A Study of Long-Term Treatment With Deferasirox in Patients With Beta-Thalassemia and Transfusional Hemosiderosis

This study has been completed.

Sponsored by:	Novartis
Information provided by:	Novartis
ClinicalTrials.gov Identifier:	NCT00171171

Purpose

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	Intervention	Phase
Beta-Thalassemia Hemosiderosis	Drug: deferasirox	Phase III

Genetics Home Reference related topics: beta thalassemia

MedlinePlus related topics: Thalassemia

Drug Information available for: Deferasirox Deferoxamine Deferoxamine mesylate 1,2-Dimethyl-3-hydroxypyrid-4-one

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study
Official Title:	A Study of Efficacy and Safety of Long-Term Treatment With Deferasirox in Patients With Beta-Thalassemia and Transfusional Hemosiderosis

Further study details as provided by Novartis:

Study Start Date:	May 2004
Estimated Study Completion Date:	August 2005

Eligibility

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

Please refer to this study by its ClinicalTrials.gov identifier: NCT00171171

Locations

Lebanon
Ali Taher
Beirut, Lebanon, Riad El Solh 1107 2020

Sponsors and Collaborators

Novartis

Investigators

Principal Investigator:

Ali Taher, Ass.Prof.

American University of Beirut Medical Center

More Information

Study ID Numbers:	CICL670A2402
Study First Received:	September 12, 2005
Last Updated:	January 25, 2007
ClinicalTrials.gov Identifier:	NCT00171171
Health Authority:	Lebanon: Institutional Review Board

Keywords provided by Novartis:

Thalassemia (beta-thal. major)
Transfusional hemosiderosis
Deferasirox
Beta-thalassemia major patients
Unable to be chelated with deferoxamine or deferiprone

Study placed in the following topic categories:

Metabolic Diseases
Deferasirox
Hematologic Diseases
Deferiprone
Beta-thalassemia
Anemia
Anemia, Hemolytic
Iron Metabolism Disorders
Thalassemia
Anemia, Hemolytic, Congenital

Thalassemia minor
Genetic Diseases, Inborn
Hemosiderosis
Beta-Thalassemia
Hemoglobinopathies
Iron Overload
Metabolic disorder
Hemoglobinopathy
Iron
Deferoxamine

Additional relevant MeSH terms:

Molecular Mechanisms of Pharmacological Action
Iron Chelating Agents
Chelating Agents
Pharmacologic Actions

ClinicalTrials.gov processed this record on January 16, 2009