A 4-year Extension Study to Core 1-year Study of Iron Chelation Therapy With Deferasirox in β-thalassemia Major Pediatric Patients With Transfusional Iron Overload.

Novartis

Status and phase: Completed; Phase 2

Conditions: Pediatric Rare Anemia; β-thalassemia Major; Transfusional Iron Overload

Treatments: Drug: Deferasirox

Study type: Interventional

Funder types: Industry

Identifiers

NCT00390858

CICL670A0106E1

Details and patient eligibility

About

In this 4-year extension study the safety, efficacy and and pharmacokinetics of deferasirox in regularly transfused pediatric patients with β-thalassemia major was assessed. Patients who successfully completed the main 1 year trial (NCT00390858) were eligible to continue in this extension trial and receive chelation therapy with deferasirox for up to 4 years.

Enrollment: 40 patients

Sex: All

Ages: 1 to 18 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Completion of the planned 12-month core trial, (NCT00390858).
• Female patients who have reached menarche and who were sexually active were to 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 from the parent or legal guardian in accordance with the national legislation.

Exclusion criteria

• Pregnant or breast feeding patients
• Patients with a history of non-compliance to medical regimens and patients who are considered by the investigator as potentially unreliable.

Other protocol-defined exclusion criteria may apply.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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40 participants in 1 patient group

Deferasirox

Experimental group

Description:

Initial dose of 10 mg/kg, dose modifications of ± 5 or 10 mg/kg were based on participant response.

Treatment:

Drug: Deferasirox