Statistical analysis and sample size
In total, 24 participants were included, with nine participants in the dose escalation arm to assess safety (part A). The randomised part B (2:1) included ten participants treated with one of two batches of ProTrans and five participants who received the placebo control. For calculation of participant numbers, part B was considered to contain three randomised groups (placebo, batch 1 ProTrans and batch 2 ProTrans), and not to include any data from part A. 