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Safety and Efficacy of Deferasirox (ICL670) in Patients With Iron Overload Resulting From Hereditary Hemochromatosis

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ClinicalTrials.gov Identifier: NCT00395629
Recruitment Status : Completed
First Posted : November 3, 2006
Results First Posted : May 18, 2011
Last Update Posted : May 30, 2011
Sponsor:
Information provided by:
Novartis

Brief Summary:
Brief Summary: This study was designed to explore a safe dose and characterize the preliminary safety and efficacy of ICL670 in adult patients with previously documented history of homozygous C282Y.

Condition or disease Intervention/treatment Phase
Iron Overload Hereditary Hemochromatosis Drug: Deferasirox (ICL670) Phase 1 Phase 2

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 49 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase I/II Open Label, Dose Escalation Trial and a Six Month Extension to Explore the Safety and Efficacy of ICL670 in Patients With Iron Overload Resulting From Hereditary Hemochromatosis.
Study Start Date : August 2006
Actual Primary Completion Date : December 2007
Actual Study Completion Date : March 2009

Resource links provided by the National Library of Medicine

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

Arm Intervention/treatment
Experimental: ICL670 (Deferasirox)
Three dose cohorts: 5 mg/kg/day, 10 mg/kg/day, 15 mg/kg/day
Drug: Deferasirox (ICL670)



Primary Outcome Measures :
  1. Absolute Change of Serum Ferritin From Baseline to the End of Extension, by Dose Cohort (Extension Per-protocol Population) [ Time Frame: 0 to 48 weeks ]
    Mean absolute change in serum ferritin from baseline to the end of the extension study.


Secondary Outcome Measures :
  1. Trough Concentrations of Deferasirox (ICL670), by Dose Cohort (Per-protocol Population) [ Time Frame: 4, 8, 12, 16, 20, and 24 weeks ]
    A blood sample was collected just prior to administration of the next dose of Deferasirox (pre-dose trough level) or approximately 24 hours after the previous dose at weeks 4, 8, 12, 16, 20 and 24. The mean trough concentration at each time point was calculated.



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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Age 18 years of age or older
  • Male or female patients homozygous for the C282Y mutation.
  • Iron overload as documented by serum ferritin and transferrin saturation
  • No known allergy or contraindication to the administration of deferasirox
  • Ability to comply with all study-related procedures, medications, and evaluations
  • Effective use of birth control measures.

Exclusion Criteria:

  • Iron overload not due to hereditary hemochromatosis
  • Males with hemoglobin <13 mg/dL, females with hemoglobin <12 mg/dL
  • Desferal treatment within 1 month of the screening visit
  • Patients currently or previously treated with deferiprone or deferasirox
  • Significant medical condition interfering with the ability to partake in this study
  • Presence of a surgical or medical condition that might significantly alter the absorption, distribution, metabolism or excretion of any study drug
  • Clinical evidence of Active Hepatitis B or C
  • Positive HIV serology
  • Pregnant or breast feeding patients
  • Patients treated with systemic investigational drug within 4 weeks prior or with topical investigational drug within 7 days prior to the screening visit

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 ClinicalTrials.gov identifier (NCT number): NCT00395629


Locations
United States, California
UC Irvine/Long Beach
Long Beach, California, United States, 90822
United States, Connecticut
University of Connecticut Health Center
Farmington, Connecticut, United States, 06030
United States, Michigan
Henry Ford Health System
Detroit, Michigan, United States, 48202
United States, Minnesota
Mayo Clinic
Rochester, Minnesota, United States, 55905
United States, Missouri
St. Louis University
St. Louis, Missouri, United States, 63110
United States, New York
Rochester General Hospital
Rochester, New York, United States, 14625
United States, North Carolina
Carolinas Medical Center
Charlotte, North Carolina, United States, 28203
United States, Ohio
Ohio State University Medical Center
Columbus, Ohio, United States, 43210
United States, South Carolina
Three Medical Park
Columbia, South Carolina, United States, 29203
Australia
Novartis Investigative Site
Brisbane, Australia
Canada, Ontario
Novartis Investigative Site
London, Ontario, Canada
France
Novartis Investigative Site
Rennes Cedex, France
Germany
Novartis Investigative Site
Chemnitz, Germany
Novartis Investigative Site
Heidelberg, Germany
Novartis Investigative Site
Magdeburg, Germany
Novartis Investigative Site
Oberhausen, Germany
Italy
Novartis Investigative Site
Modena, Italy
Novartis Investigative Site
Monza, Italy
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
Study Director: Novartis Pharmaceuticals, M.D. Novartis Pharmaceuticals

Additional Information:
Responsible Party: External Affairs, Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT00395629     History of Changes
Other Study ID Numbers: CICL670A2202
EudraCT no. 2006-002102-57 ( Registry Identifier: EudraCT )
First Posted: November 3, 2006    Key Record Dates
Results First Posted: May 18, 2011
Last Update Posted: May 30, 2011
Last Verified: May 2011

Keywords provided by Novartis:
Deferasirox
ICL670A
Iron chelators
Deferiprone
Transfusional Hemochromatosis

Additional relevant MeSH terms:
Iron Overload
Hemochromatosis
Iron Metabolism Disorders
Metabolic Diseases
Metal Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Deferasirox
Iron Chelating Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action