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| ID | Type | Description | Link |
|---|---|---|---|
| NCI-2010-02228 | Other Grant/Funding Number | NCI | |
| CCCWFU 97710 | Other Identifier | Wake Forest University Health Sciences |
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| Name | Class |
|---|---|
| National Cancer Institute (NCI) | NIH |
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RATIONALE: Deferasirox may remove excess iron from the body caused by blood transfusions.
PURPOSE: This clinical trial studies deferasirox in treating iron overload caused by blood transfusions in patients with hematologic malignancies.
PRIMARY OBJECTIVES: I. To determine the effects of the iron-chelating agent deferasirox on changes in: neutrophil function; macrophage function; lymphocyte function.
SECONDARY OBJECTIVES: I. To determine the effect of chelation on the incidence of bacterial, viral and fungal infections documented by clinical, microbiologically-proven versus radiologically-proven criteria. II. To determine the effect of iron chelation on mortality and morbidity with incidence of the following parameters: Need for hospitalization; Duration of hospitalization; Need for ventilatory support; Need for exchange transfusion/apheresis; Need for treatment with antifungals or antibiotics for documented infections.
OUTLINE: Patients receive oral deferasirox once daily for up to 6 months or until blood counts recover in the absence of disease progression or unacceptable toxicity.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Arm I | Experimental | Patients receive oral deferasirox once daily for up to 6 months or until blood counts recover in the absence of disease progression or unacceptable toxicity. |
|
| control arm | No Intervention | blood tested on healthy patients | |
| correlative | No Intervention | treated off study with or without oral deferasirox (patient choice) but lab draws to gather lab analysis |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| deferasirox | Drug | Given orally |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| Changes in Mean Neutrophil Values (as Measured by Lab) for Arm 1 (Other Arms Were Used for Calibration Only) | Changes in Neutrophils between baseline and mean neutrophils values during treatment (measured after each dose) | Baseline, up to 6 months |
| Measure | Description | Time Frame |
|---|---|---|
| Need for Hospitalization, Ventilator Support, Exchange Transfusion/Apheresis or Treatment With Antifungals or Antibiotics | Records will be assessed at baseline and prospectively while on study. | Baseline, up to 6 months |
| Cumulative Incidence of Documented Bacterial, Fungal, and Viral Infections |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Mary Ann Knovich, MD | Wake Forest University Health Sciences | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Comprehensive Cancer Center of Wake Forest University | Winston-Salem | North Carolina | 27157 | United States |
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| ID | Title | Description |
|---|---|---|
| FG000 | Arm I | Patients receive oral deferasirox once daily for up to 6 months or until blood counts recover in the absence of disease progression or unacceptable toxicity. deferasirox : Given orally laboratory biomarker analysis : Correlative studies enzyme-linked immunosorbent assay : Correlative studies |
| FG001 |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
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| laboratory biomarker analysis | Other | Correlative studies |
|
| enzyme-linked immunosorbent assay | Other | Correlative studies |
|
|
Records will be assessed at baseline and prospectively while on study. |
| Baseline, up to 6 months |
| Control Arm |
blood tested on healthy patients |
| FG002 | Correlative | treated off study with or without oral deferasirox (patient choice) but lab draws to gather lab analysis |
| COMPLETED |
|
| NOT COMPLETED |
|
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Arm I | Patients receive oral deferasirox once daily for up to 6 months or until blood counts recover in the absence of disease progression or unacceptable toxicity. deferasirox : Given orally laboratory biomarker analysis : Correlative studies enzyme-linked immunosorbent assay : Correlative studies |
| BG001 | Control Arm | blood tested on healthy patients |
| BG002 | Correlative | treated off study with or without oral deferasirox (patient choice) but lab draws to gather lab analysis |
| BG003 | 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 |
| ||||||||||||||||
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | 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 | Changes in Mean Neutrophil Values (as Measured by Lab) for Arm 1 (Other Arms Were Used for Calibration Only) | Changes in Neutrophils between baseline and mean neutrophils values during treatment (measured after each dose) | Posted | Mean | Standard Deviation | 10^9 Neutrophils per Liter | Baseline, up to 6 months |
|
|
| ||||||||||||||||||||||||||
| Secondary | Need for Hospitalization, Ventilator Support, Exchange Transfusion/Apheresis or Treatment With Antifungals or Antibiotics | Records will be assessed at baseline and prospectively while on study. | Number analyzed in rows differs from overall because data was not collected on all participants. | Posted | Count of Participants | Participants | Baseline, up to 6 months |
|
| |||||||||||||||||||||||||||
| Secondary | Cumulative Incidence of Documented Bacterial, Fungal, and Viral Infections | Records will be assessed at baseline and prospectively while on study. | Posted | Count of Participants | Participants | Baseline, up to 6 months |
|
|
6 months
only patients on Arm I (recieving drug on protocol) were tracked for toxicities
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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 | Arm I | Patients receive oral deferasirox once daily for up to 6 months or until blood counts recover in the absence of disease progression or unacceptable toxicity. deferasirox : Given orally laboratory biomarker analysis : Correlative studies enzyme-linked immunosorbent assay : Correlative studies | 4 | 4 | 4 | 4 | ||
| EG001 | Control Arm | blood tested on healthy patients | 0 | 0 | 0 | 0 | ||
| EG002 | Correlative | treated off study with or without oral deferasirox (patient choice) but lab draws to gather lab analysis | 0 | 0 | 0 | 0 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Dyspnea | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Fatigue | General disorders | Systematic Assessment |
| ||
| hyperglycemia | Investigations | Systematic Assessment |
| ||
| hyponatremia | Investigations | Systematic Assessment |
| ||
| hypokalemia | Investigations | Systematic Assessment |
| ||
| serum glutamic pyruvic transaminase | Investigations | Systematic Assessment |
| ||
| Thrombosis/thrombus/embolism | Vascular disorders | Systematic Assessment |
| ||
| Heartburn/dyspepsia | General disorders | Systematic Assessment |
| ||
| Confusion | General disorders | Systematic Assessment |
| ||
| "Distension bloating, abdominal" | General disorders | Systematic Assessment |
| ||
| Infection Bladder | Infections and infestations | Systematic Assessment |
| ||
| Cystitis | Infections and infestations | Systematic Assessment |
| ||
| Lipase | Investigations | Systematic Assessment |
| ||
| Lymphopenia | Investigations | Systematic Assessment |
| ||
| Lung infection | Infections and infestations | Systematic Assessment |
| ||
| Infection Sinus | Infections and infestations | Systematic Assessment |
| ||
| Infection with normal ANC or Grade 1 or 2 neutrophils: Nose | Injury, poisoning and procedural complications | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Low WBC | Investigations | Systematic Assessment |
| ||
| low Platelets | Investigations | Systematic Assessment |
| ||
| Low Hemoglobin | Investigations | Systematic Assessment |
| ||
| Nausea | Gastrointestinal disorders | Systematic Assessment |
| ||
| Vomiting | Gastrointestinal disorders | Systematic Assessment |
| ||
| Anorexia | General disorders | Systematic Assessment |
| ||
| Gastrointestinal - Other | General disorders | Systematic Assessment |
| ||
| Alkaline phosphatase | Investigations | Systematic Assessment |
| ||
| Creatinine | Investigations | Systematic Assessment |
| ||
| Proteinuria | Investigations | Systematic Assessment |
| ||
| Dyspnea | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Taste alteration | General disorders | Systematic Assessment |
| ||
| Insomnia | General disorders | Systematic Assessment |
| ||
| Dermatology/Skin | Skin and subcutaneous tissue disorders | Systematic Assessment |
| ||
| Fatigue | General disorders | Systematic Assessment |
| ||
| Weight loss | General disorders | Systematic Assessment |
| ||
| hyperglycemia | Investigations | Systematic Assessment |
| ||
| hypoglycemia | Investigations | Systematic Assessment |
| ||
| hypocalcemia | Investigations | Systematic Assessment |
| ||
| hypomagnesemia | Investigations | Systematic Assessment |
| ||
| hyponatremia | Investigations | Systematic Assessment |
| ||
| hypokalemia | Investigations | Systematic Assessment |
| ||
| hypoalbuminemia | Investigations | Systematic Assessment |
| ||
| hyperbilirubinemia | Investigations | Systematic Assessment |
| ||
| serum glutamic pyruvic transaminase | Investigations | Systematic Assessment |
| ||
| Constipation | Gastrointestinal disorders | Systematic Assessment |
| ||
| Pain: Head/headache | General disorders | Systematic Assessment |
| ||
| serum glutamic oxaloacetic transaminase | Investigations | Systematic Assessment |
| ||
| hypermagnesemia | Investigations | Systematic Assessment |
| ||
| hyperkalemia | Investigations | Systematic Assessment |
| ||
| Ocular/Visual - Other | Eye disorders | Systematic Assessment |
| ||
| Urinary frequency/urgency | General disorders | Systematic Assessment |
| ||
| Renal/Genitourinary - Other | General disorders | Systematic Assessment |
| ||
| Cough | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Intra-operative Injury | General disorders | Systematic Assessment |
| ||
| Musculoskeletal Soft Tissue - Other | General disorders | Systematic Assessment |
| ||
| Dry mouth | General disorders | Systematic Assessment |
| ||
| Dehydration | General disorders | Systematic Assessment |
| ||
| Pruritus/itching | General disorders | Systematic Assessment |
| ||
| Rash/desquamation | Skin and subcutaneous tissue disorders | Systematic Assessment |
| ||
| Pain: Abdomen | General disorders | Systematic Assessment |
| ||
| Ataxia | General disorders | Systematic Assessment |
| ||
| Obstruction Bladder | General disorders | Systematic Assessment |
| ||
| Pain Bladder | General disorders | Systematic Assessment |
| ||
| Amylase | Investigations | Systematic Assessment |
| ||
| Bronchospasm wheezing | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Urine color change | General disorders | Systematic Assessment |
| ||
| Dry eye syndrome | Eye disorders | Systematic Assessment |
| ||
| Anal Incontinence | General disorders | Systematic Assessment |
| ||
| gamma-Glutamyl transpeptidase | Investigations | Systematic Assessment |
| ||
| Glomerular filtration rate | Investigations | Systematic Assessment |
| ||
| Lipase | Investigations | Systematic Assessment |
| ||
| Lymphopenia | Investigations | Systematic Assessment |
| ||
| Memory impairment | General disorders | Systematic Assessment |
| ||
| Pain: Muscle | General disorders | Systematic Assessment |
| ||
| Pain Oral cavity | General disorders | Systematic Assessment |
| ||
| Pain: Extremity-limb | General disorders | Systematic Assessment |
| ||
| Lung infection | Infections and infestations | Systematic Assessment |
| ||
| Allergic rhinitis | Skin and subcutaneous tissue disorders | Systematic Assessment |
| ||
| Hyperpigmentation | General disorders | Systematic Assessment |
| ||
| Mucositis/stomatitis Oral cavity | Infections and infestations | Systematic Assessment |
| ||
| Tremor | Nervous system disorders | Systematic Assessment |
| ||
| blurred vision | Eye disorders | Systematic Assessment |
| ||
| Hemorrhage GU: Urinary | Vascular disorders | Systematic Assessment |
| ||
| Thrombosis/embolism (vascular access-related) | Vascular disorders | Systematic Assessment |
| ||
| Mucositis/stomatitis Oral cavity | Infections and infestations | Systematic Assessment |
| ||
| Infection Lip perioral | Infections and infestations | Systematic Assessment |
| ||
| Blood Infection with high grade neutropenia | Infections and infestations | Systematic Assessment |
|
adverse events were only tracked on Arm I
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Mary Ann Knovich | Comprehensive Cancer Center of Wake Forest University | 336-716-7973 | mknovich@wakehealth.edu |
| ID | Term |
|---|---|
| D015456 | Leukemia, Biphenotypic, Acute |
| D000013 | Congenital Abnormalities |
| D006646 | Histiocytosis, Langerhans-Cell |
| D054391 | Lymphoma, Extranodal NK-T-Cell |
| D017728 | Lymphoma, Large-Cell, Anaplastic |
| D007119 | Immunoblastic Lymphadenopathy |
| D064090 | Intraocular Lymphoma |
| D007946 | Leukemia, Mast-Cell |
| C535323 | Chromosome 5q Deletion Syndrome |
| D009196 | Myeloproliferative Disorders |
| D018442 | Lymphoma, B-Cell, Marginal Zone |
| D016411 | Lymphoma, T-Cell, Peripheral |
| D055728 | Primary Myelofibrosis |
| D054198 | Precursor Cell Lymphoblastic Leukemia-Lymphoma |
| D015470 | Leukemia, Myeloid, Acute |
| D002051 | Burkitt Lymphoma |
| D016403 | Lymphoma, Large B-Cell, Diffuse |
| D008228 | Lymphoma, Non-Hodgkin |
| D006689 | Hodgkin Disease |
| D016400 | Lymphoma, Large-Cell, Immunoblastic |
| D054218 | Precursor T-Cell Lymphoblastic Leukemia-Lymphoma |
| D016410 | Lymphoma, T-Cell, Cutaneous |
| D008224 | Lymphoma, Follicular |
| D020522 | Lymphoma, Mantle-Cell |
| D009182 | Mycosis Fungoides |
| D012751 | Sezary Syndrome |
| D015451 | Leukemia, Lymphocytic, Chronic, B-Cell |
| D000753 | Anemia, Refractory |
| D009101 | Multiple Myeloma |
| D008258 | Waldenstrom Macroglobulinemia |
| ID | Term |
|---|---|
| D007945 | Leukemia, Lymphoid |
| D007938 | Leukemia |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D008232 | Lymphoproliferative Disorders |
| D008206 | Lymphatic Diseases |
| D007160 | Immunoproliferative Disorders |
| D007154 | Immune System Diseases |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D017563 | Lung Diseases, Interstitial |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
| D015614 | Histiocytosis |
| D016399 | Lymphoma, T-Cell |
| D008223 | Lymphoma |
| D000072281 | Lymphadenopathy |
| D005134 | Eye Neoplasms |
| D009371 | Neoplasms by Site |
| D007951 | Leukemia, Myeloid |
| D034721 | Mastocytosis, Systemic |
| D008415 | Mastocytosis |
| D000090362 | Mast Cell Activation Disorders |
| D001855 | Bone Marrow Diseases |
| D016393 | Lymphoma, B-Cell |
| D020031 | Epstein-Barr Virus Infections |
| D006566 | Herpesviridae Infections |
| D004266 | DNA Virus Infections |
| D014777 | Virus Diseases |
| D007239 | Infections |
| D014412 | Tumor Virus Infections |
| D015448 | Leukemia, B-Cell |
| D002908 | Chronic Disease |
| D020969 | Disease Attributes |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D000740 | Anemia |
| D009190 | Myelodysplastic Syndromes |
| D054219 | Neoplasms, Plasma Cell |
| D020141 | Hemostatic Disorders |
| D014652 | Vascular Diseases |
| D002318 | Cardiovascular Diseases |
| D010265 | Paraproteinemias |
| D001796 | Blood Protein Disorders |
| D006474 | Hemorrhagic Disorders |
Not provided
Not provided
| ID | Term |
|---|---|
| D000077588 | Deferasirox |
| D004797 | Enzyme-Linked Immunosorbent Assay |
| ID | Term |
|---|---|
| D001565 | Benzoates |
| D000146 | Acids, Carbocyclic |
| D002264 | Carboxylic Acids |
| D009930 | Organic Chemicals |
| D001555 | Benzene Derivatives |
| D006841 | Hydrocarbons, Aromatic |
| D006844 | Hydrocarbons, Cyclic |
| D006838 | Hydrocarbons |
| D014230 | Triazoles |
| D001393 | Azoles |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
| D007124 | Immunoenzyme Techniques |
| D007118 | Immunoassay |
| D007158 | Immunologic Techniques |
| D008919 | Investigative Techniques |
| D007163 | Immunosorbent Techniques |
| D007150 | Immunohistochemistry |
| D015336 | Molecular Probe Techniques |
Not provided
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| Between 18 and 65 years |
|
| >=65 years |
|
| Male |
|
|
|