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Trial registered on ANZCTR


Registration number
ACTRN12622000914763
Ethics application status
Approved
Date submitted
19/06/2022
Date registered
27/06/2022
Date last updated
19/10/2023
Date data sharing statement initially provided
27/06/2022
Type of registration
Prospectively registered

Titles & IDs
Public title
Orally Administered Faecal Microbial Transplantation in the Management of Immune Checkpoint Inhibitor Associated Colitis
Scientific title
Orally Administered Faecal Microbial Transplantation in the Management of Immune Checkpoint Inhibitor Associated Colitis - A randomised control trial to assess safety and preliminary efficacy
Secondary ID [1] 307386 0
None
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Immune Checkpoint inhibitor Colitis 326698 0
Condition category
Condition code
Cancer 323945 323945 0 0
Other cancer types

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Orally administered encapsulated lyophilised faecal microbiota transplantation(FMT) administered daily over a period of 7 days. Given the nature of FMT, the "dose" of FMT will vary.
Participants will be contacted throughout the trial period to check compliance and for further reminders and arranged unused product return.
Intervention code [1] 323821 0
Treatment: Other
Comparator / control treatment
Placebo capsules will consist of microcrystalline cellulose which is encapsulated, in line with the encapsulation process used for the active FMT treatment
Control group
Placebo

Outcomes
Primary outcome [1] 331744 0
Percentage of patients in clinical remission as determined by stool count. through a patient diary
Timepoint [1] 331744 0
1 week following commencement of FMT or placebo
Secondary outcome [1] 410977 0
Percentage of patients in clinical remission as determined by the stool count through a patient diary
Timepoint [1] 410977 0
Week 2, 4, 8 and 12 following commencement of FMT or placebo
Secondary outcome [2] 410978 0
Percentage of patients in clinical response as determined by the stool count through a patient diary
Timepoint [2] 410978 0
Week 1,2,4,8 and 12 following commencement of FMT or placebo
Secondary outcome [3] 410979 0
Percentage of patients on corticosteroids assessed through a study-specific questionnaire and medical records
Timepoint [3] 410979 0
Week 2,4,6 and 12 following commencement of FMT or placebo
Secondary outcome [4] 410980 0
Median duration of corticosteroids assessed through a study-specific questionnaire and medical records
Timepoint [4] 410980 0
Week 12 following commencement of FMT or placebo
Secondary outcome [5] 410981 0
Cumulative dose of corticosteroids assessed through a study-specific questionnaire and medical records
Timepoint [5] 410981 0
Week 12 following commencement of FMT or placebo
Secondary outcome [6] 410982 0
Percentage of patients able to recommence immunotherapy assessed through a study-specific questionnaire and medical records
Timepoint [6] 410982 0
Week 12 following commencement of FMT or placebo
Secondary outcome [7] 410983 0
Change in disease activity determined by blood and stool biomarkers (C-reactive protein, faecal calprotectin)
Timepoint [7] 410983 0
Week 1, 2,4,8 and 12 following commencement of FMT or placebo
Secondary outcome [8] 410984 0
Rates of adverse events assessed through study-specific questionnaire, clinical examination and medical records.. Examples of possilbe adverse events include gastrointestinal upset, worsening colitis, transient fevers and very low risk of transmissible infection,
Timepoint [8] 410984 0
Week 1,2,4,8 and 12 following commencement of FMT or placebo
Secondary outcome [9] 410985 0
Change in patient reported outcomes assessed using PRO-2 score through a study-specific questionnaire
Timepoint [9] 410985 0
Week 1,2,4,8 and 12 following commencement of FMT or placebo

Eligibility
Key inclusion criteria
• Grade 2 or more diarrhoea (greater than 4 bowel actions more than baseline)
• Last dose of checkpoint inhibitor within 4 months of symptom onset

Either
• No response to oral glucocorticoids
OR
• Steroid intolerant
OR
• Recurrence of symptoms during or following corticosteroid wean
Or
• Recurrent episode of immune checkpoint inhibitor colitis.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
-Infective colitis
-Inflammatory bowel disease
-Previous colectomy
-Significant medical comorbidity

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Through central randomisation by computer generated list.
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Pre-established computer generated randomisation list.
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
Intervention assignment
Parallel
Other design features
Phase
Not Applicable
Type of endpoint/s
Safety/efficacy
Statistical methods / analysis
Based on previous studies using FMT in the treatment of ICI colitis and allowing for 10% drop out,s ample size calculated as 46 patients across FMT and placebo groups

Recruitment
Recruitment status
Recruiting
Date of first participant enrolment
Anticipated
1/12/2022
Actual
11/09/2023
Date of last participant enrolment
Anticipated
1/06/2024
Actual
Date of last data collection
Anticipated
1/09/2024
Actual
Sample size
Target
46
Accrual to date
2
Final
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 22587 0
St Vincent's Hospital (Darlinghurst) - Darlinghurst
Recruitment postcode(s) [1] 37841 0
2010 - Darlinghurst

Funding & Sponsors
Funding source category [1] 311663 0
Hospital
Name [1] 311663 0
St Vincent's Hospital Sydney
Country [1] 311663 0
Australia
Primary sponsor type
Hospital
Name
St Vincent's Hospital Sydney
Address
390 Victoria St, Darlinghurst, NSW, 2010
Country
Australia
Secondary sponsor category [1] 313115 0
None
Name [1] 313115 0
Address [1] 313115 0
Country [1] 313115 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 311120 0
St Vincent's Hospital HREC
Ethics committee address [1] 311120 0
St Vincent’s Hospital Translational
97-105 Boundary Street
Darlinghurst NSW 2010
Ethics committee country [1] 311120 0
Australia
Date submitted for ethics approval [1] 311120 0
09/06/2022
Approval date [1] 311120 0
20/07/2022
Ethics approval number [1] 311120 0

Summary
Brief summary
Cancer immunotherapy with immune checkpoint inhibitors (ICI) have revolutionised the treatment and outcomes for multiple cancers in the past 10 years leading to sustained remission and cure in some patients even when diagnosed with advanced disease. However, immune mediated side effects are common including diarrhoea and colitis which can affect up to 40% of patients who take ICI's. This study aims to assess whether a short course (7 days) of oral treatment with faecal microbiota transplantation (FMT) therapy has any impact upon ICI colitis symptoms in patients who are affected by this.

Who is it for?
You may be eligible for this study if you are an adult aged 18 years or older, you have experienced diarrhoea from your ICI and your last dose was within 4 months of symptom onset. Participants who are steroid intolerant, have had no response to oral glucocorticoids, have had recurrence of symptoms during or following corticosteroid wean or have had recurrent episodes of immune checkpoint inhibitor colitis will be considered for inclusion.

Study details
All participants who choose to enrol in this study will be randomly allocated by chance (similar to flipping a coin) to either the FMT group or a placebo group. Both groups will be asked to take FMT capsules each day for 7 days, and to keep a diary of their bowel movements for up to 12 weeks after enrolling in the study. Participants will also be asked to provide stool and blood samples throughout the study and may also require a colonoscopy. The overall participation will be 12 weeks.

It is hoped this research will demonstrate that it is safe and effective to use orally administered FMT to treat ICI colitis in cancer patients who have been taking immune checkpoint inhibitors. If FMT is successful, this may lead to a reduction in overall corticosteroid use, hospitalisation and an early resumption of their cancer immunotherapy for affected patients.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 120006 0
Dr Craig Haifer
Address 120006 0
Gastroenterology Department,
St Vincent's Hospital
390 Victoria St
Darlinghurst, NSW, 2010
Country 120006 0
Australia
Phone 120006 0
+61 2 83822061
Fax 120006 0
Email 120006 0
[email protected]
Contact person for public queries
Name 120007 0
Craig Haifer
Address 120007 0
Gastroenterology Department,
St Vincent's Hospital
390 Victoria St
Darlinghurst, NSW, 2010
Country 120007 0
Australia
Phone 120007 0
+61 2 83822061
Fax 120007 0
Email 120007 0
[email protected]
Contact person for scientific queries
Name 120008 0
Craig Haifer
Address 120008 0
Gastroenterology Department,
St Vincent's Hospital
390 Victoria St
Darlinghurst, NSW, 2010
Country 120008 0
Australia
Phone 120008 0
+61283822061
Fax 120008 0
Email 120008 0
[email protected]

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment
Data will be analysed as a group


What supporting documents are/will be available?

No Supporting Document Provided



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.