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Cardiac T2* in Beta-thalassemia Patients on Deferasirox Treatment

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. Identifier: NCT00447694
Recruitment Status : Completed
First Posted : March 15, 2007
Last Update Posted : October 22, 2020
Information provided by (Responsible Party):

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.

Condition or disease Intervention/treatment Phase
Beta-thalassemia Iron Overload Drug: Deferasirox Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 30 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open Label Trial Evaluating Cardiac T2* in Beta-thalassemia Patients on Deferasirox (ICL670) Treatment for 18 Months
Study Start Date : February 2006
Actual Primary Completion Date : November 2009
Actual Study Completion Date : November 2009

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Iron Thalassemia
Drug Information available for: Deferasirox

Arm Intervention/treatment
Experimental: deferasirox every day for 77 weeks
participants will be given oral deferasirox 30mg/kg/day for 77 weeks.
Drug: Deferasirox
Oral deferasirox 30mg/kg/day once per day for 77 weeks.

Primary Outcome Measures :
  1. Cardiac Iron in B thalassemia patients [ Time Frame: From baseline to week 25 and weeks 49 and 77 of the trial ]

Secondary Outcome Measures :
  1. Safety and tolerability of deferasirox [ Time Frame: throughout the study ]
  2. Changes in liver iron concentration (LIC) [ Time Frame: From baseline to week 25 and weeks 49 and 77 of trial ]
  3. Changes in ventricular ejection fraction [ Time Frame: From baseline to weeks 25, 49 and 77 ]
  4. Ferritin, NTBI (LPI and DCI)& iron metabolism parameters [ Time Frame: From Baseline through week 25, and weeks 49 and 77 of trial ]
  5. Compliance with deferasirox [ Time Frame: Throughout the study ]

Information from the National Library of Medicine

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Ages Eligible for Study:   10 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

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 identifier (NCT number): NCT00447694

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United States, California
Childrens Hospital of Los Angeles
Los Angeles, California, United States, 90027
Children's Hospital and Research Center at Oakland
Oakland, California, United States, 94609
United States, Illinois
Children's Memorial Hospital
Chicago, Illinois, United States, 60614
Sponsors and Collaborators
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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
Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: Novartis Identifier: NCT00447694    
Other Study ID Numbers: CICL670AUS04
First Posted: March 15, 2007    Key Record Dates
Last Update Posted: October 22, 2020
Last Verified: October 2020
Keywords provided by Novartis:
Iron Chelation
Iron overload
Additional relevant MeSH terms:
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Iron Overload
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hematologic Diseases
Genetic Diseases, Inborn
Iron Metabolism Disorders
Metabolic Diseases
Iron Chelating Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action