Iron Overload
Conditions
Keywords
Iron Overload
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.
Interventions
DRUGDeferiprone
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.
Sponsors
ApoPharma
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
No minimum to 10 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Occurrence of Adverse Events | 24 Weeks | Number of Adverse Events over 24 weeks |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in Serum Ferritin Concentration From Baseline. | Baseline and 24 weeks | The change in serum ferritin concentration from baseline to week 24 was measured and analyzed for all participants in the study |
Countries
Egypt, Indonesia, Malaysia
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Ferriprox Oral Solution All subjects were administered Ferriprox Oral Solution three times daily for a total daily dose of either 50, 75 or 100 mg/kg/day. Subjects were initiated at a dose of 50 mg/kg/day, but this dose could be increased after two weeks of therapy depending on the subjects' individual needs. | 100 |
| Total | 100 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Adverse Event | 2 |
| Overall Study | Lost to Follow-up | 1 |
| Overall Study | Withdrawal by Subject | 2 |
Baseline characteristics
| Characteristic | Ferriprox Oral Solution |
|---|---|
| Age Continuous | 5.1 years STANDARD_DEVIATION 2.4 |
| Region of Enrollment Egypt | 76 participants |
| Region of Enrollment Indonesia | 13 participants |
| Region of Enrollment Malaysia | 11 participants |
| serum ferritin | 2531.7 ug/L STANDARD_DEVIATION 1463 |
| Sex: Female, Male Female | 46 Participants |
| Sex: Female, Male Male | 54 Participants |
Outcome results
Primary
Occurrence of Adverse Events
Number of Adverse Events over 24 weeks
Time frame: 24 Weeks
Population: All subjects enrolled (100), had at least one dose of Ferriprox oral solution, all were included in safety analysis
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Ferriprox Oral Solution | Occurrence of Adverse Events | 212 Adverse Events |
Secondary
Change in Serum Ferritin Concentration From Baseline.
The change in serum ferritin concentration from baseline to week 24 was measured and analyzed for all participants in the study
Time frame: Baseline and 24 weeks
Population: 99 subjects had at least one post baseline measurement of serum ferritin concentration and were eligible for the efficacy analyses in the Intent to Treat population (all subjects). The Last Measurement Carried Forward methodology was used to populate any missing serum ferritin value.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Ferriprox Oral Solution | Change in Serum Ferritin Concentration From Baseline. | -355.5 ug/L | Standard Deviation 978.1 |
Comparison: Change in Serum Ferritin from baseline to week 24 was compared using regression analysis; null hypothesis was defined as no change in serum ferritin from baseline to week 24p-value: 0.0005Regression, Linear