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

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.
ClinicalTrials.gov Identifier: NCT00171821
Recruitment Status : Completed
First Posted : September 15, 2005
Last Update Posted : February 11, 2020
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Tracking Information
First Submitted Date  ICMJE September 13, 2005
First Posted Date  ICMJE September 15, 2005
Last Update Posted Date February 11, 2020
Study Start Date  ICMJE April 2005
Actual Primary Completion Date May 2009   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 21, 2010)
To evaluate if fixed starting doses of ICL670, based on transfusion history and subsequent dose titration can provide clinically acceptable chelation as measured by serum ferritin [ Time Frame: at baseline and at 52 weeks ]
Original Primary Outcome Measures  ICMJE Not Provided
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: October 26, 2010)
  • To evaluate the safety and tolerability profile of in patients treated for up to 52 weeks [ Time Frame: Monthly ]
  • Evaluate efficacy, tolerabilty and safety in the subgroup of patients with baseline LIC < 7 mg Fe/g dw [ Time Frame: Monthly ]
  • Evaluate the relationship between serum ferritin and potential surrogate markers [ Time Frame: Monthly ]
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
Descriptive Information
Brief Title  ICMJE A Study Assessing the Efficacy and Safety of Deferasirox in Patients With Transfusion-dependent Iron Overload
Official Title  ICMJE A One Year, Open-label, Single-arm, Multi-center Trial Evaluating the Efficacy and Safety of Oral ICL670 (20 mg/kg/Day) in Patients Diagnosed With Transfusion-dependent Iron Overload
Brief Summary 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 Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Transfusion-dependent Iron Overload
Intervention  ICMJE Drug: Deferasirox
Other Name: ICL670
Study Arms  ICMJE Experimental: ICL670 (Deferasirox)
Intervention: Drug: Deferasirox
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: April 15, 2013)
Original Enrollment  ICMJE Not Provided
Actual Study Completion Date  ICMJE July 2010
Actual Primary Completion Date May 2009   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.

Additional Inclusion Criteria for Adult Patients:

  • Written informed consent by the patient

Additional Inclusion Criteria for Pediatric Patients:

  • 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

Exclusion Criteria for Pediatric Patients:

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

Other protocol-defined inclusion/exclusion criteria may apply.

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 2 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Australia,   Austria,   Belgium,   China,   Denmark,   Egypt,   France,   Germany,   Greece,   Hong Kong,   Israel,   Italy,   Korea, Republic of,   Lebanon,   Malaysia,   Netherlands,   South Africa,   Spain,   Switzerland,   Taiwan,   Thailand,   Turkey,   United Kingdom
Removed Location Countries  
Administrative Information
NCT Number  ICMJE NCT00171821
Other Study ID Numbers  ICMJE CICL670A2409
2004-003953-16 ( EudraCT Number )
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Current Responsible Party Novartis ( Novartis Pharmaceuticals )
Original Responsible Party Not Provided
Current Study Sponsor  ICMJE Novartis Pharmaceuticals
Original Study Sponsor  ICMJE Novartis
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
PRS Account Novartis
Verification Date June 2013

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP