Extension Study of Iron Chelation Therapy With Deferasirox in β-thalassemia and Rare Chronic Anemia Patients

Novartis

Status and phase: Completed; Phase 2

Conditions: Anemia; Hemosiderosis

Treatments: Drug: Deferasirox

Study type: Interventional

Funder types: Industry

Identifiers

NCT00303329

CICL670A0108E1

Details and patient eligibility

About

A 1-year randomized Phase II core trial was conducted to investigate the efficacy of deferasirox in regularly transfused patients with β-thalassemia and other rare chronic anemia 2 years of age and older. Patients who successfully completed the main trial may continue in the extension trial to receive chelation therapy with deferasirox for up to 3 years. Extension was prolonged to 4 years.

The objective of this study is to assess the long-term safety and efficacy of deferasirox in these patient groups.

Enrollment: 184 patients

Sex: All

Ages: 2+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Patients completed the planned 12-month core study
• Female patients who have reached menarche and who are 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
• 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

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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184 participants in 1 patient group

Deferasirox

Experimental group

Description:

Deferasirox daily oral dose between 5-40 mg/kg/day

Treatment:

Drug: Deferasirox