METHODS
EP0060 was a Phase 2/3, multicentre, open‐label trial. The trial was conducted in accordance with the International Council for Harmonization Good Clinical Practice requirements, the ethical tenets that have their origin in the principles of the Declaration of Helsinki, and the local laws of the countries involved. Written informed consent was provided by the patient or their legal representative(s) before enrolment. Legal guardians were provided with a written explanation of the study design prior to providing consent for their child's participation. The study protocol, amendments, and patient informed consent were reviewed and approved by a national, regional, or Independent Ethics Committee or Institutional Review Board.Approximately 100 patients were planned for enrolment in two age‐based cohorts: cohort 1 was planned to include at least 40 patients aged ≥8 to <17 years, and cohort 2 was planned to include approximately 44 patients aged from ≥1 month to <8 years. Enrolment began with cohort 1. When the trial was initially conceived, the planned age range for cohort 2 was ≥4 to <8 years. A protocol amendment dated April 30, 2018 lowered the minimum age to 1 month based on newly available safety and efficacy data indicating no specific risk in this age group. After completion of the first 20 patients in cohort 1, an Independent Data Monitoring Committee (IDMC) reviewed the safety and tolerability data and recommended that additional patients could be enrolled in cohort 1 and enrolment into cohort 2 could be initiated.