Trial record 4 of 32 for:
hemochromatosis
Safety and Efficacy of Deferasirox (ICL670) in Patients With Iron Overload Resulting From Hereditary Hemochromatosis
This study has been completed.
Sponsor:
Novartis Pharmaceuticals
Information provided by:
Novartis
ClinicalTrials.gov Identifier:
NCT00395629
First received: November 1, 2006
Last updated: May 24, 2011
Last verified: May 2011
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Purpose
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.
| Condition | Intervention | Phase |
|---|---|---|
|
Iron Overload Hereditary Hemochromatosis |
Drug: Deferasirox (ICL670) |
Phase 1 Phase 2 |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| 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. |
Resource links provided by NLM:
Genetics Home Reference related topics:
hemochromatosis
Drug Information available for:
Deferasirox
U.S. FDA Resources
Further study details as provided by Novartis:
Primary Outcome Measures:
- 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 ] [ Designated as safety issue: No ]Mean absolute change in serum ferritin from baseline to the end of the extension study.
Secondary Outcome Measures:
- Trough Concentrations of Deferasirox (ICL670), by Dose Cohort (Per-protocol Population) [ Time Frame: 4, 8, 12, 16, 20, and 24 weeks ] [ Designated as safety issue: No ]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.
| Enrollment: | 49 |
| Study Start Date: | August 2006 |
| Study Completion Date: | March 2009 |
| Primary Completion Date: | December 2007 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Experimental: ICL670 (Deferasirox)
Three dose cohorts: 5 mg/kg/day, 10 mg/kg/day, 15 mg/kg/day
|
Drug: Deferasirox (ICL670) |
Eligibility| Ages Eligible for Study: | 18 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
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.
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00395629
Locations
| United States, California | |
| UC Irvine/Long Beach | |
| Long Beach, California, United States, 90822 | |
| United States, Connecticut | |
| University of Connecticut Health Center | |
| Farmington, Connecticut, United States, 06030 | |
| United States, Michigan | |
| Henry Ford Health System | |
| Detroit, Michigan, United States, 48202 | |
| United States, Minnesota | |
| Mayo Clinic | |
| Rochester, Minnesota, United States, 55905 | |
| United States, Missouri | |
| St. Louis University | |
| St. Louis, Missouri, United States, 63110 | |
| United States, New York | |
| Rochester General Hospital | |
| Rochester, New York, United States, 14625 | |
| United States, North Carolina | |
| Carolinas Medical Center | |
| Charlotte, North Carolina, United States, 28203 | |
| United States, Ohio | |
| Ohio State University Medical Center | |
| Columbus, Ohio, United States, 43210 | |
| United States, South Carolina | |
| Three Medical Park | |
| Columbia, South Carolina, United States, 29203 | |
| Australia | |
| Novartis Investigative Site | |
| Brisbane, Australia | |
| Canada, Ontario | |
| Novartis Investigative Site | |
| London, Ontario, Canada | |
| France | |
| Novartis Investigative Site | |
| Rennes Cedex, France | |
| Germany | |
| Novartis Investigative Site | |
| Chemnitz, Germany | |
| Novartis Investigative Site | |
| Heidelberg, Germany | |
| Novartis Investigative Site | |
| Magdeburg, Germany | |
| Novartis Investigative Site | |
| Oberhausen, Germany | |
| Italy | |
| Novartis Investigative Site | |
| Modena, Italy | |
| Novartis Investigative Site | |
| Monza, Italy | |
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
| Study Director: | Novartis Pharmaceuticals, M.D. | Novartis Pharmaceuticals |
More Information
Additional Information:
No publications provided
| Responsible Party: | External Affairs, Novartis Pharmaceuticals |
| ClinicalTrials.gov Identifier: | NCT00395629 History of Changes |
| Other Study ID Numbers: | CICL670A2202, EudraCT no. 2006-002102-57 |
| Study First Received: | November 1, 2006 |
| Results First Received: | December 6, 2010 |
| Last Updated: | May 24, 2011 |
| Health Authority: | United States: Food and Drug Administration Canada: Health Canada Australia: Department of Health and Ageing Therapeutic Goods Administration Germany: Federal Institute for Drugs and Medical Devices France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis) Italy: Ministry of Health |
Keywords provided by Novartis:
|
Deferasirox ICL670A Iron chelators Deferiprone Transfusional Hemochromatosis |
Additional relevant MeSH terms:
|
Hemochromatosis Iron Overload Metal Metabolism, Inborn Errors Metabolism, Inborn Errors Genetic Diseases, Inborn Iron Metabolism Disorders |
Metabolic Diseases Deferasirox Iron Chelating Agents Chelating Agents Molecular Mechanisms of Pharmacological Action Pharmacologic Actions |
ClinicalTrials.gov processed this record on May 19, 2013