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

Sponsor
Masonic Cancer Center, University of Minnesota (Other)
Overall Status
Terminated
CT.gov ID
NCT00602446
Collaborator
(none)
4
Enrollment
1
Location
1
Arm
28
Duration (Months)
0.1
Patients Per Site Per Month

Study Details

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.

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.

Study Design

Study Type:
Interventional
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 :
Aug 1, 2007
Actual Primary Completion Date :
Sep 1, 2009
Actual Study Completion Date :
Dec 1, 2009

Arms and Interventions

ArmIntervention/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 Names:
  • Exjade
  • Outcome Measures

    Primary Outcome Measures

    1. Number of Patients Not Completing Treatment [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 [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

    Criteria

    Ages Eligible for Study:
    18 Years to 70 Years
    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

    Locations

    SiteCityStateCountryPostal Code
    1Masonic Cancer Center at University of MinnesotaMinneapolisMinnesotaUnited States55455

    Sponsors and Collaborators

    • Masonic Cancer Center, University of Minnesota

    Investigators

    • Principal Investigator: Linda J. Burns, MD, Masonic Cancer Center, University of Minnesota

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Masonic Cancer Center, University of Minnesota
    ClinicalTrials.gov Identifier:
    NCT00602446
    Other Study ID Numbers:
    • CDR0000584690
    • UMN-2007LS065
    • UMN-MT2007-11R
    • NOVARTIS-CICL670AUS12
    First Posted:
    Jan 28, 2008
    Last Update Posted:
    Dec 28, 2017
    Last Verified:
    Dec 1, 2017
    Keywords provided by Masonic Cancer Center, University of Minnesota
    Additional relevant MeSH terms:

    Study Results

    Participant Flow

    Recruitment DetailsOnly one site (Masonic Cancer Center) enrolled patients in this study.
    Pre-assignment Detail4 patients were consented, however, one withdrew consent before receiving treatment.
    Arm/Group TitleDeferasirox Treated
    Arm/Group DescriptionIncludes patients that were treated with deferasirox for 6 months.
    Period Title: Overall Study
    STARTED3
    COMPLETED3
    NOT COMPLETED0

    Baseline Characteristics

    Arm/Group TitleDeferasirox Treated
    Arm/Group DescriptionIncludes patients that were treated with deferasirox for 6 months.
    Overall Participants3
    Age (Count of Participants)
    <=18 years
    0
    0%
    Between 18 and 65 years
    3
    100%
    >=65 years
    0
    0%
    Age (years) [Mean (Standard Deviation) ]
    Mean (Standard Deviation) [years]
    45.7
    (5.0)
    Sex: Female, Male (Count of Participants)
    Female
    0
    0%
    Male
    3
    100%
    Region of Enrollment (participants) [Number]
    United States
    3
    100%

    Outcome Measures

    1. Primary Outcome
    TitleNumber of Patients Not Completing Treatment
    DescriptionNumber of patients who discontinued deferasirox during 6 month daily treatment due to drug related toxicity
    Time Frame6 Months

    Outcome Measure Data

    Analysis Population Description
    Note: 1 patient had a transient decrease in hemoglobin that required discontinuation of treatment for 2 weeks; subsequently restarted at same dose and completed therapy.
    Arm/Group TitleDeferasirox Treated
    Arm/Group DescriptionIncludes patients that were treated with deferasirox for 6 months.
    Measure Participants3
    Number [Participants]
    0
    0%
    2. Secondary Outcome
    TitleReduction in Liver Iron Concentration After Study Drug
    DescriptionEfficacy 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.
    Time Frame6 Months

    Outcome Measure Data

    Analysis Population Description
    All patients included in count.
    Arm/Group TitleDeferasirox Treated
    Arm/Group DescriptionIncludes patients that were treated with deferasirox for 6 months.
    Measure Participants3
    Mean (Standard Deviation) [milligrams/gram]
    5.6
    (4.8)

    Adverse Events

    Time Frame6 Months
    Adverse Event Reporting Description
    Arm/Group TitleDeferasirox Treated
    Arm/Group DescriptionIncludes patients that were treated with deferasirox for 6 months.
    All Cause Mortality
    Deferasirox Treated
    Affected / at Risk (%)# Events
    Total/ (NaN)
    Serious Adverse Events
    Deferasirox Treated
    Affected / at Risk (%)# Events
    Total0/3 (0%)
    Other (Not Including Serious) Adverse Events
    Deferasirox Treated
    Affected / at Risk (%)# Events
    Total3/3 (100%)
    Blood and lymphatic system disorders
    Increased serum creatinine2/3 (66.7%) 2
    Gastrointestinal disorders
    Diarrhea3/3 (100%) 3
    Skin and subcutaneous tissue disorders
    Rash2/3 (66.7%) 2

    Limitations/Caveats

    [Not Specified]

    More Information

    Certain Agreements

    All Principal Investigators ARE employed by the organization sponsoring the study.

    There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

    Results Point of Contact

    Name/TitleLinda J. Burns, MD
    OrganizationMasonic Cancer Center, University of Minnesota
    Phone612-624-8144
    Emailburns019@umn.edu
    Responsible Party:
    Masonic Cancer Center, University of Minnesota
    ClinicalTrials.gov Identifier:
    NCT00602446
    Other Study ID Numbers:
    • CDR0000584690
    • UMN-2007LS065
    • UMN-MT2007-11R
    • NOVARTIS-CICL670AUS12
    First Posted:
    Jan 28, 2008
    Last Update Posted:
    Dec 28, 2017
    Last Verified:
    Dec 1, 2017