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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT00395629




Registration number
NCT00395629
Ethics application status
Date submitted
1/11/2006
Date registered
3/11/2006
Date last updated
30/05/2011

Titles & IDs
Public title
Safety and Efficacy of Deferasirox (ICL670) in Patients With Iron Overload Resulting From Hereditary Hemochromatosis
Scientific 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.
Secondary ID [1] 0 0
EudraCT no. 2006-002102-57
Secondary ID [2] 0 0
CICL670A2202
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Iron Overload 0 0
Hereditary Hemochromatosis 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders
Diet and Nutrition 0 0 0 0
Other diet and nutrition disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Deferasirox (ICL670)

Experimental: ICL670 (Deferasirox) - Three dose cohorts: 5 mg/kg/day, 10 mg/kg/day, 15 mg/kg/day


Treatment: Drugs: Deferasirox (ICL670)
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Absolute Change of Serum Ferritin From Baseline to the End of Extension, by Dose Cohort (Extension Per-protocol Population)
Timepoint [1] 0 0
0 to 48 weeks
Secondary outcome [1] 0 0
Trough Concentrations of Deferasirox (ICL670), by Dose Cohort (Per-protocol Population)
Timepoint [1] 0 0
4, 8, 12, 16, 20, and 24 weeks

Eligibility
Key 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.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 1/Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/08/2006
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/03/2009
Sample size
Target
Accrual to date
Final
49
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Novartis Investigative Site - Brisbane
Recruitment postcode(s) [1] 0 0
- Brisbane
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Connecticut
Country [3] 0 0
United States of America
State/province [3] 0 0
Michigan
Country [4] 0 0
United States of America
State/province [4] 0 0
Minnesota
Country [5] 0 0
United States of America
State/province [5] 0 0
Missouri
Country [6] 0 0
United States of America
State/province [6] 0 0
New York
Country [7] 0 0
United States of America
State/province [7] 0 0
North Carolina
Country [8] 0 0
United States of America
State/province [8] 0 0
Ohio
Country [9] 0 0
United States of America
State/province [9] 0 0
South Carolina
Country [10] 0 0
Canada
State/province [10] 0 0
Ontario
Country [11] 0 0
France
State/province [11] 0 0
Rennes Cedex
Country [12] 0 0
Germany
State/province [12] 0 0
Chemnitz
Country [13] 0 0
Germany
State/province [13] 0 0
Heidelberg
Country [14] 0 0
Germany
State/province [14] 0 0
Magdeburg
Country [15] 0 0
Germany
State/province [15] 0 0
Oberhausen
Country [16] 0 0
Italy
State/province [16] 0 0
Modena
Country [17] 0 0
Italy
State/province [17] 0 0
Monza

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Novartis Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT00395629
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Novartis Pharmaceuticals, M.D.
Address 0 0
Novartis Pharmaceuticals
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT00395629