e-Article
Long‐term survival of participants in the CENTAUR trial of sodium phenylbutyrate‐taurursodiol in amyotrophic lateral sclerosis
Document Type
article
Author
Paganoni, Sabrina; Hendrix, Suzanne; Dickson, Samuel P; Knowlton, Newman; Macklin, Eric A; Berry, James D; Elliott, Michael A; Maiser, Samuel; Karam, Chafic; Caress, James B; Owegi, Margaret Ayo; Quick, Adam; Wymer, James; Goutman, Stephen A; Heitzman, Daragh; Heiman‐Patterson, Terry D; Jackson, Carlayne E; Quinn, Colin; Rothstein, Jeffrey D; Kasarskis, Edward J; Katz, Jonathan; Jenkins, Liberty; Ladha, Shafeeq; Miller, Timothy M; Scelsa, Stephen N; Vu, Tuan H; Fournier, Christina N; Glass, Jonathan D; Johnson, Kristin M; Swenson, Andrea; Goyal, Namita A; Pattee, Gary L; Andres, Patricia L; Babu, Suma; Chase, Marianne; Dagostino, Derek; Hall, Meghan; Kittle, Gale; Eydinov, Matthew; McGovern, Michelle; Ostrow, Joseph; Pothier, Lindsay; Randall, Rebecca; Shefner, Jeremy M; Sherman, Alexander V; St Pierre, Maria E; Tustison, Eric; Vigneswaran, Prasha; Walker, Jason; Yu, Hong; Chan, James; Wittes, Janet; Yu, Zi‐Fan; Cohen, Joshua; Klee, Justin; Leslie, Kent; Tanzi, Rudolph E; Gilbert, Walter; Yeramian, Patrick D; Schoenfeld, David; Cudkowicz, Merit E
Source
Muscle & Nerve. 63(1)
Subject
Language
Abstract
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.