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

Primary Purpose

Iron Overload

Status

Completed

Phase

Phase 3

Locations

International

Study Type

Interventional

Intervention

Deferiprone

About this trial

This is an interventional treatment trial for Iron Overload focused on measuring Iron Overload

Eligibility Criteria

undefined - 10 Years (Child)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

Abo El Reish Hospital, Cairo University
Children Hospital, Ain Shams University
Cipto Mangunkusumo National Hospital
University of Malaya Medical Center

Arms of the Study

Arm 1

Arm Type

Other

Arm Label

Arm Description

Ferriprox Oral Solution single treatment

Outcomes

Primary Outcome Measures

Occurrence of Adverse Events

Number of Adverse Events over 24 weeks

Secondary Outcome Measures

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

Full Information

NCT ID

NCT00529152

First Posted

September 12, 2007

Last Updated

August 25, 2009

Sponsor

ApoPharma

1. Study Identification

Unique Protocol Identification Number

NCT00529152

Brief Title

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

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

Study Type

Interventional

2. Study Status

Record Verification Date

August 2009

Overall Recruitment Status

Completed

Study Start Date

August 2007 (undefined)

Primary Completion Date

July 2008 (Actual)

Study Completion Date

July 2008 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor

ApoPharma

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Iron Overload

Keywords

Iron Overload

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

Non-Randomized

Enrollment

100 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Arm Type

Other

Arm Description

Ferriprox Oral Solution single treatment

Intervention Type

Drug

Intervention Name(s)

Deferiprone

Other Intervention Name(s)

Ferriprox Oral Solution

Intervention Description

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.

Primary Outcome Measure Information:

Title

Occurrence of Adverse Events

Description

Number of Adverse Events over 24 weeks

Time Frame

24 Weeks

Secondary Outcome Measure Information:

Title

Change in Serum Ferritin Concentration From Baseline.

Description

The change in serum ferritin concentration from baseline to week 24 was measured and analyzed for all participants in the study

Time Frame

Baseline and 24 weeks

10. Eligibility

Sex

All

Maximum Age & Unit of Time

10 Years

Accepts Healthy Volunteers

Eligibility Criteria

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.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Prof. Mohsen S El Alfy, MD

Organizational Affiliation

Children Hospital, Ain Shams University, Cairo, Egypt

Official's Role

Principal Investigator

Facility Information:

Facility Name

Abo El Reish Hospital, Cairo University

City

Cairo

Country

Egypt

Facility Name

Children Hospital, Ain Shams University

City

Cairo

Country

Egypt

Facility Name

Cipto Mangunkusumo National Hospital

City

Jakarta

ZIP/Postal Code

10010

Country

Indonesia

Facility Name

University of Malaya Medical Center

City

Kuala Lumpur

ZIP/Postal Code

50603

Country

Malaysia

12. IPD Sharing Statement

Citations:

PubMed Identifier

20921906

Citation

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].

Results Reference

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

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