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

• ClinicalTrials.gov Identifier: NCT00879242
• Recruitment Status: Completed
• First Posted: April 9, 2009
• Last Update Posted: December 12, 2016
• Sponsor: Novartis Pharmaceuticals
• Information provided by (Responsible Party): Novartis ( Novartis Pharmaceuticals )

Tracking Information

• First Submitted Date: April 8, 2009
• First Posted Date: April 9, 2009
• Last Update Posted Date: December 12, 2016
• Study Start Date: February 2009
• Actual Primary Completion Date: May 2011 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: November 14, 2011)

Change from baseline in cardiac T2* value [ Time Frame: 52 weeks ]

Unsatisfactory response is a monthly MRI T2* improvement lower than 3% versus baseline.

• Original Primary Outcome Measures (submitted: April 8, 2009): Change from baseline in cardiac T2* value [ Time Frame: 52 weeks ]

Current Secondary Outcome Measures (submitted: April 8, 2009)

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

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: 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
• Brief Summary: This study will assess the safety and efficacy of deferasirox in patients with cardiac MRI T2* < 20 msec.
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 2
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Beta Thalassemia Transfusion Dependent

Intervention

Drug: Deferasirox

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

Study Arms

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.

Intervention: Drug: Deferasirox

• Publications *: 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.

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: November 14, 2011): 20
• Original Estimated Enrollment (submitted: April 8, 2009): 50
• Study Completion Date: Not Provided
• Actual Primary Completion Date: May 2011 (Final data collection date for primary outcome measure)

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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 10 Years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Italy

Administrative Information

• NCT Number: NCT00879242
• Other Study ID Numbers: CICL670AIT07; EUDRACT Code :2008-003230-22
• Has Data Monitoring Committee: Not Provided
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Current Responsible Party: Novartis ( Novartis Pharmaceuticals )
• Original Responsible Party: External Affairs, Novartis Pharmaceuticals
• Current Study Sponsor: Novartis Pharmaceuticals
• Original Study Sponsor: Novartis
• Collaborators: Not Provided

Investigators

• Study Director: Novartis Pharmaceuticals; Novartis Pharmaceuticals

• PRS Account: Novartis
• Verification Date: November 2011