Clinical trial NCT00395629: Safety and Efficacy of Deferasirox (ICL670) in Patients With Iron Overload Resulting From Hereditary Hemochromatosis
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.
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Iron Overload
Hereditary Hemochromatosis
Keywords: Deferasirox
ICL670A
Iron chelators
Deferiprone
Transfusional Hemochromatosis
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2006-11-03
Enrollment: 49
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility 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.

Primary outcomes:
measure: Absolute Change of Serum Ferritin From Baseline to the End of Extension, by Dose Cohort (Extension Per-protocol Population)
timeFrame: 0 to 48 weeks
Secondary outcomes:
measure: Trough Concentrations of Deferasirox (ICL670), by Dose Cohort (Per-protocol Population)
timeFrame: 4, 8, 12, 16, 20, and 24 weeks
Other outcomes:
Locations:
city: Long Beach
country: United States
facility: UC Irvine/Long Beach
state: California
city: Farmington
country: United States
facility: University of Connecticut Health Center
state: Connecticut
city: Detroit
country: United States
facility: Henry Ford Health System
state: Michigan
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
city: St. Louis
country: United States
facility: St. Louis University
state: Missouri
city: Rochester
country: United States
facility: Rochester General Hospital
state: New York
city: Charlotte
country: United States
facility: Carolinas Medical Center
state: North Carolina
city: Columbus
country: United States
facility: Ohio State University Medical Center
state: Ohio
city: Columbia
country: United States
facility: Three Medical Park
state: South Carolina
city: Brisbane
country: Australia
facility: Novartis Investigative Site
city: London
country: Canada
facility: Novartis Investigative Site
state: Ontario
city: Rennes Cedex
country: France
facility: Novartis Investigative Site
city: Chemnitz
country: Germany
facility: Novartis Investigative Site
city: Heidelberg
country: Germany
facility: Novartis Investigative Site
city: Magdeburg
country: Germany
facility: Novartis Investigative Site
city: Oberhausen
country: Germany
facility: Novartis Investigative Site
city: Modena
country: Italy
facility: Novartis Investigative Site
city: Monza
country: Italy
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals, M.D.
role: STUDY_DIRECTOR
References: