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

Phase 2

Completed

• Conditions: Beta Thalassemia Transfusion Dependent
• Interventions: Drug: Deferasirox

• Registration Number: NCT00879242
• Lead Sponsor: Novartis Pharmaceuticals

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Study Start: 2009-02
• Study First Submitted: 2009-04-08
• Study First Submitted QC: 2009-04-08
• Study First Posted: 2009-04-09
• Primary Completion: 2011-05
• Status Verified: 2011-11
• Last Update Submitted: 2016-12-08
• Last Update Posted: 2016-12-12

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 20

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%

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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.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

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

Primary Outcome Measures

Name	Time	Method
Change from baseline in cardiac T2* value	52 weeks	Unsatisfactory response is a monthly MRI T2\* improvement lower than 3% versus baseline.

Secondary Outcome Measures

Name	Time	Method
Change from baseline in cardiac functions parameters (LVEF, LVESV and LVEDV)	52 weeks
Change from baseline liver T2* value	52 weeks

Trial Locations

Locations (1)

Novartis Investigative Site; 🇮🇹 Orbassano, Italy