Long-term survival of participants in the CENTAUR trial of sodium phenylbutyrate-taurursodiol in amyotrophic lateral sclerosis


Sabrina Paganoni, Harvard Medical School
Suzanne Hendrix, Pentara Corporation
Samuel P. Dickson, Pentara Corporation
Newman Knowlton, Pentara Corporation
Eric A. Macklin, Harvard Medical School
James D. Berry, Harvard Medical School
Michael A. Elliott, Swedish Medical Center, Seattle
Samuel Maiser, Departments of Neurology
Chafic Karam, Oregon Health & Science University
James B. Caress, Wake Forest School of Medicine
Margaret Ayo Owegi, UMass Memorial Medical Center
Adam Quick, The Ohio State University College of Medicine
James Wymer, University of Florida College of Medicine
Stephen A. Goutman, University of Michigan, Ann Arbor
Daragh Heitzman, Texas Neurology
Terry D. Heiman-Patterson, Lewis Katz School of Medicine
Carlayne E. Jackson, University of Texas Health Science Center at San Antonio
Colin Quinn, University of Pennsylvania Perelman School of Medicine
Jeffrey D. Rothstein, Johns Hopkins University
Edward J. Kasarskis, University of Kentucky College of Medicine
Jonathan Katz, California Pacific Medical Center
Liberty Jenkins, California Pacific Medical Center
Shafeeq Ladha, Barrow Neurological InstituteFollow
Timothy M. Miller, Washington University School of Medicine in St. Louis
Stephen N. Scelsa, Icahn School of Medicine at Mount Sinai
Tuan H. Vu, Morsani College of Medicine
Christina N. Fournier, Emory University School of Medicine
Jonathan D. Glass, Emory University School of Medicine
Kristin M. Johnson, Ochsner Health System
Andrea Swenson, University of Iowa Carver College of Medicine
Namita A. Goyal, UCI School of Medicine

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© 2020 The Authors. Muscle & Nerve published by Wiley Periodicals LLC. An orally administered, fixed-dose coformulation of sodium phenylbutyrate-taurursodiol (PB-TURSO) significantly slowed functional decline in a randomized, placebo-controlled, phase 2 trial in ALS (CENTAUR). Herein we report results of a long-term survival analysis of participants in CENTAUR. In CENTAUR, adults with ALS were randomized 2:1 to PB-TURSO or placebo. Participants completing the 6-month (24-week) randomized phase were eligible to receive PB-TURSO in the open-label extension. An all-cause mortality analysis (35-month maximum follow-up post-randomization) incorporated all randomized participants. Participants and site investigators were blinded to treatment assignments through the duration of follow-up of this analysis. Vital status was obtained for 135 of 137 participants originally randomized in CENTAUR. Median overall survival was 25.0 months among participants originally randomized to PB-TURSO and 18.5 months among those originally randomized to placebo (hazard ratio, 0.56; 95% confidence interval, 0.34-0.92; P =.023). Initiation of PB-TURSO treatment at baseline resulted in a 6.5-month longer median survival as compared with placebo. Combined with results from CENTAUR, these results suggest that PB-TURSO has both functional and survival benefits in ALS.

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Muscle and Nerve









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