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

Phase 2

Completed

• Conditions: Thalassemia; Iron Overload
• Interventions: Drug: Combo Chelation with Deferasirox (Exjade) and Deferoxamine (DFO)

• Registration Number: NCT00901199
• Lead Sponsor: Elliott Vichinsky

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 Timeline

• Study Start: 2007-09
• Study First Submitted: 2009-05-12
• Study First Submitted QC: 2009-05-12
• Study First Posted: 2009-05-13
• Primary Completion: 2012-10
• Study Completion: 2012-10
• Results First Submitted: 2021-06-14
• Status Verified: 2021-07
• Results First Submitted QC: 2021-07-20
• Last Update Submitted: 2021-07-20
• Results First Posted: 2021-08-12
• Last Update Posted: 2021-08-12

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 22

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Deferasirox (Exjade) and Deferoxamine (DFO)	Combo Chelation with Deferasirox (Exjade) and Deferoxamine (DFO)	All subjects received Deferasirox (Exjade) and Deferoxamine (DFO) dosing based on the iron overload at baseline.

Primary Outcome Measures

Name	Time	Method
Efficacy of Combined Treatment With Deferasirox and Deferoxamine Over 12 Months	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	12 months	Comparison of average serum creatinine over 12 months of combined chelation therapy compared with baseline serum creatinine.

Trial Locations

Locations (1)

CHRCO; 🇺🇸 Oakland, California, United States