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Randomized Controlled Trial
. 2023 Apr;35(4):e14523.
doi: 10.1111/nmo.14523. Epub 2023 Jan 9.

A randomized, double-blind, placebo-controlled, phase 2b study of the efficacy and safety of velusetrag in subjects with diabetic or idiopathic gastroparesis

Thomas L Abell  1 , Braden Kuo  2 , Tuba Esfandyari  3 , Nathan D Pfeifer  4 , Maria Grimaldi  5 , Cecilia Renzulli  5 , Raffaella Tacchi  5 , Kefei Zhou  4 , Chris N Barnes  4 , Deanna D Nguyen  4 , Linda Nguyen  6 , Nicholas J Talley  7 , Richard McCallum  8
Affiliations
Randomized Controlled Trial

A randomized, double-blind, placebo-controlled, phase 2b study of the efficacy and safety of velusetrag in subjects with diabetic or idiopathic gastroparesis

Thomas L Abell et al. Neurogastroenterol Motil. 2023 Apr.

Abstract

Background: This study assessed the efficacy and safety of velusetrag-a 5-HT4 agonist with pan-gastrointestinal prokinetic activity-for gastroparesis symptom management and gastric emptying (GE).

Methods: In this multicenter, double-blind, randomized, placebo-controlled study, subjects with diabetic or idiopathic gastroparesis received velusetrag 5, 15, or 30 mg or placebo for 12 weeks. The primary efficacy outcome was a 7-day mean Gastroparesis Cardinal Symptom Index 24-h composite score (GCSI-24H) change from baseline at week 4; GE was evaluated using scintigraphy (GES) and breath tests, and safety from adverse events (AEs).

Key results: 232 subjects (183 females; 113 idiopathic gastroparesis) received treatment from February 2015 through June 2017. Least-squares mean improvement from baseline GCSI-24H (primary endpoint) at week 4 was -1.5 following velusetrag 5 mg vs -1.1 following placebo (treatment difference, -0.4; 95% confidence interval, -0.75 to -0.03; nominal p = 0.0327; Hochberg-adjusted p = 0.0980 [not significant]). Symptom improvement from baseline was achieved only with velusetrag 5 mg, which resulted in greater improvement from baseline vs placebo in all gastroparesis core symptoms, especially in subjects with idiopathic gastroparesis. Improvement from baseline GE by GES was greater in subjects receiving velusetrag (all doses) vs placebo; >70% of subjects receiving velusetrag 30 mg had GE normalization at 4 h. Treatment-emergent AEs were generally mild.

Conclusions and inferences: Velusetrag treatment was generally well-tolerated and associated with improved GE vs placebo in subjects with diabetic or idiopathic gastroparesis; however, only the lowest dose, velusetrag 5 mg, was associated with short-term improvement in gastroparesis symptoms.

Clinicaltrials: GOV: NCT02267525.

Keywords: 5-HT4 receptor agonist; GCSI-24H; gastric emptying; gastroparesis; patient-reported outcomes; velusetrag.

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References

REFERENCES

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