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

Primary Purpose

Iron Overload, Hereditary Hemochromatosis

Status
Completed
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
Deferasirox (ICL670)
Sponsored by
Novartis Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Iron Overload focused on measuring Deferasirox, ICL670A, Iron chelators, Deferiprone, Transfusional Hemochromatosis

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • UC Irvine/Long Beach
  • University of Connecticut Health Center
  • Henry Ford Health System
  • Mayo Clinic
  • St. Louis University
  • Rochester General Hospital
  • Carolinas Medical Center
  • Ohio State University Medical Center
  • Three Medical Park
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

ICL670 (Deferasirox)

Arm Description

Three dose cohorts: 5 mg/kg/day, 10 mg/kg/day, 15 mg/kg/day

Outcomes

Primary Outcome Measures

Absolute Change of Serum Ferritin From Baseline to the End of Extension, by Dose Cohort (Extension Per-protocol Population)
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)
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.

Full Information

First Posted
November 1, 2006
Last Updated
May 24, 2011
Sponsor
Novartis Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT00395629
Brief Title
Safety and Efficacy of Deferasirox (ICL670) in Patients With Iron Overload Resulting From Hereditary Hemochromatosis
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 Type
Interventional

2. Study Status

Record Verification Date
May 2011
Overall Recruitment Status
Completed
Study Start Date
August 2006 (undefined)
Primary Completion Date
December 2007 (Actual)
Study Completion Date
March 2009 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor
Novartis Pharmaceuticals

4. Oversight

Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Iron Overload, Hereditary Hemochromatosis
Keywords
Deferasirox, ICL670A, Iron chelators, Deferiprone, Transfusional Hemochromatosis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
49 (Actual)

8. Arms, Groups, and Interventions

Arm Title
ICL670 (Deferasirox)
Arm Type
Experimental
Arm Description
Three dose cohorts: 5 mg/kg/day, 10 mg/kg/day, 15 mg/kg/day
Intervention Type
Drug
Intervention Name(s)
Deferasirox (ICL670)
Primary Outcome Measure Information:
Title
Absolute Change of Serum Ferritin From Baseline to the End of Extension, by Dose Cohort (Extension Per-protocol Population)
Description
Mean absolute change in serum ferritin from baseline to the end of the extension study.
Time Frame
0 to 48 weeks
Secondary Outcome Measure Information:
Title
Trough Concentrations of Deferasirox (ICL670), by Dose Cohort (Per-protocol Population)
Description
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.
Time Frame
4, 8, 12, 16, 20, and 24 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Novartis Pharmaceuticals, M.D.
Organizational Affiliation
Novartis Pharmaceuticals
Official's Role
Study Director
Facility Information:
Facility Name
UC Irvine/Long Beach
City
Long Beach
State/Province
California
ZIP/Postal Code
90822
Country
United States
Facility Name
University of Connecticut Health Center
City
Farmington
State/Province
Connecticut
ZIP/Postal Code
06030
Country
United States
Facility Name
Henry Ford Health System
City
Detroit
State/Province
Michigan
ZIP/Postal Code
48202
Country
United States
Facility Name
Mayo Clinic
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States
Facility Name
St. Louis University
City
St. Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
Rochester General Hospital
City
Rochester
State/Province
New York
ZIP/Postal Code
14625
Country
United States
Facility Name
Carolinas Medical Center
City
Charlotte
State/Province
North Carolina
ZIP/Postal Code
28203
Country
United States
Facility Name
Ohio State University Medical Center
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43210
Country
United States
Facility Name
Three Medical Park
City
Columbia
State/Province
South Carolina
ZIP/Postal Code
29203
Country
United States
Facility Name
Novartis Investigative Site
City
Brisbane
Country
Australia
Facility Name
Novartis Investigative Site
City
London
State/Province
Ontario
Country
Canada
Facility Name
Novartis Investigative Site
City
Rennes Cedex
Country
France
Facility Name
Novartis Investigative Site
City
Chemnitz
Country
Germany
Facility Name
Novartis Investigative Site
City
Heidelberg
Country
Germany
Facility Name
Novartis Investigative Site
City
Magdeburg
Country
Germany
Facility Name
Novartis Investigative Site
City
Oberhausen
Country
Germany
Facility Name
Novartis Investigative Site
City
Modena
Country
Italy
Facility Name
Novartis Investigative Site
City
Monza
Country
Italy

12. IPD Sharing Statement

Links:
URL
http://www.novartisclinicaltrials.com/
Description
Related Info

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

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