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 >= 300?g/L and <= 1500 ?g/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 <14 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 >= 3x 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 ? History of drug or alcohol abuse within the 12 months prior to enrollment ? 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 who are found to be ineligible after screening procedures will have this documented in the case report form (CRF). These patients will not need to complete the end of study assessments.

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 ? To characterize the pharmacokinetics of ICL670 in patients with hereditary hemochromatosis;Primary end point(s): Primary ? Incidence and severity of adverse events

Secondary Outcome Measures

Name	Time	Method

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Updated 7/10/2015