Workshop | Mixed
Event Title
Proteinuria and GFR as Clinical Trial Endpoints in Focal Segmental Glomerulosclerosis: A Scientific Workshop
October 7 - 8, 2024
- Date:
- October 7 - 8, 2024
- Day1:
- - ET
- Day2:
- - ET
Attend
Dates and Time:
- Monday, October 7: noon – 5 pm
- Tuesday, October 8: 8 am – 12 pm
Location:
Bethesda Marriott, 5151 Pooks Hill Road, Bethesda, Maryland 20814
Registration:
Visit the Parasol Scientific Workshop page to register. Virtual attendance options are available.
Background:
The FDA and NephCure, the International Society of Glomerular Disease (ISGD), the Kidney Health Initiative (KHI), and the National Kidney Foundation (NKF) have expressed a willingness to leverage their combined strengths for the joint development of the substantive actions of this workshop to facilitate the development of safe and effective treatments for focal segmental glomerulosclerosis (FSGS).
FSGS is an important cause of kidney failure for which there are no FDA-approved therapies. Because of the time course for disease progression, rarity, and heterogeneity of FSGS, endpoints such as kidney failure are generally not feasible in clinical trials of FSGS. In 2019, KHI, a public-private partnership between the American Society of Nephrology and the FDA, initiated a project to identify endpoints that could be used to establish the efficacy of treatments for FSGS. The workgroup concluded that the available data support the use of complete remission of proteinuria in patients with heavy proteinuria as a surrogate endpoint for progression to kidney failure in clinical trials of FSGS. In addition, the workgroup concluded that substantial treatment effects on proteinuria short of a complete remission may also predict the effect of a treatment on progression to kidney failure; however, further work is needed to determine how such an endpoint should be defined. Specifically, to support the use of proteinuria as a reasonably likely surrogate endpoint, a better understanding of the quantitative relationship between changes in proteinuria and progression to kidney failure is needed.
The aforementioned project focused on information available in the published literature. To advance the understanding and use of proteinuria and eGFR-based endpoints as surrogate endpoints for accelerated and traditional approval in FSGS, NephCure, ISGD, KHI and NKF are facilitating new analyses of existing data from randomized controlled trials, observational studies, and registries.
The results of these analyses will be discussed at a scientific workshop, co-sponsored by the parties listed above. The statistical findings and report on the deliberations from the workshop will be made public by one or more publications in professional journals.
Goals and Objectives:
The goal of this scientific workshop is to advance the use of proteinuria and eGFR-based endpoints as surrogate endpoints for accelerated and traditional approval of new treatments for FSGS. The workshop will be used to discuss the results of the aforementioned analyses and engage in a data-driven discussion with the larger community about the use of these endpoints as surrogate endpoints for the approval of treatments for FSGS.
Who Should Attend: The primary audience includes leading academic experts, interested pharmaceutical companies, regulatory agencies, patient representatives and advocacy groups, clinicians, researchers and individuals affiliated with medical professional societies, and other government agencies and members of the healthcare sector (e.g., payers).
Contact:
If to NEPHCURE:
Joshua M Tarnoff, CEO
NephCure
1299 N. 7th St #16556
Philadelphia, PA 19122
610-540-0186 ext. 15
jtarnoff@nephcure.org
If to the International Society of Glomerular Disease:
Laurel Damashek, Executive Director
ISGD
123 Main St. #60335
Florence, MA 01062
617-285-2025
ldamashek@is-gd.org
If to the Kidney Health Initiative:
Melissa West, Senior Director for Strategic Relations and Patient Engagement
c/o American Society of Nephrology
1401 H Street, NW
Suite 900
Washington, DC 20005
(202) 740-7891
mwest@asn-online.org
If to the National Kidney Foundation (NKF):
Kerry Willis, CSO
National Kidney Foundation
30 East 33rd Street
New York NY 10016
212.889.2210 ext. 187
kerryw@kidney.org
If to FDA:
Aliza Thompson, Acting Director
Division of Cardiology and Nephrology
Building 22, Office 4160
Food and Drug Administration
10903 New Hampshire Avenue
Silver Spring, MD 21029
301-796-1957
aliza.thompson@fda.hhs.gov