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Trial registered on ANZCTR


Registration number
ACTRN12624001372572
Ethics application status
Approved
Date submitted
25/10/2024
Date registered
18/11/2024
Date last updated
18/11/2024
Date data sharing statement initially provided
18/11/2024
Type of registration
Prospectively registered

Titles & IDs
Public title
Oral vancomycin for treating atypical gut inflammation in children
Scientific title
The efficacy of oral vancomycin therapy in different phenotypes of paediatric inflammatory bowel diseases in correlation with gut microbiota composition
Secondary ID [1] 313250 0
none
Universal Trial Number (UTN)
U1111-1314-9881
Trial acronym
VIGOR
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Primary sclerosing cholangitis - ulcerative colitis 335567 0
Atypical ulcerative colitis 335568 0
Condition category
Condition code
Oral and Gastrointestinal 332138 332138 0 0
Inflammatory bowel disease

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Brief name: Oral vancomycin therapy (OVT)

International Non-proprietary Name (INN): vancomycin
- the dose administered: 50 mg/kg/day (max 1500 mg/day) in three divided doses daily
- the duration of administration: minimum of 3 months. After three months, physician will address the response. If non-responsive, OVT will be ceased. If responsive, then OVT will be continued for 6 to 12 months depending on previous history of challenges in achieving remission.
- the mode of administration: oral solution or capsule
- adherence will be monitored during clinical follow-up visits by attending physician, and during dispensing prescription renewal by pharmacist
Intervention code [1] 329829 0
Treatment: Drugs
Comparator / control treatment
Healthy children will be asked to provide stool and saliva samples to generate a group with "healthy gut and saliva microbiota" as controls. Children with atypical ulcerative colitis not responding to convential medical treatment and children with primary sclerosing chilangitis-ulcerative colitis (PSC-UC) will not be given only placebo - in that sense the OVT itself is uncontrolled.
Control group
Active

Outcomes
Primary outcome [1] 339718 0
Faecal calprotectin level <100 ug/mg (defining colitis remission)
Timepoint [1] 339718 0
3 months (primary timepoint), 6 months, 9 months and 12 months after starting OVT
Primary outcome [2] 339719 0
Change in colonoscopy Mayo score within 12 months after OVT.
Timepoint [2] 339719 0
One colonoscopy at 12 months after starting OVT and while still on OVT.
Primary outcome [3] 339720 0
Faecal microbiota composition before and after OVT vs. faecal microbiota of healthy controls
Timepoint [3] 339720 0
3 months (primary outcome), 6 months, 9 months, and 12 months after starting OVT
Secondary outcome [1] 440991 0
Change in the liver function tests in children with PSC-UC
Timepoint [1] 440991 0
Blood test at 3 months, 6 months, 9 months, and 12 months after starting OVT
Secondary outcome [2] 440992 0
Change in the elastography measurements
Timepoint [2] 440992 0
Elastography at 6 and 12 months after starting OVT
Secondary outcome [3] 440993 0
Number of participants with detected vancomycin resistant enterococcus (VRE) at faecal screening
Timepoint [3] 440993 0
3 months, 6 months, 9 months, and 12 months after starting OVT (while on OVT, and within 6 months after ceasing OVT).
Secondary outcome [4] 441469 0
Changes in the bowel wall thickness after staring OVT
Timepoint [4] 441469 0
3 months, 6 months, and 12 months after starting OVT
Secondary outcome [5] 441470 0
Saliva microbiota composition before and after OVT vs. faecal microbiota of healthy controls
Timepoint [5] 441470 0
9 months after starting OVT
Secondary outcome [6] 441803 0
Magnetic resonance cholangio-pancreatography (MRCP) findings at 12 months after starting OVT
Timepoint [6] 441803 0
12 months after starting OVT
Secondary outcome [7] 441804 0
Bile acid microbiota composition
Timepoint [7] 441804 0
12 months after starting OVT
Secondary outcome [8] 441805 0
Bile acid vancomycin concentration
Timepoint [8] 441805 0
12 months after starting OVT

Eligibility
Key inclusion criteria
• Children aged 3-17 years old with established diagnosis (of PSC-UC or conventional medical treatment resistant atypical UC: endoscopy and biopsies confirmed IBD (in PSC-UC and atypical UC); magnetic resonance cholangio pancreatography (MRCP) and/or liver biopsy confirmed PSC (in PSC-UC), MRCP ruled out PSC in atypical UC without PSC.
• Has nor received OVT preciously, but planning to be commenced on OVT for at least 3 months (or if starting with Pediatric Randomized trial of Antibiotics in acute Severe Colitis (PRASCO) - a cocktail of antibiotics for three weeks including oral vancomycin, that will be continued for at least 3 months) for treating PSC-UC or atypical UC resistant to conventional medical treatment as mentioned in the guidelines, and not for treating Clostridium difficile infection..
• Patients and/or their guardians have given an informed consent.
• No new interventions (medications or new conventional therapies) for treating IBD was given within the past 4 weeks before starting OVT.

For healthy participants:
• Children aged between 3-17 years old without inflammatory bowel diseases with similar background and age-related physical development status to the patients.
Minimum age
3 Years
Maximum age
17 Years
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
• PSC without UC, and Crohn’s disease type of IBD.
• Previous allergic reactions to vancomycin (such as vancomycin allergy) and/or other related antibiotics similar to vancomysin.
• Presence of malignant disease or potential need for liver transplantation within the following 12 months.
• For healthy controls: presence of chronic illness requiring long-term medications, use of antibiotics within the past 6 months.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Other design features
Phase
Phase 2 / Phase 3
Type of endpoint/s
Statistical methods / analysis
Demographic and clinical information will be presented using descriptive statistics measuring frequencies for categorical variables and measures of central tendency/spread for continuous variables. Continuous variables will be assessed for normal distributions using Shapiro-Wilk’s test. Mean (SD) will be used to present normally distributed continuous variables and median (IQR) for not-normally distributed ones. In order to explore the differences in various outcome measures between the three groups over time, linear mixed model analyses will be conducted. Separate random intercept/slope models with an interaction term between group and time will be built with time x group as fixed effect and subjects (participants) as random effects. Post-hoc analysis will be performed for pairwise comparison between time-points and groups. All analyses will be conducted in R (4.2.3) (https://www.r-project.org/) (R Core Team, 2023) or IBM SPSS Statistics 26.0 software.

Recruitment
Recruitment status
Not yet recruiting
Date of first participant enrolment
Anticipated
1/01/2025
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
63
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
QLD
Recruitment hospital [1] 27266 0
Queensland Children's Hospital - South Brisbane
Recruitment postcode(s) [1] 43353 0
4101 - South Brisbane

Funding & Sponsors
Funding source category [1] 317695 0
Hospital
Name [1] 317695 0
Queensland Children's Hopsital
Country [1] 317695 0
Australia
Primary sponsor type
Hospital
Name
Queensland Children's Hospital
Address
Country
Australia
Secondary sponsor category [1] 320016 0
None
Name [1] 320016 0
Address [1] 320016 0
Country [1] 320016 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 316391 0
Children’s Health Queensland Hospital and Health Service Human Research Ethics Committee
Ethics committee address [1] 316391 0
Ethics committee country [1] 316391 0
Australia
Date submitted for ethics approval [1] 316391 0
25/09/2024
Approval date [1] 316391 0
04/10/2024
Ethics approval number [1] 316391 0
HREC/2024/QCHQ/106873

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 137750 0
Dr Laura Räisänen
Address 137750 0
Queenland Children's Hospital, 501 Stanley Street, 4101 Brisbane QLD.
Country 137750 0
Australia
Phone 137750 0
+61 730697181
Fax 137750 0
Email 137750 0
laura.raisanen@tuni.fi
Contact person for public queries
Name 137751 0
Fariha Balouch
Address 137751 0
Queensland Children's Hospital, 501 Stanley Street, 4101 Brisbane QLD
Country 137751 0
Australia
Phone 137751 0
+61 730684502
Fax 137751 0
Email 137751 0
fariha.balouch@health.gov.qld.au
Contact person for scientific queries
Name 137752 0
Laura Räisänen
Address 137752 0
Queensland Children's Hospital, 501 Stanley Street, 4101 Brisbane QLD
Country 137752 0
Australia
Phone 137752 0
+61 730697181
Fax 137752 0
Email 137752 0
laura.raisanen@tuni.fi

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment
PSC-UC in children is rare, we can't make the data public due to patient confidentiality


What supporting documents are/will be available?

Doc. No.TypeCitationLinkEmailOther DetailsAttachment
24293Ethical approval  laura.raisanen@tuni.fi
24294Study protocol  laura.raisanen@tuni.fi
24295Informed consent form  laura.raisanen@tuni.fi



Results publications and other study-related documents

Documents added manually
No documents have been uploaded by study researchers.

Documents added automatically
No additional documents have been identified.