FDA EIR - Etienne M. H. Sokal, MD, PhD. - June 04, 2021

An FDA foreign clinical investigator inspection was conducted at Etienne M. H. Sokal, MD, PhD., in Brussels, Belgium, from May 31 to June 4, 2021. The inspection reviewed protocol LUM0001-304, supporting NDA 214662 under IND 119917, for a drug in Alagille Syndrome. While the site was found in general compliance and no FDA Form 483 was issued, several observations were discussed. These included investigators not consistently dating their signatures on records, insufficient documentation for a subject's vitamin D supplementation, and inconsistent use of temperature monitoring devices for investigational product shipments, or inadequate documentation of other temperature assurance methods. Additionally, some data entry errors were noted in Peds QL questionnaires, and a sponsor coding error led to the omission of raw scores for specific PedsQL data in the submission. The inspection was conducted under FDA's framework for clinical investigators (CP 7348.811). Management acknowledged the issues and committed to improving documentation practices, assessing shipping procedures, and reviewing data for omissions. Professor Sokal was reminded of his responsibility to adhere to FD & C Act regulations for FDA-regulated clinical research.