D012712 | Serum Globulins |
| D005916 | Globulins |
| D001688 | Biological Products |
| D045424 | Complex Mixtures |
| D010456 | Peptides, Cyclic |
| D047028 | Macrocyclic Compounds |
| D011083 | Polycyclic Compounds |
| D010455 | Peptides |
| D000630 | Aminopterin |
| D011622 | Pterins |
| D011621 | Pteridines |
| D006574 | Heterocyclic Compounds, 2-Ring |
| D000072471 | Heterocyclic Compounds, Fused-Ring |
| D006571 | Heterocyclic Compounds |
| D002208 | Caproates |
| D000144 | Acids, Acyclic |
| D002264 | Carboxylic Acids |
| D009930 | Organic Chemicals |
| D005227 | Fatty Acids |
| D008055 | Lipids |
| D018380 | Hematopoietic Stem Cell Transplantation |
| D033581 | Stem Cell Transplantation |
| D017690 | Cell Transplantation |
| D064987 | Cell- and Tissue-Based Therapy |
| D001691 | Biological Therapy |
| D013812 | Therapeutics |
| D014180 | Transplantation |
| D013514 | Surgical Procedures, Operative |
| D018942 | Macrolides |
| D007783 | Lactones |
| D011878 | Radiotherapy |
| D008919 | Investigative Techniques |
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 |
|
|
|
CONDITIONING REGIMEN B: Patients receive treosulfan IV on days -6 to -4 and fludarabine phosphate IV on days -6 to -2. Patients receive anti-thymocyte globulin IV on days -4 to -2. Patients undergoing umbilical cord blood transplantation will also receive low dose total-body irradiation on day -1 .
TRANSPLANTATION: Patients receive either bone marrow, peripheral blood stem cells (PBSC), or umbilical cord blood from the donor on day 0. The use of either bone marrow, PBSC, or umbilical cord blood will depend on the donor status.
Patients undergoing UCB transplantation receive cyclosporine IV over 1 hour every 8-12 hours on days -3 to 100 followed by a taper until day 180 in the absence of GVHD. Patients also receive mycophenolate mofetil IV or PO every 8 hours on days 0 to 40 followed by a taper until day 96 in the absence of GVHD.
Anti-Thymocyte Globulin: Given IV
Cyclosporine: Given IV or PO
Fludarabine Phosphate: Given IV
Laboratory Biomarker Analysis: Correlative studies
Mycophenolate Mofetil: Given IV or PO
Total-Body Irradiation: Undergo total body irradiation
Treosulfan: Given IV
Umbilical Cord Blood Transplantation: Single or double unit umbilical cord blood transplant, infused IV
|
|
CONDITIONING REGIMEN B: Patients receive treosulfan IV on days -6 to -4 and fludarabine phosphate IV on days -6 to -2. Patients receive anti-thymocyte globulin IV on days -4 to -2. Patients undergoing umbilical cord blood transplantation will also receive low dose total-body irradiation on day -1 . TRANSPLANTATION: Patients receive either bone marrow, peripheral blood stem cells (PBSC), or umbilical cord blood from the donor on day 0. The use of either bone marrow, PBSC, or umbilical cord blood will depend on the donor status. Patients undergoing UCB transplantation receive cyclosporine IV over 1 hour every 8-12 hours on days -3 to 100 followed by a taper until day 180 in the absence of GVHD. Patients also receive mycophenolate mofetil IV or PO every 8 hours on days 0 to 40 followed by a taper until day 96 in the absence of GVHD. Anti-Thymocyte Globulin: Given IV Cyclosporine: Given IV or PO Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given IV or PO Total-Body Irradiation: Undergo total body irradiation Treosulfan: Given IV Umbilical Cord Blood Transplantation: Single or double unit umbilical cord blood transplant, infused IV |
|
|
CONDITIONING REGIMEN B: Patients receive treosulfan IV on days -6 to -4 and fludarabine phosphate IV on days -6 to -2. Patients receive anti-thymocyte globulin IV on days -4 to -2. Patients undergoing umbilical cord blood transplantation will also receive low dose total-body irradiation on day -1 . TRANSPLANTATION: Patients receive either bone marrow, peripheral blood stem cells (PBSC), or umbilical cord blood from the donor on day 0. The use of either bone marrow, PBSC, or umbilical cord blood will depend on the donor status. Patients undergoing UCB transplantation receive cyclosporine IV over 1 hour every 8-12 hours on days -3 to 100 followed by a taper until day 180 in the absence of GVHD. Patients also receive mycophenolate mofetil IV or PO every 8 hours on days 0 to 40 followed by a taper until day 96 in the absence of GVHD. Anti-Thymocyte Globulin: Given IV Cyclosporine: Given IV or PO Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given IV or PO Total-Body Irradiation: Undergo total body irradiation Treosulfan: Given IV Umbilical Cord Blood Transplantation: Single or double unit umbilical cord blood transplant, infused IV |
|
|
CONDITIONING REGIMEN B: Patients receive treosulfan IV on days -6 to -4 and fludarabine phosphate IV on days -6 to -2. Patients receive anti-thymocyte globulin IV on days -4 to -2. Patients undergoing umbilical cord blood transplantation will also receive low dose total-body irradiation on day -1 .
TRANSPLANTATION: Patients receive either bone marrow, peripheral blood stem cells (PBSC), or umbilical cord blood from the donor on day 0. The use of either bone marrow, PBSC, or umbilical cord blood will depend on the donor status.
Patients undergoing UCB transplantation receive cyclosporine IV over 1 hour every 8-12 hours on days -3 to 100 followed by a taper until day 180 in the absence of GVHD. Patients also receive mycophenolate mofetil IV or PO every 8 hours on days 0 to 40 followed by a taper until day 96 in the absence of GVHD.
Anti-Thymocyte Globulin: Given IV
Cyclosporine: Given IV or PO
Fludarabine Phosphate: Given IV
Laboratory Biomarker Analysis: Correlative studies
Mycophenolate Mofetil: Given IV or PO
Total-Body Irradiation: Undergo total body irradiation
Treosulfan: Given IV
Umbilical Cord Blood Transplantation: Single or double unit umbilical cord blood transplant, infused IV
|
|
| OG001 | Regimen B (UBCT) | CONDITIONING REGIMEN B: Patients receive treosulfan IV on days -6 to -4 and fludarabine phosphate IV on days -6 to -2. Patients receive anti-thymocyte globulin IV on days -4 to -2. Patients undergoing umbilical cord blood transplantation will also receive low dose total-body irradiation on day -1 . TRANSPLANTATION: Patients receive either bone marrow, peripheral blood stem cells (PBSC), or umbilical cord blood from the donor on day 0. The use of either bone marrow, PBSC, or umbilical cord blood will depend on the donor status. Patients undergoing UCB transplantation receive cyclosporine IV over 1 hour every 8-12 hours on days -3 to 100 followed by a taper until day 180 in the absence of GVHD. Patients also receive mycophenolate mofetil IV or PO every 8 hours on days 0 to 40 followed by a taper until day 96 in the absence of GVHD. Anti-Thymocyte Globulin: Given IV Cyclosporine: Given IV or PO Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given IV or PO Total-Body Irradiation: Undergo total body irradiation Treosulfan: Given IV Umbilical Cord Blood Transplantation: Single or double unit umbilical cord blood transplant, infused IV |
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CONDITIONING REGIMEN B: Patients receive treosulfan IV on days -6 to -4 and fludarabine phosphate IV on days -6 to -2. Patients receive anti-thymocyte globulin IV on days -4 to -2. Patients undergoing umbilical cord blood transplantation will also receive low dose total-body irradiation on day -1 . TRANSPLANTATION: Patients receive either bone marrow, peripheral blood stem cells (PBSC), or umbilical cord blood from the donor on day 0. The use of either bone marrow, PBSC, or umbilical cord blood will depend on the donor status. Patients undergoing UCB transplantation receive cyclosporine IV over 1 hour every 8-12 hours on days -3 to 100 followed by a taper until day 180 in the absence of GVHD. Patients also receive mycophenolate mofetil IV or PO every 8 hours on days 0 to 40 followed by a taper until day 96 in the absence of GVHD. Anti-Thymocyte Globulin: Given IV Cyclosporine: Given IV or PO Fludarabine Phosphate: Given IV Laboratory Biomarker Analysis: Correlative studies Mycophenolate Mofetil: Given IV or PO Total-Body Irradiation: Undergo total body irradiation Treosulfan: Given IV Umbilical Cord Blood Transplantation: Single or double unit umbilical cord blood transplant, infused IV |
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