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Combination Deferasirox and Deferiprone for Severe Iron Overload in Thalassemia

This study is ongoing, but not recruiting participants.
The Cooley’s Anemia Foundation,
Ann & Robert H Lurie Children's Hospital of Chicago
Information provided by (Responsible Party):
Children's Hospital of Philadelphia Identifier:
First received: October 16, 2012
Last updated: February 15, 2017
Last verified: February 2017
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 Intervention Phase
Thalassemia Major With Severe Transfusional Iron Overload
Drug: Deferasirox and deferiprone
Phase 1
Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: No masking
Primary Purpose: Treatment
Official Title: Pilot Study of Deferasirox and Deferiprone Combination Oral Chelation for Individuals With Transfusion Dependent Thalassemia and High Iron Burden

Resource links provided by NLM:

Further study details as provided by Children's Hospital of Philadelphia:

Primary Outcome Measures:
  • Determine the safety of the combination of deferasirox and deferiprone for the treatment of subjects with thalassemia major and severe iron overload [ Time Frame: 12 months ]
    Safety and tolerability of the two oral chelators in combination based on clinical assessments (ex. incidence and severity of adverse events), physical examinations, and clinical laboratory values

Secondary Outcome Measures:
  • To obtain preliminary efficacy data on the reduction in serum ferritin level and liver iron concentration by R2 magnetic resonance imaging (MRI) and improvement in cardiac T2* MRI (estimation of cardiac iron loading) [ Time Frame: 12 months ]

Enrollment: 9
Actual Study Start Date: September 2012
Estimated Study Completion Date: August 2017
Primary Completion Date: June 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Deferasirox and deferiprone Drug: Deferasirox and deferiprone
Other Names:
  • Exjade
  • Ferriprox

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.

Ages Eligible for Study:   18 Years and older   (Adult, Senior)
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
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Please refer to this study by its identifier: NCT01709032

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
Principal Investigator: Janet L Kwiatkowski, MD, MSCE Children's Hospital of Philadlephia
  More Information

Responsible Party: Children's Hospital of Philadelphia Identifier: NCT01709032     History of Changes
Other Study ID Numbers: 12-009449
Study First Received: October 16, 2012
Last Updated: February 15, 2017

Keywords provided by Children's Hospital of Philadelphia:

Additional relevant MeSH terms:
Iron Overload
Iron Metabolism Disorders
Metabolic Diseases
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hematologic Diseases
Genetic Diseases, Inborn
Iron Chelating Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action processed this record on April 24, 2017