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