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S390 Efficacy and Safety of On-Demand Vonoprazan versus Placebo in the Treatment of Heartburn in Symptomatic Nonerosive Reflux Disease (NERD) Patients: A Phase 2 Randomized Controlled Trial

2022· article· en· W4316087129 on OpenAlexaff
Ronnie Fass, Michael F. Vaezi, Prateek Sharma, Rena Yadlapati, Barbara Hunt, Tom M. Harris, Neila Smith, Eckhard Leifke, David Armstrong

Bibliographic record

VenueThe American Journal of Gastroenterology · 2022
Typearticle
Languageen
FieldMedicine
TopicGastroesophageal reflux and treatments
Canadian institutionsMcMaster University
Fundersnot available
KeywordsMedicineHeartburnNerdPlaceboInternal medicineClinical endpointGastroenterologyRandomized controlled trialAntacidRefluxDrugDiseasePharmacologyGERD

Abstract

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Introduction: Current treatment for NERD is daily acid-suppressive therapy; however, on-demand treatment is an attractive option for long-term management. Vonoprazan, a potassium-competitive acid blocker, rapidly and profoundly suppresses gastric acid. The aim of this study was to evaluate the efficacy and safety of vonoprazan vs placebo for on-demand treatment of symptomatic NERD. Methods: This Phase 2, double-blind, placebo-controlled study (NCT04799158) enrolled NERD patients (normal endoscopy, heartburn episodes for ≥6 months, heartburn on ≥4/7 consecutive days) into a 4-week run-in period of once-daily vonoprazan 20mg. Patients without heartburn in the last 7 days of the run-in period were randomized 1:1:1:1 to receive vonoprazan 10mg, 20mg, 40mg, or placebo on-demand for 6 weeks. Patients were asked to take no more than one dose of study drug for 24h after a heartburn episode and to take no rescue antacids ≤3h after taking study drug. Patients recorded heartburn symptoms, drug and antacid use in an electronic diary. The primary endpoint was the % of evaluable heartburn episodes with complete and sustained relief (within 3h and with no further heartburn reported for 24h after taking study drug) during the on-demand treatment period. The onset of complete and sustained relief was evaluated within 30min and 1, 1.5, 2, and 3h after study drug. Results: Of 458 patients entering the run-in period, 207 (females: 125; mean age: 54y) were eligible and randomized to treatment. For the primary endpoint, in the vonoprazan 10, 20, and 40mg groups, 56.0% (201/359), 60.6% (198/327) and 70.0% (226/323) of heartburn episodes met the criteria for complete and sustained relief, respectively, vs 27.3% (101/370) for placebo (P< 0.0001 for each vonoprazan treatment vs placebo) (Table). Significant differences in complete and sustained relief occurred as early as 1h after study drug for all vonoprazan doses. In the on-demand period, 21.3% of patients receiving placebo reported a treatment-emergent adverse event (TEAE) vs 16.3%, 18.4%, and 16.7% of those on vonoprazan 10, 20, and 40mg, respectively. No TEAE was reported by >1 patient per group. No serious TEAEs were reported. Conclusion: The results suggest that on-demand vonoprazan treatment for the relief of episodic heartburn in NERD patients is efficacious and well-tolerated. On-demand vonoprazan treatment may offer NERD patients an attractive alternative to daily heartburn therapy. Table 1. - Comparison of efficacy endpoint results between the different vonoprazan doses and placebo Endpoint Vonoprazan10 mg n=52 Vonoprazan20 mg n=52 Vonoprazan40 mg n=51 Placebo n=52 Heartburn episodes with complete and sustained relief within 3 hoursa n/N evaluable episodesb (%) 201/359 (56.0) 198/327 (60.6) 226/323 (70.0) 101/370 (27.3) P-value (vs placebo) < 0.0001 < 0.0001 < 0.0001 - Timing of complete and sustained reliefa, n/N evaluable episodesb (%) Within 30 minutes 31/359 (8.6) 17/327 (5.2) 9/323 (2.8) 21/370 (5.7) P-value (vs placebo) 0.15 0.87 0.09 - Within 1 hour 101/359 (28.1) 63/327 (19.3) 74/323 (22.9) 44/370 (11.9) P-value (vs placebo) < 0.0001 0.0083 0.0002 - Within 1.5 hours 151/359 (42.1) 103/327 (31.5) 139/323 (43.0) 67/370 (18.1) P-value (vs placebo) < 0.0001 < 0.0001 < 0.0001 - Within 2 hours 182/359 (50.7) 151/327 (46.2) 187/323 (57.9) 81/370 (21.9) P-value (vs placebo) < 0.0001 < 0.0001 < 0.0001 - (a)Complete and sustained relief is defined as complete relief with no antacid taken within the indicated time frame after taking study drug and no further heartburn reported for 24 hours after taking study drug.(b)An evaluable heartburn episode is defined as any for which study drug was taken and for which the subject completed ≥1 entry in the heartburn episode diary.

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 machine prediction

Teacher imitation

Not calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.

metaresearch head score (Codex)0.002
metaresearch head score (Gemma)0.002
Version: metacan-v3-hybrid-931329e0061cValidation 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 score0.018
Threshold uncertainty score0.059

Distilled classifier scores by category (both heads)

CategoryCodexGemma
Metaresearch0.0020.002
Meta-epidemiology (narrow)0.0020.001
Meta-epidemiology (broad)0.0040.002
Bibliometrics0.0010.000
Science and technology studies0.0010.001
Scholarly communication0.0010.001
Open science0.0010.000
Research integrity0.0040.003
Insufficient payload (model declined to judge)0.0180.002

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 head0.011
GPT teacher head0.301
Teacher spread0.290 · 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 source (direct Gemma or distilled Codex), 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".

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Citations5
Published2022
Admission routes1
Has abstractyes

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