Primary endpoint
tumor, death, congenital anomaly, birth defect, infections, aseptic meningitis, disability or incapacity, allergic reactions, Cancer
ADVERSE EVENT, TUMOR, ALLERGIC REACTION, DISEASE COURSE, OBSTRUCTIVE HYDROCEPHALUS, INFECTIONS, ASEPTIC MENINGITIS, CANCER
To address the primary objective, we considered short-term (0–24 hours), mid-term (day 1 to day 14) and long-term (day 15 to week 96) safety endpoints. The short-term and mid-term safety endpoints aimed to exclude risks related to the procedure of cell injection, such as aseptic meningitis or obstructive hydrocephalus, and allergic reactions to the medicinal product. Long-term safety endpoints had the primarily purpose of excluding the presence of infections due the possible non-sterility of the cell product (a risk minimized by the extensive in vitro examinations), tumor formation (risk minimized by the in vivo evidence obtained by infusion of supra maximal doses of cells in immunosuppressed mice) as well as changes in neurological status that would not be expected based on the disease course. The documented AE severity was classified according the Common Terminology Criteria for Adverse Events (CTCAE) (grade 1=mild; grade 2=moderate; grade 3=severe; grade 4=life-threatening; grade 5=death), and the attribution to the investigational medicinal product (unrelated, unlikely, possibly, probably, definitely) was defined accordingly to National Cancer Institute guidelines of AE reporting requirements. SAE was defined as any untoward expected medical occurrence or effect that, at any dose, results in death, that is life-threatening, that requires hospitalization or prolongation of existing hospitalization, that results in persistent or significant disability or incapacity or that is a congenital anomaly or birth defect. The safety profile of 