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

Novartis

Status and phase

Completed

Phase 2

Conditions

Cardiac Iron Overload

Treatments

Drug: Deferasirox and Deferoxamine

Study type

Interventional

Funder types

Industry

Identifiers

NCT01254227

CICL670A2214

2010-021062-29 (EudraCT Number)

Details and patient eligibility

About

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.

Enrollment

60 patients

Sex

All

Ages

10+ years old

Volunteers

No Healthy Volunteers

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