Efficacy and safety study to compare deferiprone versus deferasirox in paediatric patients

Phase 1

• Conditions: chronic iron overload; MedDRA version: 20.0Level: LLTClassification code 10065974Term: Chronic iron overloadSystem Organ Class: 100000019570; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2012-000353-31-GB
• Lead Sponsor: Consorzio per Valutazioni Biologiche e Farmacologiche

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2014-08-26
• Last Update Posted: 10 September 2018

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 388

Inclusion Criteria

1. Patients of both genders aged from 1 month up to less than 18 years at the time of enrolment;
2. Patients affected by any hereditary haemoglobinopathy requiring chronic transfusion therapy and chelation, including but not limited to thalassemia syndromes and sickle cell disease;
3. Patients on current treatment with DFO or DFX or DFP in a chronic transfusion program receiving at least 150 mL/kg/year of packed red blood cells (corresponding approximately to 12 transfusions);
4. For patients naïve to chelation treatment: patients that have received at least 150 mL/kg of packed red blood cells (corresponding to approximately 12 transfusions) in a chronic transfusion program and with serum ferritin levels 800 ng/mL;
5. Until availability of results from the PK study (study DEEP-1, EudraCT no. 2012-000658-67) for patients aged from 1 month to less than 6 years: known intolerance or contraindication to deferoxamine;
6. Written informed consent and patient's informed assent according to patient?'s maturity and understanding.
Are the trial subjects under 18? yes
Number of subjects for this age range: 388
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range 0
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 0

Exclusion Criteria

1. Patients with known intolerance or contraindication to either DFP or DFX;
2. Patients receiving DFX at a dose > 40 mg/kg/day or DFP at a dose > 100 mg/kg/day at screening;
3. Platelet count <100.000/mm3 during the run-in phase;
4. Absolute neutrophils count <1.500/mm3 during the run-in phase;
5. Hb levels lower than 8g/dL during the run-in phase;
6. Evidence of abnormal liver function;
7. Iron overload from causes other than trasfusional haemosiderosis;
8. Severe heart dysfunction secondary to iron overload;
9. Serum creatinine level > ULN for age during the run-in phase;
10. History of significant medical or psychiatric disorder;
11. The patient has received another investigational drug within 30 days prior to this study;
12. Fever and other signs/symptoms of infection in the 10 days before baseline assessment;
13. Concomitant use of trivalent cation-dependent medicinal products such as aluminium-based antacids;
14. Positive test for beta-HCG or lactating female patients.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified