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Trial registered on ANZCTR


Registration number
ACTRN12623000492651p
Ethics application status
Submitted, not yet approved
Date submitted
26/04/2023
Date registered
12/05/2023
Date last updated
8/09/2024
Date data sharing statement initially provided
12/05/2023
Type of registration
Prospectively registered

Titles & IDs
Public title
Observation of Faecal Microbiota Transplantation Use for Restoration of Gut Microbiota in Gastrointestinal Disorders in the Real-World Setting (The REAL-BIOME Study). A Real-World, Prospective, Observational Study.
Scientific title
Observation of Faecal Microbiota Transplantation Use for Restoration of Gut Microbiota in Gastrointestinal Disorders in the Real-World Setting (The REAL-BIOME (Real-world Microbiome) Study).
A Phase IV, Real-World, Prospective, Open-Label, Observational Study.
Secondary ID [1] 309527 0
None
Universal Trial Number (UTN)
Trial acronym
The REAL-BIOME Study
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Gastrointestinal disorders 329808 0
Ulcerative colitis 329810 0
Crohn's disease 329811 0
Irritable bowel syndrome 329812 0
Immune checkpoint inhibitor-induced enterocolitis 329813 0
Functional constipation 329814 0
Condition category
Condition code
Oral and Gastrointestinal 326709 326709 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Oral and Gastrointestinal 326710 326710 0 0
Crohn's disease
Oral and Gastrointestinal 326711 326711 0 0
Inflammatory bowel disease

Intervention/exposure
Study type
Observational
Patient registry
False
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
The study aims to prospectively evaluate the safety and efficacy of faecal microbiota transplantation (FMT) use for restoration of the gut microbiota in gastrointestinal disorders in the real-world setting. Individuals who have been prescribed FMT by their regular specialist physician as part of their clinical care will be eligible to participate in this observational study. This use must have been approved via the TGA-managed Special Access Scheme to be eligible. FMT will not be provided as part of the study.

Patients with gastrointestinal disorders including ulcerative colitis, Crohn’s disease, irritable bowel syndrome, immune checkpoint inhibitor-associated enterocolitis, and functional constipation may be followed in this study.

Participants will be followed throughout their prescribed treatment with FMT, and will complete surveys that assess FMT safety, efficacy, tolerability/acceptability, and the impact that FMT therapy has on quality of life. Stool samples will be collected from a subset of participants to study microbial factors associated with FMT response and failure in patients with gastrointestinal disorders. Stool will be collected from up to 100 participants enrolled in the ulcerative colitis, Crohn's disease and IBS groups, depending on the number of participants that are enrolled in these groups. Stool may only be collected from participants that live in major cities.

Surveys will be completed at baseline (pre-FMT), week 8 post-FMT, and week 52 post-FMT for all participants. Additional surveys may be completed at weeks 28 and/or 40 post-FMT if participants continue on long-term FMT therapy. Participants in the ulcerative colitis, Crohn's disease and immune checkpoint inhibitor-associated enterocolitis groups will be asked to complete adverse event and FMT treatment schedule surveys at week 1 and/or week 4 post-FMT. Surveys will take approximately 5-10 minutes each, to a total of approximately 1 hours in total, however they do not all need to be completed sequentially. All surveys will be electronic and will be completed online.

No face-to-face study visits are required. The initial screening, consenting and baseline surveys will be completed via phone or video call.
Intervention code [1] 325957 0
Diagnosis / Prognosis
Comparator / control treatment
No control group
Control group
Uncontrolled

Outcomes
Primary outcome [1] 334573 0
Safety of FMT therapy for gastrointestinal disorders in the real-world setting.

Assessed using a formal adverse event / safety questionnaire developed for this study, and aligned with the Common Terminology Criteria for Adverse Events (CTCAE). Symptoms will be defined by the Medical Dictionary for Regulatory Activities (MedDRA) code.

Participants will continue to be followed by their FMT prescribing physician who will manage adverse events in line with standard of care. Expected adverse events to FMT use are generally gastrointestinal and transient in nature (for e.g. diarrhoea, stomach ache, bloating).
Timepoint [1] 334573 0
8 weeks post commencement of FMT treatment
Secondary outcome [1] 421635 0
Clinical response and/or remission following FMT therapy in gastrointestinal disorders based on disorder-specific parameters.
Ulcerative colitis group: Proportion with reduction in Simple Clinical Colitis Activity Index (SCCAI) greater than or equal to 2 points from baseline (clinical response) or SCCAI less than 2 (clinical remission)
Timepoint [1] 421635 0
8 weeks post commencement of FMT treatment.
Secondary outcome [2] 421636 0
Clinical response and/or remission following FMT therapy in gastrointestinal disorders based on disorder-specific parameters.
Crohn’s disease group: proportion with reduction in Harvey Bradshaw Index greater than or equal to 3 points from baseline (clinical response) or less than 5 (clinical remission)
Timepoint [2] 421636 0
8 weeks post commencement of FMT treatment.
Secondary outcome [3] 421637 0
Clinical response and/or remission following FMT therapy in gastrointestinal disorders based on disorder-specific parameters.
IBS group: proportion with reduction in Irritable Bowel Syndrome Severity Scoring System (IBS-SSS) greater than or equal to 50 points from baseline
Timepoint [3] 421637 0
8 weeks post commencement of FMT treatment.
Secondary outcome [4] 421638 0
Clinical response and/or remission following FMT therapy in gastrointestinal disorders based on disorder-specific parameters.
Immune Checkpoint Inhibitor-associated Enterocolitis group: Proportion with reduction in SCCAI greater than or equal to 2 points from baseline (clinical response) or SCCAI less than 2 (clinical remission)
Timepoint [4] 421638 0
8 weeks post commencement of FMT treatment.
Secondary outcome [5] 421639 0
Clinical response and/or remission following FMT therapy in gastrointestinal disorders based on disorder-specific parameters.
Functional constipation group: proportion of participants with improvement in stool frequency and/or form.
Measured using a bowel symptom survey for chronic functional constipation.
Timepoint [5] 421639 0
8 weeks post commencement of FMT treatment.
Secondary outcome [6] 421640 0
Patient perceptions of FMT tolerability and acceptability (all participants)
Measured using a 100mm visual analogue scale and tailored questionnaire.
Timepoint [6] 421640 0
Weeks 8 and 52 post commencement of FMT therapy
Secondary outcome [7] 421641 0
Change in quality of life (all participants)
Measured using the 36-item short form survey (SF-36)
Timepoint [7] 421641 0
Weeks 8 and 52 post commencement of FMT therapy
Secondary outcome [8] 421642 0
Experimental outcome: Microbial factors associated with FMT-induced clinical outcomes.
Microbial (both compositional and functional) analysis of factors associated with FMT-induced clinical outcomes. This will be assessed using metagenomic analysis of bacterial strains in the stool from a subset of patients.
Timepoint [8] 421642 0
Week 8 and week 52 post-commencement of FMT therapy.

Eligibility
Key inclusion criteria
1) Aged 18 years or older.
2) Confirmed diagnosis of ulcerative colitis, Crohn's disease, irritable bowel syndrome, immune checkpoint inhibitor-induced enterocolitis, or functional constipation.
3) Prescribed BiomeBank FMT by treating specialist physician for their gastrointestinal disorder. The prescribing physician must have received approval from the TGA to treat the patient with FMT off-label via the Special Access Scheme. This must occur before patient is eligible for the study, but is not a part of the study process.
4) Has not received FMT in 8 weeks prior to study entry.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1) Anaphylactic food allergy.
2) Pregnancy.

Study design
Purpose
Natural history
Duration
Longitudinal
Selection
Defined population
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Not yet recruiting
Date of first participant enrolment
Anticipated
4/11/2024
Actual
Date of last participant enrolment
Anticipated
20/10/2025
Actual
Date of last data collection
Anticipated
20/10/2026
Actual
Sample size
Target
150
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
ACT,NSW,NT,QLD,SA,TAS,WA,VIC

Funding & Sponsors
Funding source category [1] 313719 0
Commercial sector/Industry
Name [1] 313719 0
BiomeBank
Country [1] 313719 0
Australia
Primary sponsor type
Commercial sector/Industry
Name
BiomeBank
Address
2 Ann Nelson Drive
Thebarton, South Australia, 5031
Country
Australia
Secondary sponsor category [1] 315533 0
None
Name [1] 315533 0
Address [1] 315533 0
Country [1] 315533 0

Ethics approval
Ethics application status
Submitted, not yet approved
Ethics committee name [1] 312890 0
Bellberry Ltd.
Ethics committee address [1] 312890 0
123 Glen Osmond Road Eastwood Adelaide
South Australia 5063
Ethics committee country [1] 312890 0
Australia
Date submitted for ethics approval [1] 312890 0
18/08/2023
Approval date [1] 312890 0
Ethics approval number [1] 312890 0

Summary
Brief summary
FMT has been proven to be a safe and effective treatment for recurrent and refractory C. difficile infection and is now available within Australia for this purpose. There is also significant and growing evidence that FMT can effectively treat gastrointestinal disorders such as ulcerative colitis, Crohn’s disease, irritable bowel syndrome, immune checkpoint inhibitor-associated enterocolitis and functional constipation, and practitioners may now request access to FMT for patients with these conditions under the TGA managed Special Access Scheme-B (SAS-B) or authorised prescriber schemes. However, there is a dearth of evidence on the safety and efficacy of FMT in gastrointestinal disorders in a real-world setting.

In this a real-world, prospective, observational study, we aim to track the real-world safety and efficacy of FMT treatment in patients with gastrointestinal disorders within Australia. The results of this study may inform standard clinical care for patients with gastrointestinal disorders.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 126238 0
Dr Robert Bryant
Address 126238 0
BiomeBank
2 Ann Nelson Drive
Thebarton, South Australia, 5031
Country 126238 0
Australia
Phone 126238 0
+61 8 8152 9370
Fax 126238 0
Email 126238 0
[email protected]
Contact person for public queries
Name 126239 0
Sarah Haylock-Jacobs
Address 126239 0
BiomeBank
2 Ann Nelson Drive
Thebarton, South Australia, 5031
Country 126239 0
Australia
Phone 126239 0
+61 414 396 624
Fax 126239 0
Email 126239 0
[email protected]
Contact person for scientific queries
Name 126240 0
Sarah Haylock-Jacobs
Address 126240 0
BiomeBank
2 Ann Nelson Drive
Thebarton, South Australia, 5031
Country 126240 0
Australia
Phone 126240 0
+61 414 396 624
Fax 126240 0
Email 126240 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


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.