Clinical trial NCT00879242: Effect of Deferasirox on Patients With Cardiac MRI T2* < 20 Msec
Official title: Multicenter, Open Label, Prospective Study to Evaluate the Efficacy and Safety of Deferasirox 30 mg/kg/Day for 52 Weeks, in Transfusion-dependent Beta-thalassemic Patients With Cardiac MRI T2* < 20 Msec
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Beta Thalassemia Transfusion Dependent
Keywords: Thalassemia
cardiac MRI T2*
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2009-04-09
Enrollment: 20
Sex: ALL
Minimum age (years): 10
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This study will assess the safety and efficacy of deferasirox in patients with cardiac MRI T2\* \< 20 msec.


Eligibility criteria: Inclusion Criteria:

* β-thalassemic patients receiving regular transfusions with an history of at least 20 transfusional events
* Patients naïve for deferasirox
* Serum ferritin levels ≥1000ng/ml (average of the last 6 months assays);
* Cardiac MRI T2\* \>5 and \<20 msec.
* LVEF at MRI ≥56%

Exclusion Criteria:

* Patients that have already started deferasirox therapy
* Existing contraindications to the performance of MRI procedure, including presence of metallic prostheses not compatible with the MRI procedure other than those approved as safe for use in MRI scanners (e.g., some types of aneurysm clips, shrapnel in proximity to vital organs such as the retina), pacemaker, intractable claustrophobia and incapability to follow the instruction for the correct performance of the MRI (e.g. to maintain a supine position, to hold breath, etc.)
* Clinical conditions requiring intensive chelating therapy on the basis of Investigator's judgment
* Stable average ALT levels \>300 U/L in the preceding 12 months
* Uncontrolled systemic hypertension
* Estimated creatinine clearance \<60 ml/min
* History of nephrotic syndrome
* History of clinically significant ocular toxicity related to the chelating therapy
* Psychiatric disorders or drug dependence that might impair the capability of the patient to provide their Informed Consent or to be administered the study treatment
* Known sensitivity to study drug(s) or class of study drug(s)
* Patients with severe medical condition(s) that in the view of the investigator prohibits participation in the study
* Use of any other investigational agent in the last 30 days.

Other protocol-defined inclusion/exclusion criteria may apply.

Primary outcomes:
measure: Change from baseline in cardiac T2* value
timeFrame: 52 weeks
Secondary outcomes:
measure: Change from baseline in cardiac functions parameters (LVEF, LVESV and LVEDV)
timeFrame: 52 weeks
measure: Change from baseline liver T2* value
timeFrame: 52 weeks
Other outcomes:
Locations:
city: Cagliari
country: Italy
facility: Novartis Investigative Site
city: Orbassano
country: Italy
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References:
citation: Piga A, Longo F, Origa R, Roggero S, Pinna F, Zappu A, Castiglioni C, Cappellini MD. Deferasirox for cardiac siderosis in beta-thalassaemia major: a multicentre, open label, prospective study. Br J Haematol. 2014 Nov;167(3):423-6. doi: 10.1111/bjh.12987. Epub 2014 Jun 30. No abstract available.