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

NCT ID: NCT00600938

Last Updated: 2014-08-27

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

197 participants

Study Classification

INTERVENTIONAL

Study Start Date

2007-11-30

Study Completion Date

2013-03-31

Brief Summary

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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.

Detailed Description

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Conditions

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Transfusional Iron Overload Transfusional Hemosiderosis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Deferasirox

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

Group Type EXPERIMENTAL

Core Study: Deferasirox

Intervention Type DRUG

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

Deferasirox

Intervention Type DRUG

Deferasirox Placebo

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

Group Type ACTIVE_COMPARATOR

Core Study: Deferoxamine

Intervention Type DRUG

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

Deferoxamine

Intervention Type DRUG

Extension: deferoxamine to deferasirox

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

Group Type EXPERIMENTAL

Extension: deferoxamine to deferasirox

Intervention Type DRUG

40 mg/kg deferasirox once daily administered 30 minutes before taking food.

Extension: deferasirox to deferoxamine

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

Group Type EXPERIMENTAL

Extension: deferasirox to deferoxamine

Intervention Type DRUG

DFO at a target range of 50 mg/kg/day to 60 mg/kg/day via subcutaneous (sc) infusion lasting a period of 8 to 12 hrs administered for 5 to 7 days per week,

Interventions

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Core Study: Deferasirox

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

Intervention Type DRUG

Core Study: Deferoxamine

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

Intervention Type DRUG

Extension: deferoxamine to deferasirox

40 mg/kg deferasirox once daily administered 30 minutes before taking food.

Intervention Type DRUG

Extension: deferasirox to deferoxamine

DFO at a target range of 50 mg/kg/day to 60 mg/kg/day via subcutaneous (sc) infusion lasting a period of 8 to 12 hrs administered for 5 to 7 days per week,

Intervention Type DRUG

Deferasirox

Intervention Type DRUG

Deferoxamine

Intervention Type DRUG

Other Intervention Names

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"ICL to ICL" "DFO to DFO" "DFO to ICL" "ICL to DFO"

Eligibility Criteria

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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.
Minimum Eligible Age

10 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Novartis Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Novartis Pharmaceuticals

Role: STUDY_DIRECTOR

Novartis Pharmaceuticals

Locations

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Novartis Investigative Site

Toronto, Ontario, Canada

Site Status

Novartis Investigative Site

Nanning, Guangxi, China

Site Status

Novartis Investigative Site

Limassol, , Cyprus

Site Status

Novartis Investigative Site

Al Mansurah, , Egypt

Site Status

Novartis Investigative Site

Cairo, , Egypt

Site Status

Novartis Investigative Site

Cagliari, CA, Italy

Site Status

Novartis Investigative Site

Genova, GE, Italy

Site Status

Novartis Investigative Site

Hazmiyeh, , Lebanon

Site Status

Novartis Investigative Site

Taipei, Taiwan, Taiwan

Site Status

Novartis Investigative Site

Bangkok, , Thailand

Site Status

Novartis Investigative Site

Bangkok, , Thailand

Site Status

Novartis Investigative Site

Adana, Turkey, Turkey (Türkiye)

Site Status

Novartis Investigative Site

Ankara, Turkey, Turkey (Türkiye)

Site Status

Novartis Investigative Site

Izmir, Turkey, Turkey (Türkiye)

Site Status

Novartis Investigative Site

Antalya, , Turkey (Türkiye)

Site Status

Novartis Investigative Site

Istanbul, , Turkey (Türkiye)

Site Status

Novartis Investigative Site

Dubai, Dubai, United Arab Emirates

Site Status

Novartis Investigative Site

Leeds, West Yorkshire, United Kingdom

Site Status

Novartis Investigative Site

Leeds, , United Kingdom

Site Status

Novartis Investigative Site

London, , United Kingdom

Site Status

Novartis Investigative Site

London, , United Kingdom

Site Status

Countries

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Canada China Cyprus Egypt Italy Lebanon Taiwan Thailand Turkey (Türkiye) United Arab Emirates United Kingdom

References

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Pennell DJ, Porter JB, Piga A, Lai YR, El-Beshlawy A, Elalfy M, Yesilipek A, Kilinc Y, Habr D, Musallam KM, Shen J, Aydinok Y; CORDELIA study investigators. Sustained improvements in myocardial T2* over 2 years in severely iron-overloaded patients with beta thalassemia major treated with deferasirox or deferoxamine. Am J Hematol. 2015 Feb;90(2):91-6. doi: 10.1002/ajh.23876. Epub 2014 Nov 19.

Reference Type DERIVED
PMID: 25345697 (View on PubMed)

Pennell DJ, Porter JB, Piga A, Lai Y, El-Beshlawy A, Belhoul KM, Elalfy M, Yesilipek A, Kilinc Y, Lawniczek T, Habr D, Weisskopf M, Zhang Y, Aydinok Y; CORDELIA study investigators. A 1-year randomized controlled trial of deferasirox vs deferoxamine for myocardial iron removal in beta-thalassemia major (CORDELIA). Blood. 2014 Mar 6;123(10):1447-54. doi: 10.1182/blood-2013-04-497842. Epub 2014 Jan 2.

Reference Type DERIVED
PMID: 24385534 (View on PubMed)

Other Identifiers

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2007-000766-20

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

CICL670A2206

Identifier Type: -

Identifier Source: org_study_id

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