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

Inclusion Criteria: * Patients had to be at risk of life-threatening complications due to transfusional iron overload and be unable to tolerate therapy with any of the commercially available iron chelators (mainly deferoxamine and/or deferiprone) because of documented severe toxicity. * Patients with a degree of iron overload which was not immediately life-threatening and who were ineligible for other trials with ICL670 could also be enrolled providing they had a well-documented, sound justification for alternative chelation therapy. * Serum ferritin ≥ 8000 μg/L. * Serum ferritin \< 8000μg/L and LIC of ≥ 7 mg Fe/g dry weight. * Patients for whom ≥ 8 blood transfusions per year were required in order to maintain the Hemoglobin level at \> 9 g/dL. * Female patients who have reached menarche and who were sexually active had to use double barrier contraception (oral plus barrier contraception), or had to have undergone total hysterectomy and/or ovariectomy, or tubal ligation. * Written, voluntary informed consent. 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.