Cardiac T2* in Beta-thalassemia Patients on Deferasirox Treatment - Full Text View

Sponsor:	Novartis
Information provided by:	Novartis
ClinicalTrials.gov Identifier:	NCT00447694

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 II

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:

Genetics Home Reference related topics: beta thalassemia

MedlinePlus related topics: Iron Thalassemia

Drug Information available for: Deferasirox

U.S. FDA Resources

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 ]

Estimated 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

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):

Wood JC, Glynos T, Thompson A, Giardina P, Harmatz P, Kang BP, Paley C, Coates TD. Relationship between labile plasma iron, liver iron concentration and cardiac response in a deferasirox monotherapy trial. Haematologica. 2011 Jul;96(7):1055-8. Epub 2011 Mar 10.

Wood JC, Kang BP, Thompson A, Giardina P, Harmatz P, Glynos T, Paley C, Coates TD. The effect of deferasirox on cardiac iron in thalassemia major: impact of total body iron stores. Blood. 2010 Jul 29;116(4):537-43. Epub 2010 Apr 26.

Responsible Party:	Novartis External Affairs, Novartis
ClinicalTrials.gov Identifier:	NCT00447694 History of Changes
Other Study ID Numbers:	CICL670AUS04
Study First Received:	March 13, 2007
Last Updated:	November 18, 2009
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
Thalassemia
Iron Overload
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies

Genetic Diseases, Inborn
Iron Metabolism Disorders
Metabolic Diseases
Deferasirox
Iron Chelating Agents
Chelating Agents
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions

ClinicalTrials.gov processed this record on February 09, 2012