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

Sponsor

Novartis Pharmaceuticals (Industry)

Overall Status

Completed

CT.gov ID

NCT00879242

Collaborator

(none)

Enrollment

Locations

Arm

Patients Per Site

Study Details

Study Description

Brief Summary

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

Condition or Disease	Intervention/Treatment	Phase
Beta Thalassemia Transfusion Dependent	Drug: Deferasirox	Phase 2

Study Design

Study Type:

Interventional

Actual Enrollment :

20 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

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 Start Date :

Feb 1, 2009

Actual Primary Completion Date :

May 1, 2011

Arms and Interventions

Arm	Intervention/Treatment
Experimental: 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.	Drug: Deferasirox Dispersible tables of 125, 250 and 500 mg at the dose of 30 mg/kg/day, oral administration.

Arm

Intervention/Treatment

Experimental: 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.

Drug: Deferasirox

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

Outcome Measures

Primary Outcome Measures

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

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

Eligibility Criteria

Criteria

Ages Eligible for Study:

10 Years and Older

Sexes Eligible for Study:

All

Accepts 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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Novartis Investigative Site	Cagliari		Italy
2	Novartis Investigative Site	Orbassano		Italy

Sponsors and Collaborators

Novartis Pharmaceuticals

Investigators

Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Novartis Pharmaceuticals

ClinicalTrials.gov Identifier:

NCT00879242

Other Study ID Numbers:

CICL670AIT07
EUDRACT Code :2008-003230-22

First Posted:

Apr 9, 2009

Last Update Posted:

Dec 12, 2016

Last Verified:

Nov 1, 2011

Keywords provided by Novartis Pharmaceuticals

Thalassemia

cardiac MRI T2*

Additional relevant MeSH terms:

Thalassemia

beta-Thalassemia

Deferasirox

Study Results

No Results Posted as of Dec 12, 2016