PMID-28169285 – Plecanatide Phase III CIC Trial

PMID-28169285 – Plecanatide Phase III CIC Trial

Miner PB Jr, Koltun WD, Wiber GJ, et al. "A Randomized Phase III Clinical Trial of Plecanatide, a Uroguanylin Analog, in Patients With Chronic Idiopathic Constipation," Am J Gastroenterol, 2017;112(4):613-621.

Quick Reference

Property Value
PMID 28169285
DOI 10.1038/ajg.2016.611
Year 2017
Journal American Journal of Gastroenterology
Study Type Randomized Controlled Trial (Phase III)
Evidence Level I
Sample n=1,394 patients with CIC
Peptide(s) Studied Plecanatide

Key Findings

  • Plecanatide 3 mg and 6 mg both significantly increased durable overall CSBM responder rates vs placebo (21.0% and 19.5% vs 10.2%; P<0.001 for both)
  • Both doses significantly improved stool frequency, stool consistency, and straining
  • Onset of effect observed within the first week of treatment
  • Adverse event rates were similar across treatment and placebo groups
  • Diarrhea was the most common treatment-related adverse event (5.9% for 3 mg, 1.3% for 6 mg vs 1.3% placebo)
  • This pivotal trial supported FDA approval of plecanatide (Trulance) in 2017

Study Design

Multicenter, randomized, double-blind, placebo-controlled Phase III trial. Patients with CIC (modified Rome III criteria) were randomized 1:1:1 to plecanatide 3 mg, plecanatide 6 mg, or placebo taken once daily for 12 weeks. Primary endpoint: durable overall CSBM responder (>=3 CSBMs/week and increase of >=1 CSBM from baseline for >=9 of 12 treatment weeks).

Limitations

  • 12-week duration may not capture long-term efficacy/safety
  • Relatively modest absolute response rates (21% vs 10%)
  • Industry-sponsored trial (Synergy Pharmaceuticals)
  • Predominantly female population; generalizability to males uncertain
  • No active comparator arm

Clinical Relevance

This pivotal Phase III trial established plecanatide as an effective GC-C agonist for CIC, leading to FDA approval. Unlike linaclotide, plecanatide was designed to mimic endogenous uroguanylin with pH-dependent activation in the proximal small intestine, potentially offering a more physiological approach. The low diarrhea rate at both doses compared favorably with linaclotide, suggesting improved tolerability. This gives clinicians a second GC-C agonist option for patients who may not tolerate linaclotide.

Related

#research #RCT #evidence-level-I #gastrointestinal #plecanatide