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

Sponsor:	Novartis Pharmaceuticals
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

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

Resource links provided by NLM:

Genetics Home Reference related topics: beta thalassemia

MedlinePlus related topics: Blood Transfusion and Donation Thalassemia

Drug Information available for: Deferoxamine Deferasirox Deferoxamine mesylate

U.S. FDA Resources

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 Pharmaceuticals

Investigators

Study Chair:

Novartis

More Information

Additional Information:

Visit NovartisClinicalTrials.com: Pre-qualify for a trial, and view a list of trials and participating study centers. This link exits the ClinicalTrials.gov site

This link exits the ClinicalTrials.gov site

No publications provided by Novartis

Additional publications automatically indexed to this study by National Clinical Trials Identifier (NCT ID):

Cohen AR, Glimm E, Porter JB. Effect of transfusional iron intake on response to chelation therapy in beta-thalassemia major. Blood. 2008 Jan 15;111(2):583-7. Epub 2007 Oct 19.

Study ID Numbers:	CICL670A0107
Study First Received:	June 3, 2003
Last Updated:	September 21, 2009
ClinicalTrials.gov Identifier:	NCT00061750 History of Changes
Health Authority:	United States: Food and Drug Administration

Keywords provided by Novartis:

Thalassemia, iron overload, deferoxamine, hemosiderosis

Additional relevant MeSH terms:

Metabolic Diseases
Molecular Mechanisms of Pharmacological Action
Deferasirox
Hematologic Diseases
Iron Chelating Agents
Anemia
Anemia, Hemolytic
Iron Metabolism Disorders
Thalassemia
Pharmacologic Actions

Siderophores
Anemia, Hemolytic, Congenital
Genetic Diseases, Inborn
Hemosiderosis
Beta-Thalassemia
Hemoglobinopathies
Chelating Agents
Iron Overload
Deferoxamine

ClinicalTrials.gov processed this record on November 09, 2009