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Designing Multi-arm Multistage Adaptive Trials for Neuroprotection in Progressive Multiple Sclerosis
Vivien Li1, Baptiste Leurent1, Frederik Barkhof1
1From the Florey Institute of Neuroscience and Mental Health (V.L.), University of Melbourne; Department of Neurology (V.L.), Royal Melbourne Hospital, Australia; Department of Medical Statistics (B.L., J.M.N.) and International Statistics and Epidemiology Group (B.L.), London School of Hygiene and Tropical Medicine, UK; Department of Radiology and Nuclear Medicine, Neuroscience Campus Amsterdam (F.B.), VU University Medical Center, Amsterdam, the Netherlands; Queen Square Institute of Neurology and Centre for Medical Image Computing (F.B.), Department of Neuroinflammation, Queen Square Multiple Sclerosis Centre (M.B., O.C.), and NMR Unit, Department of Neuroinflammation (A.E.), Faculty of Brain Sciences, UCL Queen Square Institute of Neurology; MRC Clinical Trials Unit at UCL, Institute of Clinical Trials and Methodology (F.C., M.P., J.C.), and Department of Computer Science, Centre for Medical Image Computing (A.E.), University College London; National Institute for Health Research (F.B., O.C., J.C.), University College London Hospitals Biomedical Research Centre; UK Multiple Sclerosis Society (E.G., G.P., J.R.), London; Faculty of Medicine and Health Sciences (G.P.), University of East Anglia, Norwich; Statistics and Epidemiology, Division of Health Sciences (N.S.), Warwick Medical School, University of Warwick, Coventry; and Population Health Sciences Institute (J.W.), Newcastle University, UK.
This study proposes a Multi-Arm Multi-Stage (MAMS) platform trial design to speed up testing of new treatments for progressive multiple sclerosis (PMS). This efficient design reduces trial duration and participant numbers, accelerating drug discovery for PMS.
Area of Science:
- Neuroscience
- Clinical Trials Methodology
- Biostatistics
Background:
- Progressive multiple sclerosis (PMS) lacks effective treatments to slow disability progression.
- Traditional sequential clinical trials are inefficient for evaluating multiple candidate therapies for PMS.
- Multi-Arm Multi-Stage (MAMS) platform trials offer a more efficient approach to drug development.
Purpose of the Study:
- To describe and evaluate a novel MAMS trial design for progressive multiple sclerosis.
- To optimize interim and final outcome measures, sample size, and statistical considerations for PMS MAMS trials.
- To accelerate the evaluation of novel therapeutic agents for PMS.
Main Methods:
- A two-stage MAMS design was developed, incorporating input from a patient and public involvement group.
- Stage 1 interim outcome: whole brain atrophy on MRI at 18 months.
- Stage 2 final outcome: time to 6-month confirmed disability progression using a composite clinical score.
Main Results:
- Simulations indicated the MAMS design reduces trial duration and sample size by approximately one-third and half, respectively, compared to traditional trials.
- The proposed design requires approximately 1,900 participants over 6 years, assuming one treatment progresses.
- The design was positively received by individuals with multiple sclerosis.
Conclusions:
- The proposed MAMS trial design offers a substantially more efficient method for testing new PMS therapies.
- This approach accelerates the discovery of effective treatments for progressive multiple sclerosis.
- The MAMS principles may be applicable to other neurodegenerative diseases.
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