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

NCT ID: NCT00879242

Last Updated: 2016-12-12

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 20 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2009-02-28

Brief Summary

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

Conditions

Beta Thalassemia Transfusion Dependent

Keywords

Thalassemia; cardiac MRI T2*

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

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.

Group Type: EXPERIMENTAL

Deferasirox

Intervention Type: DRUG

Dispersible tables of 125, 250 and 500 mg at the dose of 30 mg/kg/day, oral administration.

Interventions

Deferasirox

Dispersible tables of 125, 250 and 500 mg at the dose of 30 mg/kg/day, oral administration.

Intervention Type: DRUG

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.

• Minimum Eligible Age: 10 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Novartis Pharmaceuticals

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Novartis Pharmaceuticals

Role: STUDY_DIRECTOR

Novartis Pharmaceuticals

Locations

Novartis Investigative Site

Cagliari, , Italy

Novartis Investigative Site

Orbassano, , Italy

Countries

Italy

References

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.

Reference Type: RESULT

PMID: 24976388 (View on PubMed)

Other Identifiers

EUDRACT Code :2008-003230-22

Identifier Type: -

Identifier Source: secondary_id

CICL670AIT07

Identifier Type: -

Identifier Source: org_study_id