Study design
Administered
ADVERSE EVENTS
A phase I/II, multinational, multicentre, open-label study (NCT02592707) was conducted in eight centres across Australia, Europe, and Canada, in accordance with the Declaration of Helsinki and the International Conference on Harmonisation Good Clinical Practice guidelines. All patients provided written informed consent. Ethical approval was obtained from relevant ethics committees. Patient enrolment was initiated on 6 March 2017, with data cut-off for this analysis on 1 April 2021.The study was conducted in two parts for safety reasons (Fig. Administered activity and peptide amounts of [Subsequently, after evaluation of safety and dosimetry data from part A by the SRC, part B was initiated. Patient safety and escalation of administered activity were evaluated by a Data Review Board (DRB) in part B. Part B assessed different administered activities and peptide amounts of [Part B consisted of 25 patients in three cohorts (cohort 1, In both parts, the interval between treatment cycles was 8 weeks, but could be extended to 12 weeks in case of inadequately recovered adverse events (AEs).Up to 55 patients were to be enrolled in the trial, a minimum of six and up to 15 in part A, and a minimum of 25 and up to 40 in part B; however, these targets were not reached due to cancelled cohorts. The study was terminated in January 2022 due to the small number of ongoing patients; all patients had completed treatment and most were already transferred to the long-term, 5-year follow-up study (20 patients were enrolled in the long-term study [NCT05017662]).Overall, three analyses were planned for the study. A first analysis was conducted with a cut-off date of 27 September 2019. A second analysis was initiated following the completion of cohorts 3 and 6 of part B of the study. A third analysis was to be performed when both parts of the study were entirely complete; however, due to cohorts 2, 4, 5, 7, and 8 not being performed, the second and third analyses were combined. The results of this combined analysis represent the complete results of both parts of this study, and are presented here. A final analysis is planned to be performed at the end of the 5-year follow-up study (estimated study completion date: April 2025).