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

A 24-Week, Open Label, Uncontrolled Study of the Safety and Efficacy of Ferriprox™ (Deferiprone) Oral Solution in Iron Overloaded Pediatric Patients With Transfusion-Dependent Anemia

• 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.

Study Overview

• Status: Completed
• Conditions: Iron Overload
• Intervention / Treatment: Drug: Deferiprone

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 Type: Interventional
• Enrollment (Actual): 100
• Phase: Phase 3

Contacts and Locations

Study Locations

• Egypt
  • Cairo, Egypt
    • Abo El Reish Hospital, Cairo University
  • Cairo, Egypt
    • Children Hospital, Ain Shams University
• Indonesia
  • Jakarta, Indonesia, 10010
    • Cipto Mangunkusumo National Hospital
• Malaysia
  • Kuala Lumpur, Malaysia, 50603
    • University of Malaya Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 10 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Other: A; Ferriprox Oral Solution single treatment	Drug: 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.; Other Names: Ferriprox Oral Solution

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Occurrence of Adverse Events	Number of Adverse Events over 24 weeks	24 Weeks

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: ApoPharma
• Investigators: Principal Investigator: Prof. Mohsen S El Alfy, MD, Children Hospital, Ain Shams University, Cairo, Egypt

Publications and helpful links

General Publications

• ElAlfy MS, Sari TT, Lee CL, Tricta F, El-Beshlawy A. The safety, tolerability, and efficacy of a liquid formulation of deferiprone in young children with transfusional iron overload. J Pediatr Hematol Oncol. 2010 Nov;32(8):601-5. doi: 10.1097/MPH.0b013e3181ec0f13. Erratum In: J Pediatr Hematol Oncol. 2011 Oct;33(7):572. El Alfy, Moshen [corrected to ElAlfy, Moshen S].

Study record dates

Study Major Dates

• Study Start: August 1, 2007
• Primary Completion (Actual): July 1, 2008
• Study Completion (Actual): July 1, 2008

Study Registration Dates

• First Submitted: September 12, 2007
• First Submitted That Met QC Criteria: September 12, 2007
• First Posted (Estimate): September 14, 2007

Study Record Updates

• Last Update Posted (Estimate): September 2, 2009
• Last Update Submitted That Met QC Criteria: August 25, 2009
• Last Verified: August 1, 2009

More Information

Terms related to this study

• Keywords: Iron Overload
• Additional Relevant MeSH Terms: Metabolic Diseases; Iron Metabolism Disorders; Iron Overload; Molecular Mechanisms of Pharmacological Action; Chelating Agents; Sequestering Agents; Iron Chelating Agents; Deferiprone
• Other Study ID Numbers: LA30-0307