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

Iron Overload Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00529152
• Other study ID #: LA30-0307
• Status: Completed
• Phase: Phase 3
• First received: September 12, 2007
• Last updated: August 25, 2009
• Start date: August 2007
• Est. completion date: July 2008

Study information

• Verified date: August 2009
• Source: ApoPharma
• Contact: n/a
• Is FDA regulated: No
• Health authority: Egypt: Ministry of Health and Population
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 100
• Est. completion date: July 2008
• Est. primary completion date: July 2008
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A to 10 Years

Eligibility

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

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Iron Overload

Intervention

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.

Locations

Country	Name	City	State
Egypt	Abo El Reish Hospital, Cairo University	Cairo
Egypt	Children Hospital, Ain Shams University	Cairo
Indonesia	Cipto Mangunkusumo National Hospital	Jakarta
Malaysia	University of Malaya Medical Center	Kuala Lumpur

Sponsors (1)

Lead Sponsor	Collaborator
ApoPharma

Countries where clinical trial is conducted

Egypt,  Indonesia,  Malaysia, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Occurrence of Adverse Events		24 Weeks	Yes
Secondary	Change in Serum Ferritin Concentration From Baseline.		Baseline and 24 weeks	No