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

This study has been terminated.
(Due to slow accrual of patients)
Sponsor:
Information provided by (Responsible Party):
Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov Identifier:
NCT00602446
First received: January 24, 2008
Last updated: November 6, 2012
Last verified: November 2012
  Purpose

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

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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

Resource links provided by NLM:


Further study details as provided by Masonic Cancer Center, University of Minnesota:

Primary Outcome Measures:
  • Number of Patients Not Completing Treatment [ Time Frame: 6 Months ] [ Designated as safety issue: Yes ]
    Number of patients who discontinued deferasirox during 6 month daily treatment due to drug related toxicity


Secondary Outcome Measures:
  • Reduction in Liver Iron Concentration After Study Drug [ Time Frame: 6 Months ] [ Designated as safety issue: No ]
    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.


Enrollment: 4
Study Start Date: August 2007
Study Completion Date: December 2009
Primary Completion Date: September 2009 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

Detailed Description:

OBJECTIVES:

Primary

  • 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.

Secondary

  • 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.

  Eligibility

Ages Eligible for Study:   18 Years to 70 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00602446

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

No publications provided

Responsible Party: Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov Identifier: NCT00602446     History of Changes
Other Study ID Numbers: CDR0000584690, UMN-2007LS065, UMN-MT2007-11R, NOVARTIS-CICL670AUS12
Study First Received: January 24, 2008
Results First Received: March 16, 2010
Last Updated: November 6, 2012
Health Authority: United States: Food and Drug Administration

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

Additional relevant MeSH terms:
Breast Neoplasms
Neoplasms
Leukemia
Lymphoma
Multiple Myeloma
Neoplasms, Plasma Cell
Plasmacytoma
Myelodysplastic Syndromes
Preleukemia
Neuroblastoma
Ovarian Neoplasms
Iron Overload
Neoplasms by Site
Breast Diseases
Skin Diseases
Neoplasms by Histologic Type
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases
Hemostatic Disorders
Vascular Diseases
Cardiovascular Diseases
Paraproteinemias
Blood Protein Disorders
Hematologic Diseases
Hemorrhagic Disorders
Bone Marrow Diseases
Precancerous Conditions
Neuroectodermal Tumors, Primitive, Peripheral

ClinicalTrials.gov processed this record on July 26, 2014