Autosomal Dominant Tubulointerstitial Kidney Disease Clinical Trial Simulator: Case Reports of Model‐Informed Drug Development
). Both slope-based eGFR change and end-of-trial eGFR measures functioned as effective surrogate endpoints. Herein, we present the model-based CTS tool developed for ADTKD and demonstrate its use in designing and evaluating clinical trial scenarios, illustrated by two representative case studies. The CTS tool provides a pragmatic framework for optimizing ADTKD trial design. By enabling scenario testing and highlighting genotype-specific considerations, it supports efficient, cost-effective planning and represents an example of model-informed drug development in rare kidney diseases.
Authors
- Kendrah Kidd (ORCID: https://orcid.org/0000-0003-3771-0949)
- Julie Roignot
- Anthony J. Bleyer (ORCID: https://orcid.org/0000-0002-2804-5273)
- Shyam Sundhar Ramesh
- Sarah Kim (ORCID: https://orcid.org/0000-0002-9179-0735)
- Sanghoon Kang (ORCID: https://orcid.org/0000-0001-6222-7479)
- Jongjin Kim
- Katherine Blakeslee
- Adrienne Williams
- Mark Rogge
Institutions
- University of Central Florida (US)
- Biogen (United States) (US)
- University of Florida (US)
- Wake Forest University (US)
Publication Details
- Journal
- CPT Pharmacometrics & Systems Pharmacology
- Published
- 2026-04-30
- DOI
- https://doi.org/10.1002/psp4.70258
- Primary Topic
- Cancer Genomics and Diagnostics
- Type
- article
- Field-Weighted Citation Impact
- 0.00