- 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
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.