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The change from baseline in PSPRS Total score are plotted at the 6 and 12 month follow-up timepoints for the PSP subjects.

Sample Sizes for PSPRS Clinical Scores

Sample size per arm required to observe 20%-80% reduction of the PSPRS Clinical scores (Gait & Midline subscore, Ocularmotor subscore, and PSPRS Total score)  in a treated group compared to an untreated group over either a 6- or 12-month period. The sample size estimations are color-coded, where the green hues are less than 50 subjects per arm.

References

Golbe, L.I, Ohman-Strickland, P,A. A clinical rating scale for progressive supranuclear palsy. Brain. 130:1552-65, 2007; doi: 10.1093/brain/awm032

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The graph illustrates the clinical progression observed in the PSP population, as measured by the PSPRS (Progressive Supranuclear Palsy Rating Scale) Total score (Golbe et. al. 2007). The trajectory of clinical decline aligns closely with the established natural history of the disease, showing an average annual increase of approximately 10 points in the PSPRS Total score. This progression rate reflects the expected deterioration in motor, cognitive, and functional abilities typically seen in PSP patients.

In conjunction with the progression data, sample size assessment has been performed. The table includes estimates of the number of participants required, per treatment arm, to detect changes ranging from 20% to 80% reduction in disease progression. The resulting sample size estimates provide critical insight into the statistical power needed to detect meaningful clinical effects, for interventional studies.

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