A Study Assessing the Efficacy and Safety of Deferasirox in Patients With Transfusion-dependent Iron Overload

NCT ID: NCT00171821

Last Updated: 2020-02-11

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

1784 participants

Study Classification

INTERVENTIONAL

Study Start Date

2005-04-30

Study Completion Date

2010-07-31

Brief Summary

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This study uses a single arm, multi-center, open-label trial design. The study will assess the efficacy and safety of 52 weeks of treatment with deferasirox (ICL670) in patients with evidence of transfusion induced iron overload.

Detailed Description

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Conditions

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Transfusion-dependent Iron Overload

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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ICL670 (Deferasirox)

Group Type EXPERIMENTAL

Deferasirox

Intervention Type DRUG

Interventions

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Deferasirox

Intervention Type DRUG

Other Intervention Names

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ICL670

Eligibility Criteria

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Inclusion Criteria

* Patients presenting with transfusion-dependent anemias (independent of underlying condition) with transfusional iron overload as shown by a serum ferritin level of ≥ 1000 ng/ml
* Patients of either gender and aged ≥ 2 years
* Female patients who have reached menarche and who are sexually active must use an effective method of contraception, or must have undergone clinically documented total hysterectomy and/or ovariectomy, or tubal ligation.


* Written informed consent by the patient


* The definition of the term "pediatric" will be in accordance with local legislation. Parents or legal guardians will be fully informed by the investigator as to the requirements of the study. The pediatric patients themselves will be informed according to their capabilities in a language and terms that they are able to understand. Written informed consent will be obtained from their parents or legal guardians on the patient's behalf in accordance with the national legislation. If capable, all patients should also personally sign their written informed assent.

Exclusion Criteria

* Non-transfusional hemosiderosis
* Patients with clinical evidence supporting the need for intensive chelation, based on the investigator's judgment
* Patients with mean levels of alanine aminotransferase (ALT) \> 300 U/l
* Patients with uncontrolled systemic hypertension
* Patients with serum creatinine above the upper limit of normal (ULN)
* Significant proteinuria as indicated by a urinary protein/creatinine ratio \> 0.5 (mg/mg) in second-voiding urine samples taken at both visits 1 and 2. A third sample is to be taken from patients in whom one ratio is \> 0.5 (mg/mg) and one is ≤ 0.5 (mg/mg) and patients in whom the urinary protein/creatinine ratio is \> 0.5 (mg/mg) in two of the three determinations are also to be excluded.
* History of nephrotic syndrome
* Patients with 3rd atrioventricular (A-V) block, clinically relevant Q-T interval prolongation as well as patients requiring treatment with digoxin and similar compounds or drugs which may induce prolongation of the Q-T interval
* Patients with a previous history of clinically relevant ocular toxicity related to iron chelation
* Systemic diseases (cardiovascular, renal, hepatic, etc.) which would prevent the patient from undergoing study treatment
* Patients with psychiatric or addictive disorders which prevent them from giving their informed consent or undergoing study treatment
* Pregnant or breast feeding patients
* Patients treated with systemic investigational drugs within the past 4 weeks or topical investigational drugs within the past 7 days
* Any other surgical or medical condition which might significantly alter the absorption, distribution, metabolism or excretion of any drug. The investigator should be guided by evidence of any of the following:

* history of inflammatory bowel syndrome, gastritis, ulcers, gastrointestinal or rectal bleeding;
* history of major gastrointestinal tract surgery such as gastrectomy, gastroenterostomy, or bowel resection;
* history of pancreatic injury or pancreatitis; indications of impaired pancreatic function/injury as indicated by abnormal lipase or amylase;
* history or presence of impaired renal function as indicated by creatinine or blood urea nitrogen (BUN) values equal or above ULN;
* history of urinary obstruction or difficulty in voiding.
* History of non-compliance to medical regimens and patients who are considered potentially unreliable and/or not cooperative
* History of drug or alcohol abuse within the 12 months prior to dosing or evidence of such abuse as indicated by the laboratory assays conducted during the run-in period
* Patients with positive test to HIV
* Life expectancy of \< 1 year


* Patient body weight which prevents the use of the smallest tablet strength (i.e. 125 mg) for proper dosing
Minimum Eligible Age

2 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

Adelaide, , Australia

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Camperdown, , Australia

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Clayton, , Australia

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Melbourne, , Australia

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Perth, , Australia

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South Brisbane, , Australia

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Westmead, , Australia

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Graz, , Austria

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Linz, , Austria

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Vienna, , Austria

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Brussels, , Belgium

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Ghent, , Belgium

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Godinne, , Belgium

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La Louvière, , Belgium

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Leuven, , Belgium

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Guangzhou, , China

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Nanjing, , China

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Shanghai, , China

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Aarhus, , Denmark

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Copenhagen, , Denmark

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Herlev, , Denmark

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Hillerød, , Denmark

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Cairo, , Egypt

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Angers, , France

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Avignon, , France

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Bobigny, , France

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Créteil, , France

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Lille, , France

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Lyon, , France

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Nice, , France

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Paris, , France

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Pessac, , France

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Rennes, , France

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Toulouse, , France

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Vandœuvre-lès-Nancy, , France

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Augsburg, , Germany

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Braunschweig, , Germany

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Dresden, , Germany

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Düsseldorf, , Germany

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Frankfurt, , Germany

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Frankfurt am Main, , Germany

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Frieburg, , Germany

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Göttingen, , Germany

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Greifswald, , Germany

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Hanover, , Germany

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Mainz, , Germany

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München, , Germany

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Ulm, , Germany

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Athens, , Greece

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Larissa, , Greece

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Pátrai, , Greece

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Thessaloniki, , Greece

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Hong Kong, , Hong Kong

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Afula, , Israel

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Jerusalem, , Israel

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Petah Tikva, , Israel

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Bologna, , Italy

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Brindisi, , Italy

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Cagliari, , Italy

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Cona, , Italy

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Genova, , Italy

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Milan, , Italy

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Napoli, , Italy

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Orbassano, , Italy

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Palermo, , Italy

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Pavia, , Italy

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Pisa, , Italy

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Reggio Calabria, , Italy

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Roma, , Italy

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Sassari, , Italy

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Hazmiyeh, , Lebanon

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Kota Bharu, , Malaysia

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Kuala Lumpur, , Malaysia

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Nijmegen, , Netherlands

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Johannesburg, , South Africa

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Parktown, , South Africa

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Seoul, , South Korea

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Barakaldo, , Spain

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Barcelona, , Spain

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Madrid, , Spain

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Seville, , Spain

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Valencia, , Spain

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Geneva, , Switzerland

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Zurich, , Switzerland

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Taichung, , Taiwan

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Taipei, , Taiwan

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Bangkok, , Thailand

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Chaingmai, , Thailand

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Adana, , Turkey (Türkiye)

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Ankara, , Turkey (Türkiye)

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Istanbul, , Turkey (Türkiye)

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Izmir, , Turkey (Türkiye)

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Leeds, , United Kingdom

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Leicester, , United Kingdom

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London, , United Kingdom

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Manchester, , United Kingdom

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Sheffield, , United Kingdom

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Countries

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Australia Austria Belgium China Denmark Egypt France Germany Greece Hong Kong Israel Italy Lebanon Malaysia Netherlands South Africa South Korea Spain Switzerland Taiwan Thailand Turkey (Türkiye) United Kingdom

References

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Porter JB, El-Alfy M, Viprakasit V, Giraudier S, Chan LL, Lai Y, El-Ali A, Han J, Cappellini MD. Utility of labile plasma iron and transferrin saturation in addition to serum ferritin as iron overload markers in different underlying anemias before and after deferasirox treatment. Eur J Haematol. 2016 Jan;96(1):19-26. doi: 10.1111/ejh.12540. Epub 2015 Jun 23.

Reference Type DERIVED
PMID: 25691036 (View on PubMed)

Lee JW, Yoon SS, Shen ZX, Ganser A, Hsu HC, El-Ali A, Habr D, Martin N, Porter JB. Hematologic responses in patients with aplastic anemia treated with deferasirox: a post hoc analysis from the EPIC study. Haematologica. 2013 Jul;98(7):1045-8. doi: 10.3324/haematol.2012.077669. Epub 2013 Apr 12.

Reference Type DERIVED
PMID: 23585526 (View on PubMed)

Pennell DJ, Porter JB, Cappellini MD, Chan LL, El-Beshlawy A, Aydinok Y, Ibrahim H, Li CK, Viprakasit V, Elalfy MS, Kattamis A, Smith G, Habr D, Domokos G, Roubert B, Taher A. Continued improvement in myocardial T2* over two years of deferasirox therapy in beta-thalassemia major patients with cardiac iron overload. Haematologica. 2011 Jan;96(1):48-54. doi: 10.3324/haematol.2010.031468. Epub 2010 Nov 11.

Reference Type DERIVED
PMID: 21071497 (View on PubMed)

Lee JW, Yoon SS, Shen ZX, Ganser A, Hsu HC, Habr D, Domokos G, Roubert B, Porter JB; EPIC study investigators. Iron chelation therapy with deferasirox in patients with aplastic anemia: a subgroup analysis of 116 patients from the EPIC trial. Blood. 2010 Oct 7;116(14):2448-54. doi: 10.1182/blood-2010-01-261289. Epub 2010 Jun 21.

Reference Type DERIVED
PMID: 20566896 (View on PubMed)

Pennell DJ, Porter JB, Cappellini MD, El-Beshlawy A, Chan LL, Aydinok Y, Elalfy MS, Sutcharitchan P, Li CK, Ibrahim H, Viprakasit V, Kattamis A, Smith G, Habr D, Domokos G, Roubert B, Taher A. Efficacy of deferasirox in reducing and preventing cardiac iron overload in beta-thalassemia. Blood. 2010 Mar 25;115(12):2364-71. doi: 10.1182/blood-2009-04-217455. Epub 2009 Dec 8.

Reference Type DERIVED
PMID: 19996412 (View on PubMed)

Cappellini MD, Porter J, El-Beshlawy A, Li CK, Seymour JF, Elalfy M, Gattermann N, Giraudier S, Lee JW, Chan LL, Lin KH, Rose C, Taher A, Thein SL, Viprakasit V, Habr D, Domokos G, Roubert B, Kattamis A; EPIC Study Investigators. Tailoring iron chelation by iron intake and serum ferritin: the prospective EPIC study of deferasirox in 1744 patients with transfusion-dependent anemias. Haematologica. 2010 Apr;95(4):557-66. doi: 10.3324/haematol.2009.014696. Epub 2009 Nov 30.

Reference Type DERIVED
PMID: 19951979 (View on PubMed)

Other Identifiers

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2004-003953-16

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

CICL670A2409

Identifier Type: -

Identifier Source: org_study_id

NCT00565578

Identifier Type: -

Identifier Source: nct_alias

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