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% |
aThe characteristics presented in this table are those that were included in the site readiness checklist