FDA EIR - John L. Berk, M.D. - July 22, 2021

An FDA inspection of Clinical Investigator John L. Berk, M.D.'s site in Boston, MA, was conducted from July 19-22, 2021. The inspection, initiated by the Center for Drug Evaluation and Research (CDER) under CPG 7348.810, evaluated compliance with regulations and Protocol ALN-TTRSC-004, a Phase 3 study for hereditary transthyretin amyloidosis (NDA 214751).

Key issues identified included inaccurate records for the 10-Meter Walk Tests, a secondary efficacy endpoint, for several subjects. Additionally, the site failed to report a protocol deviation where a subject received an unpermitted medication, a discrepancy noted by the study monitor. Concerns were also raised regarding the proper labeling, identification, and calibration of tools and instruments, such as measurement tapes and stopwatches, used to collect primary efficacy data, which is essential for data integrity.

Dr. Berk and the clinical research coordinator acknowledged the findings and committed to corrective actions. These include updating the inaccurate 10-Meter Walk Test records, officially reporting the previously undeclared protocol deviation, and ensuring all study instruments are properly labeled, identified, and calibrated for current and future studies.