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

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ClinicalTrials.gov Identifier: NCT00529152
Recruitment Status : Completed
First Posted : September 14, 2007
Results First Posted : August 12, 2009
Last Update Posted : September 2, 2009
Sponsor:
Information provided by:
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.

Condition or disease Intervention/treatment Phase
Iron Overload Drug: Deferiprone Phase 3

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  (Clinical Trial)
Actual Enrollment : 100 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (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
Study Start Date : August 2007
Actual Primary Completion Date : July 2008
Actual Study Completion Date : July 2008

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Iron
U.S. FDA Resources

Arm Intervention/treatment
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



Primary Outcome Measures :
  1. Occurrence of Adverse Events [ Time Frame: 24 Weeks ]

Secondary Outcome Measures :
  1. Change in Serum Ferritin Concentration From Baseline. [ Time Frame: Baseline and 24 weeks ]


Information from the National Library of Medicine

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Ages Eligible for Study:   up to 10 Years   (Child)
Sexes Eligible for Study:   All
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00529152


Locations
Egypt
Abo El Reish Hospital, Cairo University
Cairo, Egypt
Children Hospital, Ain Shams 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

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
First Posted: September 14, 2007    Key Record Dates
Results First Posted: August 12, 2009
Last Update Posted: September 2, 2009
Last Verified: August 2009

Keywords provided by ApoPharma:
Iron Overload

Additional relevant MeSH terms:
Iron Overload
Iron Metabolism Disorders
Metabolic Diseases
Pharmaceutical Solutions
Deferiprone
Iron Chelating Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action