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

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT01254227
First received: September 21, 2010
Last updated: July 12, 2012
Last verified: July 2012
History of Changes
  Purpose

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 Intervention Phase
Cardiac Iron Overload
 Drug: Deferasirox and Deferoxamine
 Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: 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)

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Novartis:

Primary Outcome Measures:
  • 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 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • 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 ] [ Designated as safety issue: No ]
  • Change in cardiac iron content reflected by change in MRI-measured cardiac T2* [ Time Frame: Months 6, 18 and 24 ] [ Designated as safety issue: No ]
  • change in MRI-measured parameters of the left and right ventricle (left and right heart) [ Time Frame: Months 6, 12, 18 and 24 ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: Yes ]
  • time to achieve MRI-measured cardiac T2* ≥ 10 ms [ Time Frame: 24 months ] [ Designated as safety issue: No ]

Estimated Enrollment: 45
Study Start Date: January 2011
Estimated Primary Completion Date: March 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Deferasirox
Deferoxamine combination followed by Deferasirox monotherapy
 Drug: Deferasirox and Deferoxamine

  Eligibility

Ages Eligible for Study:   10 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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

  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01254227

Locations
Canada, Ontario
Novartis Investigative Site
Toronto, Ontario, Canada, M5G 2C4
Egypt
Novartis Investigative Site
Cairo, Egypt
Greece
Novartis Investigative Site
Athens, Greece, GR-115 27
Novartis Investigative Site
Patra - RIO, Greece, GR- 26500
Italy
Novartis Investigative Site
Cagliari, CA, Italy, 09121
Novartis Investigative Site
Genova, GE, Italy, 16128
Novartis Investigative Site
Napoli, Italy, 80138
Taiwan
Novartis Investigative Site
Taipei, Taiwan, 10002
Thailand
Novartis Investigative Site
Bangkok, Thailand, 10700
Novartis Investigative Site
Bangkok, Thailand, 10330
Turkey
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 2PG
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

No publications provided

Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT01254227     History of Changes
Other Study ID Numbers: CICL670A2214, 2010-021062-29
Study First Received: September 21, 2010
Last Updated: July 12, 2012
Health Authority: United States: Food and Drug Administration
Canada: Health Canada
Turkey: Ministry of Health
Egypt: Ministry of Health and Population
Thailand: Ministry of Public Health
Taiwan: National Bureau of Controlled Drugs
Italy: The Italian Medicines Agency
Greece: National Organization of Medicines
United Kingdom: Medicines and Healthcare Products Regulatory Agency

Keywords provided by Novartis:
Cardiac iron overload,
deferasirox,
deferoxamine

Additional relevant MeSH terms:
Iron Overload
Iron Metabolism Disorders
Metabolic Diseases
Deferoxamine
Deferasirox
 Siderophores
Iron Chelating Agents
Chelating Agents
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions

ClinicalTrials.gov processed this record on May 23, 2013