ICEBOAT. A prospective, multi-center, double blind randomized trial of fecal microbiota transplantation (FMT) delivered by capsule versus placebo in severe irritable bowel syndrome (IBS)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Assistance Publique Hopitaux De Paris

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Digestive System Diseases [C06]

Trial duration

25 Sep 2025 → ongoing

Decision date (initial)

2024-10-23

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

Funding sources

French Ministry of Health (PHRC-N 2018)

External identifiers

EU CT number

2024-516099-13-00

EudraCT number

2019-003433-41

ClinicalTrials.gov

NCT06433180

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy

To evaluate the efficacy of oral capsules containing frozen fecal microbiota (FMT) vs sham FMT on IBS severity score at 12 weeks in patients with irritable bowel syndrome with severe disease refractory to conventional treatments

Secondary objectives 10

1. To evaluate the efficacy of oral capsules containing frozen fecal microbiota (FMT) vs sham FMT on IBS severity score at 12 weeks in patients with irritable bowel syndrome with severe disease refractory to conventional treatments (at least a 50 points decrease in IBS-SSS).
2. FMT success: patient’s microbiota 12 weeks after FMT closer to that of the donor than the patient’s microbiota before FMT.
3. Intestinal microbiota composition and diversity at week 12 and 24 assessed by 16s sequencing
4. Efficacy (decrease in IBS severity >75 points) at week 24 according to FMT success
5. Efficacy according to European Medical Agency endpoint in IBS (percentage of responders for IBS-D, IBS-C and IBS-M; (EMA Endpoint) defined as a patient who fulfils the response criteria (simultaneous improvement of transit and abdominal pain for at least 50% of the observation time) at week 12 and 24.
6. IBS severity at 12 weeks by donors (one donor giving FMT to several patients)
7. IBS severity (IBS-SSS) at 12 and 24 weeks by IBS subtypes according to transit pattern
8. IBS Quality of life (IBS-QoL score) at 12 weeks and 24 weeks
9. Patient’s perception of FMT (questionnaire for correct assessment of FMT or placebo and FMT acceptability)
10. Safety (Serious Adverse Events, Adverse Events) compared between groups

Conditions and MedDRA coding

Severe Irritable Bowel Syndrome (IBS)

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

1. Age >= 18 years and < 75 years
2. IBS defined according to Rome IV definition (IBS-C, IBS-D or IBS-M)
3. Severe disease (IBS-SSS >300) and refractory to at least two previous treatment strategies
4. Patient with health insurance (AME excepted)
5. Informed Written consent
6. For women with childbearing potential, efficient contraception for the duration of the participation to the study

Exclusion criteria 12

1. Other gastrointestinal disease (celiac disease, inflammatory bowel disease)
2. Participants if there is a reason to suspect an alternative diagnosis to the IBS complaints
3. Surgical intervention in the gastrointestinal region except for appendectomy, hernia repair, cholecystectomy and hemorroidectomy
4. Treatment preceding FMT with antibiotics, antifungic or probiotics treatment < 4 weeks, or factors that may affect the composition of intestinal microbiota
5. Abuse of alcohol or drugs
6. Pregnancy or breastfeeding
7. Participation in any other interventional study
8. Patients under legal protection
9. Acute COVID-19 infection
10. Presence of systemic disease, immune deficiency or treatment with immune-modulating Medication
11. Severe psychiatric disorder
12. Participants who were assessed as likely to be noncompliant (ie, not adhering to the tasks they were to perform as participants)

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Decrease in IBS severity at 12 weeks is defined by the percentage of patients having at least a 75 points decrease in IBS-SSS

Secondary endpoints 10

1. Decrease in IBS severity at 12 weeks defined by the percentage of patients having at least a 50 points decrease in IBS-SSS.
2. FMT success : The composition of the patient’s fecal microbiota 12 weeks after FMT will be compared to the patient’s microbiota before transplantation and to the donor using the Sorensen similarity index. The FMT will be considered as a success if the Sorensen index [patient after FMT vs donor] > Sorensen index [patient after FMT vs patient before FMT] and if the Sorensen index [patient after FMT vs donor] ≥ 0,6. The composition of fecal microbiota will be measured by pyrosequencing (16S RNA).
3. Intestinal microbiota composition and diversity at week 12 and 24 assessed by 16s sequencing. Microbiota composition and diversity assessed by 16s sequencing at week 12 and 24, compared to baseline and to healthy volunteers donor’s microbiota. Microbiota composition will be assessed using Qiime pipeline and analyzed at all phylogenetic levels. Diversity will be evaluated using Shannon index, Simpson index, Chao1 index and number of observed species
4. Efficacy at week 24 according to FMT success : decrease in IBS severity >75 points
5. EMA Endpoint at week 12 and 24 defined as a patient who fulfils the response criteria (simultaneous improvement of transit and abdominal pain) displayed in the following for at least 50% of the observation time
6. Percentage of responders in the different subgroups IBS-D, IBS-C and IBS-M using the primary endpoint at week 12 and 24.
7. Mean IBS-SSS (IBS severity), comparison between FMT and placebo at 12 and 24 weeks)
8. Mean IBS-QoL score (IBS Quality of life) comparison between FMT and placebo at 12 and 24 weeks (Drossman et al. 2000)
9. Patient’s perception of FMT : 1/Questionnaire for correct assessment of FMT or placebo and FMT acceptability at V2 (FMT administration) (Annex D). 2/ Questionnaire for assessment of FMT secondary effects à V3 (Annex E)
10. Safety (Serious Adverse Events, Adverse Events) compared between groups

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Assistance Publique Hopitaux De Paris

Sponsor organisation

Assistance Publique Hopitaux De Paris

Address

Porte 23, 1 Avenue Claude Vellefaux 1 Avenue Claude Vellefaux

City

Paris Cedex 10

Postcode

75475

Country

France

Scientific contact point

Organisation

Assistance Publique Hopitaux De Paris

Contact name

Coordinator Investigator

Public contact point

Organisation

Assistance Publique Hopitaux De Paris

Contact name

Coordinator Investigator

Locations

1 EU/EEA country · 8 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 5 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

2 submissions — initial application plus substantial / non-substantial modifications