hereditary hemochromatosis MedDRA version: 8.1 Level: LLT Classification code 10057874 Term: Hereditary hemochromatosis
Conditions
Interventions
Product Name: EXJADE
Product Code: ICL670
Pharmaceutical Form: Dispersible tablet
INN or Proposed INN: deferasirox
CAS Number: 201530-41-8
Current Sponsor code: ICL670
Concentration unit: mg milligram
Sponsors
Novartis Pharma Services AG
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • Age = 18 years • Male or female patients homozygous for the C282Y mutation documented by molecular diagnostic testing. • Serum ferritin value = 300µg/L and = 2000 µg/L with a transferrin saturation = 45%. • No known allergy or contraindication to the administration of deferasirox • Ability to comply with all study-related procedures, medications, and evaluations • Sexually active pre-menopausal female patients must use double-barrier contraception, oral contraceptive plus barrier contraceptive, or must have undergone clinically documented total hysterectomy and/or oophorectomy, tubal ligation. Postmenopausal is defined by amenorrhea for at least 12 months Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: • Iron overload not due to hereditary hemochromatosis • History of blood transfusion during the 6 months prior to study entry • Males with hemoglobin upper limit of normal • Patients with ALT = 2x ULN at screening • 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 (positive HBsAg with negative HBsAb) or hepatitis C (positive HCV antibody and detectable HCV RNA with ALT above the normal range) • Positive HIV serology • Patients with active inflammatory diseases that may interfere with the accurate measurement of serum ferritin • Patients with a diagnosis of a clinically relevant cataract or a previous history of clinically relevant ocular toxicity related to iron chelation • Illicit drug use and/or alcohol use, defined as no more than one drink a day for women, and no more than two drinks a day for men within the 12 months prior to enrolement. A standard drink is generally considered to be 12 ounces of beer, 5 ounces of wine, or 1.5 ounces of 80-proof distilled spirits • 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 • Patients with a known diagnosis of cirrhosis (confirmed by biopsy if available)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To explore the safety of ICL670 (dose range 5 to 20 mg/kg PO per day) in adult hemochromatosis patients homozygous for the C282Y mutation, with iron overload;Secondary Objective: • To explore the effect of ICL670 on serum ferritin • To characterize the pharmacokinetics of ICL670 in patients with hereditary hemochromatosis ;Primary end point(s): • Incidence and severity of adverse events | — |
Countries
France, Germany, Italy
Outcome results
None listed