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

This study has been completed.
Sponsor:
Information provided by:
Novartis
ClinicalTrials.gov Identifier:
NCT00395629
First received: November 1, 2006
Last updated: May 24, 2011
Last verified: May 2011
  Purpose

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
Endpoint Classification: Safety/Efficacy Study
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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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)

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
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.

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: 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
  More Information

Additional Information:
No publications provided

Responsible Party: External Affairs, Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT00395629     History of Changes
Other Study ID Numbers: CICL670A2202, EudraCT no. 2006-002102-57
Study First Received: November 1, 2006
Results First Received: December 6, 2010
Last Updated: May 24, 2011
Health Authority: United States: Food and Drug Administration
Canada: Health Canada
Australia: Department of Health and Ageing Therapeutic Goods Administration
Germany: Federal Institute for Drugs and Medical Devices
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
Italy: Ministry of Health

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

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

ClinicalTrials.gov processed this record on November 25, 2014