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Deferasirox in Treating Iron Overload Caused By Blood Transfusions in Patients With Hematologic Malignancies

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01273766
Recruitment Status : Completed
First Posted : January 10, 2011
Results First Posted : November 27, 2013
Last Update Posted : September 7, 2018
Sponsor:
Collaborator:
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Wake Forest University Health Sciences

Study Description
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Brief Summary:

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.


Condition or disease Intervention/treatment Phase
Acute Undifferentiated Leukemia Adult Acute Lymphoblastic Leukemia in Remission Adult Acute Myeloid Leukemia in Remission Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities Adult Acute Myeloid Leukemia With Del(5q) Adult Acute Myeloid Leukemia With Inv(16)(p13;q22) Adult Acute Myeloid Leukemia With t(15;17)(q22;q12) Adult Acute Myeloid Leukemia With t(16;16)(p13;q22) Adult Acute Myeloid Leukemia With t(8;21)(q22;q22) Adult Grade III Lymphomatoid Granulomatosis Adult Langerhans Cell Histiocytosis Adult Nasal Type Extranodal NK/T-cell Lymphoma Anaplastic Large Cell Lymphoma Angioimmunoblastic T-cell Lymphoma Contiguous Stage II Adult Burkitt Lymphoma Contiguous Stage II Adult Diffuse Large Cell Lymphoma Contiguous Stage II Adult Diffuse Mixed Cell Lymphoma Contiguous Stage II Adult Diffuse Small Cleaved Cell Lymphoma Contiguous Stage II Adult Immunoblastic Large Cell Lymphoma Contiguous Stage II Adult Lymphoblastic Lymphoma Contiguous Stage II Grade 1 Follicular Lymphoma Contiguous Stage II Grade 2 Follicular Lymphoma Contiguous Stage II Grade 3 Follicular Lymphoma Contiguous Stage II Mantle Cell Lymphoma Contiguous Stage II Marginal Zone Lymphoma Contiguous Stage II Small Lymphocytic Lymphoma Cutaneous B-cell Non-Hodgkin Lymphoma de Novo Myelodysplastic Syndromes Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue Hepatosplenic T-cell Lymphoma Intraocular Lymphoma Mast Cell Leukemia Myelodysplastic Syndrome With Isolated Del(5q) Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable Myeloid/NK-cell Acute Leukemia Nodal Marginal Zone B-cell Lymphoma Noncontiguous Stage II Adult Burkitt Lymphoma Noncontiguous Stage II Adult Diffuse Large Cell Lymphoma Noncontiguous Stage II Adult Diffuse Mixed Cell Lymphoma Noncontiguous Stage II Adult Diffuse Small Cleaved Cell Lymphoma Noncontiguous Stage II Adult Immunoblastic Large Cell Lymphoma Noncontiguous Stage II Adult Lymphoblastic Lymphoma Noncontiguous Stage II Grade 1 Follicular Lymphoma Noncontiguous Stage II Grade 2 Follicular Lymphoma Noncontiguous Stage II Grade 3 Follicular Lymphoma Noncontiguous Stage II Mantle Cell Lymphoma Noncontiguous Stage II Marginal Zone Lymphoma Noncontiguous Stage II Small Lymphocytic Lymphoma Noncutaneous Extranodal Lymphoma Peripheral T-cell Lymphoma Previously Treated Myelodysplastic Syndromes Primary Myelofibrosis Recurrent Adult Acute Lymphoblastic Leukemia Recurrent Adult Acute Myeloid Leukemia Recurrent Adult Burkitt Lymphoma Recurrent Adult Diffuse Large Cell Lymphoma Recurrent Adult Diffuse Mixed Cell Lymphoma Recurrent Adult Diffuse Small Cleaved Cell Lymphoma Recurrent Adult Grade III Lymphomatoid Granulomatosis Recurrent Adult Hodgkin Lymphoma Recurrent Adult Immunoblastic Large Cell Lymphoma Recurrent Adult Lymphoblastic Lymphoma Recurrent Adult T-cell Leukemia/Lymphoma Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma Recurrent Grade 1 Follicular Lymphoma Recurrent Grade 2 Follicular Lymphoma Recurrent Grade 3 Follicular Lymphoma Recurrent Mantle Cell Lymphoma Recurrent Marginal Zone Lymphoma Recurrent Mycosis Fungoides/Sezary Syndrome Recurrent Small Lymphocytic Lymphoma Refractory Anemia Refractory Multiple Myeloma Secondary Acute Myeloid Leukemia Secondary Myelofibrosis Small Intestine Lymphoma Splenic Marginal Zone Lymphoma Stage I Adult Burkitt Lymphoma Stage I Adult Diffuse Large Cell Lymphoma Stage I Adult Diffuse Mixed Cell Lymphoma Stage I Adult Diffuse Small Cleaved Cell Lymphoma Stage I Adult Hodgkin Lymphoma Stage I Adult Immunoblastic Large Cell Lymphoma Stage I Adult Lymphoblastic Lymphoma Stage I Adult T-cell Leukemia/Lymphoma Stage I Cutaneous T-cell Non-Hodgkin Lymphoma Stage I Grade 1 Follicular Lymphoma Stage I Grade 2 Follicular Lymphoma Stage I Grade 3 Follicular Lymphoma Stage I Mantle Cell Lymphoma Stage I Marginal Zone Lymphoma Stage I Multiple Myeloma Stage I Mycosis Fungoides/Sezary Syndrome Stage I Small Lymphocytic Lymphoma Stage II Adult Hodgkin Lymphoma Stage II Adult T-cell Leukemia/Lymphoma Stage II Cutaneous T-cell Non-Hodgkin Lymphoma Stage II Multiple Myeloma Stage II Mycosis Fungoides/Sezary Syndrome Stage III Adult Burkitt Lymphoma Stage III Adult Diffuse Large Cell Lymphoma Stage III Adult Diffuse Mixed Cell Lymphoma Stage III Adult Diffuse Small Cleaved Cell Lymphoma Stage III Adult Hodgkin Lymphoma Stage III Adult Immunoblastic Large Cell Lymphoma Stage III Adult Lymphoblastic Lymphoma Stage III Adult T-cell Leukemia/Lymphoma Stage III Cutaneous T-cell Non-Hodgkin Lymphoma Stage III Grade 1 Follicular Lymphoma Stage III Grade 2 Follicular Lymphoma Stage III Grade 3 Follicular Lymphoma Stage III Mantle Cell Lymphoma Stage III Marginal Zone Lymphoma Stage III Multiple Myeloma Stage III Mycosis Fungoides/Sezary Syndrome Stage III Small Lymphocytic Lymphoma Stage IV Adult Burkitt Lymphoma Stage IV Adult Diffuse Large Cell Lymphoma Stage IV Adult Diffuse Mixed Cell Lymphoma Stage IV Adult Diffuse Small Cleaved Cell Lymphoma Stage IV Adult Hodgkin Lymphoma Stage IV Adult Immunoblastic Large Cell Lymphoma Stage IV Adult Lymphoblastic Lymphoma Stage IV Adult T-cell Leukemia/Lymphoma Stage IV Cutaneous T-cell Non-Hodgkin Lymphoma Stage IV Grade 1 Follicular Lymphoma Stage IV Grade 2 Follicular Lymphoma Stage IV Grade 3 Follicular Lymphoma Stage IV Mantle Cell Lymphoma Stage IV Marginal Zone Lymphoma Stage IV Mycosis Fungoides/Sezary Syndrome Stage IV Small Lymphocytic Lymphoma Testicular Lymphoma Untreated Adult Acute Lymphoblastic Leukemia Untreated Adult Acute Myeloid Leukemia Waldenstrom Macroglobulinemia Drug: deferasirox Other: laboratory biomarker analysis Other: enzyme-linked immunosorbent assay Phase 2

Detailed Description:

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.

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 16 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Supportive Care
Official Title: Impact of Intervention With Deferasirox on the Immune Function of Patients With Hematologic Diseases and Transfusion-Related Iron Overload
Study Start Date : January 2011
Actual Primary Completion Date : March 2012
Actual Study Completion Date : December 2014

Resource links provided by the National Library of Medicine

Drug Information available for: Deferasirox
Genetic and Rare Diseases Information Center resources: Acute Lymphoblastic Leukemia Lymphoblastic Lymphoma Myeloid Leukemia Acute Myeloid Leukemia Acute Non Lymphoblastic Leukemia Multiple Myeloma Myelodysplastic Syndromes Chronic Myeloproliferative Disorders Myelodysplastic/myeloproliferative Disease Lymphosarcoma Mantle Cell Lymphoma Follicular Lymphoma B-cell Lymphoma Diffuse Large B-Cell Lymphoma Marginal Zone Lymphoma Cutaneous T-cell Lymphoma Chronic Lymphocytic Leukemia Peripheral T-cell Lymphoma Extranodal Nasal NK/T Cell Lymphoma Hodgkin Lymphoma Acute Graft Versus Host Disease Anaplastic Large Cell Lymphoma Primary Myelofibrosis Waldenstrom Macroglobulinemia Burkitt Lymphoma Mycosis Fungoides Plasmablastic Lymphoma Lymphoma, Large-cell, Immunoblastic Adult T-cell Leukemia/lymphoma Langerhans Cell Histiocytosis Sezary Syndrome Angioimmunoblastic T-cell Lymphoma Angioimmunoblastic Lymphadenopathy With Dysproteinemia Leukemia, T-cell, Chronic Lymphomatoid Granulomatosis Chronic Graft Versus Host Disease Mastocytosis

Arms and Interventions
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Arm Intervention/treatment
Experimental: 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.
 Drug: deferasirox
Given orally
Other Names:
  • Exjade
  • ICL670

Other: laboratory biomarker analysis
Correlative studies

Other: enzyme-linked immunosorbent assay
Correlative studies
Other Name: ELISA
No Intervention: control arm
blood tested on healthy patients
No Intervention: correlative
treated off study with or without oral deferasirox (patient choice) but lab draws to gather lab analysis


Outcome Measures
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Primary Outcome Measures :
  1. Changes in Mean Neutrophil Values (as Measured by Lab) for Arm 1 (Other Arms Were Used for Calibration Only) [ Time Frame: Baseline, up to 6 months ]
    Changes in Neutrophils between baseline and mean neutrophils values during treatment (measured after each dose)


Secondary Outcome Measures :
  1. Need for Hospitalization, Ventilator Support, Exchange Transfusion/Apheresis or Treatment With Antifungals or Antibiotics [ Time Frame: Baseline, up to 6 months ]
    Records will be assessed at baseline and prospectively while on study.

  2. Cumulative Incidence of Documented Bacterial, Fungal, and Viral Infections [ Time Frame: Baseline, up to 6 months ]
    Records will be assessed at baseline and prospectively while on study.


Eligibility Criteria
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Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Patients must have a pathology confirmed diagnosis of one of the following: myelodysplastic syndrome (MDS); acute leukemia; multiple myeloma; myelofibrosis; lymphoma; chronic anemia; sickle cell anemia
  • Iron score >= 2
  • Absolute Neutrophil Count (ANC) >= 1,000
  • Platelets >= 50,000
  • Albumin >= 2 g/dL
  • Alkaline phosphatase =< 5X Upper Limit of Normal (ULN)
  • Total bilirubin =< 1.5
  • Creatinine =< 2X age-appropriate Upper Limit of Normal (ULN) OR creatinine clearance >= 40 ml/min
  • Serum Glutamic Oxaloacetic Transaminase (SGOT) [AST] and Serum Glutamic Pyruvic Transaminase (SGPT) [ALT] =< 5X Upper Limit of Normal (ULN)
  • Eastern Cooperative Oncology Group(ECOG) performance status of 0 or 1
  • Women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation; should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately
  • Ability to understand and the willingness to sign a written informed consent document

Exclusion Criteria:

  • Patients with active disease undergoing chemotherapy treatment
  • Patient who have been treated with rituximab or immunomodulating drugs =< 1 month prior to enrollment
  • HIV-positive patients
  • Hepatitis-C positive patients
  • Women who are pregnant or breastfeeding
  • Patients on hemodialysis/patients with renal failure
  • Patients with sepsis or acute illness
  • Known hypersensitivity to deferasirox
  • Patients with moderate or severe hearing loss as defined by audiogram
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01273766


Locations
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United States, North Carolina
Comprehensive Cancer Center of Wake Forest University
Winston-Salem, North Carolina, United States, 27157
Sponsors and Collaborators
Wake Forest University Health Sciences
National Cancer Institute (NCI)
Investigators
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Principal Investigator: Mary Ann Knovich, MD Wake Forest University Health Sciences
More Information
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Responsible Party: Wake Forest University Health Sciences
ClinicalTrials.gov Identifier: NCT01273766    
Other Study ID Numbers: IRB00015287
NCI-2010-02228 ( Other Grant/Funding Number: NCI )
CCCWFU 97710 ( Other Identifier: Wake Forest University Health Sciences )
First Posted: January 10, 2011    Key Record Dates
Results First Posted: November 27, 2013
Last Update Posted: September 7, 2018
Last Verified: August 2018

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Burkitt Lymphoma
Mycoses
Lymphoma
Leukemia
Leukemia, Myeloid
Multiple Myeloma
Neoplasms, Plasma Cell
Leukemia, Myeloid, Acute
Lymphoma, Follicular
Preleukemia
Lymphoma, Non-Hodgkin
Neoplasm Metastasis
Precursor Cell Lymphoblastic Leukemia-Lymphoma
Leukemia, Lymphoid
Lymphoma, B-Cell
 Hodgkin Disease
Lymphoma, Mantle-Cell
Lymphoma, B-Cell, Marginal Zone
Lymphoma, T-Cell
Lymphoma, T-Cell, Peripheral
Leukemia, Lymphocytic, Chronic, B-Cell
Lymphoma, Large B-Cell, Diffuse
Lymphoma, Large-Cell, Immunoblastic
Plasmablastic Lymphoma
Mycosis Fungoides
Sezary Syndrome
Leukemia, T-Cell
Lymphoma, T-Cell, Cutaneous
Waldenstrom Macroglobulinemia
Leukemia-Lymphoma, Adult T-Cell