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The traditional way of doing a donor transplant is to give high doses of chemotherapy and radiation before giving the stem cells. However, high doses of chemotherapy and radiation can have serious side-effects. The doctors think that the transplant will be safer and more likely to be successful with reduced doses of chemotherapy and radiation. The purpose of this study is to find out how good a combination of chemotherapy and radiation at reduced doses followed by a cord blood transplant are at treating cancer.
The stem cells chosen for the transplant are from umbilical cord blood. Umbilical cord blood is collected from healthy newborn babies and frozen. One cord blood collection is called a "cord blood unit." On transplant day, the cord blood will be given through the catheter just like a blood transfusion. Transplants done this way have been successful. However, this type of transplant is fairly new. Therefore, it is important to study it so the doctors can better understand how it works.
Most blood or bone marrow transplants using donor stem cells are done as part of a study. When patients are on a study we test new ways of treating them which we think may be better than the old ways. We collect information about the result of this treatment so we can understand how well the treatment works. This is so we can learn better ways to treat our patients.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 1 | Experimental | There are three chemotherapy drugs involved. They are called fludarabine (5 doses), cyclophosphamide (1 dose), and thiotepa (2 doses). Also two days of radiation therapy. This is called Total Body Irradiation or TBI. The TBI if given for two days before, the transplant. On transplant day, the cord blood cells will be given through a catheter. The immune suppressing drugs given are called cyclosporine-A (CSA) and mycophenolate mofetil (MMF). These will be started 3 days before the transplant and will be given through the catheter. Later they can be given as tablets. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| fludarabine, cyclophosphamide, thiotepa, radiation therapy, unrelated donor umbilical cord blood graft | Drug | Cyclophosphamide 50 mg/kg/dose x 1 IV day -6 (1 dose) Fludarabine 30 mg/m2/dose x 5 IV days -6 to -2 (5 doses) Thiotepa 5 mg/kg/dose x 2 IV days -5 to -4 (2 doses) TBI 200 cGy/dose x 2 days -2 to -1 (2 doses). On transplant day, the cord blood cells will be given through your catheter. The immune suppressing drugs you will receive are called cyclosporine-A (CSA) and mycophenolate mofetil (MMF). These will be started 3 days before the transplant and will be given through your catheter. Later they can be given as tablets. |
| Measure | Description | Time Frame |
|---|---|---|
| Progression Free Survival/PFS at 1 Year Post UCBT. | To obtain a preliminary estimate of progression free survival at 1 year post UCBT. Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions | 1 year post UCBT |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Neutrophil Recovery Post Allograft for Haplo-dCBT Recipients | Successful primary donor engraftment = neutrophill recovery within the first 45 days after transplant and partial/complete donor chimerism (>/= 10%) | up to 13 days from engraftment |
| Percentage of Participants With Sustained CB-derived Neutrophil Engraftment |
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Inclusion Criteria:
have relapsed disease following initial therapy but failed to mobilize or had bone marrow involvement and therefore are not suitable for an autologous transplant OR
have CR/PR of the large cell component of their disease after either salvage chemotherapy or an autologous transplant.
be high-risk as such as p53 positivity and be in 1st CR/PR after initial therapy OR have relapsed disease following initial therapy and be in 2nd or 3rd CR/PR after salvage chemotherapy.
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Juliet Barker, MBBS | Memorial Sloan Kettering Cancer Center | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Memorial Sloan Kettering Cancer Center | New York | New York | 10065 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 23416850 | Derived | Ponce DM, Sauter C, Devlin S, Lubin M, Gonzales AM, Kernan NA, Scaradavou A, Giralt S, Goldberg JD, Koehne G, Perales MA, Young JW, Castro-Malaspina H, Jakubowski A, Papadopoulos EB, Barker JN. A novel reduced-intensity conditioning regimen induces a high incidence of sustained donor-derived neutrophil and platelet engraftment after double-unit cord blood transplantation. Biol Blood Marrow Transplant. 2013 May;19(5):799-803. doi: 10.1016/j.bbmt.2013.02.007. Epub 2013 Feb 14. |
| Label | URL |
|---|---|
| Memorial Sloan Kettering Cancer Center | View source |
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| ID | Title | Description |
|---|---|---|
| FG000 | Participants With Hematologic Malignancies | There are three chemotherapy drugs involved. They are called fludarabine (5 doses), cyclophosphamide (1 dose), and thiotepa (2 doses). Also two days of radiation therapy. This is called Total Body Irradiation or TBI. The TBI if given for two days before, the transplant. On transplant day, the cord blood cells will be given through a catheter. The immune suppressing drugs given are called cyclosporine-A (CSA) and mycophenolate mofetil (MMF). These will be started 3 days before the transplant and will be given through the catheter. Later they can be given as tablets. |
| 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 | Dec 7, 2016 |
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|
The day 100 cumulative incidence of sustained CB-derived neutrophil engraftment. |
| 100 days |
| Percentage of Participants With Sustained CB-derived Platelet Engraftment | The day 100 cumulative incidence of sustained CB-derived platelet engraftment to >/= 20 x 10^9/L | 100 days |
| COMPLETED |
|
| NOT COMPLETED |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Participants With Hematologic Malignancies | There are three chemotherapy drugs involved. They are called fludarabine (5 doses), cyclophosphamide (1 dose), and thiotepa (2 doses). Also two days of radiation therapy. This is called Total Body Irradiation or TBI. The TBI if given for two days before, the transplant. On transplant day, the cord blood cells will be given through a catheter. The immune suppressing drugs given are called cyclosporine-A (CSA) and mycophenolate mofetil (MMF). These will be started 3 days before the transplant and will be given through the catheter. Later they can be given as tablets. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Median | Full Range | 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 | 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 | Progression Free Survival/PFS at 1 Year Post UCBT. | To obtain a preliminary estimate of progression free survival at 1 year post UCBT. Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions | Posted | Number | 95% Confidence Interval | percentage of participants | 1 year post UCBT |
|
|
| ||||||||||||||||||||||||||
| Secondary | Number of Participants With Neutrophil Recovery Post Allograft for Haplo-dCBT Recipients | Successful primary donor engraftment = neutrophill recovery within the first 45 days after transplant and partial/complete donor chimerism (>/= 10%) | 43 haplo-dCBT recipients in this analysis 43 dCBT recipient | Posted | Number | participants | up to 13 days from engraftment |
|
| |||||||||||||||||||||||||||
| Secondary | Percentage of Participants With Sustained CB-derived Neutrophil Engraftment | The day 100 cumulative incidence of sustained CB-derived neutrophil engraftment. | Posted | Number | 95% Confidence Interval | Percentage of participants | 100 days |
|
| |||||||||||||||||||||||||||
| Secondary | Percentage of Participants With Sustained CB-derived Platelet Engraftment | The day 100 cumulative incidence of sustained CB-derived platelet engraftment to >/= 20 x 10^9/L | Posted | Number | 95% Confidence Interval | Percentage of participants | 100 days |
|
|
Up to 3 years
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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 | Participants With Hematologic Malignancies | There are three chemotherapy drugs involved. They are called fludarabine (5 doses), cyclophosphamide (1 dose), and thiotepa (2 doses). Also two days of radiation therapy. This is called Total Body Irradiation or TBI. The TBI if given for two days before, the transplant. On transplant day, the cord blood cells will be given through a catheter. The immune suppressing drugs given are called cyclosporine-A (CSA) and mycophenolate mofetil (MMF). These will be started 3 days before the transplant and will be given through the catheter. Later they can be given as tablets. | 36 | 86 | 41 | 86 | 47 | 86 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anorexia | Metabolism and nutrition disorders | Systematic Assessment |
| ||
| Aspiration | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Blood/Bone Marrow, other | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Creatinine | Investigations | Systematic Assessment |
| ||
| Death not assoc w CTCAE term- Death NOS | General disorders | Systematic Assessment |
| ||
| Death not assoc w CTCAE term- Multi-organ failure | General disorders | Systematic Assessment |
| ||
| Death not assoc w CTCAE term- Sudden death | General disorders | Systematic Assessment |
| ||
| Death not assoc w CTCAE term-Disease prog NOS | General disorders | Systematic Assessment |
| ||
| Dermatology/Skin, other | Skin and subcutaneous tissue disorders | Systematic Assessment |
| ||
| Diarrhea | Gastrointestinal disorders | Systematic Assessment |
| ||
| Dyspnea (shortness of breath) | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Encephalopathy | Nervous system disorders | Systematic Assessment |
| ||
| Extremity-upper (function) | Musculoskeletal and connective tissue disorders | Systematic Assessment |
| ||
| Fever (in the absence of neutropenia) | General disorders | Systematic Assessment |
| ||
| Gastrointestinal, other | Gastrointestinal disorders | Systematic Assessment |
| ||
| Hemoglobin | Investigations | Systematic Assessment |
| ||
| Hemolysis | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Hypertension | Vascular disorders | Systematic Assessment |
| ||
| Hypotension | Vascular disorders | Systematic Assessment |
| ||
| Hypoxia | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Infection w/ Gr 3/4 neut, Blood | Infections and infestations | Systematic Assessment |
| ||
| Infection, other | Infections and infestations | Systematic Assessment |
| ||
| Muscle weakness - Whole body/general | Musculoskeletal and connective tissue disorders | Systematic Assessment |
| ||
| Nausea | Gastrointestinal disorders | Systematic Assessment |
| ||
| Neurology - Other (specify) | Nervous system disorders | Systematic Assessment |
| ||
| Neuropathy: motor | Nervous system disorders | Systematic Assessment |
| ||
| Pain - Abdomen NOS | Gastrointestinal disorders | Systematic Assessment |
| ||
| Pain - Cardiac/heart | Cardiac disorders | Systematic Assessment |
| ||
| Pain - Head/headache | Nervous system disorders | Systematic Assessment |
| ||
| Pain - Urethra | Renal and urinary disorders | Systematic Assessment |
| ||
| Pulm/upp respiratory - Other (spec) | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Rash/desquamation | Skin and subcutaneous tissue disorders | Systematic Assessment |
| ||
| Renal/Genitourinary-Other Specify | Renal and urinary disorders | Systematic Assessment |
| ||
| Thrombosis/thrombus/embolism | Vascular disorders | Systematic Assessment |
| ||
| Vomiting | Gastrointestinal disorders | Systematic Assessment |
| ||
| Weight loss | Investigations | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| ALT, SGPT | Investigations | Systematic Assessment |
| ||
| AST, SGOT | Investigations | Systematic Assessment |
| ||
| Alkaline phosphatase | Investigations | Systematic Assessment |
| ||
| Bilirubin (hyperbilirubinemia) | Investigations | Systematic Assessment |
| ||
| Creatinine | Investigations | Systematic Assessment |
| ||
| Glucose, high (hyperglycemia) | Metabolism and nutrition disorders | Systematic Assessment |
| ||
| Magnesium, high (hypermagnesemia) | Metabolism and nutrition disorders | Systematic Assessment |
|
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Juliet Barker, MBBS | Memorial Sloan Kettering Cancer Center | 646-608-3756 | barkerj@mskcc.org |
| Sep 21, 2022 |
| Prot_SAP_000.pdf |
| ID | Term |
|---|---|
| D007938 | Leukemia |
| D009190 | Myelodysplastic Syndromes |
| D008228 | Lymphoma, Non-Hodgkin |
| D015464 | Leukemia, Myelogenous, Chronic, BCR-ABL Positive |
| D006689 | Hodgkin Disease |
| ID | Term |
|---|---|
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D001855 | Bone Marrow Diseases |
| D008223 | Lymphoma |
| D008232 | Lymphoproliferative Disorders |
| D008206 | Lymphatic Diseases |
| D007160 | Immunoproliferative Disorders |
| D007154 | Immune System Diseases |
| D007951 | Leukemia, Myeloid |
| D009196 | Myeloproliferative Disorders |
| D002908 | Chronic Disease |
| D020969 | Disease Attributes |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
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| ID | Term |
|---|---|
| C024352 | fludarabine |
| D003520 | Cyclophosphamide |
| D013852 | Thiotepa |
| D011878 | Radiotherapy |
| 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 |
| D013721 | Triethylenephosphoramide |
| D001388 | Aziridines |
| D001389 | Azirines |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
| D013812 | Therapeutics |
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| Unknown or Not Reported |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
|
|
|