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

Phase 1

Completed

• Conditions: Iron Overload; Hereditary Hemochromatosis
• Interventions: Drug: Deferasirox (ICL670)

• Registration Number: NCT00395629
• Lead Sponsor: Novartis Pharmaceuticals

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2006-08
• Study First Submitted: 2006-11-01
• Study First Submitted QC: 2006-11-02
• Study First Posted: 2006-11-03
• Primary Completion: 2007-12
• Study Completion: 2009-03
• Results First Submitted: 2010-12-06
• Results First Submitted QC: 2011-04-19
• Status Verified: 2011-05
• Results First Posted: 2011-05-18
• Last Update Submitted: 2011-05-24
• Last Update Posted: 2011-05-30

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 49

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

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
ICL670 (Deferasirox)	Deferasirox (ICL670)	Three dose cohorts: 5 mg/kg/day, 10 mg/kg/day, 15 mg/kg/day

Primary Outcome Measures

Name	Time	Method
Absolute Change of Serum Ferritin From Baseline to the End of Extension, by Dose Cohort (Extension Per-protocol Population)	0 to 48 weeks	Mean absolute change in serum ferritin from baseline to the end of the extension study.

Secondary Outcome Measures

Name	Time	Method
Trough Concentrations of Deferasirox (ICL670), by Dose Cohort (Per-protocol Population)	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.

Trial Locations

Locations (10)

Three Medical Park; 🇺🇸 Columbia, South Carolina, United States

Rochester General Hospital; 🇺🇸 Rochester, New York, United States

Ohio State University Medical Center; 🇺🇸 Columbus, Ohio, United States

Carolinas Medical Center; 🇺🇸 Charlotte, North Carolina, United States

St. Louis University; 🇺🇸 St. Louis, Missouri, United States

Novartis Investigative Site; 🇮🇹 Monza, Italy

UC Irvine/Long Beach; 🇺🇸 Long Beach, California, United States

University of Connecticut Health Center; 🇺🇸 Farmington, Connecticut, United States

Henry Ford Health System; 🇺🇸 Detroit, Michigan, United States

Mayo Clinic; 🇺🇸 Rochester, Minnesota, United States