Phase 2
N=49
Safety and Efficacy of Deferasirox (ICL670) in Patients With Iron Overload Resulting From Hereditary Hemochromatosis
Iron Overload · Hereditary Hemochromatosis
Bottom Line
View on ClinicalTrials.gov: NCT00395629 ↗Enrolled (actual)
49
Serious AEs
1.3%
Results posted
May 2011
Primary outcome: Primary: Absolute Change of Serum Ferritin From Baseline to the End of Extension, by Dose Cohort (Extension Per-protocol Population) — -389.1; -610.5; -571.6 µg/L
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Deferasirox (ICL670) (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Novartis Pharmaceuticals
- Primary completion
- Dec 2007
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Absolute Change of Serum Ferritin From Baseline to the End of Extension, by Dose Cohort (Extension Per-protocol Population) |
-389.1; -610.5; -571.6 | — |
| SECONDARY Trough Concentrations of Deferasirox (ICL670), by Dose Cohort (Per-protocol Population) |
9.63; 19.62; 30.48; 8.86; 23.88; 27.06 | — |
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.
Data sourced from ClinicalTrials.gov (NCT00395629). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.