A Protocol to Allow Treatment With ICL670 for Patients With or at Risk of Life-threatening Complications of Transfusional Iron Overload Who Are Unable to Tolerate Other Iron Chelators Because of Documented Severe Toxicity

Exclusion Criteria

• •Patients with transfusional iron overload who were not experiencing severe toxicities during therapy with other iron chelators (e.g. deferoxamine and/or deferiprone).
• •Patients with non-transfusional hemosiderosis.
• •Patients with severe liver failure as defined by a score of ≥ 10 points on the Child-Pugh scale.
• •Patients with serum creatinine 1.5 times the upper limit of normal (ULN) at screening.
• •Patients with a history of nephrotic syndrome.
• •Patients with a diagnosis of clinically relevant cataract or a previous history of clinically relevant ocular toxicity related to iron chelation therapy.
• •Patients with severe systemic diseases unrelated to iron overload and which would prevent them from undergoing treatment with ICL670.
• •Patients with psychiatric or addictive disorders which prevent them from giving informed consent or undergoing treatment with ICL670.
• •Pregnant or breast feeding patients.
• •Patients treated with systemic investigational drugs within the past four weeks or topical investigational drugs within the past seven days.
• •Any surgical or medical condition which might significantly alter the absorption or excretion of drugs as shown by evidence of any of the following:
• •* History of inflammatory bowel disease
• •* History of major gastrointestinal tract surgery such as gastrectomy, gastroenterostomy, or bowel resection
• •* History of pancreatic injury or pancreatitis; indication of impaired pancreatic function/injury as indicated by abnormal lipase or amylase
• •Patients being considered by the investigator as potentially unreliable and/or not cooperative with regard to the protocol.
• •History of drug or alcohol abuse within the 12 months prior to dosing.