Leo Mascarenhas, MD – FDA EIR, Jul 1, 2014

An FDA inspection of Leo Mascarenhas, MD, a clinical investigator at Children's Hospital Los Angeles, was conducted from June 17 to July 1, 2014. This pre-approval inspection, falling under Compliance Program 7348.811 for clinical and sponsor-investigators, evaluated the site's conduct of Protocol #ANBL0032 for high-risk neuroblastoma, sponsored by United Therapeutics Corporation. While no formal FDA 483 observations were issued, several critical concerns were discussed with site management. These included two instances of delayed reporting for Grade 4 adverse events to the Adverse Event Expedited Reports (AdEERS) system and one to the Institutional Review Board (IRB), exceeding protocol-mandated timeframes. The inspection also identified inaccurate reporting of white blood cell laboratory data on Case Report Forms, stemming from a failure to convert units. Significant gaps were noted in training documentation for the study's 2003 initiation and a 2010 amendment. Additionally, the investigational pharmacy lacked physical segregation for study drugs from commercial products, and a protocol amendment in 2004 was submitted to the IRB 3.5 months late. The site was advised to address these deficiencies to ensure adherence to regulatory standards and Good Clinical Practice.