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Safety & Efficacy of ICL670 vs. Deferoxamine in Beta-thalassemia Patients With Iron Overload Due to Blood Transfusions

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ClinicalTrials.gov Identifier: NCT00061750
Recruitment Status : Completed
First Posted : June 4, 2003
Last Update Posted : April 19, 2012
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Tracking Information
First Submitted Date  ICMJE June 3, 2003
First Posted Date  ICMJE June 4, 2003
Last Update Posted Date April 19, 2012
Study Start Date  ICMJE May 2003
Actual Primary Completion Date November 2004   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 14, 2006)
Demonstrate non-inferiority to deferoxamine in its effects on liver iron content (LIC)
Original Primary Outcome Measures  ICMJE Not Provided
Change History Complete list of historical versions of study NCT00061750 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: August 14, 2006)
  • Evaluate tolerability profile
  • Estimate absolute and relative change of LIC and Total body iron excretion
  • Evaluation relationship between LIC and potential surrogate markers
  • Evaluate the relationship between pharmacokinetics, pharmacodynamics and safety variable
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 Safety & Efficacy of ICL670 vs. Deferoxamine in Beta-thalassemia Patients With Iron Overload Due to Blood Transfusions
Official Title  ICMJE A Randomized, Comparative, Open Label Phase III Trial on Efficacy & Safety of Long-term Treatment With ICL670 Compared to Deferoxamine in Beta-thalassemia Patients With Transfusional Hemosiderosis
Brief Summary The purpose of this study is to deterimine if the new orally active iron chelator, ICL670, is as effective and as safe as deferoxamine in preventing accumulation of iron in the body while a patient is undergoing repeated blood transfusions.
Detailed Description Patients who require repeated blood transfusions to live accumulate iron in the body as blood cells contain iron and there is no natural body mechanism to eliminate it. After a while the iron levels get high enough to be toxic to the body. The current therapy of choice is deferoxamine, which does a good job of removing excess iron, but is difficult to administer. Deferoxamine requires subcutaneous (under the skin) infusions over 4 to 8 hours nightly 3 to 7 nights per week. In addition to the need to wear an infusion pump nightly, adverse reactions around the site of the injection are frequent.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Beta-Thalassemia
Intervention  ICMJE
  • Drug: ICL670
    Other Name: Deferasirox
  • Drug: deferoxamine
Study Arms  ICMJE
  • Experimental: ICL670
    Intervention: Drug: ICL670
  • Active Comparator: Deferoxamine
    Intervention: Drug: deferoxamine
Publications * Cohen AR, Glimm E, Porter JB. Effect of transfusional iron intake on response to chelation therapy in beta-thalassemia major. Blood. 2008 Jan 15;111(2):583-7. Epub 2007 Oct 19.

*   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 18, 2012)
595
Original Enrollment  ICMJE
 (submitted: June 23, 2005)
500
Study Completion Date  ICMJE Not Provided
Actual Primary Completion Date November 2004   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Beta-thalassemia patients already treated with or suitable for treatment with deferoxamine 20 to 40 mg/kg/day
  • Liver iron content greater than 2 mg iron/g dw as measured by liver biopsy
  • Need for regular transfusions 8 or more times per year

Exclusion Criteria:

  • Non-transfusional iron overload or transfusion-dependent anemias other than beta-thalassemia.
  • Documented toxicity to deferoxamine
  • Elevated liver enzymes in the year preceeding enrollment
  • Active hepatitis B or hepatitis C
  • HIV seropositivity
  • Elevated serum creatinine or significant proteinuria
  • History of nephrotic syndrome
  • Uncontrolled systemic hypertension
  • Fever and other signs/symptoms of infection within 10 days prior to start of the study
  • Presence of clinically relevant cataract or previous history of clinically relevant ocular toxicity related to iron chelation
  • Second or third degree AV block, clinically relevant Q-T interval prolongation, or patients requiring digoxin or other drugs that prolong the Q-T interval
  • Diseases (cardiovascular, renal, hepatic, etc.)that would prevent the patient from undergoing any of the treatment options
  • Psychiatric or additive disorders that would prevent the patient from giving informed consent
  • History of drug or alcohol abuse within the 12 months prior to the study
  • Pregnant or breast feeding patients
  • Patients treated with systemic investigational drugs within 4 weeks or topical investigational drugs within 7 days before the start of the study
  • Any surgical or medical condition that might significantly alter the absorption, distribution, metabolism or excretion of any drug, such as gastrointestinal disease or major surgery, renal disease, difficulty voiding or urinary obstruction, or impaired pancreatic function.
  • Non-compliant or unreliable patients.
  • Patients unable to undergo any study procedures such as the hearing or eye tests, or the liver echocardiography.
  • Inability to undergo a liver biopsy.
  • Patients that would need a dose of ICL670 less than 125 mg per day.
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 United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00061750
Other Study ID Numbers  ICMJE CICL670A0107
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Novartis ( Novartis Pharmaceuticals )
Study Sponsor  ICMJE Novartis Pharmaceuticals
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
PRS Account Novartis
Verification Date April 2012

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