Phase 3
N=100
Safety and Efficacy of Ferriprox™ (Deferiprone) Oral Solution in Iron Overloaded Pediatric Patients
Iron Overload
Bottom Line
View on ClinicalTrials.gov: NCT00529152 ↗Enrolled (actual)
100
Serious AEs
—
Results posted
Aug 2009
Primary outcome: Primary: Occurrence of Adverse Events — 212 Adverse Events
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Deferiprone (Drug)
- Age
- Pediatric
- Sex
- All
- Sponsor
- ApoPharma
- Primary completion
- Jul 2008
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Occurrence of Adverse Events |
212 | — |
| SECONDARY Change in Serum Ferritin Concentration From Baseline. |
-355.5 | 0.0005 sig |
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.
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) 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.
Data sourced from ClinicalTrials.gov (NCT00529152). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.