Safety and Efficacy of Ferriprox™ (Deferiprone) Oral Solution in Iron Overloaded Pediatric Patients

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

100 participants

Study Classification

INTERVENTIONAL

Study Start Date

2007-08-31

Study Completion Date

2008-07-31

Brief Summary

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* The primary objective is to assess the safety of Ferriprox oral solution for the treatment of iron overload in pediatric patients with transfusion-dependent anemia.
* The secondary objective is to assess the efficacy of Ferriprox oral solution in reducing iron overload in pediatric patients with transfusion-dependent anemia.

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Detailed Description

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This will be a multi-centre, open label, single treatment, uncontrolled study. A total of 100 iron-overloaded pediatric patients with transfusion-dependent anemia will be enrolled in the study.Eligible patients will receive Ferriprox (deferiprone) oral solution, 100 mg/mL, at a total daily dose of 75 mg/kg body weight or 100 mg/kg body weight, divided in three (3) doses, for 24 weeks.

Conditions

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Iron Overload

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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A

Ferriprox Oral Solution single treatment

Group Type OTHER

Deferiprone

Intervention Type DRUG

Ferriprox (deferiprone) oral solution will be given orally at a total daily dose of 75 mg/kg body weight or 100 mg/kg body weight, divided into 3 doses, for 24 weeks.

Interventions

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Deferiprone

Ferriprox (deferiprone) oral solution will be given orally at a total daily dose of 75 mg/kg body weight or 100 mg/kg body weight, divided into 3 doses, for 24 weeks.

Intervention Type DRUG

Other Intervention Names

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Ferriprox Oral Solution

Eligibility Criteria

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Inclusion Criteria

* Patients who are ≤ 10 years of age.
* Patients who have a confirmed diagnosis of transfusion-dependent anemia, other than Blackfan-Diamond anemia, and have chronic iron overload requiring chelation therapy.
* Patients who are in a chronic transfusion program, and who have received at least eight (8) red blood cell transfusions per year for a minimum of one year.
* Patients who are iron overloaded as assessed by serum ferritin concentration greater than 1000 µg/L.

Exclusion Criteria

* Patients who have a diagnosis of Blackfan-Diamond anemia.
* Patients who have experienced neutropenia/agranulocytosis (absolute neutrophil count (ANC) \< 1.5 x 109/L) or thrombocytopenia (platelet count \< 50.0 x 109/L).
* Patients who have had previous treatment with Ferriprox and presented serious adverse reaction or intolerance requiring withdrawal of Ferriprox.
* Patients with evidence of abnormal liver function (ALT level \> 3 times the upper limit of normal; entry may be delayed until values return to normal).
* Patients with evidence of renal failure, characterized by serum creatinine level \> 2 times the upper limit of normal; entry may be delayed until values return to normal.

Maximum Eligible Age

10 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No