An updated review of the SMA clinical trial landscape in the United States: Findings from analysis of recruitment targets on ClinicalTrials.gov and a survey of SMA clinical trial sites on factors affecting site capacity and readiness

The approval of new disease-modifying treatments, diagnostic tools, and expansion of the drug pipeline has evolved clinical trial landscape for spinal muscular atrophy (SMA) – a rare neuromuscular disease. Nonetheless, significant unmet needs for the SMA population remain and require continued evolution of the SMA clinical trial landscape. In 2023, Cure SMA developed a series of activities to assess the current SMA clinical trial landscape including the Cure SMA Capacity and Recruitment Survey. The purpose of the survey is to understand site capacity for SMA clinical trials in the US, identify factors that may limit site trial capacity and/or readiness, and identify opportunities to increase site capacity. Twenty-four (24) sites across the US with experience in conducting SMA clinical trials are represented in the results. Although existing clinical trial sites may have the capacity to take on additional SMA trials, many factors are inhibiting efficient trial management and study start-up. As recruitment needs for SMA clinical trials continue to evolve, action is needed to address the site needs – such as increased clinical staff support and bandwidth, streamlining training, and adequate funding to conduct trials – to further optimize trial site readiness and capacity. SMA clinical trial sites, sponsors, and contract research organizations will need to work together to optimize trial site readiness address challenges to capacity. • SMA research sites report having capacity for additional clinical trials • Research sites experience growing challenges to efficiently conduct SMA trials • More geographic distribution of sites needed to increase outreach to SMA community • Trial capacity needs to be monitored as treatment landscape evolves for unmet needs