Gastrointestinal Microbiota in Primary Sclerosing Cholangitis and Biliary Atresia With Vancomycin

NCT01322386 | Completed Phase 1 Results DMC

• 1 other identifier: 4751
• Study Type: interventional
• Target: 32
• Locations: 0 countries
• Sites: N/A

Brief Summary

The goals of the proposed work are two fold: Firstly, to see if the antibiotic vancomycin may be used for the early treatment of Biliary Atresia (BA) and Primary Sclerosing Cholangitis (PSC). The investigators hope to learn what effect Vancomycin has on the bacteria that are present in stool, body fluid or intestinal tissue on someone who has BA and PSC and if so by what mechanism. Secondly, the investigators hope to learn to characterize human intestinal microbial communities (microbiome: the collection or collectivity of microorganisms) using molecular methods, examine the mechanisms of interaction between host and microbiome using genomic approaches, and determine how the microbiome both preserves local health and promotes pathology. The investigators will focus on primary sclerosing cholangitis, biliary atresia, as well as states of health. The composition of the associated microbiome will be assessed based on ribosomal DNA and RNA sequences, and attention will be given to richness (diversity), evenness (relative abundance), and variation with respect to time, person, and anatomic niche. Host response at the adjacent mucosal surface will be assessed based on genome-wide gene expression patterns.

Conditions

Primary Sclerosing Cholangitis; Biliary Atresia

Keywords

PSC; BA

Outcome Measures

Primary Outcomes (1)

• Determine the Benefit of Oral Vancomycin Therapy for Primary Sclerosing Cholangitis and Biliary Atresia

  Determine the benefit of oral vancomycin therapy for Primary Sclerosing Cholangitis and Biliary Atresia through improvement of Liver function tests (LFTs) within 3 months of initiating therapy. In addition for PSC, we looked at 25% reduction of abnormal ALT \& GGT, reduction in biliary strictures and beading, and reduction of inflammation in liver biopsies and colon biopsies.

  Within 3 months of therapy

Study Arms (1)

Oral Vancomycin

EXPERIMENTAL

Vancocin

Drug: Vancomycin

Interventions

Vancomycin DRUG

Oral 50mg/Kg per day up to maximum of 1500 mg a day for three months.

Also known as: Vancocin

Oral Vancomycin

Eligibility Criteria

• Age: 1 Month - 20 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Clinical diagnosis of biliary atresia or primary sclerosing cholangitis.
• Clinical controls who are undergoing upper endoscopy or colonoscopy and do not have biliary atresia or primary sclerosing cholangitis.
• Subjects who have been on oral vancomycin for 1 year for biliary atresia or -

You may not qualify if:

• Patients that have taken antibiotics within the last 3 month will be excluded as this will alter the original bacterial flora.

Sponsors & Collaborators

• Stanford University: lead

Related Publications (2)

• Ali AH, Damman J, Shah SB, Davies Y, Hurwitz M, Stephen M, Lemos LM, Carey EJ, Lindor KD, Buness CW, Alrabadi L, Berquist WE, Cox KL. Open-label prospective therapeutic clinical trials: oral vancomycin in children and adults with primary sclerosing cholangitis. Scand J Gastroenterol. 2020 Aug;55(8):941-950. doi: 10.1080/00365521.2020.1787501. Epub 2020 Jul 7.

  PMID: 32633158 DERIVED

• Abarbanel DN, Seki SM, Davies Y, Marlen N, Benavides JA, Cox K, Nadeau KC, Cox KL. Immunomodulatory effect of vancomycin on Treg in pediatric inflammatory bowel disease and primary sclerosing cholangitis. J Clin Immunol. 2013 Feb;33(2):397-406. doi: 10.1007/s10875-012-9801-1. Epub 2012 Oct 9.

  PMID: 23054338 DERIVED

MeSH Terms

Conditions

Cholangitis, Sclerosing; Biliary Atresia

Interventions

Vancomycin

Condition Hierarchy (Ancestors)

Cholangitis; Bile Duct Diseases; Biliary Tract Diseases; Digestive System Diseases; Digestive System Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Glycopeptides; Glycoconjugates; Carbohydrates; Peptides; Amino Acids, Peptides, and Proteins

Results Point of Contact

• Title: Kenneth L Cox, MD / Principal Investigator
• Organization: Stanford University

KCox@stanfordchildrens.org

650-723-5070

Study Officials

• Kenneth L Cox, MD

  Stanford University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor of Pediatrics

Study Record Dates

• First Submitted: February 10, 2011
• First Posted: March 24, 2011
• Study Start: May 1, 2007
• Primary Completion: October 1, 2010
• Study Completion: January 1, 2012
• Last Updated: May 18, 2016
• Results First Posted: March 4, 2016

Record last verified: 2016-04

Data Sharing

• IPD Sharing: Will not share