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The purpose of this study is to look at whether the combination of lower-dose chemotherapy with two chemotherapy (anti-cancer) drugs, called busulfan and melphalan, and an antibody medication called alemtuzumab (Campath®), can prevent rejection of donor blood stem cells so that those cells take hold and build a healthy new blood cell factory after transplant. The study will also look at the safety of the combination of drugs and of the transplant of peripheral blood stem cells from a healthy relative or an unrelated donor.
Transplantation of related or unrelated allogeneic peripheral blood stem cells (PBSCs) after administration of a reduced-intensity regimen of busulfan, melphalan and alemtuzumab will be associated with satisfactory engraftment and acceptable post-transplant non-relapse mortality.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| busulfan, and melphalan, and alemtuzumab | Experimental | Three drug regimen using busulfan, and melphalan, and alemtuzumab. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| busulfan, and melphalan, and alemtuzumab | Drug | intravenous busulfan 3.2 mg/kg/dose daily for 2 days, on days -5 and -4 (i.e., 5 and 4 days, respectively, before PBSCT). intravenous melphalan 100 mg/m2 on day -3. intravenous alemtuzumab 30 mg/dose for 2 days, on days -2 and -1. |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Presence of Donor Lymphohematopoietic Chimerism (Defined as at Least 50% Donor Cells in the Peripheral Blood) in Peripheral Blood by Day +100 (i.e., 100 Days After Allogeneic PBSCT). | To determine the efficacy of related or unrelated allogeneic PBSC transplantation (PBSCT) using a preparative regimen of busulfan, melphalan and alemtuzumab, as measured by durable donor lymphohematopoietic cell engraftment. The primary efficacy endpoint is the presence of donor lymphohematopoietic chimerism (defined as at least 50% donor cells in the peripheral blood) in peripheral blood by day +100 (i.e., 100 days after allogeneic PBSCT). | Day +100 |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Relapse-free Survival. | To determine the safety of related or unrelated allogeneic PBSCT using a preparative regimen of busulfan, melphalan and alemtuzumab. The primary safety endpoint is non-relapse mortality at day +100. | Day +100 |
| Number of Participants With Event-free Survival. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Andrew M Yeager, MD | University of Arizona | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University Medical Center and UMC-North Clinic | Tucson | Arizona | 85719 | United States |
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| ID | Title | Description |
|---|---|---|
| FG000 | Busulfan, and Melphalan, and Alemtuzumab | Three drug regimen using busulfan, and melphalan, and alemtuzumab. Busulfan, and melphalan, and alemtuzumab: intravenous busulfan 3.2 mg/kg/dose daily for 2 days, on days -5 and -4 (i.e., 5 and 4 days, respectively, before PBSCT). Intravenous melphalan 100 mg/m2 on day -3. Intravenous alemtuzumab 30 mg/dose for 2 days, on days -2 and -1. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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To determine the safety of related or unrelated allogeneic PBSCT using a preparative regimen of busulfan, melphalan and alemtuzumab. The primary safety endpoint is non-relapse mortality at day +100. |
| Day +100 |
| Number of Participants With Overall Survival. | To determine the safety of related or unrelated allogeneic PBSCT using a preparative regimen of busulfan, melphalan and alemtuzumab. The primary safety endpoint is non-relapse mortality at day +100. | Day +100 |
| COMPLETED |
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | Busulfan, and Melphalan, and Alemtuzumab | Three drug regimen using busulfan, and melphalan, and alemtuzumab. Busulfan, and melphalan, and alemtuzumab: intravenous busulfan 3.2 mg/kg/dose daily for 2 days, on days -5 and -4 (i.e., 5 and 4 days, respectively, before PBSCT). Intravenous melphalan 100 mg/m2 on day -3. Intravenous alemtuzumab 30 mg/dose for 2 days, on days -2 and -1. |
| 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 |
| ||||||||||||||||||||
| 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 | Number of Participants With Presence of Donor Lymphohematopoietic Chimerism (Defined as at Least 50% Donor Cells in the Peripheral Blood) in Peripheral Blood by Day +100 (i.e., 100 Days After Allogeneic PBSCT). | To determine the efficacy of related or unrelated allogeneic PBSC transplantation (PBSCT) using a preparative regimen of busulfan, melphalan and alemtuzumab, as measured by durable donor lymphohematopoietic cell engraftment. The primary efficacy endpoint is the presence of donor lymphohematopoietic chimerism (defined as at least 50% donor cells in the peripheral blood) in peripheral blood by day +100 (i.e., 100 days after allogeneic PBSCT). | Data were collected but could not be analyzed. PI for this study has retired and any data that were gathered, are lost. No data are available for this assessment | Posted | Day +100 |
|
| |||||||||||||||||||
| Secondary | Number of Participants With Relapse-free Survival. | To determine the safety of related or unrelated allogeneic PBSCT using a preparative regimen of busulfan, melphalan and alemtuzumab. The primary safety endpoint is non-relapse mortality at day +100. | Data were collected but could not be analyzed. PI for this study has retired and any data that were gathered, are lost. No data are available for this assessment. | Posted | Day +100 |
|
| |||||||||||||||||||
| Secondary | Number of Participants With Event-free Survival. | To determine the safety of related or unrelated allogeneic PBSCT using a preparative regimen of busulfan, melphalan and alemtuzumab. The primary safety endpoint is non-relapse mortality at day +100. | Data were collected but could not be analyzed. PI for this study has retired and any data that were gathered, are lost. No data are available for this assessment | Posted | Day +100 |
|
| |||||||||||||||||||
| Secondary | Number of Participants With Overall Survival. | To determine the safety of related or unrelated allogeneic PBSCT using a preparative regimen of busulfan, melphalan and alemtuzumab. The primary safety endpoint is non-relapse mortality at day +100. | Data were collected but could not be analyzed. PI for this study has retired and any data that were gathered, are lost. No data are available for this assessment | Posted | Day +100 |
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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 | Busulfan, and Melphalan, and Alemtuzumab | Three drug regimen using busulfan, and melphalan, and alemtuzumab. Busulfan, and melphalan, and alemtuzumab: intravenous busulfan 3.2 mg/kg/dose daily for 2 days, on days -5 and -4 (i.e., 5 and 4 days, respectively, before PBSCT). Intravenous melphalan 100 mg/m2 on day -3. Intravenous alemtuzumab 30 mg/dose for 2 days, on days -2 and -1. | 7 | 16 | 3 | 16 | 16 | 16 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Pneumatosis cystoides intestinalis | Gastrointestinal disorders | Systematic Assessment |
| ||
| Fever | General disorders | Systematic Assessment |
| ||
| Abdominal Pain | Gastrointestinal disorders | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Respiratory Acidosis | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Adult Respiratory Distress Syndrome | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Hypoalbuminemia | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Alkaline phosphatase | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Respiratory Alkalosis | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Allergic rhinitis | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Anorexia | Metabolism and nutrition disorders | Systematic Assessment |
| ||
| Hyperbilirubinemia | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Bladder spasms | Renal and urinary disorders | Systematic Assessment |
| ||
| Blood/Bone marrow - other | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Hypocalcemia | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Cardiac arrhythmia | Cardiac disorders | Systematic Assessment |
| ||
| Cardiac - general | Cardiac disorders | Systematic Assessment |
| ||
| Infectious colitis | Gastrointestinal disorders | Systematic Assessment |
| ||
| Confusion | General disorders | Systematic Assessment |
| ||
| Constipation | Gastrointestinal disorders | Systematic Assessment |
| ||
| Cough | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Creatinine | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Skin - other | Skin and subcutaneous tissue disorders | Systematic Assessment |
| ||
| Diarrhea | Gastrointestinal disorders | Systematic Assessment |
| ||
| Abdominal bloating | Gastrointestinal disorders | Systematic Assessment |
| ||
| Dizziness | General disorders | Systematic Assessment |
| ||
| Dry eye | Eye disorders | Systematic Assessment |
| ||
| Dry mouth | Skin and subcutaneous tissue disorders | Systematic Assessment |
| ||
| Dry skin | Skin and subcutaneous tissue disorders | Systematic Assessment |
| ||
| Dysphagia | Nervous system disorders | Systematic Assessment |
| ||
| Dyspnea | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Edema | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Esophagitis | Gastrointestinal disorders | Systematic Assessment |
| ||
| Gait disturbance | Nervous system disorders | Systematic Assessment |
| ||
| Fatigue | General disorders | Systematic Assessment |
| ||
| Neutropenia | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Fever | General disorders | Systematic Assessment |
| ||
| Flushing | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Gastrointestinal - other | Gastrointestinal disorders | Systematic Assessment |
| ||
| Hyperglycemia | Endocrine disorders | Systematic Assessment |
| ||
| Alopecia | Skin and subcutaneous tissue disorders | Systematic Assessment |
| ||
| Dyspepsia | Gastrointestinal disorders | Systematic Assessment |
| ||
| Hemoglobin | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Bleeding - other | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Hepatobiliary/pancreas - other | Hepatobiliary disorders | Systematic Assessment |
| ||
| Hemorrhoids | Gastrointestinal disorders | Systematic Assessment |
| ||
| Hypertension | General disorders | Systematic Assessment |
| ||
| Hypotension | General disorders | Systematic Assessment |
| ||
| Hypoxia | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Infection | Infections and infestations | Systematic Assessment |
| ||
| Insomnia | General disorders | Systematic Assessment |
| ||
| Leukocytes (total WBC) | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Lymphatics - other | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Hypomagnesemia | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Anxiety | Psychiatric disorders | Systematic Assessment |
| ||
| Depression | Psychiatric disorders | Systematic Assessment |
| ||
| Mucositis/stomatitis | Skin and subcutaneous tissue disorders | Systematic Assessment |
| ||
| Metabolic - other | Metabolism and nutrition disorders | Systematic Assessment |
| ||
| Musculoskeletal - weakness/other | Musculoskeletal and connective tissue disorders | Systematic Assessment |
| ||
| Sinus reaction | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Nausea | Gastrointestinal disorders | Systematic Assessment |
| ||
| Neurology - other | Nervous system disorders | Systematic Assessment |
| ||
| Neuropathy | Nervous system disorders | Systematic Assessment |
| ||
| Neutrophils/Granulocytes | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Pain | General disorders | Systematic Assessment |
| ||
| Kidney perforation | Renal and urinary disorders | Systematic Assessment |
| ||
| Petichiae/Purpura | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Hypophosphatemia | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Platelets | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Pneumonitis/Pulmonary infiltrates | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Prutitis | General disorders | Systematic Assessment |
| ||
| Psychosis | Psychiatric disorders | Systematic Assessment |
| ||
| Pulmonary hypertension | Cardiac disorders | Systematic Assessment |
| ||
| Pulmonary/Upper respiratory - other | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Rash | Skin and subcutaneous tissue disorders | Systematic Assessment |
| ||
| Renal failure | Renal and urinary disorders | Systematic Assessment |
| ||
| Renal/Genitourinary - other | Renal and urinary disorders | Systematic Assessment |
| ||
| Rigors/chills | General disorders | Systematic Assessment |
| ||
| Seizure | Nervous system disorders | Systematic Assessment |
| ||
| Sexual/reproductive function - other | Reproductive system and breast disorders | Systematic Assessment |
| ||
| Hypernatremia | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Hyponatremia | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Somnolence/depressed level of consciousness | General disorders | Systematic Assessment |
| ||
| Atrial fibrillation | Cardiac disorders | Systematic Assessment |
| ||
| Tachycardia | Cardiac disorders | Systematic Assessment |
| ||
| Syncope | General disorders | Systematic Assessment |
| ||
| Dysgeusia | Gastrointestinal disorders | Systematic Assessment |
| ||
| Tremor | Nervous system disorders | Systematic Assessment |
| ||
| Dysuria | Renal and urinary disorders | Systematic Assessment |
| ||
| Urticaria | Renal and urinary disorders | Systematic Assessment |
| ||
| Vaginal discharge | Reproductive system and breast disorders | Systematic Assessment |
| ||
| Vascular - other | Vascular disorders | Systematic Assessment |
| ||
| Ventricular arrhythmia | Cardiac disorders | Systematic Assessment |
| ||
| Blurred vision | Eye disorders | Systematic Assessment |
| ||
| Dysarthria | Gastrointestinal disorders | Systematic Assessment |
| ||
| Vomiting | Gastrointestinal disorders | Systematic Assessment |
| ||
| Weight gain | Metabolism and nutrition disorders | Systematic Assessment |
| ||
| Weight loss | Metabolism and nutrition disorders | Systematic Assessment |
|
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Program Coordinator | University of Arizona Cancer Center | 52-626-0301 | aselegue@email.arizona.edu |
| ID | Term |
|---|---|
| D019337 | Hematologic Neoplasms |
| D009101 | Multiple Myeloma |
| D000741 | Anemia, Aplastic |
| D006457 | Hemoglobinuria, Paroxysmal |
| D055728 | Primary Myelofibrosis |
| D008223 | Lymphoma |
| D007938 | Leukemia |
| D009190 | Myelodysplastic Syndromes |
| ID | Term |
|---|---|
| D009371 | Neoplasms by Site |
| D009369 | Neoplasms |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D054219 | Neoplasms, Plasma Cell |
| D009370 | Neoplasms by Histologic Type |
| D020141 | Hemostatic Disorders |
| D014652 | Vascular Diseases |
| D002318 | Cardiovascular Diseases |
| D010265 | Paraproteinemias |
| D001796 | Blood Protein Disorders |
| D006474 | Hemorrhagic Disorders |
| D008232 | Lymphoproliferative Disorders |
| D007160 | Immunoproliferative Disorders |
| D007154 | Immune System Diseases |
| D000740 | Anemia |
| D000080983 | Bone Marrow Failure Disorders |
| D001855 | Bone Marrow Diseases |
| D000743 | Anemia, Hemolytic |
| D009196 | Myeloproliferative Disorders |
| D008206 | Lymphatic Diseases |
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| ID | Term |
|---|---|
| D002066 | Busulfan |
| D008558 | Melphalan |
| D000074323 | Alemtuzumab |
| ID | Term |
|---|---|
| D002072 | Butylene Glycols |
| D006018 | Glycols |
| D000438 | Alcohols |
| D009930 | Organic Chemicals |
| D008698 | Mesylates |
| D000476 | Alkanesulfonates |
| D017738 | Alkanesulfonic Acids |
| D000473 | Alkanes |
| D006839 | Hydrocarbons, Acyclic |
| D006838 | Hydrocarbons |
| D013451 | Sulfonic Acids |
| D013456 | Sulfur Acids |
| D013457 | Sulfur Compounds |
| D009588 | Nitrogen Mustard Compounds |
| D009150 | Mustard Compounds |
| D006846 | Hydrocarbons, Halogenated |
| D010649 | Phenylalanine |
| D024322 | Amino Acids, Aromatic |
| D000598 | Amino Acids, Cyclic |
| D000596 | Amino Acids |
| D000602 | Amino Acids, Peptides, and Proteins |
| D061067 | Antibodies, Monoclonal, Humanized |
| D000911 | Antibodies, Monoclonal |
| D000906 | Antibodies |
| D007136 | Immunoglobulins |
| D007162 | Immunoproteins |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D012712 | Serum Globulins |
| D005916 | Globulins |
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| Unknown or Not Reported |
|