A prospective, multi-center, observational study of the safety, tolerability and effectiveness of SPINRAZA in adult patients with Spinal Muscular Atrophy

PI: Craig Zaidman, MD, Department of Neurology

This is an investigator initiated 16-site, industry funded, Phase 4 clinical trial investigating the safety and efficacy of FDA-approved Spinraza for the treatment of Spinal Muscular Atrophy.

Dr. Zaidman, an investigator who was new to the role of overall study PI of a multicenter trial, reached out to Trial-CARE for project management / study coordination services. Heather Wilkins, MA from Trial-CARE began serving as the Project Manager during study start-up, and facilitated protocol development, MOP/SOP development, database design and build, and WU IRB approval. In addition, the PM brought onboard all 16 external sites, including facilitation of the sub-contracting and budget processes, regulatory file set-up and collection, and site training and initiation visits. For this study, Trial-CARE provided both clinical coordination and data coordination services, the latter in partnership with the Division of Biostatistics.

Enrollment was successfully completed as of July 1, 2021, and sites will continue to complete follow-up assessments through end of 2023.

Clincaltrials.gov NCT03709784