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

Phase 3

Completed

• Conditions: Iron Overload
• Interventions: Drug: Deferiprone

• Registration Number: NCT00529152
• Lead Sponsor: ApoPharma

Brief Summary

* 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.

Detailed Description

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.

Study Timeline

• Study Start: 2007-08
• Study First Submitted: 2007-09-12
• Study First Submitted QC: 2007-09-12
• Study First Posted: 2007-09-14
• Primary Completion: 2008-07
• Study Completion: 2008-07
• Results First Submitted: 2009-06-26
• Status Verified: 2009-08
• Results First Submitted QC: 2009-08-11
• Results First Posted: 2009-08-12
• Last Update Submitted: 2009-08-25
• Last Update Posted: 2009-09-02

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 100

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
A	Deferiprone	Ferriprox Oral Solution single treatment

Primary Outcome Measures

Name	Time	Method
Occurrence of Adverse Events	24 Weeks	Number of Adverse Events over 24 weeks

Secondary Outcome Measures

Name	Time	Method
Change in Serum Ferritin Concentration From Baseline.	Baseline and 24 weeks	The change in serum ferritin concentration from baseline to week 24 was measured and analyzed for all participants in the study

Trial Locations

Locations (4)

Children Hospital, Ain Shams University; 🇪🇬 Cairo, Egypt

Cipto Mangunkusumo National Hospital; 🇮🇩 Jakarta, Indonesia

University of Malaya Medical Center; 🇲🇾 Kuala Lumpur, Malaysia

Abo El Reish Hospital, Cairo University; 🇪🇬 Cairo, Egypt