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

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  Fig. 1. Number and types of active SMA clinical trials ​within the (a) US and (b) globally 2011–2023. New SMN Dependent – Studies exploring the safety and efficacy of investigational drugs designed to increase SMN protein levels New SMN Independent – Studies exploring the safety and efficacy of investigational drugs designed to target other systems (i.e. muscle enhancing therapy) affected by the loss of SMN protein Adjunct SMN Dependent – Studies evaluating the safety and efficacy of two approved SMN dependent treatments taken at the same time or sequentially Adjunct SMN Independent - Studies evaluating the safety and efficacy of investigational SMN independent drugs used with an approved SMN dependent treatment at the same time or sequentially Extension of SMN Dependent – studies observing and measuring long term safety, tolerability, and/or effectiveness of an SMN dependent therapy that was previously trialed Extension of SMN Independent – studies observing and measuring long term safety, tolerability, and/or effectiveness of an SMN independent therapy that was previously trialed Permutation SMN Dependent - Studies evaluating the safety and efficacy of different dosing or routes of…

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  Fig. 2. Site rating level of importance of factors enabling them to successfully run SMA trials (n = 22). The factors are ordered in the overall level of importance based on the calculated WA, which is shown for each factor. Sites had the option to select “other” and specify other factors they consider important for successful conduct of SMA clinical trials including: "association with external vendors"; "newborn screening referrals"; "past clinical trials experience"; and "the time of physical/occupational therapists to do the assessments."

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  Fig. 3. Site rating significance of factors that presented as barriers to study start-up (n = 22). The factors are ordered in the overall level of importance based on the calculated WA, which is shown for each factor.

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  Fig. 4. Site rating significance of factors limiting their ability to take on additional SMA trials (n = 22). In the survey, factors were categorized into two groups – patient related factors (highlighted in gray) and team/site related factors. The factors are ordered in the overall level of importance based on the calculated WA, which is shown for each factor. Sites had the option to select “other” and specify other factors they consider important for successful conduct of SMA clinical trials Additional site/team related factors included "needing more opportunities from industry." No additional patient related factors were provided.

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  Fig. 5. Site rating helpfulness of factors increasing their ability to take on additional SMA trials (n = 22). The factors are ordered in the overall level of importance based on the calculated WA, which is shown for each factor. Sites had the option to select “other” and specify other factors they consider important for successful conduct of SMA clinical trials including: "adequate PI effort/ support for participation to reduce clinical time" and "need for more dedicated research space (clinic/research rooms)."