Objective: To evaluate the sensitivity of routine smartphone-based performance assessments in the free-living environment as part of a randomized clinical trial in Parkinson’s disease (PD).

Background: LUMA (NCT05348785) is a Phase 2b, multicenter, randomized, double-blind, placebo-controlled study to determine safety and efficacy of BIIB122 tablets in individuals aged 30 to 80 years with early-stage PD. The primary endpoint is time to confirmed worsening in MDS-UPDRS parts II and III combined score over the treatment period. As an exploratory outcome, the study investigates the effects of BIIB122 on the longitudinal progression of motor symptoms as measured by objective performance assessments administered through a smartphone application. Digital endpoints from these assessments may be more sensitive to subtle changes in disease disability compared to clinical rating scales.

Method: Participants perform the smartphone assessments at specified clinic visits (twice at baseline, then once each at the week 24, 48, 72, and 96 visits) and on a weekly basis at home. Tests of walking, standing postural sway, pronation-supination, finger drawing, keystroke typing, finger tapping, speech production, and symbol-digit substitution are deployed through the smartphone application.

Results: The tests are being deployed at 91 sites across 11 countries and in 12 different languages. Critical steps in selecting digital endpoints with the strongest validity as indicators of worsening PD include analysis of test-retest reliability and sensitivity to longitudinal change. Reliability will be assessed using the test-retest design at the baseline clinic visit and, for unsupervised conditions, the first few weeks of remote assessments. Sensitivity to longitudinal progression relative to clinical anchors will be determined using a blinded subset of study subjects.

Conclusion: The smartphone assessment protocol allows for a good compromise between feasibility and burden to the patients and enables the selection of digital endpoints based on test-retest reliability, relationship to established anchor measures of PD severity, and sensitivity to longitudinal progression. The digital data will allow for a comparison of performance in the clinic setting versus the free-living environment, provide insight into week-by-week variability in motor function, and may provide real-world, objective evidence of treatment efficacy in early-stage PD.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/smartphone-based-digital-assessments-in-the-luma-trial-of-biib122-for-early-stage-parkinsons-disease/