Safety and Efficacy of Deferasirox (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.

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Key information

Conditions

Iron Overload Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hemochromatosis Hereditary Hemochromatosis Iron Metabolism Disorders Metabolic Diseases Metabolism, Inborn Errors Metal Metabolism, Inborn Errors Nutritional and Metabolic Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Novartis Investigative Site, Brisbane, Australia

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Deferasirox (ICL670)

Drug

Primary outcomes

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.

Secondary outcomes

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.

Sponsors and collaborators

Lead sponsor

Novartis Pharmaceuticals

Industry

Registry information

Official study 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.

Important dates

Study start: 2006
Primary completion: 2007
Study completion: 2009
First posted: Nov 3, 2006
Registry last updated: May 30, 2011

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed