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

Condition(s) targeted: Beta-Thalassemia Major

Intervention: ICL670; deferasirox (Drug)

Phase: Phase 3

Status: Active, not recruiting

Sponsored by: Novartis

Official(s) and/or principal investigator(s):
Novartis, Study Chair, Affiliation: Novartis

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.

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

Study design: Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study

Primary outcome:

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:

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

Minimum age: 3 Years. Maximum age: N/A. Gender(s): Both.

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

American University of Beirut, Beirut, Lebanon

Starting date: June 2005
Last updated: September 6, 2007