FDA EIR - Morie A. Gertz, M.D., Clinical Investigator - March 09, 2018

An FDA inspection of Clinical Investigator Morie A. Gertz, M.D., located at Mayo Clinic, was conducted from March 5-9, 2018. The inspection focused on a Phase 2/3 clinical trial for Familial Amyloid Polyneuropathy, sponsored by Ionis Pharmaceuticals, Inc., under a high-priority premarket New Drug Application (NDA) assignment and guided by FDA compliance protocols for clinical investigators. The most significant finding, formally issued on an FDA Form 483, identified that not all adverse events were captured and reported according to the study protocol, with 14 instances cited. Other issues discussed verbally included the investigator's prior failure to report non-serious adverse events to the Institutional Review Board (IRB) in annual progress reports until December 2017. Documentation inconsistencies were also noted, such as a lack of recorded verbal authorizations for adverse event changes and insufficient direct attribution for certain subject data. Furthermore, an audit revealed that several efficacy data listings were unverifiable by the site, as their scoring involved data transformation performed by a sponsor-contracted core laboratory. Dr. Gertz and his staff acknowledged all observations and committed to submitting a written response to the FDA 483, alongside implementing internal reviews and necessary corrective actions.