FDA EIR - Dr. Albert Maguire. Clinical Inv. - October 20, 2017

An FDA inspection was conducted on Clinical Investigator Dr. Albert M. Maguire at Children's Hospital of Philadelphia from October 16 to October 20, 2017. This high-priority inspection, requested by the Center for Biologics Evaluation and Research (CBER) and guided by Compliance Program Guidance Manual (CPGM) 7348.811 for Clinical Investigators, focused on the clinical study "A Safety and Efficacy Study in Subjects with Leber Congenital Amaurosis (LCA) using Adeno-Associated Viral Vector to Deliver the Gene for Human RPE65" (FDA IND Number: 13408). The inspection involved comparing sponsor-provided background materials, including protocol and data listings, with Dr. Maguire’s on-site source documents and case report forms for nine subjects. While site records consistently matched case report forms, investigators noted differences between the sponsor-provided dataset tables and the on-site data for primary and secondary efficacy endpoints such as mobility testing, visual acuity, and surgical operative findings. Despite these discrepancies in the sponsor's submission, Dr. Maguire's study coordinators successfully provided comprehensive and comparable documentation directly from the site, which aligned with their source records. Consequently, no FDA Form 483, Inspectional Observations, was issued to Dr. Maguire. A general discussion was held at the inspection's conclusion, emphasizing the importance of accurate documentation for clinical studies and timely review of adverse events, and addressing the noted differences in data provided by the sponsor to the FDA versus the site's own records.