Status:
COMPLETED
Efficacy/Safety Study of Deferiprone Compared to Deferasirox in Paediatric Patients
Lead Sponsor:
Consorzio per Valutazioni Biologiche e Farmacologiche
Collaborating Sponsors:
European Commission
Conditions:
Chronic Iron Overload
Eligibility:
All Genders
1-17 years
Phase:
PHASE3
Brief Summary
Multicentre, randomised, open label, non-inferiority active-controlled trial to evaluate efficacy and safety of a 12-months treatment with deferiprone (DFP) at dose of 75-100 mg/kg/day versus deferasi...
Detailed Description
Haemoglobinopathies are a group of inherited disorders characterized by structural variations of the haemoglobin molecule. Most of the patients affected require for survival chronic red blood cells tr...
Eligibility Criteria
Inclusion
- Patients of both genders aged from 1 month up to less than 18 years at the time of enrolment
- Patients affected by any hereditary haemoglobinopathy requiring chronic transfusion therapy and chelation, including but not limited to thalassemia syndromes and sickle cell disease
- Patients on current treatment with deferoxamine (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);
- 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;
- Until availability of results from the PK Study (Study DEEP-1, EudraCT n. 2012-000658-67) for patients aged from 1 month to less than 6 years: known intolerance or contraindication to DFO;
- Written informed consent and patient's informed assent, relating to his/her comprehension abilities and level of maturity
Exclusion
- Patients with intolerance or known contraindication to either DFP or DFX
- Patients receiving DFX at a dose \> 40 mg/kg/day or DFP at a dose \> 100 mg/kg/day at screening
- Platelet count \<100.000/mm3 during the run-in phase
- Absolute neutrophils count \<1.500/mm3 during the run-in phase
- Hb levels lower than 8g/dL during the run-in phase
- Evidence of abnormal liver function
- Iron overload from causes other than transfusional haemosiderosis
- Severe heart dysfunction secondary to iron overload
- Serum creatinine level \> ULN (Upper Limit of Normal) for age during the run-in phase
- History of significant medical or psychiatric disorder
- The patient has received another investigational drug within 30 days prior to this clinical trial
- Fever and other signs/symptoms of infection in the 10 days before baseline assessment
- Concomitant use of trivalent cation-dependent medicinal products such as aluminium-based antacids
- Positive test for β-HCG (Human chorionic gonadotropin) and lactating female patients
Key Trial Info
Start Date :
March 17 2014
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
September 21 2017
Estimated Enrollment :
435 Patients enrolled
Trial Details
Trial ID
NCT01825512
Start Date
March 17 2014
End Date
September 21 2017
Last Update
May 4 2021
Active Locations (22)
Enter a location and click search to find clinical trials sorted by distance.
1
Hospital 'Ihsan Çabej'
Lushnjë, Albania
2
Qendra Spitalore Universitare "Nene Tereza" Tirane
Tirana, Albania
3
Department of Medical and Public health Services of the Ministry of Health
Nicosia, Cyprus
4
Alexandria University Hospital - Faculty of Medicine
Alexandria, Egypt