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

Elliott Vichinsky

Status and phase: Completed; Phase 2

Conditions: Thalassemia; Iron Overload

Treatments: Drug: Combo Chelation with Deferasirox (Exjade) and Deferoxamine (DFO)

Study type: Interventional

Funder types: Other

Identifiers

NCT00901199

CICL670AUS24T

Details and patient eligibility

About

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.

Full 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.

Enrollment: 22 patients

Sex: All

Ages: 8+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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22 participants in 1 patient group

Deferasirox (Exjade) and Deferoxamine (DFO)

Experimental group

Description:

All subjects received Deferasirox (Exjade) and Deferoxamine (DFO) dosing based on the iron overload at baseline.

Treatment:

Drug: Combo Chelation with Deferasirox (Exjade) and Deferoxamine (DFO)