Fig. 1.

Trial design. *Children aged < 4 years may be included only after confirmation by the Independent Data Monitoring Committee (IDMC) upon review of the safety and bioanalytical data available when at least six children aged 4–11 years, plus at least the same number of older children and adults have completed days 45 and 90. †The unblinded interim analysis for sample size re-estimation will take place when approximately 50% of patients have completed day 45 ± 7. Depending on the results of the sample size re-estimation, the IDMC will recommend continuing with the initial sample size, increasing the sample size, or stopping the study for futility. DEB dystrophic epidermolysis bullosa, JEB junctional epidermolysis bullosa, KS Kindler syndrome, SoC standard of care