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Table 3 Characteristics of Sites that Participated in 2018 Readiness Program Pilot (N = 9)

From: The SMA Clinical Trial Readiness Program: creation and evaluation of a program to enhance SMA trial readiness in the United States

Characteristicsa # of Sites with % of Sites with
Minimum Criteria
 Clinical research infrastructure 9 100%
 Seeing SMA patients for research or care 9 100%
Site Research Capabilities and Experience
 Dedicated clinical research unit 8 89%
 Clinical trial experience (any) 9 100%
 Neuromuscular clinical trial experience 5 56%
 Conducting other SMA research studies (not clinical trials) 4 44%
 Active enrollment for other SMA research studies (not clinical trials) 3 33%
Patient Population
 Children 8 89%
 Adults 6 67%
Principal Investigator Experience
 SMA clinical trial experience 5 56%
 Other neuromuscular clinical trial experience (outside of SMA) 7 78%
 Certified Principal Investigator 0 0%
Clinical Research Coordinator Experience
 SMA clinical trial experience 2 22%
 Other neuromuscular clinical trial experience (outside of SMA) 5 56%
 Coordinator(s) has completed ACRP CRC or SOCRA certification 6 67%
Physical Therapist Experience
 SMA-specific motor function outcome measures (clinical evaluation) 9 100%
 SMA-specific motor function outcome measures (clinical trials) 5 56%
 Other neuromuscular disease outcome measures (not SMA) 8 89%
 Completed reliability training for motor function outcome measures 8 89%
Staff Training Related to Conduct of Clinical Research
 Staff involved in clinical research have conducted all or majority of the listed training programs 9 100%
Clinical Trial Operations
 IRB (Centralized or Local) 9 100%
 Well-documented informed consent process 9 100%
 Established, well-documented approach for ensuring adherence to study protocol 9 100%
 Established and well-documented approach to PI oversight 9 100%
  1. aThe characteristics presented in this table are those that were included in the site readiness checklist