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

This study has been completed.
Sponsor:
Information provided by:
ApoPharma
ClinicalTrials.gov Identifier:
NCT00529152
First received: September 12, 2007
Last updated: August 25, 2009
Last verified: August 2009
History of Changes
  Purpose
  • 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.

Condition Intervention Phase
Iron Overload
 Drug: Deferiprone
 Phase 3

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

Resource links provided by NLM:

Drug Information available for: Deferiprone
U.S. FDA Resources

Further study details as provided by ApoPharma:

Primary Outcome Measures:
  • Occurrence of Adverse Events [ Time Frame: 24 Weeks ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Change in Serum Ferritin Concentration From Baseline. [ Time Frame: Baseline and 24 weeks ] [ Designated as safety issue: No ]

Enrollment: 100
Study Start Date: August 2007
Study Completion Date: July 2008
Primary Completion Date: July 2008 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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 Name: Ferriprox Oral Solution

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.

  Eligibility

Ages Eligible for Study:   up to 10 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00529152

Locations
Egypt
Children Hospital, Ain Shams University
Cairo, Egypt
Abo El Reish Hospital, Cairo University
Cairo, Egypt
Indonesia
Cipto Mangunkusumo National Hospital
Jakarta, Indonesia, 10010
Malaysia
University of Malaya Medical Center
Kuala Lumpur, Malaysia, 50603
Sponsors and Collaborators
ApoPharma
Investigators
Principal Investigator: Prof. Mohsen S El Alfy, MD Children Hospital, Ain Shams University, Cairo, Egypt
  More Information

No publications provided by ApoPharma

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Responsible Party: Dian Shaw, ApoPharma Inc.
ClinicalTrials.gov Identifier: NCT00529152     History of Changes
Other Study ID Numbers: LA30-0307
Study First Received: September 12, 2007
Results First Received: June 26, 2009
Last Updated: August 25, 2009
Health Authority: Egypt: Ministry of Health and Population

Keywords provided by ApoPharma:
Iron Overload

Additional relevant MeSH terms:
Iron Overload
Iron Metabolism Disorders
Metabolic Diseases
Deferiprone
 Iron Chelating Agents
Chelating Agents
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions

ClinicalTrials.gov processed this record on June 18, 2013