Abstract Details

Title Examination of Participant Demographics and ALS Symptom Characteristics from Pooled Resource Open-Access ALS Clinical Trials and National ALS Registry

Topic General Neurology

Presentation(s) P4 - Poster Session 4 (8:00 AM-9:00 AM)

Poster/Presentation
Number 6-003

Objective To identify potential underrepresentation of participant demographic niche and symptom onset/duration characteristics in Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT) compared to the web-portal National ALS Registry (Registry).

Background Amyotrophic lateral sclerosis (ALS) is a rare neurodegenerative disease of lower/upper motor neurons. The Registry is a multi-faceted ALS research platform that collects patient samples for research via the National ALS Biorepository, assists researchers with their recruitment for clinical/epidemiological studies, and collects/disseminates data to researchers. PRO-ACT is a database procured from ALS clinical studies and offers a platform for an aggregated characterization of trial enrollees.

Design/Methods Registry data (2010-2019) were deduplicated/deidentified for registrants who self-enrolled in the web portal, and information about demographics and ALS symptom onset site/duration (prior to diagnosis) were gathered. The latest available PRO-ACT data were accessed, and predictors were extracted for data comparison. Means or median, percentages, and 95% CIs were calculated for a descriptive summary, and distribution differences in the study populations were evaluated.

Results Mean age of enrollees at the time of diagnosis for Registry was higher compared to PRO-ACT (59.5 vs. 54.7 years old). Likewise, age at the time of symptom onset was also greater in this cohort. Greater percentages of younger (≤59) but smaller percentages of older (60+) participants were represented in PRO-ACT. Minority enrollment was greater in the Registry participants. Percentages of patients reporting bulbar or limb onset were similar for both study populations, but self-noted symptom duration was slightly longer for the Registry enrollees (10.8 vs. 9.0 months).

Conclusions Significant difference in the age distribution and minority representation of enrollees were noted between the study populations. Skewness of data to a younger cohort in PRO-ACT may be explained by eligibility criteria inherent in clinical trials. Addressing the observed discrepancy in Registry and PRO-ACT enrollment can provide better understanding of disease heterogeneity and biomarker determination.

Authors/Disclosures
Moon Han, PhD (Agency for Toxic Substances and Disease Registry) PRESENTER	Dr. Han has nothing to disclose.
Jaime Raymond	Jaime Raymond has nothing to disclose.
Theodore Larson	Theodore Larson has nothing to disclose.
Paul Mehta	Paul Mehta has nothing to disclose.
D Kevin Horton	No disclosure on file

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