Efficacy and Safety Study of Deferasirox in Patients With Non-transfusion Dependent Thalassemia

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE4

Total Enrollment

134 participants

Study Classification

INTERVENTIONAL

Study Start Date

2012-12-06

Study Completion Date

2019-01-17

Brief Summary

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Assessed the efficacy of deferasirox in patients with non-transfusion dependent thalassemia based on change in liver iron concentration from baseline after 52 weeks of treatment. Provided further assessment of the long-term efficacy and safety of deferasirox in NTDT patients with iron overload (LIC ≥ 5 mg Fe/g liver dw and SF ≥ 300 ng/mL) for up to 260 weeks.

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Detailed Description

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Conditions

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Non-transfusion Dependent Thalassemia

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Deferasirox

All patients were treated with 10mg/kg/day deferasirox with dose adjustments after 4 weeks of treatment according to baseline Liver Iron Concentration (LIC).

Group Type OTHER

deferasirox

Intervention Type DRUG

Deferasirox dispersible tablets at strengths of 125 mg, 250 mg, and 500 mg were administered by oral daily dosing.

Interventions

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deferasirox

Deferasirox dispersible tablets at strengths of 125 mg, 250 mg, and 500 mg were administered by oral daily dosing.

Intervention Type DRUG

Other Intervention Names

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ICL670

Eligibility Criteria

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Inclusion Criteria

Non-transfusion dependent congenital or chronic anemia inclusive of beta-thalassemia intermedia, HbE beta-thalassemia or alpha-thalassemia intermedia (HbH disease)/ Liver iron concentration \>/= 5 mg Fe/g dw Serum Ferritin \>/= 300 ng/mL

Exclusion Criteria

HbS-beta Thalassemia, anticipated regular transfusion program during the study, blood transfusion 6 months prior to study start, significant proteinuria, creatinine clearance \</= 40 ml/min, serum creatinine \> ULN, ALT \>5 x ULN, active hepatitis B or C, cirrhosis

Pediatrics Only:

A patient's weight of at least 20 kg is required to allow dosing of 5 mg/kg with one tablet of 125 mg

Minimum Eligible Age

10 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No