Journal article
Long-term survival of participants in the CENTAUR trial of sodium phenylbutyrate-taurursodiol in amyotrophic lateral sclerosis
Muscle & nerve, Vol.63(1), pp.31-39
01/01/2021
DOI: 10.1002/mus.27091
PMCID: PMC7820979
PMID: 33063909
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.
Details
- Title: Subtitle
- Long-term survival of participants in the CENTAUR trial of sodium phenylbutyrate-taurursodiol in amyotrophic lateral sclerosis
- Creators
- Sabrina Paganoni - Harvard UniversitySuzanne Hendrix - Pentara Corp, Millcreek, UT USASamuel P. Dickson - Pentara Corp, Millcreek, UT USANewman Knowlton - Pentara Corp, Millcreek, UT USAEric A. Macklin - Harvard UniversityJames D. Berry - Harvard UniversityMichael A. Elliott - Washington University in St. LouisSamuel Maiser - Hennepin Healthcare, Dept Neurol, Minneapolis, MN USAChafic Karam - Oregon Health & Science UniversityJames B. Caress - Wake Forest UniversityMargaret Ayo Owegi - University of Massachusetts AmherstAdam Quick - The Ohio State UniversityJames Wymer - University of FloridaStephen A. Goutman - University of Michigan–Ann ArborDaragh Heitzman - Ohio State Univ, Coll Med, Dept Neurol, Columbus, OH 43210 USATerry D. Heiman-Patterson - Temple UniversityCarlayne E. Jackson - The University of Texas Health Science Center at San AntonioColin Quinn - University of PennsylvaniaJeffrey D. Rothstein - Johns Hopkins UniversityEdward J. Kasarskis - University of KentuckyJonathan Katz - California Pacific Medical CenterLiberty Jenkins - California Pacific Medical CenterShafeeq Ladha - Barrow Neurological InstituteTimothy M. Miller - Washington University in St. LouisStephen N. Scelsa - Icahn School of Medicine at Mount SinaiTuan H. Vu - University of South FloridaChristina N. Fournier - Emory UniversityJonathan D. Glass - Emory UniversityKristin M. Johnson - Ochsner Health SystemAndrea Swenson - Emory UniversityNamita A. Goyal - University of California, IrvineGary L. Pattee - Ochsner Hlth Syst, Dept Neurol, New Orleans, LA USAPatricia L. Andres - Univ Iowa, Dept Neurol, Carver Coll Med, Iowa City, IA 52242 USASuma Babu - Harvard UniversityMarianne Chase - Harvard UniversityDerek Dagostino - Harvard UniversityMeghan Hall - Barrow Neurological InstituteGale Kittle - Barrow Neurological InstituteMatthew Eydinov - Harvard UniversityMichelle McGovern - Harvard UniversityJoseph Ostrow - Harvard UniversityLindsay Pothier - Harvard UniversityRebecca Randall - Barrow Neurological InstituteJeremy M. Shefner - Barrow Neurological InstituteAlexander Sherman - Harvard UniversityMaria E. St Pierre - Harvard Med Sch, Massachusetts Gen Hosp, Sean M Healey & AMG Ctr, Boston, MA 02115 USAEric Tustison - Harvard UniversityPrasha Vigneswaran - Harvard UniversityJason Walker - Harvard UniversityHong Yu - Harvard UniversityJames Chan - Harvard UniversityJanet Wittes - Stat Collaborat Inc, Washington, DC USAZi-Fan Yu - Stat Collaborat Inc, Washington, DC USAJoshua Cohen - Amylyx Pharmaceut Inc, Cambridge, MA USAJustin Klee - Amylyx Pharmaceut Inc, Cambridge, MA USAKent Leslie - Amylyx Pharmaceut Inc, Cambridge, MA USARudolph E. Tanzi - Harvard UniversityWalter Gilbert - Harvard UniversityPatrick D. Yeramian - Amylyx Pharmaceut Inc, Cambridge, MA USADavid Schoenfeld - Harvard UniversityMerit E. Cudkowicz - Harvard University
- Resource Type
- Journal article
- Publication Details
- Muscle & nerve, Vol.63(1), pp.31-39
- DOI
- 10.1002/mus.27091
- PMID
- 33063909
- PMCID
- PMC7820979
- NLM abbreviation
- Muscle Nerve
- ISSN
- 0148-639X
- eISSN
- 1097-4598
- Publisher
- Wiley
- Number of pages
- 9
- Grant note
- Amylyx Pharmaceuticals, Inc.; Takeda Pharmaceutical Company Ltd ALS Finding A Cure Foundation ALS Association
- Language
- English
- Date published
- 01/01/2021
- Academic Unit
- Neurology
- Record Identifier
- 9984303022902771
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