Evaluating Use of Deferasirox as Compared to Deferoxamine in Treating Cardiac Iron Overload (CORDELIA)

Novartis

Status and phase: Completed; Phase 2

Conditions: Transfusional Hemosiderosis; Transfusional Iron Overload

Treatments: Drug: Core Study: Deferoxamine; Drug: Extension: deferasirox to deferoxamine; Drug: Extension: deferoxamine to deferasirox; Drug: Core Study: Deferasirox; Drug: Deferoxamine; Drug: Deferasirox

Study type: Interventional

Funder types: Industry

Identifiers

NCT00600938

2007-000766-20 (EudraCT Number)

CICL670A2206

Details and patient eligibility

About

This is a clinical research study in patients who have iron overload in the heart due to chronic blood transfusions.

The study will have 2 treatment groups and will compare the safety and efficacy of chelation therapy with a medicine called deferasirox (ICL670) with another medicine called deferoxamine (DFO). The study is aimed at finding out which of the two medicines is the best for treating iron overload in the heart.

Patients will be treated for 12 months (core study phase). Patients who complete the core study phase will be offered to continue their study treatment in a 12 months extension phase. During the core and extension, the effects of treatment on iron overload in the heart and the liver will be evaluated using specific magnetic resonance imaging (MRI) assessments.

Enrollment: 197 patients

Sex: All

Ages: 10 to 65 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Male or female patients, aged 10 years and above, with β-thalassemia major or DBA or sideroblastic anemia on chronic transfusion therapy, having given written consent to participate in the study.
• Patients with cardiac iron as measured by a myocardial T2* value that is ≥ 6ms but not ≥ 20 ms.
• Patients with a lifetime history of at least 50 units of red cell transfusions, and must be receiving at least ≥10 units/yr of red blood cells transfusions.
• Patients with a left ventricular ejection fraction (LVEF) ≥ 56 % as determined by cardiovascular magnetic resonance (CMR).
• Patients with liver iron content (LIC) value ≥ 3 mg Fe / g dw, as determined by liver MRI.

Exclusion criteria

• Patients with clinical symptoms of cardiac dysfunction.
• Patients unable to undergo study assessments including MRI
• Patients participating in another clinical trial or receiving an investigational drug.

Other protocol-defined inclusion/exclusion criteria may apply.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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197 participants in 4 patient groups

Deferasirox

Experimental group

Description:

20 mg/kg/day once daily (od) for 2 weeks, followed by 30 mg/kg/day od for 1 week and a subsequent continuation of 40 mg/kg/day

Treatment:

Drug: Deferasirox

Drug: Core Study: Deferasirox

Deferasirox Placebo

Active Comparator group

Description:

50 mg/kg/day to 60 mg/kg/day infused subcutaneously in 8- to 12-hour intervals administered 5 to 7 days/week

Treatment:

Drug: Deferoxamine

Drug: Core Study: Deferoxamine

Extension: deferoxamine to deferasirox

Experimental group

Description:

"DFO to ICL" (patients who switched from DFO to deferasirox in extension)

Treatment:

Drug: Extension: deferoxamine to deferasirox

Extension: deferasirox to deferoxamine

Experimental group

Description:

"ICL to DFO" (patients who switched from deferasirox to DFO in extension)

Treatment:

Drug: Extension: deferasirox to deferoxamine