Combo Chelation Trial

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Elliott Vichinsky, Children's Hospital & Research Center Oakland
ClinicalTrials.gov Identifier:
NCT00901199
First received: May 12, 2009
Last updated: August 5, 2013
Last verified: August 2013
  Purpose

This is a small pilot study looking at the safety of giving combination chelation with Exjade and Desferal to 15 patients. The hypothesis is that combination chelation is safe in decreasing overall iron in patients with thalassemia.


Condition Intervention Phase
Thalassemia
Iron Overload
Drug: Combo Chelation with Deferasirox (Exjade) and Desferal (DFO)
Phase 4

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: 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

Resource links provided by NLM:


Further study details as provided by Children's Hospital & Research Center Oakland:

Primary Outcome Measures:
  • Monitoring kidney function with urine protein/creatine levels [ Time Frame: with transfusion ] [ Designated as safety issue: Yes ]

Enrollment: 22
Study Start Date: September 2007
Study Completion Date: October 2012
Primary Completion Date: October 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Child Cohort
This is the cohort in the study for children ages 8-18 years old. All subjects in this arm must have Liver Iron by SQUID of between 5-15mg/g dry liver and have a documented endocrinopathy or cardiac finding (low T2* or decreased cardiac function). All subjects in this arm will receive 7 days per week of Exjade and 3-5 days per week of Desferal.
Drug: Combo Chelation with Deferasirox (Exjade) and Desferal (DFO)
All subjects will be given Exjade 7 days per week 20-30mg/kg and Deferoxamine 3-7 days per week 50mg/kg. Days per week are dependent on how much iron overload subject has.
Other Names:
  • Exjade, ICL670 (deferisirox)
  • DFO, Desferal (deferoxamine)
Moderate Adult Cohort
Adults in this arm will have moderate iron overload,defined as SQUID of 5-15mg/g dry weight. They will also have to have a documented endocrinopathy or cardiac finding (low T2*). All subjects in this cohort will receive 7 days per week of Exjade (20-30mg/kg) and Desferal (50mg/kg)3-5 days per week.
Drug: Combo Chelation with Deferasirox (Exjade) and Desferal (DFO)
All subjects will be given Exjade 7 days per week 20-30mg/kg and Deferoxamine 3-7 days per week 50mg/kg. Days per week are dependent on how much iron overload subject has.
Other Names:
  • Exjade, ICL670 (deferisirox)
  • DFO, Desferal (deferoxamine)
Adults cohort with high iron overload
Adults with high iron overload defined as over 15mg/g dry liver. No cardiac finding or endocrinopathy necessary. Subjects in this cohort will receive Exjade 20-30mg/kg 7 days per week and Desferal (50mg/kg)5-7 days per week.
Drug: Combo Chelation with Deferasirox (Exjade) and Desferal (DFO)
All subjects will be given Exjade 7 days per week 20-30mg/kg and Deferoxamine 3-7 days per week 50mg/kg. Days per week are dependent on how much iron overload subject has.
Other Names:
  • Exjade, ICL670 (deferisirox)
  • DFO, Desferal (deferoxamine)

  Eligibility

Ages Eligible for Study:   8 Years and older
Genders Eligible for Study:   Both
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00901199

Locations
United States, California
CHRCO
Oakland, California, United States, 94609
Sponsors and Collaborators
Children's Hospital & Research Center Oakland
  More Information

No publications provided by Children's Hospital & Research Center Oakland

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Elliott Vichinsky, Director, Hematology/Oncology, Children's Hospital & Research Center Oakland
ClinicalTrials.gov Identifier: NCT00901199     History of Changes
Other Study ID Numbers: CICL670AUS24T
Study First Received: May 12, 2009
Last Updated: August 5, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by Children's Hospital & Research Center Oakland:
Thalassemia
Chelation
Iron Overload
Thalassemia patients with Iron overload

Additional relevant MeSH terms:
Iron Overload
Thalassemia
Anemia
Anemia, Hemolytic
Anemia, Hemolytic, Congenital
Genetic Diseases, Inborn
Hematologic Diseases
Hemoglobinopathies
Iron Metabolism Disorders
Metabolic Diseases
Deferasirox
Deferoxamine
Iron
Chelating Agents
Growth Substances
Iron Chelating Agents
Micronutrients
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Physiological Effects of Drugs
Sequestering Agents
Siderophores
Trace Elements

ClinicalTrials.gov processed this record on October 20, 2014