Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12624000467538
Ethics application status
Approved
Date submitted
20/03/2024
Date registered
16/04/2024
Date last updated
16/04/2024
Date data sharing statement initially provided
16/04/2024
Type of registration
Prospectively registered

Titles & IDs
Public title
Faecal microbiota transplantation for primary sclerosing cholangitis
Scientific title
Lyophylised encapsulated faecal microbiota transplantation for patients with primary sclerosing cholangitis: a phase 1 study to evaluate safety
Secondary ID [1] 311712 0
none
Universal Trial Number (UTN)
Trial acronym
FMT-PSC
Linked study record

Health condition
Health condition(s) or problem(s) studied:
primary sclerosing cholangitis 333190 0
Condition category
Condition code
Oral and Gastrointestinal 329875 329875 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Oral and Gastrointestinal 329876 329876 0 0
Inflammatory bowel disease
Inflammatory and Immune System 329877 329877 0 0
Other inflammatory or immune system disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Faecal microbiota transplantation capsules:
a) 6 capsules per day in the 2 week induction period (amounting to approximately 4 grams of stool per day)
b) followed by 2 capsules per day during the 22 week maintenance period (amounting to approximately 1.3 grams of stool per day)

Adherence to treatment will be measured using a self-reporting as well as return of capsules.
Intervention code [1] 328175 0
Treatment: Other
Comparator / control treatment
No control group
Control group
Uncontrolled

Outcomes
Primary outcome [1] 337645 0
Safety
Timepoint [1] 337645 0
From week 0 to week 4 post intervention commencement, participants will be asked about adverse events during a weekly study visit (in person or via phone). Thereafter, participants will be asked about adverse events during 4 weekly study visits (in person) from week 8 to week 24 post intervention commencement. Participants will be asked to contact a study coordinator and report any adverse events at any time that they arise throughout the trial.
Secondary outcome [1] 432682 0
Tolerability
Timepoint [1] 432682 0
Weekly from week 0 - 4, then 4 weekly until week 24 post intervention commencement
Secondary outcome [2] 432833 0
Adherence to FMT capsules
Timepoint [2] 432833 0
Self-reported capsule administration recorded daily by the participant until week 24 post intervention commencement; Administration record and unused capsules returned to a study coordinator at 4 weekly intervals post intervention commencement until week 24 post intervention commencement
Secondary outcome [3] 432834 0
Change in alkaline phosphatase (ALP)
Timepoint [3] 432834 0
Baseline, week 0, week 2, week 4, and then every 4 weeks until week 24 post intervention commencement
Secondary outcome [4] 432835 0
Change in gamma glutamyltransferase (GGT)
Timepoint [4] 432835 0
Baseline, week 0, week 2, week 4, and then every 4 weeks until week 24 post intervention commencement
Secondary outcome [5] 432836 0
Change in PSC Mayo Risk Score
Timepoint [5] 432836 0
Baseline, week 12 and week 24 post intervention commencement
Secondary outcome [6] 432837 0
Change in Enhanced liver fibrosis (ELF) score
Timepoint [6] 432837 0
Baseline, week 24 post intervention commencement
Secondary outcome [7] 432838 0
Change in liver stiffness
Timepoint [7] 432838 0
Baseline, week 24 post intervention commencement
Secondary outcome [8] 432839 0
Magnetic resonance imaging (MRI) progression
Timepoint [8] 432839 0
Baseline, week 24 post intervention commencement
Secondary outcome [9] 432840 0
Clinically significant ascending cholangitis
Timepoint [9] 432840 0
From week 0 to week 4 post intervention commencement, participants will be asked about complications of PSC during a weekly study visit (in person or via phone). Thereafter, participants will be asked about complications during 4 weekly study visits (in person) from week 8 to week 24 post intervention commencement. Participants will be asked to contact a study coordinator and report any complications of PSC at any time that they arise throughout the trial.
Secondary outcome [10] 432841 0
PSC-related clinical progression
Timepoint [10] 432841 0
From week 0 to week 4 post intervention commencement, participants will be asked about clinical progression of PSC during a weekly study visit (in person or via phone). Thereafter, participants will be asked about clinical progression during 4 weekly study visits (in person) from week 8 to week 24 post intervention commencement. Participants will be asked to contact a study coordinator and report any features of clinical progression of PSC at any time that they arise throughout the trial.
Secondary outcome [11] 432842 0
Gastrointestinal inflammation
Timepoint [11] 432842 0
Baseline, week 2, week 12, and week 24 post intervention commencement
Secondary outcome [12] 432844 0
Sonographic inflammatory bowel disease (IBD) activity
Timepoint [12] 432844 0
Baseline, week 12, week 24 post intervention commencement
Secondary outcome [13] 432845 0
Endoscopic IBD activity
Timepoint [13] 432845 0
Baseline, week 24 post intervention commencement
Secondary outcome [14] 432846 0
Quality of life
Timepoint [14] 432846 0
Baseline, week 2, week 12, week 24 post intervention commencement
Secondary outcome [15] 432849 0
Dietary intake and patterns
Timepoint [15] 432849 0
Baseline, week 0, week 12, week 24 post intervention commencement
Secondary outcome [16] 433688 0
Clinical inflammatory bowel disease activity for participants with ulcerative colitis
Timepoint [16] 433688 0
Baseline, week 2, week 12, and week 24 post intervention commencement
Secondary outcome [17] 433689 0
Clinical inflammatory bowel disease activity for participants with ulcerative colitis
Timepoint [17] 433689 0
Baseline, week 2, week 12, and week 24 post intervention commencement
Secondary outcome [18] 433690 0
Clinical inflammatory bowel disease activity for participants with Crohn's disease
Timepoint [18] 433690 0
Baseline, week 2, week 12, and week 24 post intervention commencement
Secondary outcome [19] 433691 0
Clinical inflammatory bowel disease activity for participants with Crohn's disease
Timepoint [19] 433691 0
Baseline, week 2, week 12, and week 24 post intervention commencement
Secondary outcome [20] 433692 0
Change in fatigue
Timepoint [20] 433692 0
Baseline, week 2, week 12 and week 24 post intervention commencement
Secondary outcome [21] 433693 0
Change in pruritis
Timepoint [21] 433693 0
Baseline, week 2, week 12 and week 24 post intervention commencement
Secondary outcome [22] 433694 0
Change in pain
Timepoint [22] 433694 0
Baseline, week 2, week 12 and week 24 post intervention commencement
Secondary outcome [23] 433695 0
Change in microbial composition
Timepoint [23] 433695 0
Baseline, week 0, week 2, week 12, week 24 post intervention commencement
Secondary outcome [24] 433696 0
Change in microbial metabolic function
Timepoint [24] 433696 0
Baseline, week 0, week 2, week 12, week 24 post intervention commencement

Eligibility
Key inclusion criteria
1. Diagnosis of PSC (small and large-duct) established for 6 months or more
2. Age 16-75 years
3. If taking ursodeoxycholic acid then stable dose at less than 25mg/kg/day for more than 12 weeks prior to study entry
4. Laboratory parameters:
o ALP elevated above the upper limit of normal (above 110 U/L)
o International normalized ratio less than or equal to 1.3
o eGFR more than 60mL/min/1.73m2
o Albumin more than 32 g/L
o Platelets more than 120 x 109/L
Minimum age
16 Years
Maximum age
75 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
• Serum ALP variation > 30% between tests at two time-points 1 week apart during screening (unless both figures within normal range)
• Childs B or C cirrhosis (clinical parameters as assessed by the treating clinician) or clinically significant portal hypertension
• Total bilirubin > 1.5 x ULN unless due to Gilbert’s syndrome
• Liver transplantation
• Subtotal or total colectomy
• Active malignancy (cholangiocarcinoma or colorectal cancer)
• Presence of positive antimitochondrial antibody or features to suggest overlap autoimmune hepatitis
• Clinically significant dominant stricture deemed likely to require intervention within 3 months
• Ascending cholangitis within 3 months prior to enrolment
• Presence of a percutaneous drain or biliary stent
• Other causes of liver disease (alcohol induced hepatitis, viral hepatitis, primary biliary cholangitis, hemochromatosis, Wilson’s disease, Alpha-1 antitrypsin deficiency, non-alcoholic fatty liver disease)
• Pregnant or intending to become pregnant within 24 weeks
• Currently breastfeeding
• Immunodeficiency (beyond that associated with IBD-related therapy)
• Prebiotic, probiotic or antibiotic use within 4 weeks of enrolment
• Allergy or intolerance to vancomycin, metronidazole or neomycin
• Corticosteroid therapy of prednisolone >25mg daily (or equivalent)
• Anticoagulant therapy or dual antiplatelet therapy
• Active illicit drug use, narcotic drug use or alcohol consumption of a dependent nature
• Active IBD which has required change in therapy in the last 3 months or thought to require change in medication or surgery within 3 months of study entry
• Any medical condition that the treating gastroenterologist deems to pose a theoretical risk to the participant undergoing FMT
• Past colonic dysplasia apart from low grade dysplasia arising in tubular adenoma or sessile serrated adenoma
• Presence of IgG4-related disease
• Anaphylaxis to any food products
• Coeliac disease
• Participant unable to provide informed consent

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
Single group
Other design features
Phase
Phase 1
Type of endpoint/s
Safety
Statistical methods / analysis

Recruitment
Recruitment status
Not yet recruiting
Date of first participant enrolment
Anticipated
29/04/2024
Actual
Date of last participant enrolment
Anticipated
31/03/2025
Actual
Date of last data collection
Anticipated
30/09/2025
Actual
Sample size
Target
20
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
SA

Funding & Sponsors
Funding source category [1] 316041 0
Charities/Societies/Foundations
Name [1] 316041 0
Gastroenterological Society of Australia and Dr Falk
Country [1] 316041 0
Australia
Primary sponsor type
Government body
Name
Central Adelaide Local Health Network
Address
Country
Australia
Secondary sponsor category [1] 318203 0
None
Name [1] 318203 0
Address [1] 318203 0
Country [1] 318203 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 314863 0
Central Adelaide Local Health Network HREC
Ethics committee address [1] 314863 0
Ethics committee country [1] 314863 0
Australia
Date submitted for ethics approval [1] 314863 0
27/11/2023
Approval date [1] 314863 0
19/12/2023
Ethics approval number [1] 314863 0

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

Contacts
Principal investigator
Name 132970 0
Dr Ryan Mathias
Address 132970 0
The Queen Elizabeth Hospital, 28 Woodville Road, Woodville South, SA, 5011
Country 132970 0
Australia
Phone 132970 0
+614 35 597 597
Fax 132970 0
Email 132970 0
[email protected]
Contact person for public queries
Name 132971 0
Dr Damjana Bogatic
Address 132971 0
The Queen Elizabeth Hospital, 28 Woodville Road, Woodville South, SA, 5011
Country 132971 0
Australia
Phone 132971 0
+614 22 715 671
Fax 132971 0
Email 132971 0
[email protected]
Contact person for scientific queries
Name 132972 0
Dr Damjana Bogatic
Address 132972 0
The Queen Elizabeth Hospital, 28 Woodville Road, Woodville South, SA, 5011
Country 132972 0
Australia
Phone 132972 0
+614 22 715 671
Fax 132972 0
Email 132972 0
[email protected]

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
De-identified data underlying published results
When will data be available (start and end dates)?
December 2025 to December 2030
Available to whom?
Researchers who provide a methodologically sound proposal
Available for what types of analyses?
Meta-analyses, systematic reviews
How or where can data be obtained?
Contact principal investigator via email on [email protected]
Subject to approval by principle investigator


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.