A Randomized, Double-Blind, Phase 3 Safety and Efficacy Study of Ridinilazole Versus Vancomycin for Treatment of Clostridioides difficile Infection: Clinical Outcomes With Microbiome and Metabolome Correlates of Response

Clin Infect Dis. 2024 Jun 14;78(6):1462-1472. doi: 10.1093/cid/ciad792.

Abstract

Background: Exposure to antibiotics predisposes to dysbiosis and Clostridioides difficile infection (CDI) that can be severe, recurrent (rCDI), and life-threatening. Nonselective drugs that treat CDI and perpetuate dysbiosis are associated with rCDI, in part due to loss of microbiome-derived secondary bile acid (SBA) production. Ridinilazole is a highly selective drug designed to treat CDI and prevent rCDI.

Methods: In this phase 3 superiority trial, adults with CDI, confirmed with a stool toxin test, were randomized to receive 10 days of ridinilazole (200 mg twice daily) or vancomycin (125 mg 4 times daily). The primary endpoint was sustained clinical response (SCR), defined as clinical response and no rCDI through 30 days after end of treatment. Secondary endpoints included rCDI and change in relative abundance of SBAs.

Results: Ridinilazole and vancomycin achieved an SCR rate of 73% versus 70.7%, respectively, a treatment difference of 2.2% (95% CI: -4.2%, 8.6%). Ridinilazole resulted in a 53% reduction in recurrence compared with vancomycin (8.1% vs 17.3%; 95% CI: -14.1%, -4.5%; P = .0002). Subgroup analyses revealed consistent ridinilazole benefit for reduction in rCDI across subgroups. Ridinilazole preserved microbiota diversity, increased SBAs, and did not increase the resistome. Conversely, vancomycin worsened CDI-associated dysbiosis, decreased SBAs, increased Proteobacteria abundance (∼3.5-fold), and increased the resistome.

Conclusions: Although ridinilazole did not meet superiority in SCR, ridinilazole greatly reduced rCDI and preserved microbiome diversity and SBAs compared with vancomycin. These findings suggest that treatment of CDI with ridinilazole results in an earlier recovery of gut microbiome health. Clinical Trials Registration.Ri-CoDIFy 1 and 2: NCT03595553 and NCT03595566.

Keywords: Clostridioides difficile; bile acids; microbiome; ridinilazole; vancomycin.

Publication types

  • Clinical Trial, Phase III
  • Randomized Controlled Trial
  • Multicenter Study

MeSH terms

  • Adult
  • Aged
  • Anti-Bacterial Agents* / adverse effects
  • Anti-Bacterial Agents* / therapeutic use
  • Benzimidazoles
  • Clostridioides difficile* / drug effects
  • Clostridium Infections* / drug therapy
  • Clostridium Infections* / microbiology
  • Double-Blind Method
  • Dysbiosis / chemically induced
  • Female
  • Gastrointestinal Microbiome* / drug effects
  • Humans
  • Male
  • Metabolome / drug effects
  • Middle Aged
  • Oxadiazoles / adverse effects
  • Oxadiazoles / therapeutic use
  • Pyridines
  • Treatment Outcome
  • Vancomycin* / adverse effects
  • Vancomycin* / therapeutic use

Substances

  • Vancomycin
  • ridinilazole
  • Anti-Bacterial Agents
  • Oxadiazoles
  • Benzimidazoles
  • Pyridines

Associated data

  • ClinicalTrials.gov/NCT03595553
  • ClinicalTrials.gov/NCT03595566