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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

September 12, 2007
August 25, 2009
August 2007
July 2008   (final data collection date for primary outcome measure)
Occurrence of Adverse Events [ Time Frame: 24 Weeks ] [ Designated as safety issue: Yes ]
The patient's tolerance of treatment will be determined, as assessed by the occurrence of adverse events [ Time Frame: 24 Weeks ]
Complete list of historical versions of study NCT00529152 on ClinicalTrials.gov Archive Site
Change in Serum Ferritin Concentration From Baseline. [ Time Frame: Baseline and 24 weeks ] [ Designated as safety issue: No ]
The efficacy endpoint will be the change in serum ferritin concentration from baseline. [ Time Frame: 24 Weeks ]
Not Provided
Not Provided
 
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.

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.

Interventional
Phase 3
Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Iron Overload
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
A
Ferriprox Oral Solution single treatment
Intervention: Drug: Deferiprone
El Alfy M, 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.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
100
July 2008
July 2008   (final data collection date for primary outcome measure)

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.
Both
up to 10 Years
No
Contact information is only displayed when the study is recruiting subjects
Egypt,   Indonesia,   Malaysia
 
NCT00529152
LA30-0307
Yes
Dian Shaw, ApoPharma Inc.
ApoPharma
Not Provided
Principal Investigator: Prof. Mohsen S El Alfy, MD Children Hospital, Ain Shams University, Cairo, Egypt
ApoPharma
August 2009

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP