Clinical trial NCT00529152: 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
Overall status: COMPLETED
Phase: PHASE3
Conditions: Iron Overload
Keywords: Iron Overload
Lead sponsor: ApoPharma
Sponsor class: INDUSTRY
First posted: 2007-09-14
Enrollment: 100
Sex: ALL
Maximum age (years): 10
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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.

Primary outcomes:
measure: Occurrence of Adverse Events
timeFrame: 24 Weeks
Secondary outcomes:
measure: Change in Serum Ferritin Concentration From Baseline.
timeFrame: Baseline and 24 weeks
Other outcomes:
Locations:
city: Cairo
country: Egypt
facility: Abo El Reish Hospital, Cairo University
city: Cairo
country: Egypt
facility: Children Hospital, Ain Shams University
city: Jakarta
country: Indonesia
facility: Cipto Mangunkusumo National Hospital
city: Kuala Lumpur
country: Malaysia
facility: University of Malaya Medical Center
Study officials:
affiliation: Children Hospital, Ain Shams University, Cairo, Egypt
name: Prof. Mohsen S El Alfy, MD
role: PRINCIPAL_INVESTIGATOR
References:
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.