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

This study has been completed.
Information provided by:
Novartis Identifier:
First received: November 1, 2006
Last updated: May 24, 2011
Last verified: May 2011
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 Intervention Phase
Iron Overload
Hereditary Hemochromatosis
Drug: Deferasirox (ICL670)
Phase 1
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: 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.

Resource links provided by NLM:

Further study details as provided by Novartis:

Primary Outcome Measures:
  • 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:
  • 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.

Enrollment: 49
Study Start Date: August 2006
Study Completion Date: March 2009
Primary Completion Date: December 2007 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: ICL670 (Deferasirox)
Three dose cohorts: 5 mg/kg/day, 10 mg/kg/day, 15 mg/kg/day
Drug: Deferasirox (ICL670)


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

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.

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Please refer to this study by its identifier: NCT00395629

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
Novartis Investigative Site
Brisbane, Australia
Canada, Ontario
Novartis Investigative Site
London, Ontario, Canada
Novartis Investigative Site
Rennes Cedex, France
Novartis Investigative Site
Chemnitz, Germany
Novartis Investigative Site
Heidelberg, Germany
Novartis Investigative Site
Magdeburg, Germany
Novartis Investigative Site
Oberhausen, Germany
Novartis Investigative Site
Modena, Italy
Novartis Investigative Site
Monza, Italy
Sponsors and Collaborators
Novartis Pharmaceuticals
Study Director: Novartis Pharmaceuticals, M.D. Novartis Pharmaceuticals
  More Information

Additional Information:
Responsible Party: External Affairs, Novartis Pharmaceuticals Identifier: NCT00395629     History of Changes
Other Study ID Numbers: CICL670A2202
EudraCT no. 2006-002102-57 ( Registry Identifier: EudraCT )
Study First Received: November 1, 2006
Results First Received: December 6, 2010
Last Updated: May 24, 2011

Keywords provided by Novartis:
Iron chelators
Transfusional Hemochromatosis

Additional relevant MeSH terms:
Iron Overload
Iron Metabolism Disorders
Metabolic Diseases
Metal Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Iron Chelating Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action processed this record on April 28, 2017