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Sample size estimations for MRI-monitored trials of MS comparing new vs standard treatments
M P Sormani1, M Rovaris, F Bagnato
1Neuroimaging Research Unit, Department of Neuroscience, Scientific Institute and University Ospedale San Raffaele, Milan, Italy.
Abstract:
The authors estimated the sample sizes needed for exploratory trials of MS assessing the efficacy of new treatments in reducing the number of new enhancing lesions vs those of interferon-beta or glatiramer acetate. The sample sizes per arm ranged from 868 (effect: 20%) to 94 (effect: 50%) for patients with relapsing-remitting MS and from 2,484 (effect: 20%) to 361 (effect: 50%) for patients with secondary progressive MS. In MS, exploratory trials of new vs available therapies require large numbers of patients, even when MR end-points are used.
Insights
Exploratory trials for multiple sclerosis (MS) treatments need large patient numbers, even with MRI endpoints. Sample sizes vary based on disease type and treatment effect size, ranging from 94 to over 2,400 patients per arm.
Area of Science:
- Neurology
- Clinical Trials
- Biostatistics
Background:
- Multiple Sclerosis (MS) treatment research requires robust clinical trial designs.
- Assessing novel MS therapies against established treatments like interferon-beta and glatiramer acetate is crucial.
- Magnetic Resonance (MR) imaging endpoints are increasingly utilized in MS clinical trials.
Purpose of the Study:
- To estimate the sample sizes required for exploratory clinical trials in MS.
- To compare the sample size needs for new MS treatments versus existing therapies (interferon-beta, glatiramer acetate).
- To evaluate sample size requirements based on different effect sizes and MS subtypes.
Main Methods:
- Sample size calculations were performed for exploratory MS trials.
- The study considered new treatments aimed at reducing new enhancing lesions.
- Calculations were stratified by MS disease course (relapsing-remitting MS and secondary progressive MS) and anticipated treatment effect (20% to 50%).
Main Results:
- Sample sizes per arm for relapsing-remitting MS ranged from 868 (20% effect) to 94 (50% effect).
- Sample sizes per arm for secondary progressive MS ranged from 2,484 (20% effect) to 361 (50% effect).
- Even with MR endpoints, substantial patient numbers are necessary for exploratory MS trials.
Conclusions:
- Exploratory trials for novel MS therapies necessitate large sample sizes.
- The required patient numbers are significantly influenced by the expected treatment effect size and the specific MS population studied.
- These findings highlight the resource-intensive nature of developing new MS treatments.