Safety and Efficacy of Deferasirox in Patients With Transfusion Dependent Iron Overload - a Non-comparative Extension Study

Novartis

Status and phase: Completed; Phase 3; Phase 2

Conditions: Liver Iron Overload

Treatments: Drug: Deferasirox

Study type: Interventional

Funder types: Industry

Identifiers

NCT01033747

CICL670A0105E2

Details and patient eligibility

About

The purpose of this study is to assess the safety and the effects on liver iron of Deferasirox when given for a long treatment period in patients with transfusion dependent iron overload.

Enrollment: 70 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Patients Currently participating in the 9-month comparative prolongation of extension phase of the original study.
• Patients currently participating in the food-effect sub-study, according to amendment 3.
• Ability to provide written informed consent prior to participation in this non-comparative extension study.
• Female patients sexually active must use double-barrier contraception, oral contraceptive plus barrier contraceptive, or must have undergone clinically documented total hysterectomy and/or ovariectomy, or tubal ligation.
• Body weight of at least 35 kg.

Exclusion criteria

• Pregnant or breastfeeding patients.
• History of non-compliance to medical regimens and patients who are considered potentially unreliable.
• Proteinuria > 300 mg/L second void morning urine.
• Patients with serum creatinine above the upper limit normal.

Other protocol-defined inclusion/exclusion criteria may apply.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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70 participants in 2 patient groups

Deferasirox

Experimental group

Description:

Deferasirox group consists of all participants who were initially randomized to 10 and 20 mg/kg/day deferasirox orally daily in the main study and remained on the same treatment during the comparative prolongation study (NCT00379483) and at the beginning of the 5-year non-comparative study

Treatment:

Drug: Deferasirox

Drug: Deferasirox

Deferasirox Crossover

Experimental group

Description:

Deferasirox Crossover group consists of participants who were initially randomized to 40 mg/kg/day deferoxamine (DFO)subcutaneously in the main study and comparative prolongation study and crossed over to 5mg/kg/day to 30 mg/kg/day deferasirox orally daily at the beginning of the 5-year non-comparative extension study

Treatment:

Drug: Deferasirox

Drug: Deferasirox