Efficacy and Safety of Deferasirox in Patients With Chronic Anemia and Transfusional Hemosiderosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00631163
Recruitment Status : Completed
First Posted : March 7, 2008
Last Update Posted : November 16, 2016
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Brief Summary:

The overall purpose of this trial is to further evaluate the efficacy and safety of deferasirox, dosed initially according to the transfusional iron intake, in patients with transfusion dependant anemia related to disorders other than β-thalassemia and sickle cell disease.

During the study, the dose will be adjusted based on serum Ferritin.The overall purpose of the extension is to allow further treatment of patients who have already completed the core study, and to enable collection of long term efficacy and safety data. Patients will continue to receive Deferasirox at the dose they received at the end of the core study.

Condition or disease Intervention/treatment Phase
Chronic Anemia Transfusional Hemosiderosis Drug: Deferasirox (ICL670) Phase 2

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 114 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Multi-Center, Open-label, Non Comparative, Phase II Trial on Efficacy and Safety of ICL670 Given for 1 Year With Dose Adjustments Based on Serum Ferritin in Patients With Chronic Anemia and Transfusional Hemosiderosis Including an Additional 1 Year Extension.
Study Start Date : October 2007
Actual Primary Completion Date : January 2011
Actual Study Completion Date : February 2012

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Anemia Iron
Drug Information available for: Deferasirox
U.S. FDA Resources

Arm Intervention/treatment
Experimental: Deferasirox

The recommended initial daily dose of Deferasirox is 20 mg/kg body weight for most patients.

An initial daily dose of 30 mg/kg or 10mg/kg should be considered for patients requiring more intensive or less intensive chelation, respectively

Drug: Deferasirox (ICL670)
The recommended initial daily dose of Deferasirox is 20 mg/kg body weight.
Other Name: ICL670

Primary Outcome Measures :
  1. Change of Liver iron concentration measured by R2 MRI over a period of 1 year treatment. [ Time Frame: 12 months, 24 months ]

Secondary Outcome Measures :
  1. Change of serum ferritin concentration over a period of 1 year treatment. [ Time Frame: 12 months, 24 months ]
  2. Evaluation of safety and tolerability of deferasirox in Japanese patients. [ Time Frame: 12 months, 24 months ]
  3. Evaluation of Iron balance after 1 year of treatment [ Time Frame: 12 months, 24 months ]
  4. Evaluation of serum ferritin as a marker for accurate monitoring of chelation therapy [ Time Frame: 12 months, 24 months ]
  5. Evaluation of ophthalmologic safety of deferasirox [ Time Frame: 12 months, 24 months ]

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Ages Eligible for Study:   2 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion criteria (Core):

  • Patients with transfusional iron overload due to:
  • low or intermediate (INT-1) risk Myelodysplastic Syndrome (MDS)determined via International Prognosis Scoring System (IPSS) criteria
  • other congenital or acquired anemias excluding B-thalassemia and sickle cell disease
  • Lifetime transfusion history of ≥20 unit (approximately 100 mL/kg) of packed red blood cells or showing evidence of iron overload (serum ferritin >1000 µg/L).
  • Able to provide written informed consent
  • Life expectancy ≥ 12 months If patient was previously treated with deferiprone, a washout period of one month should occur before the first dose of deferasirox

Inclusion criteria (Extension):

  • Patients completing the planned 12-month core study (CICL670A2204).
  • Written informed consent obtained from the patient and/or legal guardian on the patient's behalf in accordance with national legislation.

Exclusion criteria (Core and Extension):

  • Patients with β-thalassemia, sickle cell disease or myelodysplastic syndrome with an IPSS score being Intermediate-2 or High.
  • Patients with serum creatinine > ULN
  • Patients with ALT(SGPT) levels > 5 x ULN
  • Significant proteinuria as indicated by a urinary protein/creatinine ratio >0.5 mg/mg in a non-first void urine sample on two assessments during the screening period.
  • History of HIV positive test result , or of clinical or laboratory evidence of active Hepatitis B or Hepatitis C (HBsAg in the absence of HBsAb OR HCV Ab positive with HCV RNA positive and ALT above the normal range)
  • Patients on investigational MDS therapies, including lenalidomide, thalidomide, azacitidine and arsenic trioxide, must have a ≥ 4 week washout period prior to the first dose of study drug.
  • Patients with systemic uncontrolled hypertension
  • Patients with unstable cardiac disease not controlled by standard medical therapy
  • Systemic disease (cardiovascular, renal, hepatic, etc.) which would prevent study treatment
  • Pregnancy (as documented in required screening laboratory test) or breast feeding.
  • Patients treated with systemic investigational drug within the past 4 weeks or topical investigational drug within the past 7 days
  • Other surgical or medical condition which might significantly alter the absorption, distribution, metabolism or excretion of study drug
  • Patients being considered by the investigator potentially unreliable and/or not cooperative with regard to the study protocol
  • History of hypersensitivity to any of the study drug or excipients
  • Sexually active pre-menopausal female patients without adequate contraception. Female patients must use effective contraception or must have undergone clinically documented total hysterectomy and/or oophorectomy, tubal ligation or be postmenopausal defined by amenorrhea for at least 12 months.

Other protocol defined inclusion/exclusion criteria may apply.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00631163

Novartis Investigative Site
Nagoya, Aichi, Japan, 453-8511
Novartis Investigative Site
Nagoya, Aichi, Japan, 466-8560
Novartis Investigative Site
Fukuoka-city, Fukuoka, Japan, 814-0180
Novartis Investigative Site
Sapporo-city, Hokkaido, Japan, 060-8543
Novartis Investigative Site
Nishinomiya, Hyogo, Japan, 663-8501
Novartis Investigative Site
Kahoku-gun, Ishikawa, Japan, 920-0293
Novartis Investigative Site
Kanazawa, Ishikawa, Japan, 920-8641
Novartis Investigative Site
OsakaSayama, Osaka, Japan, 589-8511
Novartis Investigative Site
Suita-city, Osaka, Japan, 565-0871
Novartis Investigative Site
Shimotsuka-gun, Tochigi, Japan, 321-0293
Novartis Investigative Site
Simotsuke-city, Tochigi, Japan, 329-0498
Novartis Investigative Site
Bunkyo-ku, Tokyo, Japan, 113-8655
Novartis Investigative Site
Cyuo-ku, Tokyo, Japan, 104-8560
Novartis Investigative Site
Minato-ku, Tokyo, Japan, 108-8639
Novartis Investigative Site
Shinagawa-ku, Tokyo, Japan, 141-8625
Novartis Investigative Site
Shinjuku-ku, Tokyo, Japan, 160-0023
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Shinjuku-ku, Tokyo, Japan, 162-8666
Novartis Investigative Site
Hiroshima, Japan, 734-8551
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Kumamoto, Japan, 860-0811
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Kyoto, Japan, 606-8507
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Nagasaki, Japan, 852-8501
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Toyama, Japan, 930-8550
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Warszawa, Poland, 02-097
Novartis Investigative Site
Warszawa, Poland, 02-776
Novartis Investigative Site
Singapore, Singapore, 169608
Novartis Investigative Site
Salamanca, Castilla y Leon, Spain, 37007
Novartis Investigative Site
Valencia, Spain, 46026
Novartis Investigative Site
Adana, Turkey, 01330
Novartis Investigative Site
Ankara, Turkey, 06100
Novartis Investigative Site
Istanbul, Turkey, 34093
Novartis Investigative Site
Izmir, Turkey, 35040
Sponsors and Collaborators
Novartis Pharmaceuticals
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
Study Director: Novartis Pharmaceuticals Novartis Pharmeceuticals

Additional Information:
Publications of Results:
Responsible Party: Novartis Pharmaceuticals Identifier: NCT00631163     History of Changes
Obsolete Identifiers: NCT01199003
Other Study ID Numbers: CICL670A2204
2006-003337-32 ( EudraCT Number )
First Posted: March 7, 2008    Key Record Dates
Last Update Posted: November 16, 2016
Last Verified: November 2016

Keywords provided by Novartis ( Novartis Pharmaceuticals ):
Iron Overload,
Chronic Anemia,
Transfusional hemosiderosis,

Additional relevant MeSH terms:
Hematologic Diseases
Iron Overload
Iron Metabolism Disorders
Metabolic Diseases
Iron Chelating Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action