PI-Labile Plasma Iron in Deferasirox-Treated Thalassemic Patients - ND

• Conditions: thalassemic patients diagnosed with chronic iron overload due to regular blood transfusion; MedDRA version: 9.1Level: LLTClassification code 10043388Term: Thalassaemia

• Registration Number: EUCTR2007-004164-51-IT
• Lead Sponsor: FONDAZIONE ITALIANA LEONARDO GIAMBRONEPER LA GUARIGIONE DALLA TALASSEMIA - ONLUS

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2009-03-23
• Last Update Posted: 19 March 2012

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: Not specified

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 <1,000 µg/l in patient effectively managed with other chelating therapies but for whom the physicians consider a change appropriate Written informed consent by the patient or by their parents or legal guardians on the patient?s behalf, and child?s or teenager?s assent (if applicable), in accordance with the national legislation. Female patients who have reached menarche and who are sexually active must use an effective method of contraception, or must have undergone clinically documented total hysterectomy and/or ovariectomy, or tubal ligation.
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Non transfusional hemosiderosis Renal impairment and/or with estimated creatinine clearance <60 ml/min at baseline Clinically significant proteinuria Severe liver impairment and/or ALT/AST >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

Study & Design

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

Primary Outcome Measures

Name	Time	Method
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