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

Iron Overload Clinical Trial

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.

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00395629
• Other study ID #: CICL670A2202
• Secondary ID: EudraCT no. 2006
• Status: Completed
• Phase: Phase 1/Phase 2
• First received: November 1, 2006
• Last updated: May 24, 2011
• Start date: August 2006
• Est. completion date: March 2009

Study information

• Verified date: May 2011
• Source: Novartis
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug AdministrationCanada: Health CanadaAustralia: Department of Health and Ageing Therapeutic Goods AdministrationGermany: Federal Institute for Drugs and Medical DevicesFrance: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)Italy: Ministry of Health
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 49
• Est. completion date: March 2009
• Est. primary completion date: December 2007
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

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 Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Hemochromatosis
• Hereditary Hemochromatosis
• Iron Overload

Intervention

Drug:
• Deferasirox (ICL670)

Locations

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

Sponsors (1)

Lead Sponsor	Collaborator
Novartis Pharmaceuticals

Countries where clinical trial is conducted

United States,  Australia,  Canada,  France,  Germany,  Italy, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	0 to 48 weeks	No
Secondary	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.	4, 8, 12, 16, 20, and 24 weeks	No

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