Journal article
Effect of sodium phenylbutyrate/taurursodiol on tracheostomy/ventilation-free survival and hospitalisation in amyotrophic lateral sclerosis: long-term results from the CENTAUR trial
Journal of neurology, neurosurgery and psychiatry, Vol.93(8), pp.871-875
05/16/2022
DOI: 10.1136/jnnp-2022-329024
PMCID: PMC9304116
PMID: 35577511
Abstract
BackgroundCoformulated sodium phenylbutyrate/taurursodiol (PB/TURSO) was shown to prolong survival and slow functional decline in amyotrophic lateral sclerosis (ALS).ObjectiveDetermine whether PB/TURSO prolonged tracheostomy/ventilation-free survival and/or reduced first hospitalisation in participants with ALS in the CENTAUR trial.MethodsAdults with El Escorial Definite ALS ≤18 months from symptom onset were randomised to PB/ TURSO or placebo for 6 months. Those completing randomised treatment could enrol in an open-label extension (OLE) phase and receive PB/TURSO for ≤30 months. Times to the following individual or combined key events were compared in the originally randomised treatment groups over a period spanning trial start through July 2020 (longest postrandomisation follow-up, 35 months): death, tracheostomy, permanent assisted ventilation (PAV) and first hospitalisation.ResultsRisk of any key event was 47% lower in those originally randomised to PB/TURSO (n=87) versus placebo (n=48, 71% of whom received delayed-start PB/TURSO in the OLE phase) (HR=0.53; 95% CI 0.35 to 0.81; p=0.003). Risks of death or tracheostomy/PAV (HR=0.51; 95% CI 0.32 to 0.84; p=0.007) and first hospitalisation (HR=0.56; 95% CI 0.34 to 0.95; p=0.03) were also decreased in those originally randomised to PB/TURSO.ConclusionsEarly PB/TURSO prolonged tracheostomy/PAV-free survival and delayed first hospitalisation in ALS.Trial registration number NCT03127514; NCT03488524.
Details
- Title: Subtitle
- Effect of sodium phenylbutyrate/taurursodiol on tracheostomy/ventilation-free survival and hospitalisation in amyotrophic lateral sclerosis: long-term results from the CENTAUR trial
- Creators
- Sabrina Paganoni - Clinical Research InstituteSuzanne Hendrix - Pentara Corporation, Millcreek, Utah, USA.Samuel P Dickson - Pentara Corporation, Millcreek, Utah, USA.Newman Knowlton - Pentara Corporation, Millcreek, Utah, USA.James D Berry - Clinical Research InstituteMichael A Elliott - Swedish Medical CenterSamuel Maiser - Hennepin Healthcare Research InstituteChafic Karam - University of PennsylvaniaJames B Caress - Wake Forest UniversityMargaret Ayo Owegi - Memorial Medical CenterAdam Quick - The Ohio State UniversityJames Wymer - University of FloridaStephen A Goutman - University of Michigan–Ann ArborDaragh Heitzman - Texas NeurologyTerry D Heiman-Patterson - Temple UniversityCarlayne Jackson - The University of Texas Health Science Center at San AntonioColin Quinn - University of PennsylvaniaJeffrey D Rothstein - Department of Neurology, Johns Hopkins University Brain Science Institute, Baltimore, Maryland, USA.Edward J Kasarskis - University of KentuckyJonathan Katz - California Pacific Medical CenterLiberty Jenkins - California Pacific Medical CenterShafeeq S Ladha - Barrow Neurological InstituteTimothy M Miller - Hope Center for Neurological DisordersStephen N Scelsa - Icahn School of Medicine at Mount SinaiTuan H Vu - University of South FloridaChristina Fournier - Emory UniversityKristin M Johnson - Ochsner Health SystemAndrea Swenson - University of IowaNamita Goyal - University of California, IrvineGary L Pattee - Urology AssociatesSuma Babu - Clinical Research InstituteMarianne Chase - Harvard Medical SchoolDerek Dagostino - Clinical Research InstituteMeghan Hall - Barrow Neurological InstituteGale Kittle - Barrow Neurological InstituteMathew Eydinov - Clinical Research InstituteJoseph Ostrow - Clinical Research InstituteLindsay Pothier - Clinical Research InstituteRebecca Randall - Worldwide Clinical TrialsJeremy M Shefner - Barrow Neurological InstituteAlexander V Sherman - Clinical Research InstituteEric Tustison - Clinical Research InstitutePrasha Vigneswaran - Clinical Research InstituteHong Yu - Clinical Research InstituteJoshua Cohen - Amylyx Pharmaceuticals Inc, Cambridge, Massachusetts, USA.Justin Klee - Amylyx Pharmaceuticals Inc, Cambridge, Massachusetts, USA.Rudolph Tanzi - Department of Neurology, Genetics and Aging Research Unit, McCance Center for Brain Health, Massachusetts General Hospital, Harvard University, Boston, Massachusetts, USA.Walter Gilbert - Carl M. Loeb University Professor Emeritus and Chair of the Society of Fellows at Harvard, Harvard University, Cambridge, Massachusetts, USA.Patrick Yeramian - Amylyx Pharmaceuticals Inc, Cambridge, Massachusetts, USA.Merit Cudkowicz - Harvard Medical School
- Resource Type
- Journal article
- Publication Details
- Journal of neurology, neurosurgery and psychiatry, Vol.93(8), pp.871-875
- DOI
- 10.1136/jnnp-2022-329024
- PMID
- 35577511
- PMCID
- PMC9304116
- NLM abbreviation
- J Neurol Neurosurg Psychiatry
- ISSN
- 0022-3050
- eISSN
- 1468-330X
- Publisher
- BMJ Publishing Group Ltd
- Grant note
- Not Applicable / The ALS Association Not Applicable / ALS Finding a Cure Not Applicable / Amylyx Pharmaceuticals, Inc.
- Language
- English
- Date published
- 05/16/2022
- Academic Unit
- Neurology
- Record Identifier
- 9984303018502771
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