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

This study has been completed.

Sponsored by: Novartis
Information provided by: Novartis
ClinicalTrials.gov Identifier: NCT00061750
  Purpose

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 Intervention Phase
Beta-Thalassemia
Drug: ICL670
Drug: deferoxamine
Phase III

Genetics Home Reference related topics:   beta thalassemia   

MedlinePlus related topics:   Blood Transfusion and Donation    Thalassemia   

ChemIDplus related topics:   Deferasirox    Deferoxamine    Deferoxamine mesylate   

U.S. FDA Resources

Study Type:   Interventional
Study Design:   Treatment, Randomized, Open Label, Active Control, Parallel Assignment, Safety/Efficacy Study
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

Further study details as provided by Novartis:

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

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

Estimated Enrollment:   500
Study Start Date:   May 2003

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.

  Eligibility
Ages Eligible for Study:   2 Years and older
Genders Eligible for Study:   Both
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

Please refer to this study by its ClinicalTrials.gov identifier: NCT00061750

Locations
United States, California
Children's Hospital Oakland    
      Oakland, California, United States, 94609
Stanford Hospital    
      Stanford, California, United States, 94305-5208
Children's Hospital of Los Angeles    
      Los Angeles, California, United States, 90027-6062
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

Investigators
Study Chair:     Novartis     Novartis    
  More Information

Publications indexed to this study:

Study ID Numbers:   CICL670A0107
First Received:   June 3, 2003
Last Updated:   August 14, 2006
ClinicalTrials.gov Identifier:   NCT00061750
Health Authority:   United States: Food and Drug Administration

Keywords provided by Novartis:
Thalassemia, iron overload, deferoxamine, hemosiderosis  

Study placed in the following topic categories:
Deferasirox
Hematologic Diseases
Beta-thalassemia
Anemia
Anemia, Hemolytic
Thalassemia
Anemia, Hemolytic, Congenital
Thalassemia minor
Genetic Diseases, Inborn
Hemosiderosis
Beta-Thalassemia
Hemoglobinopathies
Iron Overload
Hemoglobinopathy
Iron
Deferoxamine

Additional relevant MeSH terms:
Molecular Mechanisms of Pharmacological Action
Iron Chelating Agents
Chelating Agents
Pharmacologic Actions
Siderophores

ClinicalTrials.gov processed this record on August 28, 2008




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