Trial reportMuscle & nerve2021
Long-term survival of participants in the CENTAUR trial of sodium phenylbutyrate-taurursodiol in amyotrophic lateral sclerosis.
Registry-linked trialOpen access · hybridAbstract readRandomized Controlled Trial
In one paragraph
Trial report in Muscle & nerve, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03488524 (Evaluation of the Safety, Tolerability, Efficacy, and Activity of AMX0035, a Fixed Combination of Phenylbutyrate), which is not on this map. Cited by 97 papers, 4 of them syntheses that pooled it.
0numbers the graph read from it
0cells of the map it votes in
97citing papers in PubMed, 4 pooled it
9.7field-weighted citation impact, top 1% of its field
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Evaluation of the Safety, Tolerability, Efficacy, and Activity of AMX0035, a Fixed Combination of Phenylbutyrate (PB) and Tauroursodeoxycholic Acid (TUDCA), for Treatment of Amyotrophic Lateral Sclerosis: Open-Label Extension
Who cites it
97 citing papers in PubMed, 4 syntheses or guidelines pooled it, 174 citations in OpenAlex.
- Biomarkers in ALS trials: from discovery to clinical utility.Frontiers in neuroscience · 2025Pooled it
- The Role of Short-Chain Fatty Acids in Microbiota-Gut-Brain Cross-Talk with a Focus on Amyotrophic Lateral Sclerosis: A Systematic Review.International journal of molecular sciences · 2023Pooled it
- Pooled it
- Perampanel for amyotrophic lateral sclerosis: A systematic review and meta-analysis.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2022Pooled it
- Bile acid metabolites predict multiple sclerosis progression and supplementation is safe in progressive disease.Med (New York, N.Y.) · 2025Trial
- Tetramethylpyrazine Nitrone in Amyotrophic Lateral Sclerosis: A Randomized Clinical Trial.JAMA network open · 2025Trial
- Analysis of sodium phenylbutyrate and taurursodiol survival effect in ALS using external controls.Annals of clinical and translational neurology · 2023Trial
- Impact of Early Non-Invasive Ventilation in Amyotrophic Lateral Sclerosis: A multicenter Randomized Controlled Trial.Journal of neuromuscular diseases · 2023Trial
- Survival analyses from the CENTAUR trial in amyotrophic lateral sclerosis: Evaluating the impact of treatment crossover on outcomes.Muscle & nerve · 2022Trial
- Long-term survival of participants in the CENTAUR trial of sodium phenylbutyrate-taurursodiol in amyotrophic lateral sclerosis.Muscle & nerve · 2021Trial
- Article
- Sensory Cortical Hyperexcitability in Amyotrophic Lateral Sclerosis Involves a Broad Hand Representation Within the Primary Somatosensory Cortex.Muscle & nerve · 2026Article
- Review
- Extracellular Pgk1 or Its Derived Short Peptide Interacted with Membrane-Associated Enolase 2 Receptor: A Potential Therapy for ALS Motor Neuron Degeneration.Biomolecules · 2026Review
- Efficacy of Sodium Phenylbutyrate-Taurursodiol in Amyotrophic Lateral Sclerosis: A Systematic Review and Meta-Analysis.Annals of Indian Academy of Neurology · 2026Article
- T-2 Toxin Neurotoxicity: Molecular Mechanisms and Emerging Chemoprotective Strategies.Antioxidants (Basel, Switzerland) · 2026Review
- Therapeutic Challenges and Future Breakthroughs in Amyotrophic Lateral Sclerosis: From Precision Medicine to Innovative Trial Design.Drug design, development and therapy · 2026Review
- The HDAC Inhibitor Butyrate Reduces SOD1 Aggregation and Improves Motor Function inInternational journal of cell biology · 2026Article
- Development of Antisense Oligonucleotide Gapmers for Amyotrophic Lateral Sclerosis.Methods in molecular biology (Clifton, N.J.) · 2026Article
- Navigating the landscape of protein folding and proteostasis: from molecular chaperones to therapeutic innovations.Signal transduction and targeted therapy · 2025Review
37 more citing papers are in PubMed but not listed here.
Corrections and comments
- Commented on by
Authors and funding
61 authors at 20 institutions in 2 countries.
Sabrina PaganoniSean M. Healey & AMG Center for ALS & the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts.ORCID 0000-0003-0505-1168
Suzanne HendrixPentara Corporation, Millcreek, Utah.
Samuel P DicksonPentara Corporation, Millcreek, Utah.
Newman KnowltonPentara Corporation, Millcreek, Utah.
Eric A MacklinDepartment of Medicine, Biostatistics Center, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts.
James D BerrySean M. Healey & AMG Center for ALS & the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts.
Michael A ElliottSwedish Neuroscience Institute, Seattle, Washington.
Samuel MaiserDepartments of Neurology and Medicine, Hennepin Healthcare, Minneapolis, Minnesota.
Chafic KaramDepartment of Neurology, Oregon Health & Science University, Portland, Oregon.
James B CaressDepartment of Neurology, Wake Forest School of Medicine, Winston-Salem, North Carolina.
Margaret Ayo OwegiDepartment of Neurology, University of Massachusetts Memorial Medical Center, Worcester, Massachusetts.
Adam QuickDepartment of Neurology, The Ohio State University College of Medicine, Columbus, Ohio.
James WymerDepartment of Neurology, University of Florida College of Medicine, Gainesville, Florida.
Stephen A GoutmanDepartment of Neurology, University of Michigan, Ann Arbor, Michigan.ORCID 0000-0001-8780-6637
Daragh HeitzmanTexas Neurology, Dallas, Texas.
Terry D Heiman-PattersonDepartment of Neurology, Lewis Katz School of Medicine, Temple University, Philadelphia, Pennsylvania.
Carlayne E JacksonDepartment of Neurology, The University of Texas Health Science Center at San Antonio, San Antonio, Texas.
Colin QuinnDepartment of Neurology, University of Pennsylvania Perelman School of Medicine, Philadelphia, Pennsylvania.
Jeffrey D RothsteinBrain Science Institute and Department of Neurology, Johns Hopkins University, Baltimore, Maryland.
Edward J KasarskisDepartment of Neurology, University of Kentucky College of Medicine, Lexington, Kentucky.
Jonathan KatzCalifornia Pacific Medical Center Research Institute and Forbes Norris MDA/ALS Research and Treatment Center, San Francisco, California.
Liberty JenkinsCalifornia Pacific Medical Center Research Institute and Forbes Norris MDA/ALS Research and Treatment Center, San Francisco, California.
Shafeeq LadhaDepartment of Neurology, Gregory W. Fulton ALS Center, Barrow Neurological Institute, Phoenix, Arizona.
Timothy M MillerDepartment of Neurology, Washington University School of Medicine in St. Louis, St. Louis, Missouri.
Stephen N ScelsaDepartment of Neurology, Mount Sinai Beth Israel, Icahn School of Medicine at Mount Sinai, New York, New York.
Tuan H VuDepartment of Neurology, University of South Florida Morsani College of Medicine, Tampa, Florida.
Christina N FournierDepartments of Neurology and Pathology, Emory University School of Medicine, Atlanta, Georgia.
Jonathan D GlassDepartments of Neurology and Pathology, Emory University School of Medicine, Atlanta, Georgia.
Kristin M JohnsonDepartment of Neurology, Ochsner Health System, New Orleans, Louisiana.
Andrea SwensonDepartment of Neurology, University of Iowa Carver College of Medicine, Iowa City, Iowa.
Namita A GoyalDepartment of Neurology, University of California, Irvine School of Medicine, Irvine, California.
Gary L PatteeNeurology Associates, Lincoln, Nebraska.
Patricia L AndresIndependent Consultant, Nobleboro, Maine.
Suma BabuSean M. Healey & AMG Center for ALS & the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts.
Marianne ChaseSean M. Healey & AMG Center for ALS & the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts.
Derek DagostinoSean M. Healey & AMG Center for ALS & the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts.
Meghan HallDepartment of Neurology, Gregory W. Fulton ALS Center, Barrow Neurological Institute, Phoenix, Arizona.
Gale KittleDepartment of Neurology, Gregory W. Fulton ALS Center, Barrow Neurological Institute, Phoenix, Arizona.
Matthew EydinovSean M. Healey & AMG Center for ALS & the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts.
Michelle McGovernSean M. Healey & AMG Center for ALS & the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts.
Joseph OstrowSean M. Healey & AMG Center for ALS & the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts.
Lindsay PothierSean M. Healey & AMG Center for ALS & the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts.
Rebecca RandallDepartment of Neurology, Gregory W. Fulton ALS Center, Barrow Neurological Institute, Phoenix, Arizona.
Jeremy M ShefnerDepartment of Neurology, Gregory W. Fulton ALS Center, Barrow Neurological Institute, Phoenix, Arizona.
Alexander V ShermanSean M. Healey & AMG Center for ALS & the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts.
Maria E St PierreSean M. Healey & AMG Center for ALS & the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts.
Eric TustisonSean M. Healey & AMG Center for ALS & the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts.
Prasha VigneswaranSean M. Healey & AMG Center for ALS & the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts.
Jason WalkerSean M. Healey & AMG Center for ALS & the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts.
Hong YuSean M. Healey & AMG Center for ALS & the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts.
James ChanDepartment of Medicine, Biostatistics Center, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts.
Janet WittesStatistics Collaborative, Inc., Washington, District of Columbia.
Zi-Fan YuStatistics Collaborative, Inc., Washington, District of Columbia.
Joshua CohenAmylyx Pharmaceuticals, Inc., Cambridge, Massachusetts.
Justin KleeAmylyx Pharmaceuticals, Inc., Cambridge, Massachusetts.
Kent LeslieAmylyx Pharmaceuticals, Inc., Cambridge, Massachusetts.
Rudolph E TanziSean M. Healey & AMG Center for ALS & the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts.
Walter GilbertHarvard University, Cambridge, Massachusetts.
Patrick D YeramianAmylyx Pharmaceuticals, Inc., Cambridge, Massachusetts.
David SchoenfeldDepartment of Medicine, Biostatistics Center, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts.
Merit E CudkowiczSean M. Healey & AMG Center for ALS & the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts.
Massachusetts General Hospital · USBarrow Neurological Institute · USAmplyx Pharmaceuticals (United States) · USHarvard University · USTetraLogic Pharmaceuticals (United States) · USCalifornia Pacific Medical Center · USEmory University · USStatistics Collaborative · USHarvard University Press · USHennepin Healthcare Research Institute · USJohns Hopkins University · USMcBride and Associates (United States) · USMemorial Medical Center · USMount Sinai Beth Israel · USOchsner Health System · USOldham Council · GBOregon Health & Science University · USSwedish Medical Center · USTemple University · USTexas Neurology · US
Funding
University of California Health Participation in the National COVID Cohort Collaborative (N3C)UL1TR001414 · NCATS · UNIVERSITY OF CALIFORNIA-IRVINE · PI COOPER, DAN M, VILAIN, ERIC J. · 2015 to 2023
$35.1MWake Forest Clinical and Translational Science AwardUL1TR001420 · NCATS · WAKE FOREST UNIVERSITY HEALTH SCIENCES · PI ARD, JAMY D, FOLEY, KRISTIE L · 2015 to 2023
$32.3MNCATS NIH HHS UL1 TR001414NCATS NIH HHS UL1 TR001420
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.
Indexed as
AdolescentAdultAgedAged, 80 and overAmyotrophic Lateral SclerosisDouble-Blind MethodFemaleHumansMaleMiddle AgedNeuroprotective AgentsPhenylbutyratesTaurochenodeoxycholic AcidTimeYoung Adult4-phenylbutyric acidNeuroprotective AgentsPhenylbutyratesTaurochenodeoxycholic Acidursodoxicoltaurineamyotrophic lateral sclerosisCENTAURmotor neuron diseasesodium phenylbutyrate-taurursodiolsurvival analysis
Identifiers
What OpenQuestion holds
Textmetadata
LicenceCC BY-NC
Read underepoch 390
Registered trials
NCT03488524ClinicalTrials.gov
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.