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Cardiac T2* in Beta-thalassemia Patients on Deferasirox Treatment

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ClinicalTrials.gov Identifier: NCT00447694
Recruitment Status : Completed
First Posted : March 15, 2007
Results First Posted : June 7, 2021
Last Update Posted : June 14, 2021
Information provided by (Responsible Party):

Tracking Information
First Submitted Date  ICMJE March 13, 2007
First Posted Date  ICMJE March 15, 2007
Results First Submitted Date  ICMJE May 11, 2021
Results First Posted Date  ICMJE June 7, 2021
Last Update Posted Date June 14, 2021
Study Start Date  ICMJE February 2006
Actual Primary Completion Date November 2009   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 11, 2021)
Magnetic Resonance Imaging (MRI) T2* and Absolute Change From Baseline in MRI T2* [ Time Frame: From Baseline to 25, 49, 77 Week ]
Cardiac T2* was measured in the short axis plane at the widest point of a 4-chamber localizer using custom breath-hold R2* gradient echo sequences modeled after techniques used by Anderson et al (2001) and Westwood et al (2003).
Original Primary Outcome Measures  ICMJE
 (submitted: March 13, 2007)
Cardiac Iron in B thalassemia patients
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: May 11, 2021)
  • Change From Baseline in Liver Iron Concentration (LIC) Was Measured by MRI R2 From Absolute Change From Baseline to 101 Weeks [ Time Frame: From Baseline to 25, 49, 77 Week ]
    MRI evaluation of liver iron concentration has been validated by liver biopsy (St Pierre et al 2005). Studies comparing T2* values of liver iron concentration (LIC) with LIC as assessed by biopsy have confirmed that T2* values reflect liver iron content (Wood et al 2003b). Direct tissue-validation of cardiac T2* measurements in humans has not been performed because endomyocardial biopsy is a dangerous and unreliable indicator of cardiac iron overload (Olson et al 1989, Fitchett et al 1980). However, it has been shown that cardiac T2* accurately reflects cardiac iron in a gerbil iron cardiomyopathy model (Wood et al 2004b). T2* measurements have shown excellent inter-scanner and inter-exam reproducibility, making them suitable for longitudinal monitoring (Westwood et al 2003a, Westwood et al 2003b).
  • Left Ventricular Ejection Fraction (LVEF) and Change in Left Ventricular Ejection Fraction From Baseline to 101 Weeks [ Time Frame: From Baseline to 25, 49, 77 Week ]
    Magnetic resonance imaging (MRI)-measured cardiac T2* and cardiac function reflected by left and right ventricle ejection fraction. A standardized MRI protocol for T2* acquisition technique will be used in the centers. Images will be reviewed centrally by an expert MRI reader.
  • Serum Ferritin and Changes From Baseline in Serum Ferritin During Study [ Time Frame: From Baseline to 25, 49, 77 Week ]
    Serum ferritin will be assessed at each study visit. Analysis was performed in Completer population consists of those participants who had a Week 77 MRI.
Original Secondary Outcome Measures  ICMJE
 (submitted: March 13, 2007)
  • Safety and tolerabilty of deferasirox
  • Changes in liver iron concentration (LIC)
  • Changes in ventricular ejection fraction
  • Ferritin, NTBI (LPI and DCI)& iron metabolism parameters
  • Compliance with deferasirox
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
Descriptive Information
Brief Title  ICMJE Cardiac T2* in Beta-thalassemia Patients on Deferasirox Treatment
Official Title  ICMJE An Open Label Trial Evaluating Cardiac T2* in Beta-thalassemia Patients on Deferasirox (ICL670) Treatment for 18 Months
Brief Summary The purpose of this trial is to evaluate changes in cardiac iron as measured by MRI T2* in beta-thalassemia patients with deferasirox treatment.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Beta-thalassemia
  • Iron Overload
Intervention  ICMJE Drug: Deferasirox
Oral deferasirox 30mg/kg/day once per day for 77 weeks.
Study Arms  ICMJE Experimental: deferasirox every day for 77 weeks
Participants received Deferasirox 30 milligrams per kilogram per day (mg/kg/day) orally once daily (OD), 30 minutes before breakfast, preferably around the same time every morning if possible. Deferasirox tablets were dropped into water or orange juice, or apple juice and stirred until completely dispersed. For doses less than 1 gram (g), tablets were dissolved in at least 100 milliliter (mL) of liquid; for doses of 1 to 3 g, tablets were dissolved in at least 200 mL. After tablets were fully disintegrated, the liquid was promptly consumed.
Intervention: Drug: Deferasirox
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: March 13, 2007)
Original Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE November 2009
Actual Primary Completion Date November 2009   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Male or female β-thalassemia outpatients on chronic transfusion therapy (defined as > 8 transfusions per year)
  • Lifetime minimum of 100 previous packed red blood cell transfusions
  • Patients currently on chelation therapy will require a one day wash out prior to the first dose of study drug
  • Age ≥ 10 years
  • Sexually active females of childbearing potential must have a negative serum or urine pregnancy test and use an effective method of contraception, or must have undergone clinically documented total hysterectomy.

Exclusion Criteria:

  • Ejection Fraction < 56 % measured using steady-state free precession imaging by MRI
  • Contraindication to MRI, including cardiac pacemaker, brain aneurysm clip, implanted neurostimulator, insulin pump, cochlear implant, metal slivers in the eyes, intrauterine device or any other MRI incompatible metal implants or intractable claustrophobia
  • Abnormal laboratory values as defined by the protocol
  • Clinical or laboratory evidence of active Hepatitis B or Hepatitis C
  • History of HIV positive test result (ELISA or Western blot)
  • Uncontrolled systemic hypertension
  • Second or third degree A-V block
  • Life-threatening arrhythmias, including sustained ventricular tachycardia and aborted sudden death, within the last year
  • History of cardiac conditions or unstable cardiac disease not controlled by standard medical therapy
  • History of clinically relevant ocular toxicity related to iron chelation
  • Systemic diseases (cardiovascular, renal, hepatic, etc.) which would prevent study treatment
  • Pregnancy or breast feeding (documented negative pregnancy test required for study entry)
  • Patients enrolled in an ongoing clinical trial of deferasirox (ICL670) cannot be withdrawn in order to participate in this study
  • Treatment with systemic investigational drug within the past 4 weeks or topical investigational drug within the past 7 days
  • Other surgical or medical condition which might significantly alter the absorption, distribution, metabolism or excretion of any drug
  • History of non-compliance to medical regimens or patients who are considered potentially unreliable and/or not cooperative
  • Other inclusion/exclusion criteria may apply
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 10 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
Administrative Information
NCT Number  ICMJE NCT00447694
Other Study ID Numbers  ICMJE CICL670AUS04
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Current Responsible Party Novartis
Original Responsible Party Not Provided
Current Study Sponsor  ICMJE Novartis
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Thomas Coates, MD Childresn's Hospital of Los Angeles
Principal Investigator: Alexis Thompson, MD Children's Memorial Hospital of Chicago
Principal Investigator: Paul Harmatz, MD Children's Hospital and Research Center at Oakland
PRS Account Novartis
Verification Date June 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP