Sex, racial, and ethnic disparities in motor neuron disease: clinical trial enrolment

Abstract

Objective

Motor neuron disease (MND) is a group of neurological diseases, the majority being amyotrophic lateral sclerosis (ALS), with varying clinical presentations across demographics. Clinical trial enrollment reflecting global disease burden improves understanding of diverse presentations and aids personalized therapy development. We assessed the sex, racial, and ethnic composition of MND/ALS clinical trial participants relative to global disease burdens.

Methods

We searched ‘motor neuron disease OR amyotrophic lateral sclerosis’ on ClinicalTrials.gov from 02/2000–04/2024. We extracted trial (start year, study site, sponsor location, phase, masking, intervention) and demographic data (sex, race, ethnicity) from randomized interventional studies. We obtained sex-based MND/ALS disease burden estimates from the Global Burden of Disease database. For females, we calculated pooled participation-to-prevalence ratio (PPR) with 95% confidence intervals (CIs), with PPR of 0.8–1.2 indicating adequate enrollment. We used Kruskal–Wallis tests to compare demographic groups across trial characteristics.

Results

Of 85 trials, females comprised 37.47% (n = 5011) of 13,372 participants; the pooled female PPR was 0.97 (95% CI: 0.77–1.16). Of 41 trials (9340 participants) reporting race, 121 (1.30%) participants were Black or African American, 16 (0.17%) American Indian or Alaskan Native, and 6 (0.06%) Native Hawaiian or Other Pacific Islander. 24 trials (595 participants) reported ethnicity, with a minority of Hispanic participants (n = 153; 2.57%).

Conclusions

MND/ALS clinical trials had adequate female enrollment relative to global disease burdens. Race and ethnicity data were underreported. However, there were enrollment disparities of racial and ethnic groups. Increased trial leadership diversity, equitable enrollment policies, and addressing barriers to participation could improve enrollment diversity.

Keywords:

• Motor neuron disease
• amyotrophic lateral sclerosis
• MND
• ALS
• disparity

Author contributions

CT and BT designed the work. CT drafted the main manuscript text. MW and HS conducted all data collection. BT conducted the statistical analysis and interpretation of the data. BT, AA, and CBT substantively revised the work. All authors reviewed and approved the final manuscript prior to submission. All authors agree to be accountable for all aspects of the work and ensure all questions related to the work are resolved.

Declaration of interest

The authors report no conflicts of interest. The authors alone are responsible for the content and writing of this article.

Data availability statement

The authors confirm that the data supporting the findings of this study are available within the article and its materials.