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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 infrastructure9100%
 Seeing SMA patients for research or care9100%
Site Research Capabilities and Experience
 Dedicated clinical research unit889%
 Clinical trial experience (any)9100%
 Neuromuscular clinical trial experience556%
 Conducting other SMA research studies (not clinical trials)444%
 Active enrollment for other SMA research studies (not clinical trials)333%
Patient Population
Principal Investigator Experience
 SMA clinical trial experience556%
 Other neuromuscular clinical trial experience (outside of SMA)778%
 Certified Principal Investigator00%
Clinical Research Coordinator Experience
 SMA clinical trial experience222%
 Other neuromuscular clinical trial experience (outside of SMA)556%
 Coordinator(s) has completed ACRP CRC or SOCRA certification667%
Physical Therapist Experience
 SMA-specific motor function outcome measures (clinical evaluation)9100%
 SMA-specific motor function outcome measures (clinical trials)556%
 Other neuromuscular disease outcome measures (not SMA)889%
 Completed reliability training for motor function outcome measures889%
Staff Training Related to Conduct of Clinical Research
 Staff involved in clinical research have conducted all or majority of the listed training programs9100%
Clinical Trial Operations
 IRB (Centralized or Local)9100%
 Well-documented informed consent process9100%
 Established, well-documented approach for ensuring adherence to study protocol9100%
 Established and well-documented approach to PI oversight9100%
  1. aThe characteristics presented in this table are those that were included in the site readiness checklist