Safety of Deferasirox (ICL670) and Deferoxamine (Desferal or DFO) Combined Chelation Therapy in Patients With Transfusion Dependent Thalassemia and Iron Overload
Combo Chelation Trial
Sponsors
Source
Children's Hospital & Research Center Oakland
Oversight Info
Has Dmc
Yes
Brief Summary
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.
Overall Status
Completed
Start Date
2007-09-01
Completion Date
2012-10-01
Primary Completion Date
2012-10-01
Phase
Phase 4
Study Type
Interventional
Primary Outcome
Measure |
Time Frame |
|
Monitoring kidney function with urine protein/creatine levels |
with transfusion |
Enrollment
22
Conditions
Intervention
Intervention Type
Drug
Intervention Name
Description
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.
Arm Group Label
Child Cohort
Moderate Adult Cohort
Adults cohort with high iron overload
Other Name
Exjade, ICL670 (deferisirox)
DFO, Desferal (deferoxamine)
Eligibility
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
Gender
All
Minimum Age
8 Years
Maximum Age
N/A
Healthy Volunteers
No
Location
Facility |
|
CHRCO Oakland California 94609 United States |
Location Countries
Country
United States
Verification Date
2013-08-01
Lastchanged Date
N/A
Firstreceived Date
N/A
Responsible Party
Responsible Party Type
Principal Investigator
Investigator Affiliation
Children's Hospital & Research Center Oakland
Investigator Full Name
Elliott Vichinsky
Investigator Title
Director, Hematology/Oncology
Keywords
Has Expanded Access
No
Condition Browse
Number Of Arms
3
Intervention Browse
Mesh Term
Iron
Deferasirox
Deferoxamine
Arm Group
Arm Group Label
Child Cohort
Arm Group Type
Other
Description
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.
Arm Group Label
Moderate Adult Cohort
Arm Group Type
Other
Description
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.
Arm Group Label
Adults cohort with high iron overload
Arm Group Type
Other
Description
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.
Firstreceived Results Date
N/A
Firstreceived Results Disposition Date
N/A
Study Design Info
Allocation
Non-Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
None (Open Label)
Study First Submitted
May 12, 2009
Study First Submitted Qc
May 12, 2009
Study First Posted
May 13, 2009
Last Update Submitted
August 5, 2013
Last Update Submitted Qc
August 5, 2013
Last Update Posted
August 7, 2013
ClinicalTrials.gov processed this data on August 29, 2018
Conditions
Conditions usually refer to a disease, disorder, syndrome, illness, or injury. In ClinicalTrials.gov,
conditions include any health issue worth studying, such as lifespan, quality of life, health risks, etc.
Interventions
Interventions refer to the drug, vaccine, procedure, device, or other potential treatment being studied.
Interventions can also include less intrusive possibilities such as surveys, education, and interviews.
Study Phase
Most clinical trials are designated as phase 1, 2, 3, or 4, based on the type of questions
that study is seeking to answer:
In Phase 1 (Phase I) clinical trials, researchers test a new drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
In Phase 2 (Phase II) clinical trials, the study drug or treatment is given to a larger group of people (100-300) to see if it is effective and to further evaluate its safety.
In Phase 3 (Phase III) clinical trials, the study drug or treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
In Phase 4 (Phase IV) clinical trials, post marketing studies delineate additional information including the drug's risks, benefits, and optimal use.
These phases are defined by the Food and Drug Administration in the Code of Federal Regulations.
In Phase 1 (Phase I) clinical trials, researchers test a new drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
In Phase 2 (Phase II) clinical trials, the study drug or treatment is given to a larger group of people (100-300) to see if it is effective and to further evaluate its safety.
In Phase 3 (Phase III) clinical trials, the study drug or treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
In Phase 4 (Phase IV) clinical trials, post marketing studies delineate additional information including the drug's risks, benefits, and optimal use.
These phases are defined by the Food and Drug Administration in the Code of Federal Regulations.