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.

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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
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article

Autosomal Dominant Tubulointerstitial Kidney Disease Clinical Trial Simulator: Case Reports of Model‐Informed Drug Development

Kendrah Kidd, Julie Roignot, Anthony J. Bleyer, Shyam Sundhar Ramesh et al.
CPT Pharmacometrics & Systems Pharmacology
Cancer Genomics and Diagnostics
article

Autosomal Dominant Tubulointerstitial Kidney Disease Clinical Trial Simulator: Case Reports of Model‐Informed Drug Development

Kendrah Kidd, Julie Roignot, Anthony J. Bleyer, Shyam Sundhar Ramesh, Sarah Kim, Sanghoon Kang, Jongjin Kim, Katherine Blakeslee, Adrienne Williams, Mark Rogge
article en

Abstract

). 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.

CPT Pharmacometrics & Systems PharmacologyVol. 15(5)
University of Central Florida (US), Biogen (United States) (US), University of Florida (US), Wake Forest University (US)
Good health and well-being
Openalex Percentile: Top 11%
Cancer Genomics and Diagnostics
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Autosomal Dominant Tubulointerstitial Kidney Disease Clinical Trial Simulator: Case Reports of Model‐Informed Drug Development — Kendrah Kidd, Julie Roignot, et al. · CPT Pharmacometrics & Systems Pharmacology (2026) | TGRS Research Map | TGRS