Resubmission of the “Choices” clinical trial to CTIS, after its initial rejection, is being prepared by the European HIT-CF Europe project team, giving promising signals for the start of the trial at the beginning of the new year.
Individuals selected for the trial at participating European centres have already been notified and HIT-CF is negotiating with other partners to set up trials for those unable to participate in CHOICES. A total of 52 patients, selected from European centres, will be enrolled in the trial, which will examine the effect of a triple combination of CFTR modifiers on increasing the number of functional CFTR channels on the cell surface. In addition, at the same time as the study medicines were being tested , Tezacaftor/Ivacaftor (Symkevi) treatment was also tested in organoids and the individualised response of organoids to Symkevi has been disclosed to HIT-CF participants not eligible for the Choices study.