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
31/10/2024
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
Sub-study: Profiling the portal vein in primary sclerosing cholangitis
Scientific title
Lyophylised encapsulated faecal microbiota transplantation for patients with primary sclerosing cholangitis: a phase 1 study to evaluate safety
Sub-study: Profiling the portal circulation in primary sclerosing cholangitis
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.

The sub-study will not involve an additional intervention or exposure, but will involve additional samples to be taken, including portal venous blood, peripheral blood, stool and urine and baseline and at 24 weeks after study enrolment.
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
Secondary outcome [25] 441166 0
Baseline composition of microbial constituents and products within the portal venous blood (sub-study)
Timepoint [25] 441166 0
Secondary outcome [26] 441167 0
Baseline composition of microbial constituents and products within the portal venous blood (sub-study)
Timepoint [26] 441167 0
At baseline
Secondary outcome [27] 441168 0
Baseline metabolic and immune function of the portal venous blood (sub-study)
Timepoint [27] 441168 0
At baseline
Secondary outcome [28] 441169 0
Baseline metabolic and immune function of the portal venous blood (sub-study)
Timepoint [28] 441169 0
At baseline
Secondary outcome [29] 441170 0
Change in composition of microbial constituents and products within the portal venous blood after FMT (sub-study)
Timepoint [29] 441170 0
At baseline
Secondary outcome [30] 441171 0
Change in composition of microbial constituents and products within the portal venous blood after FMT (sub-study)
Timepoint [30] 441171 0
Week 24 after intervention commencement
Secondary outcome [31] 441172 0
Change in metabolic and immune function of the portal venous blood after FMT (sub-study)
Timepoint [31] 441172 0
Week 24 after intervention commencement
Secondary outcome [32] 441173 0
Change in metabolic and immune function of the portal venous blood after FMT (sub-study)
Timepoint [32] 441173 0
Week 24 after 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

For those enrolling in the sub-study, additional exclusion criteria include:
• Any medical conditions which increase the risk of bleeding including haemophilia or other known specific clotting factor deficiencies
• Portal vein thrombosis
• Portal hypertension (clinically, on FibroScan, endoscopic features, or endoscopic ultrasound features)

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
15/11/2024
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
Funding source category [2] 317734 0
Commercial sector/Industry
Name [2] 317734 0
BiomeBank
Country [2] 317734 0
Australia
Primary sponsor type
Commercial sector/Industry
Name
BiomeBank
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
ryan.mathias@sa.gov.au
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
damjana.bogatic@sa.gov.au
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
damjana.bogatic@sa.gov.au

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 ryan.mathias@sa.gov.au
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.