Design of clinical trials in acute kidney injury: Report from an NIDDK workshop on trial methodology

Paul M. Palevsky; Bruce A. Molitoris; Mark D. Okusa; Adeera Levin; Sushrut S. Waikar; Ron Wald; Glenn M. Chertow; Patrick T. Murray; Chirag R. Parikh; Andrew D. Shaw; Alan S. Go; Sarah G. Faubel; John A. Kellum; Vernon M. Chinchilli; Kathleen D. Liu; Alfred K. Cheung; Steven D. Weisbord; Lakhmir S. Chawla; James S. Kaufman; Prasad Devarajan; Robert M. Toto; Chi Yuan Hsu; Tom Greene; Ravindra L. Mehta; John B. Stokes; Aliza M. Thompson; B. Taylor Thompson; Christof S. Westenfelder; James A. Tumlin; David G. Warnock; Sudhir V. Shah; Yining Xie; Emily G. Duggan; Paul L. Kimmel; Robert A. Star

doi:10.2215/CJN.12791211

Design of clinical trials in acute kidney injury: Report from an NIDDK workshop on trial methodology

Paul M. Palevsky, Bruce A. Molitoris, Mark D. Okusa, Adeera Levin, Sushrut S. Waikar, Ron Wald, Glenn M. Chertow, Patrick T. Murray, Chirag R. Parikh, Andrew D. Shaw, Alan S. Go, Sarah G. Faubel, John A. Kellum, Vernon M. Chinchilli, Kathleen D. Liu, Alfred K. Cheung, Steven D. Weisbord, Lakhmir S. Chawla, James S. Kaufman, Prasad DevarajanRobert M. Toto, Chi Yuan Hsu, Tom Greene, Ravindra L. Mehta, John B. Stokes, Aliza M. Thompson, B. Taylor Thompson, Christof S. Westenfelder, James A. Tumlin, David G. Warnock, Sudhir V. Shah, Yining Xie, Emily G. Duggan, Paul L. Kimmel, Robert A. Star

Research output: Contribution to journal › Article › peer-review

82 Scopus citations

Abstract

Acute kidney injury (AKI) remains a complex clinical problem associated with significant short-term morbidity and mortality and lacking effective pharmacologic interventions. Patients with AKI experience longer-term risks for progressive chronic ESRD, which diminish patients' health-related quality of life and create a larger burden on the healthcare system. Although experimental models have yielded numerous promising agents, translation into clinical practice has been unsuccessful, possibly because of issues in clinical trial design, such as delayed drug administration,masking of therapeutic benefit by adverse events, and inadequate sample size. To address issues of clinical trial design, the National Institute of Diabetes and Digestive and Kidney Diseases sponsored a workshop titled "Clinical Trials in Acute Kidney Injury: Current Opportunities and Barriers" in December 2010. Workshop participants included representatives fromacademia, industry, and government agencies whose areas of expertise spanned basic science, clinical nephrology, critical care medicine, biostatistics, pharmacology, and drug development. This document summarizes the discussions of collaborative workgroups that addressed issues related to patient selection, study endpoints, the role of novel biomarkers, sample size and power calculations, and adverse events and pilot/feasibility studies in prevention and treatment of AKI. Companion articles outline the discussions of workgroups for model trials related to prevention or treatment of established AKI in different clinical settings, such as in patients with sepsis.

Original language	English (US)
Pages (from-to)	844-850
Number of pages	7
Journal	Clinical Journal of the American Society of Nephrology
Volume	7
Issue number	5
DOIs	https://doi.org/10.2215/CJN.12791211
State	Published - May 1 2012
Externally published	Yes

ASJC Scopus subject areas

Epidemiology
Critical Care and Intensive Care Medicine
Nephrology
Transplantation

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Palevsky, P. M., Molitoris, B. A., Okusa, M. D., Levin, A., Waikar, S. S., Wald, R., Chertow, G. M., Murray, P. T., Parikh, C. R., Shaw, A. D., Go, A. S., Faubel, S. G., Kellum, J. A., Chinchilli, V. M., Liu, K. D., Cheung, A. K., Weisbord, S. D., Chawla, L. S., Kaufman, J. S., ... Star, R. A. (2012). Design of clinical trials in acute kidney injury: Report from an NIDDK workshop on trial methodology. Clinical Journal of the American Society of Nephrology, 7(5), 844-850. https://doi.org/10.2215/CJN.12791211

Palevsky, PM, Molitoris, BA, Okusa, MD, Levin, A, Waikar, SS, Wald, R, Chertow, GM, Murray, PT, Parikh, CR, Shaw, AD, Go, AS, Faubel, SG, Kellum, JA, Chinchilli, VM, Liu, KD, Cheung, AK, Weisbord, SD, Chawla, LS, Kaufman, JS, Devarajan, P, Toto, RM, Hsu, CY, Greene, T, Mehta, RL, Stokes, JB, Thompson, AM, Taylor Thompson, B, Westenfelder, CS, Tumlin, JA, Warnock, DG, Shah, SV, Xie, Y, Duggan, EG, Kimmel, PL & Star, RA 2012, 'Design of clinical trials in acute kidney injury: Report from an NIDDK workshop on trial methodology', Clinical Journal of the American Society of Nephrology, vol. 7, no. 5, pp. 844-850. https://doi.org/10.2215/CJN.12791211

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title = "Design of clinical trials in acute kidney injury: Report from an NIDDK workshop on trial methodology",

abstract = "Acute kidney injury (AKI) remains a complex clinical problem associated with significant short-term morbidity and mortality and lacking effective pharmacologic interventions. Patients with AKI experience longer-term risks for progressive chronic ESRD, which diminish patients' health-related quality of life and create a larger burden on the healthcare system. Although experimental models have yielded numerous promising agents, translation into clinical practice has been unsuccessful, possibly because of issues in clinical trial design, such as delayed drug administration,masking of therapeutic benefit by adverse events, and inadequate sample size. To address issues of clinical trial design, the National Institute of Diabetes and Digestive and Kidney Diseases sponsored a workshop titled {"}Clinical Trials in Acute Kidney Injury: Current Opportunities and Barriers{"} in December 2010. Workshop participants included representatives fromacademia, industry, and government agencies whose areas of expertise spanned basic science, clinical nephrology, critical care medicine, biostatistics, pharmacology, and drug development. This document summarizes the discussions of collaborative workgroups that addressed issues related to patient selection, study endpoints, the role of novel biomarkers, sample size and power calculations, and adverse events and pilot/feasibility studies in prevention and treatment of AKI. Companion articles outline the discussions of workgroups for model trials related to prevention or treatment of established AKI in different clinical settings, such as in patients with sepsis.",

author = "Palevsky, {Paul M.} and Molitoris, {Bruce A.} and Okusa, {Mark D.} and Adeera Levin and Waikar, {Sushrut S.} and Ron Wald and Chertow, {Glenn M.} and Murray, {Patrick T.} and Parikh, {Chirag R.} and Shaw, {Andrew D.} and Go, {Alan S.} and Faubel, {Sarah G.} and Kellum, {John A.} and Chinchilli, {Vernon M.} and Liu, {Kathleen D.} and Cheung, {Alfred K.} and Weisbord, {Steven D.} and Chawla, {Lakhmir S.} and Kaufman, {James S.} and Prasad Devarajan and Toto, {Robert M.} and Hsu, {Chi Yuan} and Tom Greene and Mehta, {Ravindra L.} and Stokes, {John B.} and Thompson, {Aliza M.} and {Taylor Thompson}, B. and Westenfelder, {Christof S.} and Tumlin, {James A.} and Warnock, {David G.} and Shah, {Sudhir V.} and Yining Xie and Duggan, {Emily G.} and Kimmel, {Paul L.} and Star, {Robert A.}",

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AU - Palevsky, Paul M.

AU - Molitoris, Bruce A.

AU - Okusa, Mark D.

AU - Levin, Adeera

AU - Waikar, Sushrut S.

AU - Wald, Ron

AU - Chertow, Glenn M.

AU - Murray, Patrick T.

AU - Parikh, Chirag R.

AU - Shaw, Andrew D.

AU - Go, Alan S.

AU - Faubel, Sarah G.

AU - Kellum, John A.

AU - Chinchilli, Vernon M.

AU - Liu, Kathleen D.

AU - Cheung, Alfred K.

AU - Weisbord, Steven D.

AU - Chawla, Lakhmir S.

AU - Kaufman, James S.

AU - Devarajan, Prasad

AU - Toto, Robert M.

AU - Hsu, Chi Yuan

AU - Greene, Tom

AU - Mehta, Ravindra L.

AU - Stokes, John B.

AU - Thompson, Aliza M.

AU - Taylor Thompson, B.

AU - Westenfelder, Christof S.

AU - Tumlin, James A.

AU - Warnock, David G.

AU - Shah, Sudhir V.

AU - Xie, Yining

AU - Duggan, Emily G.

AU - Kimmel, Paul L.

AU - Star, Robert A.

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Design of clinical trials in acute kidney injury: Report from an NIDDK workshop on trial methodology

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