Effect of Deferasirox on Patients With Cardiac MRI T2* < 20 Msec

Novartis

Status and phase: Completed; Phase 2

Conditions: Beta Thalassemia Transfusion Dependent

Treatments: Drug: Deferasirox

Study type: Interventional

Funder types: Industry

Identifiers

NCT00879242

CICL670AIT07

EUDRACT Code :2008-003230-22

Details and patient eligibility

About

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

Enrollment: 20 patients

Sex: All

Ages: 10+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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20 participants in 1 patient group

Deferasirox

Experimental group

Description:

30 mg/kg/day. The daily dose can be increased to 40 mg/kg in case of unsatisfactory response after 12 weeks of treatment.

Treatment:

Drug: Deferasirox