Trial design
ADVERSE EVENTS
This was a Phase I/II single-site study, sponsored by NextCell Pharma, Stockholm, Sweden, and conducted at Karolinska University Hospital, Stockholm, Sweden. The study protocol was published in advance [Randomisation was performed with a web-based randomisation system with a randomisation code created prior to the start of the study. The randomisation was made in blocks, with participants randomised to batch 1 ProTrans, batch 2 ProTrans or placebo treatment. Randomisation envelopes were kept at the clinic in a locked room, with study staff opening a randomisation envelope at the baseline visit.The primary safety outcome was evaluated through the registration of adverse events (AEs). The primary efficacy variable was the comparison of the intervention (ProTrans) vs placebo as measured by the ∆-change in C-peptide AUC (0–120 min) for a mixed meal tolerance test (MMTT) at day 372 following ProTrans/placebo infusion when compared with pretreatment. Secondary outcome measures assessed the number of insulin-independent participants and the number of participants with daily insulin needs <0.25 U/kg, insulin requirement/kg body weight, HbA