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Status unknownNCT05376228PSC-VancUpdated Jun 1, 2022

A Systems Biology Approach for Identification of Host and Microbial Mechanisms and Druggable Targets for the Treatment of PSC-IBD

An observational study in Inflammatory Bowel Diseases and Primary Sclerosing Cholangitis, sponsored by University Hospital Birmingham NHS Foundation Trust. Status unknown at 1 site in United Kingdom. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2022-06-01.

Sponsored by University Hospital Birmingham NHS Foundation Trust · Observational

The sponsor has not verified this record recently (last verified May 2022), so the status shown — last known as Recruiting — may be out of date.
Study type
Observational
Model
Case-only
Time perspective
Prospective
Enrollment
15
Ages
18 Years and older
Sex
All
01

Study summary

Primary sclerosing cholangitis (PSC) is the classical hepatobiliary manifestation of inflammatory bowel disease (IBD). Although rare, PSC is associated with significant and disproportionate unmet needs; with heightened risks of colorectal cancer and colectomy, and greater all-cause mortality rates compared to matched IBD patients. Unfortunately, no medical therapy has been proven to slow disease progression in PSC-IBD, and liver transplantation is the only lifesaving intervention for patients.

The strong association between PSC and IBD has led to several pathogenic hypotheses, in which dysregulated mucosal immune responses are proposed to contribute. Of note, the investigators recently identified distinct mucosal transcriptomic profiles in PSC-IBD; with regards bile acid metabolism, bile acid signalling, and a central role of enteric dysbiosis. In parallel, pilot data from other groups have shown that treatment with oral vancomycin (a non-absorbable, gut-specific antibiotic) attenuates colonic inflammation and improves biochemical markers of cholestasis in PSC. However, there is no mechanistic data exploring the host-microbial alterations under vancomycin treatment in PSC-IBD, neither the impact of vancomycin on bile acid circulation. The investigators of this study hypothesize that oral vancomycin attenuates colonic mucosal inflammation in PSC-IBD, by restoring gut microbiota mediated bile acid homeostatic pathways. Through these means the study aims to identify druggable gut microbial and host molecular pathways associated with bile acid mediated colonic mucosal inflammation in PSC-IBD.

Read the detailed description

Primary sclerosing cholangitis (PSC) is the classical hepatobiliary manifestation of inflammatory bowel disease (IBD). Although rare, PSC is associated with significant and disproportionate unmet needs; with heightened risks of colorectal cancer and colectomy, and greater all-cause mortality rates compared to age- and sex-matched IBD controls. Unfortunately, no medical therapy has been proven to slow disease progression in PSC-IBD, and liver transplantation is the only lifesaving intervention for patients. The strong association between PSC and IBD has led to several pathogenic hypotheses, in which dysregulated mucosal immune responses are proposed to contribute. Of note, the investigators of this study recently identified distinct mucosal transcriptomic profiles in PSC-IBD; with regards bile acid metabolism, bile acid signalling, and a central role of enteric dysbiosis. In parallel, pilot data from other groups have shown that treatment with oral vancomycin (a non-absorbable, gut-specific antibiotic) attenuates colonic inflammation and improves biochemical markers of cholestasis in PSC. However, there is no mechanistic data exploring the host-microbial alterations under vancomycin treatment in PSC-IBD, neither the impact of vancomycin on bile acid circulation.

In this study, fifteen PSC-IBD patients will be recruited through a large tertiary referral centre, who are undergoing lower gastrointestinal examination as per routine standard of care. Participants will be offered 4 weeks of treatment with oral vancomycin, and stool samples collected at different timepoints to evaluate changes in metagenomic, metatranscriptomic, and bile acid profiles. Colonic biopsies will be collected at baseline and at week 4 (flexible sigmoidoscopy) and subjected to FACS sorted RNA sequencing to identify changes in colonic epithelial cell pathways. Multi-omics data integration will be performed to uncover combinations of predictive profiles, model microbial networks, and host transcriptomic changes implicated in the response to oral vancomycin. This study will inform the downstream identification of specific host molecular and microbial pathways that has a potential for development of therapeutic targets for PSC-IBD in clinical practice.

02

Conditions studied

  • Inflammatory Bowel Diseases
  • Primary Sclerosing Cholangitis

Keywords

  • primary sclerosing cholangitis
  • inflammatory bowel disease
  • microbiome
  • vancomycin
03

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No
Sampling method
Non-probability sample

Study population

15 patients with active PSC-IBD will undergo 4 weeks of treatment with open label (standard of care) oral vancomycin.

Inclusion criteria

  • Patients with confirmed diagnosis of primary sclerosing cholangitis and concurrent colitis
  • Mild to moderately active colitis based on partial Mayo score of ≥3 and ≤6
  • Scheduled for a standard of care lower GI endoscopy as part of disease assessment / surveillance

Exclusion criteria

Exclusion Criteria:

  • History of previous colectomy
  • Isolated small bowel disease
  • Stricturing , fistulating or perianal phenotype
  • Use of antibiotics and/or probiotics in the prior 3 months
  • Use of steroids in last 2 weeks
  • Commenced thiopurines / methotrexate in last 3 months
  • History of intolerance to oral vancomycin
  • Decompensated liver disease (Child C cirrhosis)
  • Active infectious diarrhoea
04

Study design

Observational model
Case-only
Time perspective
Prospective
Enrollment
15 participants (estimated)
Patient registry
No

Interventions

  • DrugOral Vancomycin

    As part of standard of care

05

What researchers measure

Primary outcomes

  1. Identify stool metagenomic, metatranscriptomic and bile acid profiles following treatment with oral vancomycin in PSC-IBD

    Time frame: 12 months

  2. Investigate changes in colonic mucosal epithelial bile acid and immunological pathways through FACS sorted RNA-sequencing following oral vancomycin

    Time frame: 12 months

  3. Identification of druggable host molecular and microbial targets through multi-omic integration analysis

    Time frame: 12 months

06

Study locations

1 of 1 sites recruiting
  • University Hospitals Birmingham NHS Foundation Trust
    Birmingham, B15 2GW, United Kingdom
    • Nabil Quraishi · Contact · nabil.quraishi@nhs.net · 01213712000
    • Nabil Quraishi · Principal investigator
    • Palak J Trivedi · Principal investigator
    • Tariq Iqbal · Principal investigator
    • Naveen Sharma · Sub investigator
    • Andrew Beggs · Sub investigator
    • Christopher Quince · Sub investigator
    • Matthew Brookes · Sub investigator
    • Rachel Cooney · Sub investigator
    Recruiting
07

References and documents

Publications

  • Quraishi MN, Acharjee A, Beggs AD, Horniblow R, Tselepis C, Gkoutos G, Ghosh S, Rossiter AE, Loman N, van Schaik W, Withers D, Walters JRF, Hirschfield GM, Iqbal TH. A Pilot Integrative Analysis of Colonic Gene Expression, Gut Microbiota, and Immune Infiltration in Primary Sclerosing Cholangitis-Inflammatory Bowel Disease: Association of Disease With Bile Acid Pathways. J Crohns Colitis. 2020 Jul 30;14(7):935-947. doi: 10.1093/ecco-jcc/jjaa021. PubMed 32016358 ↗

Individual participant data

Plan to share: Undecided

08

Registry details

Key details

Study ID
NCT05376228
Lead sponsor
University Hospital Birmingham NHS Foundation Trust
Responsible party
Mohammed Nabil Quraishi (Chief Investigator and Consultant Gastroenterologist, University Hospital Birmingham NHS Foundation Trust) — Principal investigator
First posted
May 17, 2022
Start date
Feb 1, 2022
Primary completion
Feb 1, 2023 (estimated)
Completion
Apr 1, 2023 (estimated)
Last update
Jun 1, 2022

Study contacts

Nabil Quraishi, PhD, MRCP
Contact
nabil.quraishi@nhs.net
01213712000
Palak Trivedi, PhD, MRCP
Contact
p.j.trivedi@bham.ac.uk
01213712000

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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