FARGO is a randomised, phase IIa, multi-centre, placebo-controlled trial to compare Faecal Microbiota Transplant (FMT) with placebo in patients with primary sclerosing cholangitis (PSC) and concomitant inflammatory bowel disease.
Primary Sclerosing Cholangitis (PSC) is a chronic liver disease and there is no medical therapy proven to slow disease progression. Many patients with PSC also develop inflammatory bowel disease (IBD). It has been shown that there is an imbalance of 'normal' microbiome (e.g. bacteria, viruses, fungi) in the gut of people with PSC and IBD. This imbalance is believed to contribute to the development (and possibly worsening) of liver disease in PSC. It is believed that Faecal Microbiota Transplant (FMT) treatment can restore the balance in the gut microbiome and that this can lead to reduction in symptoms of PSC and IBD and improve quality of life. FMT involves the transplantation of faeces (or stool) from a healthy individual to a person with PSC. FMT is prepared from stool collected from unrelated, anonymous, healthy donors. The stool is treated in a laboratory at the University of Birmingham. The donors are carefully screened and the donated stool carefully tested to ensure that it is as clean and safe as possible before it is made into doses of FMT suitable for treatment purposes. Data from treatment with FMT in other conditions including Inflammatory Bowel Disease (IBD), Clostridioides difficile (C.diff) infection and PSC has shown FMT to be safe. The primary aim of the FARGO trial is to determine the efficacy of FMT in patients with PSC. The FARGO trial will recruit 58 patients. Half will be randomised to FMT and half to placebo. The trial will be offered at a number of hospitals across England. Patients will be involved in the trial for 50 weeks, including a 2-week screening, 8 treatments over 8 weeks, and follow-up to 48 weeks post-randomisation. Trial visits will include the collection of health history, blood tests, stool tests, pregnancy tests (if applicable), medication reviews, disease specific measures, patient questionnaires and possible symptom and side-effect review. Research blood, urine, stool and colonic biopsy samples will also be collected.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
58
FMT is 50mL aliquots of filtered suspension of stool (0.6g/mL). FMT for administration via colonoscopy will be made up of 150g stool in 250mL (5 aliquots). FMT for administration via enema will be made up of 30g stool in 50mL (1 aliquot) made up to 100mL by the addition of 50mL normal saline
FMT Placebo is 50mL aliquots containing 0.9% w/v saline and glycerol in a ratio of 9 parts saline: 1 part glycerol v/v. FMT Placebo for administration via colonoscopy will be made up of 250mL (5 aliquots). FMT Placebo for administration via enema will be made up of 50mL (1 aliquot) made up to 100mL by the addition of 50mL normal saline
Queen Elizabeth Hospital Birmingham, University Hospitals Birmingham NHS Foundation Trust
Birmingham, United Kingdom
RECRUITINGSt Mark's Hospital, London North West University Healthcare NHS Trust
London, United Kingdom
NOT_YET_RECRUITINGRoyal Free Hospital, Royal Free London NHS Foundation Trust
London, United Kingdom
Serum Alkaline Phosphatase (ALP)
Reduction in serum ALP values from baseline, measured at 48 weeks following the first dose of FMT or FMT Placebo.
Time frame: ALP at Screening (Week -2), Week 1, Week 3, Week 5, Week 8, Week 12, Week 24, Week 36 and Week 48
Patient-reported outcome (PRO) measures
Patient-reported outcome (PRO) measure: Primary Sclerosing Cholangitis -Patient Reported Outcome (PSC-PRO) Questionnaire
Time frame: PSC-PRO at Screening (Week -2), Week 8, Week 12, Week 24, Week 36 and Week 48
Patient-reported outcome (PRO) measures
Patient-reported outcome (PRO) measure: short-form (SF)-36 Questionnaire
Time frame: SF-36 at Screening (Week -2), Week 8, Week 12, Week 24, Week 36 and Week 48
Patient-reported outcome (PRO) measures
Patient-reported outcome (PRO) measures: 5 dimensional itch scale (5D itch) Questionnaire
Time frame: 5D-Itch at Screening (Week -2), Week 8, Week 12, Week 24, Week 36 and Week 48
Patient-reported outcome (PRO) measures
Patient-reported outcome (PRO) measures: Short Inflammatory Bowel Disease Questionnaire (SIBDQ)
Time frame: SIBDQ at Screening (Week -2), Week 8, Week 12, Week 24, Week 36 and Week 48
Surrogate biomarkers of liver fibrosis
Surrogate biomarkers of liver fibrosis: vibration-controlled transient elastography (VCTE)
Time frame: Surrogate biomarkers of liver fibrosis: VCTE at Screening (Week -2) and Week 48
Surrogate biomarkers of liver fibrosis
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King's College Hospital, King's College Hospital NHS Foundation Trust
London, United Kingdom
RECRUITINGJohn Radcliffe Hospital, Oxford University Hospitals NHS Foundation Trust
Oxford, United Kingdom
RECRUITINGSurrogate biomarkers of liver fibrosis: enhanced liver fibrosis score (ELF)
Time frame: Surrogate biomarkers of liver fibrosis: ELF at Week 1, Week 5, Week 12, and Week 48
Routine liver laboratory parameters
Routine liver laboratory parameters: serum alanine transaminase (ALT)
Time frame: Routine liver laboratory parameters: ALT at Screening (Week -2), Week 1, Week 3, Week 5, Week 8, Week 12, Week 24, Week 36 and Week 48
Routine liver laboratory parameters
Routine liver laboratory parameters: serum aspartate aminotransferase (AST)
Time frame: Routine liver laboratory parameters: AST at Screening (Week -2), Week 1, Week 3, Week 5, Week 8, Week 12, Week 24, Week 36 and Week 48
Routine liver laboratory parameters
Routine liver laboratory parameters: bilirubin
Time frame: Routine liver laboratory parameters: bilirubin at Screening (Week -2), Week 1, Week 3, Week 5, Week 8, Week 12, Week 24, Week 36 and Week 48
Routine liver laboratory parameters
Routine liver laboratory parameters: gamma glutamyl transferase
Time frame: Routine liver laboratory parameters: gamma glutamyl transferase at Screening (Week -2), Week 1, Week 3, Week 5, Week 8, Week 12, Week 24, Week 36 and Week 48
Routine liver laboratory parameters
Routine liver laboratory parameters: albumin
Time frame: Routine liver laboratory parameters: albumin at Screening (Week -2), Week 1, Week 3, Week 5, Week 8, Week 12, Week 24, Week 36 and Week 48
PSC specific prognostic scores
PSC specific prognostic scores: the UK-PSC Score
Time frame: PSC specific prognostic scores: UK-PSC Score at Screening (Week -2), Week 1, Week 3, Week 5, Week 8, Week 12, Week 24, Week 36 and Week 48
PSC specific prognostic scores
PSC specific prognostic scores: the Amsterdam-Oxford PSC score
Time frame: PSC specific prognostic scores: Amsterdam-Oxford PSC Score at Screening (Week -2), Week 1, Week 3, Week 5, Week 8, Week 12, Week 24, Week 36 and Week 48
Quantitative assessment of Inflammatory Bowel Disease (IBD) activity
Quantitative assessment of IBD activity: The Mayo colitis score
Time frame: Quantitative assessment of IBD activity: Mayo colitis score at Week 1, Week 12 and Week 48
Quantitative assessment of IBD activity
Quantitative assessment of IBD activity: The Simple Endoscopic Score for Crohn's Disease (SES-CD);
Time frame: Quantitative assessment of IBD activity: SES-CD at Week 1, Week 12 and Week 48
Quantitative assessment of IBD activity
Quantitative assessment of IBD activity: faecal calprotectin
Time frame: Quantitative assessment of IBD activity: faecal calprotectin at Screening (Week -2), Week 1, Week 5, Week 12 and Week 48
The incidence of clinical non-trial endpoint events
The incidence of clinical non-trial endpoint events: acute cholangitis flares (including those that are resistant to single course antibiotic treatment), acute colitis flares, and episodes/time to hepatic decompensation
Time frame: The incidence of clinical non-trial endpoint events at Screening (Week -2), Week 1, Week 2, Week 3, Week 4, Week 5, Week 6, Week 7, Week 8, Week 12, Week 24, Week 36 and Week 48
The incidence of trial endpoint events
The incidence of trial endpoint events: cholangiocarcinoma / hepatopancreatobiliary malignancy, referral for liver transplantation, colonic resection or colorectal cancer, and/or mortality
Time frame: The incidence of trial endpoint events at Screening (Week -2), Week 1, Week 2, Week 3, Week 4, Week 5, Week 6, Week 7, Week 8, Week 12, Week 24, Week 36 and Week 48
Occurrence of adverse events
Occurrence of adverse events as measured by CTCAE v5.0
Time frame: Occurrence of adverse events at Screening (Week -2), Week 1, Week 2, Week 3, Week 4, Week 5, Week 6, Week 7, Week 8, Week 12, Week 24, Week 36 and Week 48