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

Thalassemia Clinical Trial

Official title:

Safety of Deferasirox (ICL670) and Deferoxamine (Desferal or DFO) Combined Chelation Therapy in Patients With Transfusion Dependent Thalassemia and Iron Overload

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00901199
• Other study ID #: CICL670AUS24T
• Status: Completed
• Phase: Phase 2
• Start date: September 2007
• Est. completion date: October 2012

Study information

• Verified date: July 2021
• Source: UCSF Benioff Children's Hospital Oakland
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 22
• Est. completion date: October 2012
• Est. primary completion date: October 2012
• Accepts healthy volunteers: No
• Gender: All
• Age group: 8 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Iron Overload
• Thalassemia

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.

Locations

Country	Name	City	State
United States	CHRCO	Oakland	California

Sponsors (1)

Lead Sponsor	Collaborator
Elliott Vichinsky

Country where clinical trial is conducted

United States, 

References & Publications (1)

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 — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	12 months
Primary	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.	12 months