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Long‐term survival of participants in the CENTAUR trial of sodium phenylbutyrate‐taurursodiol in amyotrophic lateral sclerosis
Author(s) -
Pagai 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,
HeimanPatterson 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 Christi.,
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 ZiFan,
Cohen Joshua,
Klee Justin,
Leslie Kent,
Tanzi Rudolph E.,
Gilbert Walter,
Yeramian Patrick D.,
Schoenfeld David,
Cudkowicz Merit E.
Publication year - 2021
Publication title -
muscle and nerve
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.025
H-Index - 145
eISSN - 1097-4598
pISSN - 0148-639X
DOI - 10.1002/mus.27091
Subject(s) - hazard ratio , medicine , placebo , randomized controlled trial , amyotrophic lateral sclerosis , randomization , confidence interval , pathology , disease , alternative medicine
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.