Extension Study on Efficacy and Safety of Deferasirox Treatment in Patients With Beta-thalassemia and Transfusional Hemosiderosis - Full Text View

Sponsor:	Novartis Pharmaceuticals
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

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

Resource links provided by NLM:

Genetics Home Reference related topics: beta thalassemia

MedlinePlus related topics: Thalassemia

Drug Information available for: Deferasirox

U.S. FDA Resources

Further study details as provided by Novartis:

Primary Outcome Measures:

Liver Iron Concentration measured by liver MRI (or if not practicable liver biopsy) [ Time Frame: at 1 and 2 years ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:

Ongoing monitoring and recording of all adverse events and serious adverse events [ Time Frame: ongoing ] [ Designated as safety issue: Yes ]
Hematology, blood chemistry and urine renal function values at monthly intervals [ Time Frame: monthly ] [ Designated as safety issue: No ]
surrogate markers, such as serum ferritin, serum iron, serum transferrin (TRF) and transferrin saturation at monthly intervals [ Time Frame: monthly ] [ Designated as safety issue: No ]
Measurement of vital signs and performance of physical examinations at 12-weekly intervals [ Time Frame: Every 12 weeks ] [ Designated as safety issue: No ]
Total body iron excretion rate (estimated based on the iron influx as determined by the amount of red cells transfused and the change in total body iron stores) at 1-year and 2-years [ Time Frame: At 1 year & at 2 years ] [ Designated as safety issue: No ]

Enrollment:	247
Study Start Date:	June 2005
Primary Completion Date:	May 2008 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
ICL670: Experimental	Drug: ICL670; deferasirox

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 Pharmaceuticals

Investigators

Study Director:

Novartis Pharmaceuticals

More Information

No publications provided

Responsible Party:	Novartis Pharmaceuticals ( External Affairs )
Study ID Numbers:	CICL670A2402E1
Study First Received:	September 12, 2005
Last Updated:	November 18, 2009
ClinicalTrials.gov Identifier:	NCT00171301 History of Changes
Health Authority:	Lebanon: Institutional Review Board

Keywords provided by Novartis:

Transfusional hemosiderosis
Beta-thalassemia major
Deferasirox

Additional relevant MeSH terms:

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

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

ClinicalTrials.gov processed this record on November 27, 2009