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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

Tracking Information
First Submitted Date  ICMJE May 12, 2009
First Posted Date  ICMJE May 13, 2009
Results First Submitted Date  ICMJE June 14, 2021
Results First Posted Date  ICMJE August 12, 2021
Last Update Posted Date August 12, 2021
Study Start Date  ICMJE September 2007
Actual Primary Completion Date October 2012   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: July 20, 2021)
  • 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.
  • 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.
Original Primary Outcome Measures  ICMJE
 (submitted: May 12, 2009)
Monitoring kidney function with urine protein/creatine levels [ Time Frame: with transfusion ]
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Combined Chelation Therapy in Patients With Transfusion Dependent Thalassemia and Iron Overload
Official Title  ICMJE Safety of Deferasirox (ICL670) and Deferoxamine (Desferal or DFO) Combined Chelation Therapy in Patients With Transfusion Dependent Thalassemia and Iron Overload
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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Thalassemia
  • Iron Overload
Intervention  ICMJE 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)
Study Arms  ICMJE Experimental: Deferasirox (Exjade) and Deferoxamine (DFO)
All subjects received Deferasirox (Exjade) and Deferoxamine (DFO) dosing based on the iron overload at baseline.
Intervention: Drug: Combo Chelation with Deferasirox (Exjade) and Deferoxamine (DFO)
Publications * 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.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: August 5, 2013)
22
Original Estimated Enrollment  ICMJE
 (submitted: May 12, 2009)
15
Actual Study Completion Date  ICMJE October 2012
Actual Primary Completion Date October 2012   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 8 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00901199
Other Study ID Numbers  ICMJE CICL670AUS24T
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Current Responsible Party Elliott Vichinsky, UCSF Benioff Children's Hospital Oakland
Original Responsible Party Elliott Vichinsky MD, Children's Hospital and Research Center Oakland
Current Study Sponsor  ICMJE Elliott Vichinsky
Original Study Sponsor  ICMJE UCSF Benioff Children's Hospital Oakland
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Elliot Vichinsky, MD UCSF Benioff Children's Hospital Oakland
PRS Account UCSF Benioff Children's Hospital Oakland
Verification Date July 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP