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| ID | Type | Description | Link |
|---|---|---|---|
| MSKCC-07006 |
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| Name | Class |
|---|---|
| National Cancer Institute (NCI) | NIH |
The purpose of this study in patients needing treatment for AL amyloidosis is to see how well treatment with IV melphalan works and then, if some clonal plasma cells are still present about 2 to 3 months after melphalan treatment, to see how well treatment with bortezomib and dexamethasone works to reduce the rest of the clonal plasma cell disease.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| All Patients | Experimental | All patients treated with Melphalan with Stem Cell Transplant and Adjuvant Bortezomib and Dexamethasone for Recently Diagnosed Untreated Patients with Systemic Light-Chain (AL) Amyloidosis |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| bortezomib | Drug | Given IV |
| |
| dexamethasone |
| Measure | Description | Time Frame |
|---|---|---|
| Hematologic and Organ Response | patients will be assessed for hematologic response (the response of the clonal plasma cell disease). If the plasma cell disease persists, then they will receive 6 cycles of adjuvant therapy with bortezomib and dexamethasone; patients with peripheral neuropathy will receive dexamethasone alone because of the risk of neuropathy associated with bortezomib. Symptomatic organ involvement with amyloid as defined below. Patients must have symptomatic involvement of no more than 2 of the following 4 visceral organ-systems: kidneys, liver/GI, peripheral/autonomic nervous system, and heart. | 2-3 months post transplant |
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DISEASE CHARACTERISTICS:
Histologically confirmed amyloidosis
Diagnosed within the past 12 months
Clonal plasma cell disorder, as demonstrated by any of the following:
Negative genetic testing for hereditary forms of amyloidosis
No amyloid-specific syndrome (e.g., carpal tunnel syndrome or skin purpura) as the only evidence of disease
No advanced cardiac amyloidosis
Must have symptomatic involvement of no more than 2 of the following visceral organ systems:
No persistent pleural effusions
No clinically overt multiple myeloma with > 30% plasma cells in the bone marrow or lytic bone lesions
Able to undergo autologous stem cell transplantation
PATIENT CHARACTERISTICS:
SWOG performance status 0-3
Not pregnant or nursing
Negative pregnancy test
Fertile patients must use effective contraception
Bilirubin < 2.0 mg/dL
Creatinine clearance < 51 mL/min allowed
LVEF > 45% by echocardiogram
No New York Heart Association class III-IV congestive heart failure
No history of cardiac syncope
No recurrent symptomatic arrhythmias
No oxygen-dependent restrictive cardiomyopathy
No myocardial infarction within the past 6 months
Pulmonary diffusion capacity > 50% predicted by pulmonary function testing
No uncontrolled infection
No other active malignancy, except for any of the following:
No hypersensitivity to bortezomib, boron, or mannitol
No HIV positivity
No serious medical or psychiatric illness that would preclude study compliance
PRIOR CONCURRENT THERAPY:
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| Name | Affiliation | Role |
|---|---|---|
| Heather Landau, 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 | United States |
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| ID | Title | Description |
|---|---|---|
| FG000 | All Patients | All patients treated with Melphalan with Stem Cell Transplant and Adjuvant Bortezomib and Dexamethasone for Recently Diagnosed Untreated Patients with Systemic Light-Chain (AL) Amyloidosis |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Drug |
Given orally |
|
| COMPLETED |
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| NOT COMPLETED |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | All Patients | All patients treated with Melphalan with Stem Cell Transplant and Adjuvant Bortezomib and Dexamethasone for Recently Diagnosed Untreated Patients with Systemic Light-Chain (AL) Amyloidosis |
| 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 |
|
| 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 | Hematologic and Organ Response | patients will be assessed for hematologic response (the response of the clonal plasma cell disease). If the plasma cell disease persists, then they will receive 6 cycles of adjuvant therapy with bortezomib and dexamethasone; patients with peripheral neuropathy will receive dexamethasone alone because of the risk of neuropathy associated with bortezomib. Symptomatic organ involvement with amyloid as defined below. Patients must have symptomatic involvement of no more than 2 of the following 4 visceral organ-systems: kidneys, liver/GI, peripheral/autonomic nervous system, and heart. | Posted | Number | participants | 2-3 months post transplant |
|
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|
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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 | All Patients | All patients treated with Melphalan with Stem Cell Transplant and Adjuvant Bortezomib and Dexamethasone for Recently Diagnosed Untreated Patients with Systemic Light-Chain (AL) Amyloidosis | 20 | 40 | 40 | 40 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Arthritis (non-septic) | General disorders | CTC-3.0 | Systematic Assessment |
| |
| Atrial fibrillation | Cardiac disorders | CTC-3.0 | Systematic Assessment |
| |
| CNS cerebrovascular ischemia | Nervous system disorders | CTC-3.0 | Systematic Assessment |
| |
| Cardiac Arrhythmia, other | Cardiac disorders | CTC-3.0 | Systematic Assessment |
| |
| Cardiac General, other | Cardiac disorders | CTC-3.0 | Systematic Assessment |
| |
| Coagulation, other | Blood and lymphatic system disorders | CTC-3.0 | Systematic Assessment |
| |
| Conduction Abnormality NOS | General disorders | CTC-3.0 | Systematic Assessment |
| |
| Death not associated with CTCAE term- Death NOS | General disorders | CTC-3.0 | Systematic Assessment |
| |
| Dehydration | Gastrointestinal disorders | CTC-3.0 | Systematic Assessment |
| |
| Dyspnea (shortness of breath) | Respiratory, thoracic and mediastinal disorders | CTC-3.0 | Systematic Assessment |
| |
| Febrile neutropenia | General disorders | CTC-3.0 | Systematic Assessment |
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| Fever (in the absence of neutropenia) | General disorders | CTC-3.0 | Systematic Assessment |
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| Hemorrhage, Upper GI NOS | Gastrointestinal disorders | CTC-3.0 | Systematic Assessment |
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| Hemorrhage/Bleeding, other | General disorders | CTC-3.0 | Systematic Assessment |
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| Hypotension | Cardiac disorders | CTC-3.0 | Systematic Assessment |
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| Hypoxia | General disorders | CTC-3.0 | Systematic Assessment |
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| Neutrophil-Pneumonia (lung) | Infections and infestations | CTC-3.0 | Systematic Assessment |
| |
| Neutrophil-Up airway NOS | Infections and infestations | CTC-3.0 | Systematic Assessment |
| |
| Infection, other | Infections and infestations | CTC-3.0 | Systematic Assessment |
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| Nausea | Gastrointestinal disorders | CTC-3.0 | Systematic Assessment |
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| Ocular/Visual - Other | General disorders | CTC-3.0 | Systematic Assessment |
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| Platelets | Blood and lymphatic system disorders | CTC-3.0 | Systematic Assessment |
| |
| Pulm/upper respiratory - Other | Respiratory, thoracic and mediastinal disorders | CTC-3.0 | Systematic Assessment |
| |
| Rash/desquamation | Skin and subcutaneous tissue disorders | CTC-3.0 | Systematic Assessment |
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| Renal failure | Renal and urinary disorders | CTC-3.0 | Systematic Assessment |
| |
| Restrictive cardiomyopathy | Cardiac disorders | CTC-3.0 | Systematic Assessment |
| |
| Sodium, low (hyponatremia) | Metabolism and nutrition disorders | CTC-3.0 | Systematic Assessment |
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| Supraventricular tachycardia | Cardiac disorders | CTC-3.0 | Systematic Assessment |
| |
| Syncope (fainting) | Nervous system disorders | CTC-3.0 | Systematic Assessment |
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| Thrombosis/thrombus/embolism | Respiratory, thoracic and mediastinal disorders | CTC-3.0 | Systematic Assessment |
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| Vasovagal episode | Nervous system disorders | CTC-3.0 | Systematic Assessment |
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| Ventricular arrhythmia- Ventricular tachycardia | Cardiac disorders | CTC-3.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Albumin, low (hypoalbuminemia) | Metabolism and nutrition disorders | CTC-3.0 | Systematic Assessment |
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| Alkaline phosphatase | Metabolism and nutrition disorders | CTC-3.0 | Systematic Assessment |
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| ALT, SGPT | Blood and lymphatic system disorders | CTC-3.0 | Systematic Assessment |
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| AST, SGOT | Blood and lymphatic system disorders | CTC-3.0 | Systematic Assessment |
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| Bicarbonate, serum-low | Metabolism and nutrition disorders | CTC-3.0 | Systematic Assessment |
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| Bilirubin (hyperbilirubinemia) | Metabolism and nutrition disorders | CTC-3.0 | Systematic Assessment |
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| Cholesterol,high(hypercholestremia) | General disorders | CTC-3.0 | Systematic Assessment |
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| Constipation | Gastrointestinal disorders | CTC-3.0 | Systematic Assessment |
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| Cough | Respiratory, thoracic and mediastinal disorders | CTC-3.0 | Systematic Assessment |
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| Creatinine | Metabolism and nutrition disorders | CTC-3.0 | Systematic Assessment |
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| Cushingoid appearance | General disorders | CTC-3.0 | Systematic Assessment |
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| Dehydration | Gastrointestinal disorders | CTC-3.0 | Systematic Assessment |
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| Diarrhea | Gastrointestinal disorders | CTC-3.0 | Systematic Assessment |
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| Distension/bloating, abdominal | Gastrointestinal disorders | CTC-3.0 | Systematic Assessment |
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| Dyspnea (shortness of breath) | Respiratory, thoracic and mediastinal disorders | CTC-3.0 | Systematic Assessment |
| |
| Fatigue (asthenia, lethargy, malaise) | General disorders | CTC-3.0 | Systematic Assessment |
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| Glucose, high (hyperglycemia) | Metabolism and nutrition disorders | CTC-3.0 | Systematic Assessment |
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| Glucose, low (hypoglycemia) | Metabolism and nutrition disorders | CTC-3.0 | Systematic Assessment |
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| Hemoglobin | Blood and lymphatic system disorders | CTC-3.0 | Systematic Assessment |
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| Infection, other | Infections and infestations | CTC-3.0 | Systematic Assessment |
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| Prothrombin Time international normalized ratio | Blood and lymphatic system disorders | CTC-3.0 | Systematic Assessment |
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| Leukocytes (total WBC) | Blood and lymphatic system disorders | CTC-3.0 | Systematic Assessment |
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| Lipase | General disorders | CTC-3.0 | Systematic Assessment |
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| Lymphopenia | Blood and lymphatic system disorders | CTC-3.0 | Systematic Assessment |
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| Magnesium, high (hypermagnesemia) | Metabolism and nutrition disorders | CTC-3.0 | Systematic Assessment |
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| Magnesium, low (hypomagnesemia) | Metabolism and nutrition disorders | CTC-3.0 | Systematic Assessment |
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| Nausea | Gastrointestinal disorders | CTC-3.0 | Systematic Assessment |
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| Neuropathy: sensory | Nervous system disorders | CTC-3.0 | Systematic Assessment |
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| Neutrophils/granulocytes | Blood and lymphatic system disorders | CTC-3.0 | Systematic Assessment |
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| Pain - Neuralgia/peripheral nerve | Nervous system disorders | CTC-3.0 | Systematic Assessment |
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| Phosphate, low (hypophosphatemia) | Metabolism and nutrition disorders | CTC-3.0 | Systematic Assessment |
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| Platelets | Blood and lymphatic system disorders | CTC-3.0 | Systematic Assessment |
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| Potassium, high (hyperkalemia) | Metabolism and nutrition disorders | CTC-3.0 | Systematic Assessment |
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| Potassium, low (hypokalemia) | Metabolism and nutrition disorders | CTC-3.0 | Systematic Assessment |
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| Pruritus/itching | Skin and subcutaneous tissue disorders | CTC-3.0 | Systematic Assessment |
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| Partial thromboplastin time | Blood and lymphatic system disorders | CTC-3.0 | Systematic Assessment |
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| Rash/desquamation | Skin and subcutaneous tissue disorders | CTC-3.0 | Systematic Assessment |
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| Renal failure | Renal and urinary disorders | CTC-3.0 | Systematic Assessment |
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| Sodium, low (hyponatremia) | Metabolism and nutrition disorders | CTC-3.0 | Systematic Assessment |
| |
| Syncope (fainting) | Nervous system disorders | CTC-3.0 | Systematic Assessment |
| |
| Weight gain | General disorders | CTC-3.0 | Systematic Assessment |
|
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Heather Landau | Memorial Sloan Kettering Cancer Center | 212-639-8808 | LandauH@mskcc.org |
| ID | Term |
|---|---|
| D009101 | Multiple Myeloma |
| D054219 | Neoplasms, Plasma Cell |
| D000075363 | Immunoglobulin Light-chain Amyloidosis |
| ID | Term |
|---|---|
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D020141 | Hemostatic Disorders |
| D014652 | Vascular Diseases |
| D002318 | Cardiovascular Diseases |
| D010265 | Paraproteinemias |
| D001796 | Blood Protein Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D006474 | Hemorrhagic Disorders |
| D008232 | Lymphoproliferative Disorders |
| D007160 | Immunoproliferative Disorders |
| D007154 | Immune System Diseases |
| D000686 | Amyloidosis |
| D057165 | Proteostasis Deficiencies |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
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| ID | Term |
|---|---|
| D000069286 | Bortezomib |
| D003907 | Dexamethasone |
| ID | Term |
|---|---|
| D001897 | Boronic Acids |
| D000148 | Acids, Noncarboxylic |
| D000143 | Acids |
| D007287 | Inorganic Chemicals |
| D001896 | Boron Compounds |
| D009930 | Organic Chemicals |
| D011719 | Pyrazines |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
| D011246 | Pregnadienetriols |
| D011245 | Pregnadienes |
| D011278 | Pregnanes |
| D013256 | Steroids |
| D000072473 | Fused-Ring Compounds |
| D011083 | Polycyclic Compounds |
| D013259 | Steroids, Fluorinated |
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| Title | Measurements |
|---|---|
|