A phase I/II open label, dose escalation trial to explore the safety and efficacy of ICL670 in patients with iron overload resulting from hereditary hemochromatosis

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 48

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

• Iron overload not due to hereditary hemochromatosis
• History of blood transfusion during the 6 months prior to study entry
• Males with hemoglobin <13 mg/dL, females with hemoglobin <12 mg/dL
• Treatment with phlebotomy within 2 weeks of screening visit
• Desferal treatment within 1 month of the screening visit
• Patients currently or previously treated with deferiprone or deferasirox (ICL670)
• Patients with serum creatinine > 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)

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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<br>• To characterize the pharmacokinetics of ICL670 in patients with hereditary hemochromatosis<br>;Primary end point(s): • Incidence and severity of adverse events

Secondary Outcome Measures

Name	Time	Method

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