Background: Amyotrophic lateral sclerosis (ALS) is a clinically heterogeneous, fatal motor neuron disease. Natural history studies are critical to improving understanding of disease progression and accelerating development of novel therapies. ASSESS ALL ALS (ASSESS), part of the NIH-funded ALL ALS Consortium, is a longitudinal observational study designed to reduce barriers related to geography and disease severity, supporting broad research participation and multimodal data collection. ASSESS enables harmonized data and biospecimen sharing to accelerate collaborative research.
Objectives: Describe cohort characteristics, baseline functional status, and patient-reported outcomes of the first 785 ASSESS participants.
Methods: ASSESS enrolls in 3 cohorts across 36 U.S. Clinical Sites: individuals with ALS attending in-person visits (on-site), individuals with ALS participating remotely (remote), and controls. Participants complete in-clinic or remote visits every 3-4 months over 2 years. Biospecimens collected include blood and optional CSF. Real-time data is captured into a centralized EDC and coordinates biospecimen storage and distribution through a central NIH-supported biorepository.
Results: As of November 2025, 785 participants were enrolled across 47 U.S. states and Puerto Rico: 311 on-site, 271 remote, 203 controls. Mean age at enrollment was 62 for on-site and remote and 51 for controls. 56% of symptomatic ALS participants self-identified as male (53% on-site, 60% remote) compared to 35% of controls. 86% of participants self-identified as white (86% on-site, 88% remote, 81% control) with 5-8% of Hispanic or Latino ancestry. Biospecimens collected include blood from 513 participants and CSF from 75. Among symptomatic participants, mean baseline ALSFRS-R score was 33.05 ± 8.83, consistent with moderate functional impairment. Baseline UCLA Loneliness Scale scores indicated low perceived loneliness (32.66 ± 9.94), Hospital Anxiety and Depression Scale scores were consistent with borderline symptom levels (10.84 ± 8.83).
Conclusion: ASSESS plans to enroll 1,800 participants by September 2026. Early data demonstrates the feasibility of capturing multidimensional clinical, psychosocial, and biospecimen data from a large, geographically diverse cohort. Descriptive analyses inform strategies to ensure the cohort reflects the U.S. ALS population. Shared data and biospecimens will support analyses of ALS heterogeneity and disease trajectories.