Results of a 2023 Spinal Muscular Atrophy (SMA) Clinical Trial Site Capacity and Recruitment Survey


Clinical Trials

Poster Number: S107


Fatou Sarr, Faegre Drinker Biddle & Reath, Ilse Peterson, MPH, Faegre Drinker Biddle & Reath, Jackie Glascock, PhD, Cure SMA Elk Grove, Mary Curry, ND, Cure SMA Elk Grove, Mary Schroth, MD, FAAP, FCCP, Cure SMA

Cure SMA conducted a survey 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.

Learnings from a similar 2017 survey, assessment of the current and projected SMA trial recruitment needs based on information from, and discussions with individuals from industry sponsors and trial sites informed the survey development. The survey was open from June 28 – August 1, 2023 and distributed to individuals at US sites conducting trials for SMA investigated drugs.

24 SMA clinical trial sites are represented in the results (n=22 unique site completed responses and two unique site partial responses). All but three respondents reported having capacity to conduct additional SMA trials. Limited staff bandwidth (Weighted Average [WA] = 2.11 of 3 where 1 = not at all and 3 = a great deal) and contract negotiations (WA = 2.09) presented as the most significant barriers to study start-up. Increasing the number of clinical trial staff (WA = 3.64 of 5 where 1 = not helpful and 5 = extremely helpful) (especially for clinical research coordinators (WA = 3.88) and physical therapists (WA = 3.43)) and streamlining PT training for trials (WA = 3.71) were reported as the most helpful factors to increase site capacity for new SMA clinical trials.

As recruitment needs for SMA clinical trials continues to increase, action is needed to address the ongoing needs of research staff/facilities and further optimize trial site readiness and capacity.

Funding for this research was provided by the 2023 SMA Industry Collaboration; members include Cure SMA, Biogen, Scholar Rock, Novartis Gene Therapies, Biohaven Pharmaceuticals, Epirium Bio, Genentech/Roche, and SMA Europe.