Motor Neurone Disease - Systematic Multi-Arm Adaptive Randomised Trial
Status:
Recruiting
Trial end date:
2026-12-01
Target enrollment:
Participant gender:
Summary
MND-SMART is investigating whether selected drugs can slow down the progression of motor
neurone disease (MND) and improve survival.
The study is 'multi-arm' meaning more than one treatment will be tested at the same time. In
the first instance the trial will have 3 arms; drug 1, drug 2 and placebo (dummy drug). This
allows the evaluation of drug 1 versus placebo and separately drug 2 versus placebo.
Participants will be randomly allocated to either drug 1, drug 2 or placebo. Medicines being
tested are already approved for use in other conditions.
MND-SMART has an 'adaptive' design. This means medicines being studied can change according
to emerging results. Treatments shown to be ineffective can be dropped and new drugs can be
added over the duration of the study. This will allow many treatments, over time, to be
efficiently and definitively evaluated.
The first medicines being tested have been selected following a rigorous process involving a
systematic, unbiased, and comprehensive review of past clinical trials data, as well as
information from pre-clinical research (studies in laboratories), for MND and other related
neurodegenerative disorders. Drugs have been ranked for inclusion in MND-SMART by a group of
independent MND experts according to set criteria. These include consideration of how the
drugs work, their safety profiles, and the quality of previous studies.
New drugs will be selected for investigation in MND-SMART based on continuous review of
constantly updated scientific evidence as well as findings from state-of-the-art human stem
cell based drug discovery platforms.
Phase:
Phase 2/Phase 3
Details
Lead Sponsor:
University of Edinburgh
Collaborators:
NHS Lothian University College, London University of Warwick