Trials in MND
MOTOR NEURON DISEASE, DISEASE
Despite 125 clinical trials registered between 2008 and 2019, involving 15,647 people with MND and evaluating 76 investigative medicinal products (IMPs) [MND–SMART (Motor Neuron Disease–Systematic Multi-Arm Randomised Adaptive Trial) is a phase 3 UK-wide innovative adaptive platform trial, clinicaltrials.gov (NCT04302870) and EudraCT (Trial record number: 2019-000099-41) recruiting people with a clinical diagnosis of MND, irrespective of genetic status.MND–SMART aims to evaluate a series of drugs over the next two decades, within an adaptive platform trial protocol. Adaptive design trials use the ongoing results of the trial to feedback recommendations on the continuation or early stopping of candidate drugs, with the platform aspect ensuring new candidate drugs can be introduced during the course of the trial. All IMPs are administered in a liquid form, to ensure participants can continue to take them if difficulties with swallowing develop or progress. Broad inclusion criteria promotes wide participation and ensures that large numbers of people living with MND are eligible, with no sub-type exclusions to capture disease heterogeneity and improve the generalisability of findings.