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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
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 Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Thalassemia
Iron Overload
Intervention Drug: Combo Chelation with Deferasirox (Exjade) and Deferoxamine (DFO)
Enrollment 22
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Deferasirox (Exjade) and Deferoxamine (DFO)
Hide Arm/Group Description

Patients will receive combined chelation therapy using Deferasirox (Exjade) and Deferoxamine (DFO) with dosing based on liver iron concentration at baseline.

Patients with moderate iron overload,defined as liver iron concentration of 5-15mg/g plus a documented endocrinopathy or cardiac iron (low MRI T2*), will receive 7 days per week of Deferasirox (Exjade) 20-30 mg/kg and Deferoxamine (DFO) 50 mg/kg 3-5 days per week.

Patients with high iron overload, defined as liver iron concentration over 15mg/g will receive Deferasirox (Exjade) 20-30 mg/kg 7 days per week and Deferoxamine (DFO) 50 mg/kg 5-7 days per week.

Period Title: Overall Study
Started 22
Completed 18
Not Completed 4
Arm/Group Title Deferasirox (Exjade) and Deferoxamine (DFO)
Hide Arm/Group Description

Patients will receive combined chelation therapy using Deferasirox (Exjade) and Deferoxamine (DFO) with dosing based on liver iron concentration at baseline.

Patients with moderate iron overload,defined as liver iron concentration of 5-15mg/g plus a documented endocrinopathy or cardiac iron (low MRI T2*), will receive 7 days per week of Deferasirox (Exjade) 20-30 mg/kg and Deferoxamine (DFO) 50 mg/kg 3-5 days per week.

Patients with high iron overload, defined as liver iron concentration over 15mg/g will receive Deferasirox (Exjade) 20-30 mg/kg 7 days per week and Deferoxamine (DFO) 50 mg/kg 5-7 days per week.

Overall Number of Baseline Participants 22
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Age, Categorical Number Analyzed 22 participants
<=18 years
6
  27.3%
Between 18 and 65 years
16
  72.7%
>=65 years
0
   0.0%
Sex/Gender, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Sex/Gender Not Collected Number Analyzed 22 participants
22
 100.0%
1.Primary Outcome
Title Efficacy of Combined Treatment With Deferasirox and Deferoxamine Over 12 Months
Hide 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
Hide Outcome Measure Data
Hide Analysis Population Description
Eighteen subjects completed one year of therapy. Four subjects did not complete the study for the following reasons: lost to follow-up (1), abdominal pain (1), death (1), and non-compliance (1).
Arm/Group Title Deferasirox (Exjade) and Desferal (DFO)
Hide Arm/Group Description:
Subjects were treated with Deferasirox (Exjade) and Desferal (DFO) with dosing based on baseline iron overload.
Overall Number of Participants Analyzed 18
Median (Full Range)
Unit of Measure: mg/g
-6.2
(-23.1 to 2.7)
2.Primary Outcome
Title Change in Serum Creatinine During 12 Months Combined Chelation Therapy
Hide Description Comparison of average serum creatinine over 12 months of combined chelation therapy compared with baseline serum creatinine.
Time Frame 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Deferasirox (Exjade) and Desferal (DFO)
Hide Arm/Group Description:
Subjects were treated with Deferasirox (Exjade) and Desferal (DFO) with dosing based on baseline iron overload.
Overall Number of Participants Analyzed 22
Mean (95% Confidence Interval)
Unit of Measure: mg/dl
0.09
(0.05 to 0.12)
Time Frame 1 year
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Deferasirox (Exjade) and Deferoxamine (DFO)
Hide Arm/Group Description Treatment with Deferasirox (Exjade) and Deferoxamine (DFO) with dosing based on baseline iron overload.
All-Cause Mortality
Deferasirox (Exjade) and Deferoxamine (DFO)
Affected / at Risk (%)
Total   1/22 (4.55%)    
Hide Serious Adverse Events
Deferasirox (Exjade) and Deferoxamine (DFO)
Affected / at Risk (%) # Events
Total   2/22 (9.09%)    
Cardiac disorders   
Death * [1]  1/22 (4.55%)  1
Hepatobiliary disorders   
Cholelithiasis * [2]  1/22 (4.55%)  1
Infections and infestations   
Central venous line infection *  1/22 (4.55%)  1
*
Indicates events were collected by non-systematic assessment
[1]
Baseline liver iron 24.6 mg/g, cardiac MRI T2* 4.8 ms, ejection fraction 67%. Two months into the study, presented to ER with abdominal pain, ascites, diarrhea, and leukocytosis. Expired during emergency colectomy from cardiac arrhythmias
[2]
Cholecystectomy
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 2.5%
Deferasirox (Exjade) and Deferoxamine (DFO)
Affected / at Risk (%) # Events
Total   1/22 (4.55%)    
Blood and lymphatic system disorders   
Transfusion reaction *  1/22 (4.55%)  1
*
Indicates events were collected by non-systematic assessment
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Ashutosh Lal, MD
Organization: Children's Hospital & Research Center at Oakland
Phone: 510-428-3172
EMail: ashutosh.lal@ucsf.edu
Layout table for additonal information
Responsible Party: Elliott Vichinsky, UCSF Benioff Children's Hospital Oakland
ClinicalTrials.gov Identifier: NCT00901199    
Other Study ID Numbers: CICL670AUS24T
First Submitted: May 12, 2009
First Posted: May 13, 2009
Results First Submitted: June 14, 2021
Results First Posted: August 12, 2021
Last Update Posted: August 12, 2021