Eligibility Module
The Eligibility Module contains detailed information about who can participate in the clinical trial. This includes eligibility criteria, age restrictions, gender requirements, healthy volunteer status, and study population descriptions, helping researchers understand who is eligible to participate in the study.
Eligibility Module path is as follows:
Study -> Protocol Section -> Eligibility Module
Eligibility Module
Ignite Creation Date:
2025-12-26 @ 11:12 PM
Ignite Modification Date:
2025-12-26 @ 11:12 PM
NCT ID:
NCT01825512
Eligibility Criteria:
Inclusion Criteria:
* 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 Criteria:
* 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
Healthy Volunteers:
False
Sex:
ALL
Minimum Age:
1 Month
Maximum Age:
17 Years
Study:
NCT01825512
Study Brief:
Protocol Section:
NCT01825512