biostudies-literatureUnknown89(2)van Eijk RPA<h4>Objectives</h4>Plasma creatinine is a predictor of survival in amyotrophic lateral sclerosis (ALS). It remains, however, to be established whether it can monitor disease progression and serve as surrogate endpoint in clinical trials.<h4>Methods</h4>We used clinical trial data from three cohorts of clinical trial participants in the LITRA, EMPOWER and PROACT studies. Longitudinal associations between functional decline, muscle strength and survival with plasma creatinine were assessed. Results were translated to trial design in terms of sample size and power.<h4>Results</h4>A total of 13 564 measurements were obtained for 1241 patients. The variability between patients in rate of decline was lower in plasma creatinine than in ALS functional rating scale-Revised (ALSFRS-R; p<0.001). The Journal of neurology, neurosurgery, and psychiatry156-161https://www.ebi.ac.uk/biostudies/studies/S-EPMC5800333biostudies-literatureMonitoring disease progression with plasma creatinine in amyotrophic lateral sclerosis clinical trials.PMC5800333van den Berg LHFerguson TAEijkemans MJCNikolakopoulos SVeldink JHvan Eijk RPAfalseMonitoring disease progression with plasma creatinine in amyotrophic lateral sclerosis clinical trials.<h4>Objectives</h4>Plasma creatinine is a predictor of survival in amyotrophic lateral sclerosis (ALS). It remains, however, to be established whether it can monitor disease progression and serve as surrogate endpoint in clinical trials.<h4>Methods</h4>We used clinical trial data from three cohorts of clinical trial participants in the LITRA, EMPOWER and PROACT studies. Longitudinal associations between functional decline, muscle strength and survival with plasma creatinine were assessed. Results were translated to trial design in terms of sample size and power.<h4>Results</h4>A total of 13 564 measurements were obtained for 1241 patients. The variability between patients in rate of decline was lower in plasma creatinine than in ALS functional rating scale-Revised (ALSFRS-R; p<0.001). The 2018-01-01T00:00:00Z2018 Feb2026-06-17T06:10:34.347Z2026-06-17T03:07:44.678ZS-EPMC58003332908486810.1136/jnnp-2017-317077