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Closed due to poor accrual
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Approximately 30% of patients who are candidates for bone marrow transplants do not have an HLA-matched, or close to matched, donor available. For this reason, doctors have been testing ways to make transplants from HLA-partially matched donors as safe and effective as transplants from HLA-matched donors.
This study is being done to test the safety and the treatment results of a specific kind of transplant. In this transplant, blood from two donors will be used. Each donor will share one half of your HLA type. Blood from both donors will be transplanted at the same time.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| REGIMEN 1 | Experimental | REGIMEN 1: Patients undergo hyperfractionated TBI TID for a total of 11-12 doses on days -10 to -7 and receive thiotepa IV over 4 hours QD on days -6 and -5, fludarabine phosphate IV over 30 minutes QD on days -6 to -2, and anti-thymocyte globulin IV on days -4 to -2. TRANSPLANTATION: Patients undergo CD34-selected allogeneic PBSCT on day 0. |
|
| Regimen 2 | Experimental | To be given to patients non-malignant, life-threatening diseases and patients with hematologic malignancies, with extensive prior therapy and comorbidities who are unable to receive TBI, consists of Melphalan 70mg/m2 IV x 2 days, thiotepa 5mg/kg IV x 2 days (or 10mg/kg x 1 day), and fludarabine 25 mg/m2 IV x 5 days. TRANSPLANTATION: Patients undergo CD34-selected allogeneic PBSCT on day 0. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| total-body irradiation (TBI) | Radiation |
| ||
| thiotepa |
| Measure | Description | Time Frame |
|---|---|---|
| Efficacy of HLA-haploidentical Biparental T-cell Depleted CD34+ Peripheral Blood Stem Cell Transplants | Efficacy is measured by:
| 1 year |
| Measure | Description | Time Frame |
|---|---|---|
| Evaluation of Recipients Post Transplant | The levels of engraftment and persistence of hematopoietic cells and their myeloid and lymphoid progressing from each donor post transplant. The tolerance or reactivity of engrafted T cells from each donor detected in the blood at 3, 6, and thereafter every 3-6 months until normal, post transplant against host cells and cells derived from the other parent as measured by standard mixed lymphocyte culture and cell mediated cytolysis assays. |
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Inclusion Criteria:
AML in 1st remission - for patients whose AML does not have 'good risk' cytogenetic features (i.e. t 8;21, t15;17, inv 16).
Cardiac: asymptomatic or if symptomatic then LVEF at rest must be > 50% and must improve with exercise.
Exclusion Criteria:
Donor Inclusion Criteria:
Donor Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Richard O'Reilly, MD | Memorial Sloan Kettering Cancer Center | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Memorial Sloan Kettering Cancer Center | New York | New York | 10065-0009 | United States |
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| Label | URL |
|---|---|
| Memorial Sloan Kettering Cancer Center | View source |
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| ID | Title | Description |
|---|---|---|
| FG000 | REGIMEN 1 | REGIMEN 1: Patients undergo hyperfractionated TBI TID for a total of 11-12 doses on days -10 to -7 and receive thiotepa IV over 4 hours QD on days -6 and -5, fludarabine phosphate IV over 30 minutes QD on days -6 to -2, and anti-thymocyte globulin IV on days -4 to -2. TRANSPLANTATION: Patients undergo CD34-selected allogeneic PBSCT on day 0. total-body irradiation (TBI) thiotepa fludarabine phosphate anti-thymocyte globulin allogeneic hematopoietic stem cell transplantation peripheral blood stem cell transplantation laboratory biomarker analysis |
| 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 | Jul 30, 2015 |
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|
| fludarabine phosphate | Drug |
|
| melphalan | Drug |
|
| anti-thymocyte globulin | Biological |
|
| allogeneic hematopoietic stem cell transplantation | Procedure |
|
| peripheral blood stem cell transplantation | Biological |
|
| laboratory biomarker analysis | Other |
|
| 1 year |
| FG001 | Regimen 2 | To be given to patients non-malignant, life-threatening diseases and patients with hematologic malignancies, with extensive prior therapy and comorbidities who are unable to receive TBI, consists of Melphalan 70mg/m2 IV x 2 days, thiotepa 5mg/kg IV x 2 days (or 10mg/kg x 1 day), and fludarabine 25 mg/m2 IV x 5 days. TRANSPLANTATION: Patients undergo CD34-selected allogeneic PBSCT on day 0. thiotepa fludarabine phosphate melphalan anti-thymocyte globulin allogeneic hematopoietic stem cell transplantation peripheral blood stem cell transplantation laboratory biomarker analysis |
| COMPLETED |
|
| NOT COMPLETED |
|
Zero participants were enrolled to Regimen 1
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| ID | Title | Description |
|---|---|---|
| BG000 | REGIMEN 1 | REGIMEN 1: Patients undergo hyperfractionated TBI TID for a total of 11-12 doses on days -10 to -7 and receive thiotepa IV over 4 hours QD on days -6 and -5, fludarabine phosphate IV over 30 minutes QD on days -6 to -2, and anti-thymocyte globulin IV on days -4 to -2. TRANSPLANTATION: Patients undergo CD34-selected allogeneic PBSCT on day 0. total-body irradiation (TBI) thiotepa fludarabine phosphate anti-thymocyte globulin allogeneic hematopoietic stem cell transplantation peripheral blood stem cell transplantation laboratory biomarker analysis |
| BG001 | Regimen 2 | To be given to patients non-malignant, life-threatening diseases and patients with hematologic malignancies, with extensive prior therapy and comorbidities who are unable to receive TBI, consists of Melphalan 70mg/m2 IV x 2 days, thiotepa 5mg/kg IV x 2 days (or 10mg/kg x 1 day), and fludarabine 25 mg/m2 IV x 5 days. TRANSPLANTATION: Patients undergo CD34-selected allogeneic PBSCT on day 0. thiotepa fludarabine phosphate melphalan anti-thymocyte globulin allogeneic hematopoietic stem cell transplantation peripheral blood stem cell transplantation laboratory biomarker analysis |
| 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, 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 | Efficacy of HLA-haploidentical Biparental T-cell Depleted CD34+ Peripheral Blood Stem Cell Transplants | Efficacy is measured by:
| Due to the small accrual on study, as well as patient course following treatment on study, the primary objectives of this study were not able to be analyzed. | Posted | 1 year |
|
| ||||||||||||||||||||||
| Secondary | Evaluation of Recipients Post Transplant | The levels of engraftment and persistence of hematopoietic cells and their myeloid and lymphoid progressing from each donor post transplant. The tolerance or reactivity of engrafted T cells from each donor detected in the blood at 3, 6, and thereafter every 3-6 months until normal, post transplant against host cells and cells derived from the other parent as measured by standard mixed lymphocyte culture and cell mediated cytolysis assays. | Due to the small accrual on study, as well as patient course following treatment on study, the primary objectives of this study were not able to be analyzed. | Posted | 1 year |
|
Up to 24 months after transplant
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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 | Regimen 2 | To be given to patients non-malignant, life-threatening diseases and patients with hematologic malignancies, with extensive prior therapy and comorbidities who are unable to receive TBI, consists of Melphalan 70mg/m2 IV x 2 days, thiotepa 5mg/kg IV x 2 days (or 10mg/kg x 1 day), and fludarabine 25 mg/m2 IV x 5 days. TRANSPLANTATION: Patients undergo CD34-selected allogeneic PBSCT on day 0. | 3 | 3 | 2 | 3 | 3 | 3 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Acidosis | Metabolism and nutrition disorders | CTC-4.0 | Systematic Assessment |
| |
| Bronchopulmonary hemorrhage | Respiratory, thoracic and mediastinal disorders | CTC-4.0 | Systematic Assessment |
| |
| Hypotension | Vascular disorders | CTC-4.0 | Systematic Assessment |
| |
| Hypoxia | Reproductive system and breast disorders | CTC-4.0 | Systematic Assessment |
| |
| Multi-organ failure | General disorders | CTC-4.0 | Systematic Assessment |
| |
| Myelitis | Infections and infestations | CTC-4.0 | Systematic Assessment |
| |
| Nervous system disorders - Other, specify | Nervous system disorders | CTC-4.0 | Systematic Assessment |
| |
| Pancreatitis | Gastrointestinal disorders | CTC-4.0 | Systematic Assessment |
| |
| Renal and urinary disorders - Other, specify | Renal and urinary disorders | CTC-4.0 | Systematic Assessment |
| |
| Sinus tachycardia | Cardiac disorders | CTC-4.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Lymphocyte count decreased | Investigations | CTC-4.0 | Systematic Assessment |
| |
| Platelet count decreased | Investigations | CTC-4.0 | Systematic Assessment |
| |
| White blood cell decreased | Investigations | CTC-4.0 | Systematic Assessment |
| |
| Anemia | Blood and lymphatic system disorders | CTC-4.0 | Systematic Assessment |
| |
| Hypokalemia | Metabolism and nutrition disorders | CTC-4.0 | Systematic Assessment |
| |
| Neutrophil count decreased | Investigations | CTC-4.0 | Systematic Assessment |
| |
| Activated partial thromboplastin time prolonged | Investigations | CTC-4.0 | Systematic Assessment |
| |
| Hyperglycemia | Metabolism and nutrition disorders | CTC-4.0 | Systematic Assessment |
| |
| Hyperkalemia | Metabolism and nutrition disorders | CTC-4.0 | Systematic Assessment |
| |
| Hypocalcemia | Metabolism and nutrition disorders | CTC-4.0 | Systematic Assessment |
| |
| Hypophosphatemia | Metabolism and nutrition disorders | CTC-4.0 | Systematic Assessment |
| |
| Lipase increased | Investigations | CTC-4.0 | Systematic Assessment |
| |
| Serum amylase increased | Investigations | CTC-4.0 | Systematic Assessment |
|
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Richard O'Reilly, MD | Memorial Sloan Kettering Cancer Center | 646-888-2157 | oreillyr@mskcc.org |
| Mar 19, 2018 |
| Prot_SAP_000.pdf |
| ID | Term |
|---|---|
| D009190 | Myelodysplastic Syndromes |
| D008228 | Lymphoma, Non-Hodgkin |
| ID | Term |
|---|---|
| D001855 | Bone Marrow Diseases |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D008223 | Lymphoma |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D008232 | Lymphoproliferative Disorders |
| D008206 | Lymphatic Diseases |
| D007160 | Immunoproliferative Disorders |
| D007154 | Immune System Diseases |
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| ID | Term |
|---|---|
| D014916 | Whole-Body Irradiation |
| D013852 | Thiotepa |
| C042382 | fludarabine phosphate |
| D008558 | Melphalan |
| D000961 | Antilymphocyte Serum |
| D036102 | Peripheral Blood Stem Cell Transplantation |
| ID | Term |
|---|---|
| D011878 | Radiotherapy |
| D013812 | Therapeutics |
| D008919 | Investigative Techniques |
| D063088 | Phosphoramides |
| D009943 | Organophosphorus Compounds |
| D009930 | Organic Chemicals |
| D013721 | Triethylenephosphoramide |
| D001388 | Aziridines |
| D001389 | Azirines |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
| D009588 | Nitrogen Mustard Compounds |
| D009150 | Mustard Compounds |
| D006846 | Hydrocarbons, Halogenated |
| D006838 | Hydrocarbons |
| D010649 | Phenylalanine |
| D024322 | Amino Acids, Aromatic |
| D000598 | Amino Acids, Cyclic |
| D000596 | Amino Acids |
| D000602 | Amino Acids, Peptides, and Proteins |
| D007106 | Immune Sera |
| D000906 | Antibodies |
| D007136 | Immunoglobulins |
| D007162 | Immunoproteins |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D012712 | Serum Globulins |
| D005916 | Globulins |
| D001688 | Biological Products |
| D045424 | Complex Mixtures |
| D018380 | Hematopoietic Stem Cell Transplantation |
| D033581 | Stem Cell Transplantation |
| D017690 | Cell Transplantation |
| D064987 | Cell- and Tissue-Based Therapy |
| D001691 | Biological Therapy |
| D014180 | Transplantation |
| D013514 | Surgical Procedures, Operative |
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| 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 |
|
|