Extension Study on Efficacy and Safety of Deferasirox Treatment in Patients With Beta-Thalassemia and Transfusional Hemosiderosis

This study is ongoing, but not recruiting participants.

Sponsored by:	Novartis
Information provided by:	Novartis
ClinicalTrials.gov Identifier:	NCT00171301

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 is a 1-year extension study and will evaluate the long-term efficacy, safety and tolerability of deferasirox.

Condition	Intervention	Phase
Beta-Thalassemia Major	Drug: ICL670; deferasirox	Phase III

Genetics Home Reference related topics: beta thalassemia

MedlinePlus related topics: Thalassemia

Drug Information available for: Deferasirox

U.S. FDA Resources

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

Further study details as provided by Novartis:

Primary Outcome Measures:

To allow patients treated with ICL670 in the core protocol to continue iron chelation therapy
To evaluate the long-term safety and tolerability profile of ICL670

Secondary Outcome Measures:

To estimate the absolute and relative changes of liver iron content (LIC)
To evaluate the role of serum ferritin and other surrogate markers
To assess drug usage compliance

Estimated Enrollment:	250
Study Start Date:	June 2005

Eligibility

Ages Eligible for Study:	3 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria

Patients completing the planned 12-month core study
Female patients who have reached menarche and who are sexually active must use an effective method of contraception, or must have undergone clinically documented total hysterectomy and/or ovariectomy, or tubal ligation
Written informed consent obtained from the patient and/or legal guardian on the patient's behalf in accordance with the national legislation Exclusion Criteria
Pregnant or breast feeding patients.
Patients being considered by the investigator potentially unreliable and/or not cooperative with regard to the core study protocol, or the planned extension protocol

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00171301

Locations

Lebanon
American University of Beirut
Beirut, Lebanon

Sponsors and Collaborators

Novartis

Investigators

Study Chair:

Novartis

More Information

Study ID Numbers:	CICL670A2402E1
Study First Received:	September 12, 2005
Last Updated:	September 6, 2007
ClinicalTrials.gov Identifier:	NCT00171301
Health Authority:	Lebanon: Institutional Review Board

Keywords provided by Novartis:

Transfusional hemosiderosis
Beta-thalassemia major
Deferasirox

Study placed in the following topic categories:

Metabolic Diseases
Deferasirox
Hematologic Diseases
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

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