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Cardiac T2* in Beta-thalassemia Patients on Deferasirox Treatment

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Novartis
ClinicalTrials.gov Identifier:
NCT00447694
First received: March 13, 2007
Last updated: April 25, 2012
Last verified: April 2012
  Purpose

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 Intervention Phase
Beta-thalassemia
Iron Overload
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: Treatment
Official Title: An Open Label Trial Evaluating Cardiac T2* in Beta-thalassemia Patients on Deferasirox (ICL670) Treatment for 18 Months

Resource links provided by NLM:


Further study details as provided by Novartis:

Primary Outcome Measures:
  • Cardiac Iron in B thalassemia patients [ Time Frame: From baseline to week 25 and weeks 49 and 77 of the trial ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Safety and tolerabilty of deferasirox [ Time Frame: throughout the study ] [ Designated as safety issue: Yes ]
  • Changes in liver iron concentration (LIC) [ Time Frame: From baseline to week 25 and weeks 49 and 77 of trial ] [ Designated as safety issue: Yes ]
  • Changes in ventricular ejection fraction [ Time Frame: From baseline to weeks 25, 49 and 77 ] [ Designated as safety issue: Yes ]
  • Ferritin, NTBI (LPI and DCI)& iron metabolism parameters [ Time Frame: From Baseline through week 25, and weeks 49 and 77 of trial ] [ Designated as safety issue: Yes ]
  • Compliance with deferasirox [ Time Frame: Throughout the study ] [ Designated as safety issue: Yes ]

Enrollment: 30
Study Start Date: February 2006
Study Completion Date: November 2009
Primary Completion Date: November 2009 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: deferasirox every day for 77 weeks
particpants will be given oral deferasirox 30mg/kg/day for 77 weeks.
Drug: Deferasirox
Oral deferasirox 30mg/kg/day once per day for 77 weeks.

  Eligibility

Ages Eligible for Study:   10 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  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: NCT00447694

Locations
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
Novartis
Investigators
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
  More Information

No publications provided by Novartis

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Novartis
ClinicalTrials.gov Identifier: NCT00447694     History of Changes
Other Study ID Numbers: CICL670AUS04
Study First Received: March 13, 2007
Last Updated: April 25, 2012
Health Authority: United States: Food and Drug Administration

Keywords provided by Novartis:
Iron Chelation
Deferasirox
Chelator
Desferal
beta-thalassemia
Iron overload

Additional relevant MeSH terms:
Beta-Thalassemia
Iron Overload
Thalassemia
Anemia
Anemia, Hemolytic
Anemia, Hemolytic, Congenital
Genetic Diseases, Inborn
Hematologic Diseases
Hemoglobinopathies
Iron Metabolism Disorders
Metabolic Diseases
Deferasirox
Chelating Agents
Iron Chelating Agents
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Sequestering Agents

ClinicalTrials.gov processed this record on November 25, 2014