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

May 24, 2011 updated by: Novartis Pharmaceuticals

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.

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

49

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

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

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (ADULT, OLDER_ADULT)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NON_RANDOMIZED
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
EXPERIMENTAL: ICL670 (Deferasirox)
Three dose cohorts: 5 mg/kg/day, 10 mg/kg/day, 15 mg/kg/day
Drug: Deferasirox (ICL670)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
0 to 48 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
4, 8, 12, 16, 20, and 24 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Novartis Pharmaceuticals

Investigators

  • Study Director: Novartis Pharmaceuticals, M.D., Novartis Pharmaceuticals

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

August 1, 2006

Primary Completion (ACTUAL)

December 1, 2007

Study Completion (ACTUAL)

March 1, 2009

Study Registration Dates

First Submitted

November 1, 2006

First Submitted That Met QC Criteria

November 2, 2006

First Posted (ESTIMATE)

November 3, 2006

Study Record Updates

Last Update Posted (ESTIMATE)

May 30, 2011

Last Update Submitted That Met QC Criteria

May 24, 2011

Last Verified

May 1, 2011

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CICL670A2202
  • EudraCT no. 2006-002102-57 (REGISTRY: EudraCT)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Iron Overload

Clinical Trials on Deferasirox (ICL670)

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Norway

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe