Overview
Sponsor-declared trial summary
Phase
Therapeutic exploratory (Phase II)
Status
Authorised, recruitment pending
Participants planned
1
Countries
1
Sites
2
non-alcoholic steatohepatitis
- Assess the efficiency of the FMT in capsule form compared to the placebo regarding liver improvement passed 72 weeks in patients with NASH. - Assess the security and tolerability of the FMT in capsule form in patients with NASH during 72 weeks of treatment.
Key facts
- Sponsor
- Mikrobiomik Healthcare Company S.L.
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Nutritional and Metabolic Diseases [C18]
- Decision date (initial)
- 2025-02-17
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- No
- Vulnerable population
- No
- Funding sources
- Mikrobiomik Healthcare Company S.L.
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Efficacy, Therapy, Safety
- Assess the efficiency of the FMT in capsule form compared to the placebo regarding liver improvement passed 72 weeks in patients with NASH.
- Assess the security and tolerability of the FMT in capsule form in patients with NASH during 72 weeks of treatment.
Secondary objectives 6
- Assess the NASH changes in the liver histology with the FMT in capsule form in comparison to the placebo after 72 weeks.
- Assess the effect of the FMT in capsule form compared to the placebo regarding the liver fibrosis measured by changes in non-invasive markers.
- Assess the effect of the FMT in capsule form compared to the placebo on the fat content.
- Determine the effect of the FMT in capsule form on different metabolic factors after 72 weeks of treatment.
- Determine the effect of the FMT in capsule form on the cardiovascular risk (CVR) after 72 weeks of treatment.
- Assess the effect of the FMT in capsule form on the health results perceived by the patients.
Conditions and MedDRA coding
non-alcoholic steatohepatitis
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 22.0 | PT | 10053219 | Non-alcoholic steatohepatitis | 100000004871 |
Study design 1 period
| # | Title | Allocation | Blinding | Roles blinded | Arms |
|---|---|---|---|---|---|
| 1 | Overall trial The study will consist of two phases; an initial screening phase and a treatment and follow-up phase.
The initial screening phase will begin when a patient signs the Informed Consent (IC), and has a total duration of 12 weeks.
The treatment and follow-up phase will begin 12 weeks after the IC is signed. At the beginning of the treatment phase, the patient will be randomized and assigned to one of the two study groups. The duration of the treatment period is 48 weeks, during which the patient will receive the study treatment in 5 doses, an initial dose of 24 capsules of investigational drug/placebo, and 4 reminder doses of 12 capsules/dose of investigational drug/placebo. Between each of the treatment doses there will be a period of 12 weeks. The total duration of the follow-up period is 24 weeks.
|
Randomised Controlled | Double | [{"id":86300,"code":2,"name":"Investigator"},{"id":86299,"code":1,"name":"Subject"}] | Arm 1: Experimental group Arm 2: Control group |
Regulatory references
- Plan to share IPD
- Yes
- IPD plan description
- Data requests can be submitted after article publication. Access to trial IPD can be requested by qualified researchers engaging in independent and scientific research, and will be provided following review and approval of a research proposal and Statistical Analysis Plan (SAP) and execution of a Data Sharing Agreement (DSA).
| EU CT number | Title | Sponsor |
|---|---|---|
| 2022-500185-94-00 | Randomized, double-blind and multicentric study to assess the efficiency, security and tolerability of the faecal microbiota transfer in capsule form vs. placebo for the treatment of patients with non-alcoholic steatohepatitis (EMOTION) | Mikrobiomik Healthcare Company S.L. |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 6
- Patients of both genders between 18-75 years of age (both included)
- Body mass index <40 kg/m2
- NASH histological diagnosis, accepted by the EASL and the AASLD, of a liver biopsy obtained up to 24 weeks prior to signing the informed consent form
- NASH histological activity score (NAS) ≥ 4, with a score of 1 or more in each subcomponent (steatosis, lobular inflammation and bulging of liver cells)
- Histological evidence of stage 1 fibrosis (with perisinusoidal or portal fibrosis), stage 2 fibrosis (perisinusoidal and portal/periportal fibrosis) or stage 3 fibrosis (bridging fibrosis) as defined by the NASH CRN fibrosis score
- In the case of women and men of childbearing age, for safety, those who agree to follow the required contraceptive measures after signing the informed consent form until the first visit of the follow-up period
Exclusion criteria 20
- Evidence of having another type of liver disease
- High alcohol intake history (daily consumption > 30 g/day for men and > 20 g/day for women)
- Weight loss of over 5% in the 3 months prior to the screening
- Subjects with HbA1c > 9,5%. In the case of subjects with an HbA1c > 9,5% during the selection visit, a repeated test can be performed during the window of selection. A repeated result of HbA1c > 9,5% shall result in exclusion
- Diabetic patients with: •Insulin treatment. • Changes in the antidiabetic medicine in the 4 months prior to the liver biopsy according to the following conditions: -Dose modification of the treatment with Glucagon- like peptide-1 (GLP-1) agonists. -Implementation of the treatment with a new antidiabetic
- History of bariatric surgery
- Cirrhosis
- Portal thrombosis
- Known or suspected hepatocellular carcinoma
- Clinically significant gastrointestinal, cardiovascular, neurological, psychiatric, metabolic, kidney, liver, respiratory, inflammatory, or infectious disease, according to what the investigator determines
- An estimated glomerular filtration rate (eGFR) <45 ml / min / 1,73 m2 (calculated according to the Chronic Kidney Disease Epidemiology Collaboration method [CKD-EPI])
- Medical conditions that lower life expectancy to less than 2 years, including cancer
- Presence of a hereditary or acquired immunodeficiency
- Inflammatory bowel disease diagnosis (Crohn’s disease, ulcerative colitis, microscopic colitis), active irritable bowel syndrome (in the last 2 years according to the Rome IV criteria), celiac disease not well controlled with a gluten-free diet, active gastroparesis, toxic megacolon
- Major intra-abdominal surgery in the 2 months prior to the randomization of the patient in the study
- Intake of antibiotics in the 8 weeks prior to the screening date
- Intake of commercialized probiotics/prebiotics/symbiotic in the 4 weeks prior to the screening date
- Pregnancy or breastfeeding
- Any other condition that, according to the investigator, could impede or hinder compliance
- Use of medication with a potential steatogenic effect (corticosteroids, valproic acid, amiodarone and/or tamoxifen) in the 6 months prior to the first dose of the study drug
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 2
- -Patient ratio with a NASH resolution without fibrosis worsening. -Patient ratio without fibrosis nor activity worsening. -Patient ratio with a MRI-PDFF betterment and without a fibrosis or activity worsening.
- Occurrence of adverse events (AE), severe AE (SAE), AE that result in the interruption of the study’s treatment, AE of special interest, and changes in the vital signs and the laboratory results during 72 weeks of treatment
Secondary endpoints 12
- Patient ratio with betterment regarding the lobular inflammation and/or ballooning without fibrosis worsening
- Changes in fibrosis biomarkers
- MRI-PDFF
- Changes in anthropometric measurements
- Changes in lipid profile
- Changes in inflammation biomarkers
- Changes in insulin resistance
- Changes in vital signs
- Changes in emerging CVR factors
- Changes in the carotid ultrasound
- Changes in the SF-36 questionnaire
- Changes in the CLDQ-NAFLD questionnaire
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
Lyophilized capsules of fecal microbiota
PRD9559185 · Product
- Active substance
- Allogeneic Faecal Microbiota, Pooled
- Pharmaceutical form
- CAPSULE
- Route of administration
- ORAL USE
- Max daily dose
- 6 g gram(s)
- Max total dose
- 6 g gram(s)
- Max treatment duration
- 48 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- MIKROBIOMIK HEALTHCARE COMPANY S.L.
- Paediatric formulation
- No
- Orphan designation
- No
Placebo 1
microcrystalline cellulose Ph Eur, in hypromellose capsules
N/A · Product
- Other product name
- N/A
- Pharmaceutical form
- N/A
- ATC code
- N/A — N/A
- Marketing authorisation
- N/A
- MA holder
- N/A
- MA country
- Iceland
- Paediatric formulation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Mikrobiomik Healthcare Company S.L.
- Sponsor organisation
- Mikrobiomik Healthcare Company S.L.
- Address
- Astondo Bidea 612
- City
- Derio
- Postcode
- 48160
- Country
- Spain
Scientific contact point
- Organisation
- Mikrobiomik Healthcare Company S.L.
- Contact name
- Clinical trial information desk
Public contact point
- Organisation
- Mikrobiomik Healthcare Company S.L.
- Contact name
- Clinical trial information desk
Third parties 1
| Organisation | City, country | Duties |
|---|---|---|
| Sermes Planificacion S.L. ORG-100008509
|
Madrid, Spain | On site monitoring, Code 10, Code 11, Code 12, Code 5, Data management, Code 8 |
Locations
1 EU/EEA country · 2 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Spain | Authorised, recruitment pending | 1 | 2 |
| Rest of world | — | 0 | — |
Investigational sites
Hospital Universitario Marques De Valdecilla
Digestive, Avenida Valdecilla Sn, 39008, Santander
Hospital Universitario Puerta De Hierro De Majadahonda
Gastroenterology and Hepatology, Calle De Manuel De Falla 1, 28222, Majadahonda
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.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_NASH_Protoc 2022-500185-94-00_for pub | 4 |
| Recruitment arrangements (for publication) | 20220310 NASH selection procedure material for pub | 1 |
| Subject information and informed consent form (for publication) | NASH ICF Additional studies_for pub | 2 |
| Subject information and informed consent form (for publication) | NASH ICF_for pub | 2 |
| Synopsis of the protocol (for publication) | NASH protoc summary | 2 |
Application history
1 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-11-28 | Spain | Acceptable with conditions 2025-02-17
|
2025-02-17 |