Kim Annink

182 Chapter 8 will be 37% and 27% in the allopurinol group. Therefore, we calculated with a two- sided X ²-test (alpha=0.05, power 80%) a sample size of 682 infants (341 per treatment group) in which the primary outcome should be ascertained. Assuming a drop-out rate of 10% for loss to follow-up, a total of 760 infants need to be enrolled. And assuming that 10% of the parents will refuse participation after the initial dose of the study drug, 846 infants have to be randomized (see Figure 2). We estimate a recruitment of about 35 patients per month in approximately 70 study centers (the recruitment of additional study sites is ongoing) and therefore recruitment will last 24 months. Data analysis All statistical analyses will be described in detail in a statistical analysis plan completed before closure of the database. Monitoring safety An independent Data Monitoring Committee (DMC) will monitor the participants’ well-being and the overall risk/benefit-ratio of the study. National monitors will monitor the accuracy and completeness of the data and the safety issues such as the presence of serious adverse events. Regulatory aspects Trial sponsor Sponsor of the ALBINO trial is the University Hospital Tuebingen, Geissweg 3, 72076 Tuebingen, Germany. Contact is available under albino@med.uni-tuebingen.de. Orphan Drug Designation The Committee for Orphan Medicinal Products (COMP) has given a positive advice to ACE Pharmaceuticals for the orphan drug designation for allopurinol sodium for treatment of perinatal asphyxia (EU/3/15/1493) and an Orphan Drug Designation has been granted by the European Medicines Agency. The public summary is available at: https://www.ema.europa.eu/documents/ orphan-designation/eu/3/15/1493-public- summary-opinion-orphan-designation-allopurinol-sodium-treatment-perinatal- asphyxia_en.pdf