About
Hours and details
I have had a long-standing and wide-ranging interest in ADPKD, including basic investigations addressing mechanisms of ion transport by cystic epithelia, clinical investigations describing causes of mortality in the ADPKD ESRD population, and participation and direction of clinical trials in ADPKD. I have participated in all of the major interventional clinical trials in ADPKD serving as the Boston site Principal Investigator and member of the Steering Committees for the HALT PKD study, the TEMPO 3/4 and REPRISE trials of tolvaptan in ADPKD, the TAME-PKD study of metformin, and the Venglustat STAGED-PKD study. I also served as Boston site PI for the Tesevatinib (Kadmon) and Bardoxolone (Falcon) studies. I have been a member of the Scientific Advisory Committee of the Polycystic Kidney Disease Foundation from 1999 to 2017 (Chairman from 2006-2010. Over the past 10 years, I initiated and co-led the PKD Outcomes Consortium and have been the principal individual involved in bringing together the contributors from academia, pharma, NIH, FDA, EMA, CDISC, and C-Path. Total kidney volume has been approved as a prognostic biomarker by both the FDA and the EMA. Dr. Perrone continues to serve as the co-Director of the PKD Outcomes Consortium which meets on a monthly basis to discuss new approaches to achieving regulatory goals for ADPKD and ARPKD. Recently, the Critical Path Institute convened a regulatory summit in PKD for which Dr. Perrone was deeply involved in planning and organization. I am very familiar and experienced with care of PKD patients, USRDS data analysis, performing clinical trials, biomarker development, background in basic science, development of patient assessment tools, ability to recruit participants to clinical trials in ADPKD, and expertise and experience working with multicenter clinical trials and large consortia.
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- Physician