Comparison of Demographics: National Amyotrophic Lateral Sclerosis Registry and Clinical Trials Data

Objective To characterize the participant demographics in the Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT) database compared with the web-portal National Amyotrophic Lateral Sclerosis (ALS) Registry (the Registry).

Methods Demographics and ALS symptom information were compared between the self-reported registrant data in the Registry web portal (2010–2021) and the latest available PRO-ACT data (updated August 2022), which is a collection of clinical trials data.

Results Greater percentages of younger (≤ 59 years old) but smaller percentages of older (60 + years old) participants were represented in PRO-ACT compared to Registry.

Enrollment for minority race groups was greater in the Registry portal data, but race information was largely missing/unknown in PRO-ACT database.

Median age at the time of diagnosis and age at the time of symptom onset were significantly higher for Registry enrollees compared to the participants of PRO-ACT.

Symptom onset sites were similarly reported, but duration between self-noted symptom onset and diagnosis was slight, but significantly longer for the Registry enrollees (11 vs. 9 months).

Hispanic were as likely as non-Hispanic to participate in research studies, based on the Registry data.

Conclusion There was a notable difference in the age distribution and minority representation of enrollees between the PRO-ACT and Registry study populations.

Age distribution in the PRO-ACT database skewed to a younger and less diverse cohort.

Despite the clinical heterogeneity and complex disease mechanism of ALS, identifying the underrepresented demographic niche in the PRO-ACT and Registry study populations can help improve patient participation and criteria for patient selection to enhance generalizability.