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Record W4321077653 · doi:10.1038/s41591-022-02200-8

Tropifexor for nonalcoholic steatohepatitis: an adaptive, randomized, placebo-controlled phase 2a/b trial

2023· article· en· W4321077653 on OpenAlexfundno aff
Arun J. Sanyal, Patricia López, Eric Lawitz, Kathryn Jean Lucas, Juergen Loeffler, Won Kim, George Boon‐Bee Goh, Jee‐Fu Huang, Carla Serra, Pietro Andreoné, Yi‐Cheng Chen, Stanley H. Hsia, Vlad Ratziu, Diego Aizenberg, Hiroshi Tobita, Aasim Sheikh, John M. Vierling, Yoon Jun Kim, Hideyuki Hyogo, Dean Tai, Zachary Goodman, F Schaefer, Ian R. I. Carbarns, Sophie Lamle, Miljen Martić, Nikolai V. Naoumov, Clifford A. Brass

Bibliographic record

VenueNature Medicine · 2023
Typearticle
Languageen
FieldMedicine
TopicLiver Disease Diagnosis and Treatment
Canadian institutionsnot available
FundersJanssen PharmaceuticalsNational Institute on Alcohol Abuse and AlcoholismNovartis PharmaAstraZenecaAllerganRegeneron PharmaceuticalsNational Institutes of HealthNovo NordiskVirginia Commonwealth UniversityEisaiIntercept PharmaceuticalsMallinckrodt PharmaceuticalsIonis PharmaceuticalsGenentechBoston Scientific CorporationValeant Pharmaceuticals InternationalGilead SciencesNational Institute of Diabetes and Digestive and Kidney DiseasesSanofiAmgenNGM BiopharmaceuticalsPfizerCelgeneEli Lilly and CompanyBristol-Myers Squibb
KeywordsPlaceboTolerabilityMedicineInternal medicineNonalcoholic steatohepatitisGastroenterologyAdverse effectRandomized controlled trialFarnesoid X receptorNonalcoholic fatty liver diseaseFatty liverPathologyChemistry

Abstract

fetched live from OpenAlex

Abstract The multimodal activities of farnesoid X receptor (FXR) agonists make this class an attractive option to treat nonalcoholic steatohepatitis. The safety and efficacy of tropifexor, an FXR agonist, in a randomized, multicenter, double-blind, three-part adaptive design, phase 2 study, in patients with nonalcoholic steatohepatitis were therefore assessed. In Parts A + B, 198 patients were randomized to receive tropifexor (10–90 μg) or placebo for 12 weeks. In Part C, 152 patients were randomized to receive tropifexor 140 µg, tropifexor 200 µg or placebo (1:1:1) for 48 weeks. The primary endpoints were safety and tolerability to end-of-study, and dose response on alanine aminotransferase (ALT), aspartate aminotransferase (AST) and hepatic fat fraction (HFF) at week 12. Pruritus was the most common adverse event in all groups, with a higher frequency in the 140- and 200-µg tropifexor groups. Decreases from baseline in ALT and HFF were greater with tropifexor versus placebo at week 12, with a relative decrease in least squares mean from baseline observed with all tropifexor doses for ALT (tropifexor 10–90-μg dose groups ranged from −10.7 to −16.5 U l −1 versus placebo (−7.8 U l −1 ) and tropifexor 140- and 200-μg groups were −18.0 U l −1 and −23.0 U l −1 , respectively, versus placebo (−8.3 U l −1 )) and % HFF (tropifexor 10–90-μg dose groups ranged from −7.48% to −15.04% versus placebo (−6.19%) and tropifexor 140- and 200-μg groups were −19.07% and −39.41%, respectively, versus placebo (−10.77%)). Decreases in ALT and HFF were sustained up to week 48; however, similar trends in AST with tropifexor at week 12 were not observed. As with other FXR agonists, dose-related pruritus was frequently observed. Clinicaltrials.gov registration: NCT02855164

Fetched live from OpenAlex and de-inverted. Abstracts are not stored in this database: the inverted indexes are 8.6 GB of the frame’s 9.3 GB of text, and the host has 13 GB free.

How this classification was reachedexpand

Full frame distilled prediction

Teacher imitation

Not calibrated prevalence, not ground truth. Human validation pending. Learned from the 10,348 direct Codex labels and 10,348 direct Gemma labels. Candidate is the union of thresholded teacher heads; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels or direct frontier model labels.

metaresearch head score (Codex)0.001
metaresearch head score (Gemma)0.002
Version: codex-gemma-dda1882f352aValidation status: machine_predicted_unvalidated
Candidate categoriesMeta-epidemiology (narrow)
Consensus categoriesnone
DomainCandidate signal: none · Consensus signal: none
Study designCandidate signal: Randomized trial · Consensus signal: Randomized trial
GenreCandidate signal: Empirical · Consensus signal: Empirical
Teacher disagreement score0.025
Threshold uncertainty score1.000

Codex and Gemma teacher scores by category

CategoryCodexGemma
Metaresearch0.0010.002
Meta-epidemiology (narrow)0.0000.000
Meta-epidemiology (broad)0.0020.001
Bibliometrics0.0000.000
Science and technology studies0.0000.000
Scholarly communication0.0000.000
Open science0.0000.000
Research integrity0.0000.000
Insufficient payload (model declined to judge)0.0000.000

Machine scores (provisional)

The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.

Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.

Opus teacher head0.032
GPT teacher head0.378
Teacher spread0.346 · how far apart the two teachers sit on this one work
Validation statusscore_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from it

Classification

machine, unvalidated

Machine predicted; a candidate call from one teacher head, not a consensus.

Study designRandomized trial
Domainnot available
GenreEmpirical

How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".

Quick stats

Citations163
Published2023
Admission routes1
Has abstractyes

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