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Combined Chelation Therapy in Patients With Transfusion Dependent Thalassemia and Iron Overload

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ClinicalTrials.gov Identifier: NCT00901199
Recruitment Status : Completed
First Posted : May 13, 2009
Results First Posted : August 12, 2021
Last Update Posted : August 12, 2021
Sponsor:
Information provided by (Responsible Party):
Elliott Vichinsky, UCSF Benioff Children's Hospital Oakland

Study Description
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Brief Summary:
This is a pilot study looking at the safety and efficacy of giving combination chelation with deferasirox and deferoxamine. The hypothesis is that combination chelation is safe in decreasing overall iron in patients with thalassemia.

Condition or disease Intervention/treatment Phase
Thalassemia Iron Overload Drug: Combo Chelation with Deferasirox (Exjade) and Deferoxamine (DFO) Phase 2

Detailed Description:
This was a phase two pilot clinical trial designed to evaluate the safety and efficacy of the combination of deferasirox and deferoxamine in transfusion dependent thalassemia with a range of systemic iron burden. The duration of combined therapy was 12 months. Changes in liver iron concentration, ferritin, myocardial iron, and serum creatinine was monitored.
Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 22 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Safety of Deferasirox (ICL670) and Deferoxamine (Desferal or DFO) Combined Chelation Therapy in Patients With Transfusion Dependent Thalassemia and Iron Overload
Study Start Date : September 2007
Actual Primary Completion Date : October 2012
Actual Study Completion Date : October 2012

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Iron Thalassemia

Arms and Interventions
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Arm Intervention/treatment
Experimental: Deferasirox (Exjade) and Deferoxamine (DFO)
All subjects received Deferasirox (Exjade) and Deferoxamine (DFO) dosing based on the iron overload at baseline.
 Drug: Combo Chelation with Deferasirox (Exjade) and Deferoxamine (DFO)
All subjects will be given Deferasirox 20-30 mg/kg for 7 days per week. All subject will be given Deferoxamine 50 mg/kg for 3-7 days per week. The number of days for Deferoxamine will be determined by liver iron concentration at baseline.
Other Names:
  • Deferasirox, (Exjade)
  • Deferoxamine (DFO)


Outcome Measures
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Primary Outcome Measures :
  1. Efficacy of Combined Treatment With Deferasirox and Deferoxamine Over 12 Months [ Time Frame: 12 months ]
    Change in liver iron concentration from baseline to 12 months with the use of combined chelation therapy. The change was calculated as the liver iron concentration at 12 months minus the value at baseline.

  2. Change in Serum Creatinine During 12 Months Combined Chelation Therapy [ Time Frame: 12 months ]
    Comparison of average serum creatinine over 12 months of combined chelation therapy compared with baseline serum creatinine.


Eligibility Criteria
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Ages Eligible for Study:   8 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Transfusion Dependent Thalassemia
  • If iron between 5-15 mg/g dry liver by SQUID, subject must have a documented endocrinopathy or cardiac finding
  • Older than 8 years

Exclusion Criteria:

  • Participating on another interventional clinical trial
Contacts and Locations
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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 ClinicalTrials.gov identifier (NCT number): NCT00901199


Locations
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United States, California
CHRCO
Oakland, California, United States, 94609
Sponsors and Collaborators
Elliott Vichinsky
Investigators
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Principal Investigator: Elliot Vichinsky, MD UCSF Benioff Children's Hospital Oakland
More Information
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Publications of Results:

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Responsible Party: Elliott Vichinsky, Director, Hematology/Oncology, UCSF Benioff Children's Hospital Oakland
ClinicalTrials.gov Identifier: NCT00901199    
Other Study ID Numbers: CICL670AUS24T
First Posted: May 13, 2009    Key Record Dates
Results First Posted: August 12, 2021
Last Update Posted: August 12, 2021
Last Verified: July 2021
Keywords provided by Elliott Vichinsky, UCSF Benioff Children's Hospital Oakland:
Thalassemia
Chelation
Iron Overload
Thalassemia patients with Iron overload
Additional relevant MeSH terms:
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Thalassemia
Iron Overload
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
Iron Metabolism Disorders
 Metabolic Diseases
Deferasirox
Deferoxamine
Iron Chelating Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action
Siderophores