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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01254227
Recruitment Status : Completed
First Posted : December 6, 2010
Last Update Posted : November 5, 2014
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Cardiac Iron Overload Drug: Deferasirox and Deferoxamine Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 60 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase II, Open-label, Single-arm, Multicenter Study to Evaluate the Efficacy and Safety of Deferasirox in Combination With Deferoxamine Followed by Deferasirox Monotherapy in Patients With Severe Cardiac Iron Overload Due to Chronic Blood Transfusion (HYPERION)
Study Start Date : January 2011
Actual Primary Completion Date : November 2013
Actual Study Completion Date : November 2013

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Iron

Arm Intervention/treatment
Experimental: Deferasirox
Deferoxamine combination followed by Deferasirox monotherapy
Drug: Deferasirox and Deferoxamine

Primary Outcome Measures :
  1. change in cardiac iron content as measured by T2*.Cardiac T2* is the relaxation time, a cardiac magnetic resonance parameter expressed in milliseconds (ms)inversely correlated to the amount of iron in the heart. [ Time Frame: month 12 ]

Secondary Outcome Measures :
  1. Percentage of patients achieving MRI-measured cardiac T2* ≥ 10 ms (but at least 10% relative increase in cardiac T2* from baseline) [ Time Frame: Months 6, 12, 18 and 24 ]
  2. Change in cardiac iron content reflected by change in MRI-measured cardiac T2* [ Time Frame: Months 6, 18 and 24 ]
  3. change in MRI-measured parameters of the left and right ventricle (left and right heart) [ Time Frame: Months 6, 12, 18 and 24 ]
  4. occurrence and severity of adverse events and of abnormal laboratory and special safety assessments (laboratory safety values, ECG, vital signs, auditory and ophthalmic examinations) [ Time Frame: 24 months ]
  5. time to achieve MRI-measured cardiac T2* ≥ 10 ms [ Time Frame: 24 months ]

Information from the National Library of Medicine

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Ages Eligible for Study:   10 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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)
  • 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

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01254227

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Canada, Ontario
Novartis Investigative Site
Toronto, Ontario, Canada, M5G 2C4
Novartis Investigative Site
Cairo, Egypt
Novartis Investigative Site
Athens, GR, Greece, GR-115 27
Novartis Investigative Site
Patra - RIO, GR, Greece, 265 04
Novartis Investigative Site
Cagliari, CA, Italy, 09121
Novartis Investigative Site
Genova, GE, Italy, 16128
Novartis Investigative Site
Napoli, Italy, 80138
Novartis Investigative Site
Taipei, Taiwan, 10002
Novartis Investigative Site
Bangkok, Thailand, 10330
Novartis Investigative Site
Bangkok, Thailand, 10700
Novartis Investigative Site
Adana, Turkey, 01330
Novartis Investigative Site
Antalya, Turkey, 07070
Novartis Investigative Site
Istanbul, Turkey, 34093
Novartis Investigative Site
Izmir, Turkey, 35040
United Kingdom
Novartis Investigative Site
London, United Kingdom, NW1 2BU
Sponsors and Collaborators
Novartis Pharmaceuticals
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Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: Novartis Pharmaceuticals Identifier: NCT01254227    
Other Study ID Numbers: CICL670A2214
2010-021062-29 ( EudraCT Number )
First Posted: December 6, 2010    Key Record Dates
Last Update Posted: November 5, 2014
Last Verified: November 2014
Keywords provided by Novartis ( Novartis Pharmaceuticals ):
Cardiac iron overload,
Additional relevant MeSH terms:
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Iron Overload
Iron Metabolism Disorders
Metabolic Diseases
Iron Chelating Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action