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

• 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: Elliott Vichinsky
• Information provided by (Responsible Party): Elliott Vichinsky, UCSF Benioff Children's Hospital Oakland

Tracking Information

• First Submitted Date: May 12, 2009
• First Posted Date: May 13, 2009
• Results First Submitted Date: June 14, 2021
• Results First Posted Date: August 12, 2021
• Last Update Posted Date: August 12, 2021
• Study Start Date: September 2007
• Actual Primary Completion Date: October 2012 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (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 (submitted: May 12, 2009): Monitoring kidney function with urine protein/creatine levels [ Time Frame: with transfusion ]
• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• 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
• 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: Interventional
• Study Phase: Phase 2
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment

Condition

• Thalassemia
• Iron Overload

Intervention

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

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.

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: August 5, 2013): 22
• Original Estimated Enrollment (submitted: May 12, 2009): 15
• Actual Study Completion Date: October 2012
• Actual Primary Completion Date: October 2012 (Final data collection date for primary outcome measure)

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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 8 Years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT00901199
• Other Study ID Numbers: CICL670AUS24T
• Has Data Monitoring Committee: Yes
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: 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: Elliott Vichinsky
• Original Study Sponsor: UCSF Benioff Children's Hospital Oakland
• Collaborators: Not Provided

Investigators

• Principal Investigator: Elliot Vichinsky, MD; UCSF Benioff Children's Hospital Oakland

• PRS Account: UCSF Benioff Children's Hospital Oakland
• Verification Date: July 2021