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Combination Deferasirox and Deferiprone for Severe Iron Overload in Thalassemia

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: NCT01709032
Recruitment Status : Completed
First Posted : October 17, 2012
Results First Posted : March 6, 2019
Last Update Posted : March 6, 2019
The Cooley's Anemia Foundation,
Ann & Robert H Lurie Children's Hospital of Chicago
Information provided by (Responsible Party):
Children's Hospital of Philadelphia

Brief Summary:
We hypothesize that the combination treatment with deferasirox and deferiprone will be well tolerated and will result in significant improvement in cardiac and liver iron levels.

Condition or disease Intervention/treatment Phase
Thalassemia Major With Severe Transfusional Iron Overload Drug: Deferasirox and deferiprone Phase 1 Phase 2

Detailed Description:
Death and disability from iron related damage to the heart remain the most serious issue facing transfusion-dependent patients with thalassemia. However, over the past decade there have been several reports of improved survival and fewer cardiac complications. This improvement may be related to the availability of three chelators and also the accurate measurement of iron stores in various organs (e.g. heart and liver) with magnetic resonance imaging, which allows for personalized, tailored medical care for patients. The chelator characteristics, side effect profiles, and ability to remove iron from specific organs differ among the chelators, suggesting that combination therapy may be beneficial. Using two drugs at lower doses may be more tolerable than escalating doses of a single drug and may improve iron removal. The combination of deferoxamine and deferiprone has been shown to be particularly beneficial for reducing cardiac iron, but it requires a painful injection/infusion, which hinders adherence. This pilot study aims to investigate the safety of an oral-only combination chelator regimen (deferasirox and deferiprone) in individuals with thalassemia major with poorly controlled iron overload and to assess how well this chelator combination lowers iron stores over one year.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 9 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Pilot Study of Deferasirox and Deferiprone Combination Oral Chelation for Individuals With Transfusion Dependent Thalassemia and High Iron Burden
Actual Study Start Date : September 2012
Actual Primary Completion Date : August 1, 2015
Actual Study Completion Date : August 1, 2017

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Iron Thalassemia

Arm Intervention/treatment
Experimental: Deferasirox and deferiprone Drug: Deferasirox and deferiprone
Other Names:
  • Exjade
  • Ferriprox

Primary Outcome Measures :
  1. Number of Participants With Improvement in Liver Iron Concentration [ Time Frame: 12 months ]
    Determine the safety of the combination of Deferasirox and Deferiprone for the treatment of subjects with Thalassemia Major and Severe Iron Overload by assessing change in liver iron concentration from baseline to follow-up

Secondary Outcome Measures :
  1. Number of Participants With Improvement in Cardiac T2* MRI [ Time Frame: 12 months ]
    Improvement in Cardiac T2* MRI from baseline to determine if there is a reduction of cardiac iron burden.

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Alpha or beta thalassemia
  • Receiving chronic transfusions (at least 20 transfusions in lifetime) with iron overload requiring treatment with chelation
  • Serum ferritin >500 ng/ml
  • Liver iron concentration equal to or greater than 10 mg/g dw (by R2 MRI) or 7 to 10 mg/g dw (by R2 MRI) and not improving OR cardiac T2* between 6 and <20 ms
  • Women of childbearing age must have a negative pregnancy test
  • Agree to use approved method of contraception for the duration of the study
  • Subjects must have a good understanding of the study and be willing to comply with study procedures

Exclusion Criteria:

  • Subjects with past history of unexplained neutropenia (ANC < 1500/mcL), clinically significant renal disease (creatinine above the upper limit of normal), proteinuria >300 mg/L, clinically significant liver disease (ALT > 5x upper limit of normal), pulmonary or cardiovascular disease
  • History of other clinically relevant oral, endocrine, neurologic, psychiatric, immunologic, bone marrow or skin disorder that contraindicates dosing with deferasirox or deferiprone
  • History of adverse reaction or known allergy to either deferasirox or deferiprone necessitating drug discontinuation
  • Currently receiving treatment for active hepatitis
  • Use of any investigational agent in the past 30 days
  • Cardiac T2* <6 ms, left ventricular ejection fraction < 56%, and/or arrhythmia (certain subjects may be eligible if they have already had a trial of deferoxamine and deferiprone). Subjects who refuse to use deferoxamine after extensive consultation with at least 2 health care providers will also be allowed to participate.
  • Pregnant or breastfeeding females
  • Unwilling or unable to comply with study related procedures

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): NCT01709032

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United States, Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
United States, Pennsylvania
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Sponsors and Collaborators
Children's Hospital of Philadelphia
The Cooley's Anemia Foundation,
Ann & Robert H Lurie Children's Hospital of Chicago
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Principal Investigator: Janet L Kwiatkowski, MD, MSCE Children's Hospital of Philadlephia
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Responsible Party: Children's Hospital of Philadelphia Identifier: NCT01709032    
Other Study ID Numbers: 12-009449
First Posted: October 17, 2012    Key Record Dates
Results First Posted: March 6, 2019
Last Update Posted: March 6, 2019
Last Verified: February 2019
Keywords provided by Children's Hospital of Philadelphia:
Additional relevant MeSH terms:
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Iron Overload
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hematologic Diseases
Genetic Diseases, Inborn
Iron Metabolism Disorders
Metabolic Diseases
Iron Chelating Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action