A Study of Long-term Treatment With Deferasirox in Patients With Beta-thalassemia and Transfusional Hemosiderosis

Novartis

Status and phase: Completed; Phase 3

Conditions: Hemosiderosis; Beta-Thalassemia

Treatments: Drug: deferasirox

Study type: Interventional

Funder types: Industry

Identifiers

NCT00171171

CICL670A2402

Details and patient eligibility

About

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 will evaluate the efficacy, safety and tolerability of deferasirox.

Enrollment: 252 patients

Sex: All

Ages: 2+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Beta-thalassemia outpatients with transfusional hemosiderosis and unable to be chelated with deferoxamine (DFO) due to DFO being contra-indicated and/or due to documented unacceptable toxicity of DFO or documented poor response to DFO despite proper compliance, or documented non-compliance to DFO, with serum ferritin ≥ 500 ng/ml and LIC ≥ 2 mg/Fe/g dw liver
• Beta-thalassemia outpatients with transfusional hemosiderosis treated with deferiprone

Exclusion criteria

• Means levels of ALT > 300 U/L
• Serum creatinine above upper limit of normal
• Active hepatitis C or chronic hepatitis B receiving specific treatment

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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252 participants in 1 patient group

Deferasirox

Experimental group

Treatment:

Drug: deferasirox