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Record W4362619875 · doi:10.1097/hep.0000000000000395

Seladelpar efficacy and safety at 3 months in patients with primary biliary cholangitis: ENHANCE, a phase 3, randomized, placebo-controlled study

2023· article· en· W4362619875 on OpenAlexaff
Gideon M. Hirschfield, Mitchell L. Shiffman, Aliya Gulamhusein, Kris V. Kowdley, John M. Vierling, Cynthia Levy, Andreas E. Kremer, Ehud Zigmond, Pietro Andreoné, Stuart C. Gordon, Christopher L. Bowlus, Eric Lawitz, Richard Aspinall, Daniel S. Pratt, K. L. Raikhelson, M.S. González-Huezo, Michael A. Heneghan, Sook‐Hyang Jeong, Alma Laura Ladrón De Guevara, Marlyn J. Mayo, George Ν. Dalekos, Joost P.H. Drenth, Ewa Janczewska, Barbara Leggett, Frederik Nevens, Vı́ctor Vargas, Eli Zuckerman, Christophe Corpechot, E Fassio, Holger Hinrichsen, Pietro Invernizzi, Palak Trivedi, Lisa Forman, David E.J. Jones, Stephen Ryder, Mark G. Swain, Alexandra Steinberg, Pol Boudes, Yun‐Jung Choi, Charles A. McWherter

Bibliographic record

VenueHepatology · 2023
Typearticle
Languageen
FieldMedicine
TopicLiver Diseases and Immunity
Canadian institutionsUniversity of CalgaryMcGill University Health CentreUniversity of TorontoUniversity Health Network
FundersCilagJanssen PharmaceuticalsNovartis PharmaGenentechNorgineAOP OrphanEisaiSwedish Orphan BiovitrumNovo NordiskDr. Falk PharmaAbbott LaboratoriesNational Institute for Health and Care ResearchPfizerModernaCelgeneBioCrystLoxo OncologyAlnylam PharmaceuticalsCook MedicalIpsenGilead SciencesGlaxoSmithKlineU.S. Department of JusticeAllerganAstraZenecaBristol-Myers SquibbEli Lilly and CompanyNGM BiopharmaceuticalsAmgenKowa CompanyExelixisSanofi
KeywordsMedicinePlaceboRandomized controlled trialBiliary tractInternal medicineGastroenterologyPathologyAlternative medicine

Abstract

fetched live from OpenAlex

BACKGROUND AND AIMS: ENHANCE was a phase 3 study that evaluated efficacy and safety of seladelpar, a selective peroxisome proliferator-activated receptor-δ (PPAR) agonist, versus placebo in patients with primary biliary cholangitis with inadequate response or intolerance to ursodeoxycholic acid (UDCA). APPROACH AND RESULTS: Patients were randomized 1:1:1 to oral seladelpar 5 mg (n=89), 10 mg (n=89), placebo (n=87) daily (with UDCA, as appropriate). Primary end point was a composite biochemical response [alkaline phosphatase (ALP) < 1.67×upper limit of normal (ULN), ≥15% ALP decrease from baseline, and total bilirubin ≤ ULN] at month 12. Key secondary end points were ALP normalization at month 12 and change in pruritus numerical rating scale (NRS) at month 6 in patients with baseline score ≥4. Aminotransferases were assessed. ENHANCE was terminated early following an erroneous safety signal in a concurrent, NASH trial. While blinded, primary and secondary efficacy end points were amended to month 3. Significantly more patients receiving seladelpar met the primary end point (seladelpar 5 mg: 57.1%, 10 mg: 78.2%) versus placebo (12.5%) ( p < 0.0001). ALP normalization occurred in 5.4% ( p =0.08) and 27.3% ( p < 0.0001) of patients receiving 5 and 10 mg seladelpar, respectively, versus 0% receiving placebo. Seladelpar 10 mg significantly reduced mean pruritus NRS versus placebo [10 mg: -3.14 ( p =0.02); placebo: -1.55]. Alanine aminotransferase decreased significantly with seladelpar versus placebo [5 mg: 23.4% ( p =0.0008); 10 mg: 16.7% ( p =0.03); placebo: 4%]. There were no serious treatment-related adverse events. CONCLUSIONS: Patients with primary biliary cholangitis (PBC) with inadequate response or intolerance to UDCA who were treated with seladelpar 10 mg had significant improvements in liver biochemistry and pruritus. Seladelpar appeared safe and well tolerated.

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)0.000
Version: codex-gemma-dda1882f352aValidation status: machine_predicted_unvalidated
Candidate categoriesnone
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 score0.066
Threshold uncertainty score0.602

Codex and Gemma teacher scores by category

CategoryCodexGemma
Metaresearch0.0010.000
Meta-epidemiology (narrow)0.0000.000
Meta-epidemiology (broad)0.0010.000
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 head0.008
GPT teacher head0.267
Teacher spread0.260 · 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.

The models applied no category: nothing in the taxonomy fit this work.
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

Citations113
Published2023
Admission routes1
Has abstractyes

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