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A Study of Long-term Treatment With Deferasirox in Patients With Beta-thalassemia and Transfusional Hemosiderosis

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: NCT00171171
Recruitment Status : Completed
First Posted : September 15, 2005
Last Update Posted : March 1, 2017
Sponsor:
Information provided by:
Novartis

Study Description
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Brief Summary:
Because patients with beta-thalassemia are unable to actively eliminate iron from the body, toxic and eventually lethal levels of iron can accumulate as a result of repeated blood transfusions. This study will evaluate the efficacy, safety and tolerability of deferasirox.

Condition or disease Intervention/treatment Phase
Beta-Thalassemia Hemosiderosis Drug: deferasirox Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 252 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Study of Efficacy and Safety of Long-term Treatment With Deferasirox in Patients With Beta-thalassemia and Transfusional Hemosiderosis
Study Start Date : May 2004
Actual Primary Completion Date : November 2006

Resource links provided by the National Library of Medicine

Drug Information available for: Deferasirox

Arms and Interventions
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Arm Intervention/treatment
Experimental: Deferasirox Drug: deferasirox


Outcome Measures
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Primary Outcome Measures :
  1. Liver Iron Concentration (as measured by biopsy) [ Time Frame: at baseline and after 1 yeor of ICL670 treatment ]

Secondary Outcome Measures :
  1. Iron balance, i.e. Total Body Iron Excretion (based on the iron influx as determined by the amount of red cells transfused and the change in total body iron (TBI) stores) [ Time Frame: after 1 year of ICL670 treatment ]
  2. Putative surrogate markers such as serum ferritin serum iron, serum transferrin and transferrin saturation [ Time Frame: monthly assessments for 1 year ]
  3. Adverse events [ Time Frame: during 1 year ICL670 treatment ]
  4. hematology and biochemistry, urinalysis [ Time Frame: monthly assessments for 1 year ICL670 treatment ]
  5. ECG and Echocardiography [ Time Frame: 6-monthly for 1 year ICL670 treatment ]

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 outpatients with transfusional hemosiderosis and unable to be chelated with deferoxamine (DFO) due to DFO being contra-indicated and/or due to documented unacceptable toxicity of DFO or documented poor response to DFO despite proper compliance, or documented non-compliance to DFO, with serum ferritin ≥ 500 ng/ml and LIC ≥ 2 mg/Fe/g dw liver
  • Beta-thalassemia outpatients with transfusional hemosiderosis treated with deferiprone

Exclusion Criteria:

  • Means levels of ALT > 300 U/L
  • Serum creatinine above upper limit of normal
  • Active hepatitis C or chronic hepatitis B receiving specific treatment
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): NCT00171171


Locations
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Egypt
Novartis Investigative Site
Cairo, Egypt
Lebanon
Ali Taher
Beirut, Lebanon, Riad El Solh 1107 2020
Oman
Novartis Investigative Site
Muscat, Oman
Saudi Arabia
Novartis Investigative Site
Riyadh, Saudi Arabia
Syrian Arab Republic
Novartis Investigative Site
Damascus, Syrian Arab Republic
Sponsors and Collaborators
Novartis
Investigators
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Principal Investigator: Ali Taher, Ass.Prof. American University of Beirut Medical Center
More Information
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Additional Information:

Publications of Results:

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Responsible Party: External Affairs, Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT00171171    
Other Study ID Numbers: CICL670A2402
First Posted: September 15, 2005    Key Record Dates
Last Update Posted: March 1, 2017
Last Verified: November 2016
Keywords provided by Novartis:
Thalassemia (beta-thal. major)
Transfusional hemosiderosis
Deferasirox
Beta-thalassemia major patients
Unable to be chelated with deferoxamine or deferiprone
Additional relevant MeSH terms:
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Thalassemia
beta-Thalassemia
Hemochromatosis
Hemosiderosis
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
 Iron Overload
Iron Metabolism Disorders
Metabolic Diseases
Metal Metabolism, Inborn Errors
Metabolism, Inborn Errors
Deferasirox
Iron Chelating Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action