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Deferasirox in Treating Patients With Iron Overload After Undergoing a Donor Stem Cell Transplant

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ClinicalTrials.gov Identifier: NCT00602446
Recruitment Status : Terminated (Due to slow accrual of patients)
First Posted : January 28, 2008
Results First Posted : March 30, 2010
Last Update Posted : December 28, 2017
Information provided by (Responsible Party):

Study Description
Brief Summary:

RATIONALE: Deferasirox may be effective in treating iron overload caused by blood transfusions in patients who have undergone donor stem cell transplant.

PURPOSE: This phase II trial is studying the side effects and how well deferasirox works in treating patients with iron overload after donor stem cell transplant.

Condition or disease Intervention/treatment Phase
Breast Cancer Iron Overload Leukemia Lymphoma Multiple Myeloma and Plasma Cell Neoplasm Myelodysplastic Syndromes Neuroblastoma Ovarian Cancer Drug: deferasirox Phase 2

Detailed Description:



  • To evaluate the safety of deferasirox given over 6 months in reducing liver iron concentration in patients with transfusional iron overload after undergoing allogeneic hematopoietic stem cell transplantation.


  • To evaluate the efficacy of deferasirox in reducing liver iron overload in these patients.

OUTLINE: This is a multicenter study.

Patients receive oral deferasirox once daily for 6 months in the absence of disease progression or unacceptable toxicity.

After completion of study therapy, patients are followed at 4 weeks.

Study Design

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 4 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Supportive Care
Official Title: Open-Label Single-Arm Pilot Study of Deferasirox (Exjade®) in Adult Allogeneic Hematopoietic Stem Cell Transplant Recipients With Transfusional Iron Overload
Study Start Date : August 2007
Primary Completion Date : September 2009
Study Completion Date : December 2009

Arms and Interventions

Arm Intervention/treatment
Experimental: Deferasirox Treated
Includes patients that were treated with deferasirox for 6 months.
Drug: deferasirox
20 mg/kg once daily orally for 6 months
Other Name: Exjade

Outcome Measures

Primary Outcome Measures :
  1. Number of Patients Not Completing Treatment [ Time Frame: 6 Months ]
    Number of patients who discontinued deferasirox during 6 month daily treatment due to drug related toxicity

Secondary Outcome Measures :
  1. Reduction in Liver Iron Concentration After Study Drug [ Time Frame: 6 Months ]
    Efficacy as measured by reduction in liver iron concentration (LIC) after 6 months of the study drug compared to baseline (LIC at baseline minus LIC at 6 months). This shows the mean reduction for the 3 subjects treated in this study.

Eligibility Criteria

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Ages Eligible for Study:   18 Years to 70 Years   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Confirmed diagnosis of iron overload, defined as serum ferritin > 1,000 ng/mL and liver iron concentration ≥ 5 mg iron/g on tissue proton transverse relaxation rates Magnetic Resonance Imaging (MRI)
  • Underwent prior allogeneic hematopoietic stem cell transplantation (HSCT) using either myeloablative or reduced-intensity conditioning at least 12 months ago
  • No evidence of relapse or progression of the primary disease for which allogeneic HSCT was performed
  • Patients who have become red-cell transfusion independent (i.e., no red cell transfusions within the past 3 months) as well as patients who require red cell transfusions are eligible
  • Meets one of the following criteria:

    • Ineligible for phlebotomy (hemoglobin < 11 g/dL, poor intravenous access, or unable to undergo phlebotomy every 4 weeks)
    • Have failed treatment with phlebotomy (serum ferritin > 50% of baseline after 3 months of phlebotomy)
    • Refused phlebotomy
  • ECOG performance status of 0-2
  • Life expectancy ≥ 6 months
  • Adequate renal function defined as serum creatinine < or = 1.6 mg/dL and creatinine clearance of > or = 60 ml/min calculated using the Crockcroft-Gault formula on 2 occasions within 30 days of enrollment
  • Sexually active men and women must use an effective method of contraception. Alternatively, women must have undergone clinically documented total hysterectomy and/or oophorectomy, or tubal ligation or be postmenopausal.
  • Must be able to give written informed consent.
  • Prior therapy with deferoxamine allowed provided it was completed ≥ 12 months ago

Exclusion Criteria:

  • Contraindication for performing MRI or inability to undergo MRI because of claustrophobia or weight (>350 pounds).
  • Inability to take medications orally.
  • Uncontrolled bacterial, viral, or fungal infection
  • ANC ≥ 1,000/mm³
  • Hemoglobin ≥ 8.0 g/dL
  • Platelet count ≥ 50,000/mm³
  • Aspartate aminotransferance (AST) and alanine aminotransferase (ALT) ≤ 5 times the upper limit of normal
  • Less than 4 weeks since prior and no concurrent systemic investigational drug
  • Less than 7 days since prior and no concurrent topical investigational drug. Concurrent non-investigational medications needed to treat concomitant medical conditions are allowed, with the exception of other chelating agents. Concurrent growth factors such as epoetin alfa, darbepoetin alfa, filgrastim (G-CSF), and sargramostim (GM-CSF) allowed. Concurrent irradiated packed red-cell and platelet transfusions allowed as clinically indicated. Concurrent low-doses of vitamin C supplements (≤ 200 mg/day) allowed.
  • Concurrent iron supplements or multivitamins with iron.
  • Aluminum-containing antacid therapies may not be taken simultaneously with deferasirox, but may be taken 2 hours before or after administration of deferasirox
  • On dialysis or status post-renal transplantation
  • Pregnant or nursing
Contacts and Locations

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): NCT00602446

United States, Minnesota
Masonic Cancer Center at University of Minnesota
Minneapolis, Minnesota, United States, 55455
Sponsors and Collaborators
Masonic Cancer Center, University of Minnesota
Principal Investigator: Linda J. Burns, MD Masonic Cancer Center, University of Minnesota
More Information

Responsible Party: Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov Identifier: NCT00602446     History of Changes
Other Study ID Numbers: CDR0000584690
UMN-2007LS065 ( Other Identifier: Masonic Cancer Center, University of Minnesota )
UMN-MT2007-11R ( Other Identifier: Blood and Marrow Transplantation Program )
NOVARTIS-CICL670AUS12 ( Other Identifier: Novartis Pharmaceuticals )
First Posted: January 28, 2008    Key Record Dates
Results First Posted: March 30, 2010
Last Update Posted: December 28, 2017
Last Verified: December 2017

Keywords provided by Masonic Cancer Center, University of Minnesota:
iron overload

Additional relevant MeSH terms:
Multiple Myeloma
Myelodysplastic Syndromes
Iron Overload
Neoplasms, Plasma Cell
Neoplasms by Histologic Type
Hemostatic Disorders
Vascular Diseases
Cardiovascular Diseases
Blood Protein Disorders
Hematologic Diseases
Hemorrhagic Disorders
Lymphoproliferative Disorders
Immunoproliferative Disorders
Immune System Diseases
Bone Marrow Diseases
Precancerous Conditions
Neuroectodermal Tumors, Primitive, Peripheral
Neuroectodermal Tumors, Primitive
Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Iron Metabolism Disorders
Metabolic Diseases