GLOBAL PHASE III RARE DISEASE STUDY EXCEEDS DATABASE LOCK TARGET DURING COVID-19

• Utilized web-based clinical trial management software, to track site specific COVID-19 restrictions — provided updates to the study team on what each site permitted on a weekly basis
• Developed site specific plans for remote based monitoring and source data verification and ensured site source documentation process forms (SSDPFs) were updated for source sharing

— Use of Fervor Site Source Portal which allowed sites to upload source documents for CRA’s to review remotely

— Video-based monitoring

— Sites emailed redacted certified copies

• Updated protocol language to allow for COVID mitigations with regulatory submissions globally — Increased visit windows

— Allowed for home health visits

— Allowed for telehealth visits

— Allowed for local lab testing when site didn’t allow subject visits

• Developed remote consenting guidance for both sites and subjects
• Developed source data verification plan that allowed for focus on critical data for primary endpoints

— Set required % source data verification for critical data and overall source data verification

• Added detailed language to the Monitoring Plan – defining the source data verification plan and provided

detailed guidance to CRAs

• Developed Database Lock Plan to detail timelines, source data verification plan, roles and responsibilities

for database lock, and contingency planning

• Created detailed metrics reporting and reviewed weekly as it related to the database lock to prioritize

sites of concern and necessary follow-up