Back to resultsCombined Chelation Therapy in Patients With Transfusion Dependent Thalassemia and Iron Overload
Primary Purpose
Thalassemia, Iron Overload
Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Combo Chelation with Deferasirox (Exjade) and Deferoxamine (DFO)
Sponsored by
About this trial
This is an interventional treatment trial for Thalassemia focused on measuring Thalassemia, Chelation, Iron Overload, Thalassemia patients with Iron overload
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
Sites / Locations
- CHRCO
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Deferasirox (Exjade) and Deferoxamine (DFO)
Arm Description
All subjects received Deferasirox (Exjade) and Deferoxamine (DFO) dosing based on the iron overload at baseline.
Outcomes
Primary Outcome Measures
Efficacy of Combined Treatment With Deferasirox and Deferoxamine Over 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.
Change in Serum Creatinine During 12 Months Combined Chelation Therapy
Comparison of average serum creatinine over 12 months of combined chelation therapy compared with baseline serum creatinine.
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT00901199
Brief Title
Combined Chelation Therapy in Patients With Transfusion Dependent Thalassemia and Iron Overload
Official Title
Safety of Deferasirox (ICL670) and Deferoxamine (Desferal or DFO) Combined Chelation Therapy in Patients With Transfusion Dependent Thalassemia and Iron Overload
Study Type
Interventional
2. Study Status
Record Verification Date
July 2021
Overall Recruitment Status
Completed
Study Start Date
September 2007 (undefined)
Primary Completion Date
October 2012 (Actual)
Study Completion Date
October 2012 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor-Investigator
Name of the Sponsor
Elliott Vichinsky
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
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.
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Thalassemia, Iron Overload
Keywords
Thalassemia, Chelation, Iron Overload, Thalassemia patients with Iron overload
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
22 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Deferasirox (Exjade) and Deferoxamine (DFO)
Arm Type
Experimental
Arm Description
All subjects received Deferasirox (Exjade) and Deferoxamine (DFO) dosing based on the iron overload at baseline.
Intervention Type
Drug
Intervention Name(s)
Combo Chelation with Deferasirox (Exjade) and Deferoxamine (DFO)
Other Intervention Name(s)
Deferasirox, (Exjade), Deferoxamine (DFO)
Intervention Description
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.
Primary Outcome Measure Information:
Title
Efficacy of Combined Treatment With Deferasirox and Deferoxamine Over 12 Months
Description
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.
Time Frame
12 months
Title
Change in Serum Creatinine During 12 Months Combined Chelation Therapy
Description
Comparison of average serum creatinine over 12 months of combined chelation therapy compared with baseline serum creatinine.
Time Frame
12 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
8 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Elliot Vichinsky, MD
Organizational Affiliation
UCSF Benioff Children's Hospital Oakland
Official's Role
Principal Investigator
Facility Information:
Facility Name
CHRCO
City
Oakland
State/Province
California
ZIP/Postal Code
94609
Country
United States
12. IPD Sharing Statement
Citations:
PubMed Identifier
23151373
Citation
Lal A, Porter J, Sweeters N, Ng V, Evans P, Neumayr L, Kurio G, Harmatz P, Vichinsky E. Combined chelation therapy with deferasirox and deferoxamine in thalassemia. Blood Cells Mol Dis. 2013 Feb;50(2):99-104. doi: 10.1016/j.bcmd.2012.10.006. Epub 2012 Nov 11.
Results Reference
result
Learn more about this trial
Combined Chelation Therapy in Patients With Transfusion Dependent Thalassemia and Iron Overload
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