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

Sponsored by:	Novartis
Information provided by:	Novartis
ClinicalTrials.gov Identifier:	NCT00395629

Purpose

Brief Summary This study is being carried out in order to test a new oral treatment for iron overload in patients with Hereditary Hemochromatosis. The study is also being carried out to find out if this medication is tolerable, that is, it doesn't have a lot of side effects.

Condition	Intervention	Phase
Hereditary Hemochromatosis	Drug: deferasirox	Phase II

Genetics Home Reference related topics: hemochromatosis

MedlinePlus related topics: Hemochromatosis

Drug Information available for: Deferasirox

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study
Official Title:	A Phase I/II Open Label, Dose-Escalation Trial to Explore the Safety and Efficacy of ICL670 in Patients With Iron Overload Resulting From Hereditary Hemochromatosis

Further study details as provided by Novartis:

Primary Outcome Measures:

Incidence and severity of adverse events

Secondary Outcome Measures:

Efficacy based on serum ferritin
Pharmacokinetic parameters (PK) of deferasirox

Estimated Enrollment:	40
Study Start Date:	September 2006
Estimated Study Completion Date:	January 2008

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
Deferoxamine mesylate 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
Active Hepatitis
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

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, 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
Francisco Gonzales, M.D., PA
Columbia, South Carolina, United States, 29203
Australia

Herston, Australia
Canada, Ontario

London, Ontario, Canada
France

Rennes Cedex, France
Germany

Magdeburg, Germany

Oberhausen, Germany

Heidleberg, Germany

Chemnitz, Germany
Italy

Modena, Italy

Milano, Italy

Sponsors and Collaborators

Novartis

Investigators

Principal Investigator:

Prad Phatak, M.D.

Rochester General Hospital

More Information

Study ID Numbers:	CICL670A2202
Study First Received:	November 1, 2006
Last Updated:	May 29, 2008
ClinicalTrials.gov Identifier:	NCT00395629
Health Authority:	United States: Institutional Review Board; Germany: Federal Institute for Drugs and Medical Devices; France: Safety and health products agency; Italy: Italian Agency of Medicine

Keywords provided by Novartis:

Hemochromatosis, iron overload, ICL670, deferasirox

Study placed in the following topic categories:

Metabolism, Inborn Errors
Metabolic Diseases
Genetic Diseases, Inborn
Deferasirox
Hemochromatosis, type 3

Hemochromatosis
Iron Metabolism Disorders
Iron Overload
Metabolic disorder
Iron

Additional relevant MeSH terms:

Molecular Mechanisms of Pharmacological Action
Iron Chelating Agents
Chelating Agents
Metal Metabolism, Inborn Errors
Pharmacologic Actions

ClinicalTrials.gov processed this record on January 16, 2009