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Safety & Efficacy of ICL670 vs. Deferoxamine in Beta-thalassemia Patients With Iron Overload Due to Blood Transfusions

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.
 
ClinicalTrials.gov Identifier: NCT00061750
Recruitment Status : Completed
First Posted : June 4, 2003
Last Update Posted : April 19, 2012
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Study Description
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Brief Summary:
The purpose of this study is to deterimine if the new orally active iron chelator, ICL670, is as effective and as safe as deferoxamine in preventing accumulation of iron in the body while a patient is undergoing repeated blood transfusions.

Condition or disease Intervention/treatment Phase
Beta-Thalassemia Drug: ICL670 Drug: deferoxamine Phase 3

Detailed Description:
Patients who require repeated blood transfusions to live accumulate iron in the body as blood cells contain iron and there is no natural body mechanism to eliminate it. After a while the iron levels get high enough to be toxic to the body. The current therapy of choice is deferoxamine, which does a good job of removing excess iron, but is difficult to administer. Deferoxamine requires subcutaneous (under the skin) infusions over 4 to 8 hours nightly 3 to 7 nights per week. In addition to the need to wear an infusion pump nightly, adverse reactions around the site of the injection are frequent.
Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 595 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Randomized, Comparative, Open Label Phase III Trial on Efficacy & Safety of Long-term Treatment With ICL670 Compared to Deferoxamine in Beta-thalassemia Patients With Transfusional Hemosiderosis
Study Start Date : May 2003
Actual Primary Completion Date : November 2004

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Iron Thalassemia

Arms and Interventions
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Arm Intervention/treatment
Experimental: ICL670 Drug: ICL670
Other Name: Deferasirox
Active Comparator: Deferoxamine Drug: deferoxamine


Outcome Measures
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Primary Outcome Measures :
  1. Demonstrate non-inferiority to deferoxamine in its effects on liver iron content (LIC)

Secondary Outcome Measures :
  1. Evaluate tolerability profile
  2. Estimate absolute and relative change of LIC and Total body iron excretion
  3. Evaluation relationship between LIC and potential surrogate markers
  4. Evaluate the relationship between pharmacokinetics, pharmacodynamics and safety variable

Eligibility Criteria
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Ages Eligible for Study:   2 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Beta-thalassemia patients already treated with or suitable for treatment with deferoxamine 20 to 40 mg/kg/day
  • Liver iron content greater than 2 mg iron/g dw as measured by liver biopsy
  • Need for regular transfusions 8 or more times per year

Exclusion Criteria:

  • Non-transfusional iron overload or transfusion-dependent anemias other than beta-thalassemia.
  • Documented toxicity to deferoxamine
  • Elevated liver enzymes in the year preceeding enrollment
  • Active hepatitis B or hepatitis C
  • HIV seropositivity
  • Elevated serum creatinine or significant proteinuria
  • History of nephrotic syndrome
  • Uncontrolled systemic hypertension
  • Fever and other signs/symptoms of infection within 10 days prior to start of the study
  • Presence of clinically relevant cataract or previous history of clinically relevant ocular toxicity related to iron chelation
  • Second or third degree AV block, clinically relevant Q-T interval prolongation, or patients requiring digoxin or other drugs that prolong the Q-T interval
  • Diseases (cardiovascular, renal, hepatic, etc.)that would prevent the patient from undergoing any of the treatment options
  • Psychiatric or additive disorders that would prevent the patient from giving informed consent
  • History of drug or alcohol abuse within the 12 months prior to the study
  • Pregnant or breast feeding patients
  • Patients treated with systemic investigational drugs within 4 weeks or topical investigational drugs within 7 days before the start of the study
  • Any surgical or medical condition that might significantly alter the absorption, distribution, metabolism or excretion of any drug, such as gastrointestinal disease or major surgery, renal disease, difficulty voiding or urinary obstruction, or impaired pancreatic function.
  • Non-compliant or unreliable patients.
  • Patients unable to undergo any study procedures such as the hearing or eye tests, or the liver echocardiography.
  • Inability to undergo a liver biopsy.
  • Patients that would need a dose of ICL670 less than 125 mg per day.
Contacts and Locations
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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 ClinicalTrials.gov identifier (NCT number): NCT00061750


Locations
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United States, California
Children's Hospital of Los Angeles
Los Angeles, California, United States, 90027-6062
Children's Hospital Oakland
Oakland, California, United States, 94609
Stanford Hospital
Stanford, California, United States, 94305-5208
United States, Illinois
Children's Memorial Hospital
Chicago, Illinois, United States, 60614-3394
United States, Massachusetts
Children's Hospital Boston
Boston, Massachusetts, United States, 02115
United States, New York
Weill Medical College of Cornell University
New York, New York, United States, 10021
United States, Pennsylvania
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104-4318
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
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Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT00061750    
Other Study ID Numbers: CICL670A0107
First Posted: June 4, 2003    Key Record Dates
Last Update Posted: April 19, 2012
Last Verified: April 2012
Keywords provided by Novartis ( Novartis Pharmaceuticals ):
Thalassemia, iron overload, deferoxamine, hemosiderosis
Additional relevant MeSH terms:
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Thalassemia
beta-Thalassemia
Iron Overload
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
 Iron Metabolism Disorders
Metabolic Diseases
Deferasirox
Deferoxamine
Iron Chelating Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action
Siderophores