A Phase 2a randomized, single-center, double-blind, placebo-controlled study to evaluate the safety and preliminary efficacy of oral iOWH032 against cholera diarrhea in a controlled human infection model

PLoS Negl Trop Dis. 2021 Nov 18;15(11):e0009969. doi: 10.1371/journal.pntd.0009969. eCollection 2021 Nov.

Abstract

Cholera remains a major cause of infectious diarrhea globally. Despite the increased availability of cholera vaccines, there is still an urgent need for other effective interventions to reduce morbidity and mortality. Furthermore, increased prevalence of antibiotic-resistant Vibrio cholerae threatens the use of many drugs commonly used to treat cholera. We developed iOWH032, a synthetic small molecule inhibitor of the cystic fibrosis transmembrane conductance regulator chloride channel, as an antisecretory, host-directed therapeutic for cholera. In the study reported here, we tested iOWH032 in a Phase 2a cholera controlled human infection model. Forty-seven subjects were experimentally infected with V. cholerae El Tor Inaba strain N16961 in an inpatient setting and randomized to receive 500 mg iOWH032 or placebo by mouth every 8 hours for 3 days to determine the safety and efficacy of the compound as a potential treatment for cholera. We found that iOWH032 was generally safe and achieved a mean (± standard deviation) plasma level of 4,270 ng/mL (±2,170) after 3 days of oral dosing. However, the median (95% confidence interval) diarrheal stool output rate for the iOWH032 group was 25.4 mL/hour (8.9, 58.3), compared to 32.6 mL/hour (15.8, 48.2) for the placebo group, a reduction of 23%, which was not statistically significant. There was also no significant decrease in diarrhea severity and number or frequency of stools associated with iOWH032 treatment. We conclude that iOWH032 does not merit future development for treatment of cholera and offer lessons learned for others developing antisecretory therapeutic candidates that seek to demonstrate proof of principle in a cholera controlled human infection model study. Trial registration: This study is registered with ClinicalTrials.gov as NCT04150250.

Publication types

  • Clinical Trial, Phase II
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Administration, Oral
  • Adolescent
  • Adult
  • Cholera / drug therapy*
  • Cholera / metabolism
  • Cholera / microbiology
  • Cystic Fibrosis Transmembrane Conductance Regulator / antagonists & inhibitors
  • Cystic Fibrosis Transmembrane Conductance Regulator / metabolism
  • Diarrhea / drug therapy*
  • Diarrhea / metabolism
  • Diarrhea / microbiology
  • Double-Blind Method
  • Female
  • Humans
  • Hydroxyquinolines / administration & dosage*
  • Hydroxyquinolines / adverse effects
  • Male
  • Oxadiazoles / administration & dosage*
  • Oxadiazoles / adverse effects
  • Vibrio cholerae / physiology
  • Young Adult

Substances

  • CFTR protein, human
  • Hydroxyquinolines
  • Oxadiazoles
  • Cystic Fibrosis Transmembrane Conductance Regulator
  • IOWH-032
  • 5-carboxy-8-hydroxyquinoline

Associated data

  • ClinicalTrials.gov/NCT04150250

Grants and funding

This study was supported by a grant to PATH from the UK government (grant #300341-111) to E.L.d.H. and R.K.M.C.. https://www.gov.uk/government/organisations/foreign-commonwealth-development-office The funders had no role in study design, data collection and analysis, decision to publish, or preparation of the manuscript.