thalassemic patients diagnosed with chronic iron overload due to regular blood transfusion MedDRA version: 9.1 Level: LLT Classification code 10043388 Term: Thalassaemia
Conditions
Interventions
Trade Name: EXJADE
Pharmaceutical Form: Tablet
INN or Proposed INN: DEFERASIROX
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 125-
Trade Name: EXJADE
Pharmaceut
Sponsors
FONDAZIONE ITALIANA "LEONARDO GIAMBRONE"PER LA GUARIGIONE DALLA TALASSEMIA - ONLUS
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: M/F >= 2 years of age Thalassemia Major or Intermedia regularly transfused Patients potentially eligible to the treatment with deferasirox History of at least 20 blood transfusions (equivalent to approximately 100 mL/kg of PRBC) or evidence from clinical monitoring that chronic iron overload is present (e.g. serum ferritin >1,000 µg/l); Serum ferritin could be =65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Non transfusional hemosiderosis Renal impairment and/or with estimated creatinine clearance 500 U/L Pregnant or breast feeding patients Hypersensitivity to the active substance or to any of the excipients Concomitant use of other iron chelating agents Evidence of chelation-related cataracts or hearing loss within 4 weeks prior to baseline Patient unwilling or unable to comply with the protocol
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary goal of this study will be to assess LPI levels before and after deferasirox chelation therapy in thalassemic patients undergoing regular blood transfusion. LPI plasma levels pre- and post-treatment will be monitored in these patients every 2 months for a year, with a check point after 6 months.;Secondary Objective: 1. to evaluate the daily profile of LPI before and after deferasirox therapy; 2. to ascertain the patient?s compliance to the deferasirox therapy by measuring LPI plasma levels; 3. to evaluate the efficacy, safety and acceptance of the deferasirox therapy; 4. to establish whether any clinical endpoints correlate with measurable LPI levels; 5. to evaluate a vascular response with a vasodilatation test with and without induction of ischaemia;Primary end point(s): analyzing values of the IPA obtained with a method semi-quantitative fluorescent highlighting action ossido-riduttiva of the IPA | — |
Countries
Italy
Outcome results
None listed