4 results
Assess the efficacy and safety of eculizumab in pediatric patients with aHUS to control TMA as characterized by thrombocytopenia, hemolysis and renal impairment.
Assess the efficacy of eculizumab in adult patients with aHUS to control TMA as characterized by thrombocytopenia, hemolysis and renal impairment.
Stratum 1:To show that a non total body irradiation (TBI) containing conditioning (Flu/Thio/ivBu) results in a non inferior survival as compared to conditioning with TBI/Etoposide in children older than 4 years after HSCT from a Human leucocyte…
Primary Objective: Proof of safety and tolerability of home parenteral nutrition (HPN) with an Omega-3 fatty acid enriched MCT/LCT lipid emulsion in adult patients with chronic intestinal failure (CIF) in need of long-term HPNSecondary Objectives:…