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

NCT00395629 | Completed Phase 1 Results

• 2 other identifiers: CICL670A2202EudraCT no. 2006-002102-57
• Study Type: interventional
• Target: 49
• Locations: 6 countries
• Sites: 18

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.

Conditions

Iron Overload; Hereditary Hemochromatosis

Keywords

Deferasirox; ICL670A; Iron chelators; Deferiprone; Transfusional Hemochromatosis

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

• Trough Concentrations of Deferasirox (ICL670), by Dose Cohort (Per-protocol Population)

  4, 8, 12, 16, 20, and 24 weeks

Study Arms (1)

ICL670 (Deferasirox)

EXPERIMENTAL

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

Drug: Deferasirox (ICL670)

Interventions

Deferasirox (ICL670) DRUG

ICL670 (Deferasirox)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (18)

UC Irvine/Long Beach

Long Beach, California, 90822, United States

Location

University of Connecticut Health Center

Farmington, Connecticut, 06030, United States

Location

Henry Ford Health System

Detroit, Michigan, 48202, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

St. Louis University

St Louis, Missouri, 63110, United States

Location

Rochester General Hospital

Rochester, New York, 14625, United States

Location

Carolinas Medical Center

Charlotte, North Carolina, 28203, United States

Location

Ohio State University Medical Center

Columbus, Ohio, 43210, United States

Location

Three Medical Park

Columbia, South Carolina, 29203, United States

Location

Novartis Investigative Site

Brisbane, Australia

Location

Novartis Investigative Site

London, Ontario, Canada

Location

Novartis Investigative Site

Rennes, France

Location

Novartis Investigative Site

Chemnitz, Germany

Location

Novartis Investigative Site

Heidelberg, Germany

Location

Novartis Investigative Site

Magdeburg, Germany

Location

Novartis Investigative Site

Oberhausen, Germany

Location

Novartis Investigative Site

Modena, Italy

Location

Novartis Investigative Site

Monza, Italy

Location

Related Links

• Related Info

MeSH Terms

Conditions

Iron Overload; Hemochromatosis

Interventions

Deferasirox

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Benzoates; Acids, Carbocyclic; Carboxylic Acids; Organic Chemicals; Benzene Derivatives; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Triazoles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Results Point of Contact

• Title: Study Director
• Organization: Novartis Pharmaceuticals

862-778-8300

Study Officials

• Novartis Pharmaceuticals, M.D.

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY

Study Record Dates

• First Submitted: November 1, 2006
• First Posted: November 3, 2006
• Study Start: August 1, 2006
• Primary Completion: December 1, 2007
• Study Completion: March 1, 2009
• Last Updated: May 30, 2011
• Results First Posted: May 18, 2011

Record last verified: 2011-05

Locations

DE (4); IT (2); AU (1); FR (1); CA (1); US (9)