Efficacy and Safety of Deferasirox in Combination With Deferoxamine Followed by Deferasirox Monotherapy in Severe Cardiac Iron Overload

Novartis Pharmaceuticals60 enrolled

Overview

This study will evaluate the efficacy and safety of deferasirox in combination with deferoxamine followed be deferasirox monotherapy in patients with severe iron overload due to chronic blood transfusions.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Cardiac Iron Overload

Interventions

Deferasirox and DeferoxamineDRUG

Eligibility

Sex: ALLMin age: 10 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Patients with β-thalassemia major or Diamond-Blackfan anemia (DBA) or congenital sideroblastic anemia on chronic transfusion therapy * Myocardial T2\* value that is ≥ 5 and \< 10 ms * Left ventricular ejection fraction (LVEF) ≥ 56% as determined by Magnetic resonance imaging (MRI) * Liver Iron Concentration (LIC) ≥ 7 mg Fe /g dw as determined by R2 MRI. * Lifetime history of at least 50 units of red blood cell transfusions, and must be receiving at least ≥ 8 units/yr of red blood cell transfusions * Serum ferritin ≥ 1000 ng/mL Exclusion Criteria: * Patients with clinical symptoms of cardiac dysfunction (shortness of breath at rest or exertion, orthopnea, exercise intolerance, lower extremity edema, arrhythmias) * Patients unable to undergo study assessments including MRI * Patients with serum creatinine greater than Upper limit of normal ULN)range or with significant proteinuria as indicated by a urinary protein/creatinine ratio (UPCR) ≥1.0 mg/mg in a non-first void urine sample at baseline. Other protocol-defined inclusion/exclusion criteria may apply

Locations (15)

Novartis Investigative Site

Toronto, Ontario, Canada

Novartis Investigative Site

Cairo, Egypt

Novartis Investigative Site

Athens, GR, Greece

Outcomes

Primary Outcomes

Change in Cardiac Iron Content From Baseline to Month 12

Cardiac T2\* is the most sensitive and reproducible test in detecting myocardial iron load. A cardiac T2\* value of \<10 ms is defined as severe cardiac iron overload. Participants who do not have baseline T2\* or do not have any post-baseline T2\* are excluded from the analysis.

Time frame: From Baseline to Month 12

Secondary Outcomes

Percentage of Participants With T2*>=10 ms and at Least 10% Relative Increase From Baseline at Month 6, 12, 18 and 24

The number of evaluable participants at each visit were used as the denominator for the calculation of proportion at each visit.

Time frame: From the Months 6, 12, 18 and 24

Change in Cardiac Iron Content From Baseline to Month 6,18 and 24

The change in cardiac iron content was calculated as ratio of Cardiac T2\* at different time points; the efficacy endpoint analyses were performed on the Full Analysis Set (FAS).

Time frame: From Baseline to Months 6, 18 and 24

Change in Left Ventricular Ejection Fraction (LVEF) From Baseline to Month 6, 12, 18 and 24

Magnetic resonance imaging (MRI)-measured cardiac T2\* and cardiac function reflected by left and right ventricle ejection fraction. A standardized MRI protocol for T2\* acquisition technique will be used in the centers. Images will be reviewed centrally by an expert MRI reader.

Time frame: From the Months 6, 12, 18 and 24

Change in Right Ventricular Ejection Fraction (RVEF) From Baseline to Month 6, 12, 18 and 24

Data from ClinicalTrials.gov

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