Funding for this research was provided by:
Wellcome Trust (210752/Z/18/Z)
Received: 24 May 2020
Accepted: 16 February 2021
First Online: 27 February 2021
Ethics approval and consent to participate
: Ethical approval was not required as treatment was provided on a compassionate basis due to lack of alternative established treatments and was approved by the institution’s Drugs and Therapeutics Committee. The orphan use of the drug was also reported to the Health Product Regulatory Agency or Ireland, and their adverse event reporting system was adhered to throughout the treatment period.
: Written informed consent was obtained from patients or their guardians.
: Miransertib was provided by ArQule Inc. (Burlington MA) on a compassionate managed patient access programme basis for the duration of the study period.