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

Study Overview
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Brief Summary
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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.

Official Title
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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.

Conditions
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Iron Overload, Hereditary Hemochromatosis

Intervention / Treatment
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* Drug: Deferasirox (ICL670)


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: ICL670 (Deferasirox)<br>Three dose cohorts: 5 mg/kg/day, 10 mg/kg/day, 15 mg/kg/day | Drug: Deferasirox (ICL670)<br> <br> |

What is the study measuring?
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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) | 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) | 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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Deferasirox, ICL670A, Iron chelators, Deferiprone, Transfusional Hemochromatosis