FMT in IT-refractory HCC - FAB-HCC Pilot Study

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Medical University Of Vienna

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Digestive System Diseases [C06]

Trial duration

4 Aug 2023 → 17 Mar 2025

Decision date (initial)

2024-12-10

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

External identifiers

EU CT number

2024-516835-29-00

EudraCT number

2022-000234-42

ClinicalTrials.gov

NCT05750030

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Therapy, Efficacy

Safety as measured by incidence and severity of treatment-related adverse events, with severity determined according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 5.0

Secondary objectives 4

1. Efficacy as assessed by best radiological response according to mRECIST and RECIST v1.1 criteria
2. Efficacy as assessed by objective response rate and disease control rate
3. Efficacy as assessed by progression-free survival and overall survival
4. Quality of life assessed by EQ-5D-5L questionnaire

Conditions and MedDRA coding

Hepatocellular Carcinoma

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 12

1. Signed informed consent form
2. Age ≥ 18 years
3. Histologically or radiologically confirmed HCC
4. Patients with progressive disease (according to mRECIST) during treatment with atezolizumab/bevacizumab (without or with prior complete or partial response as best radiological response according to mRECIST) OR patients with stable disease as best radiological response (according to mRECIST) after the first 12 months of atezolizumab/bevacizumab treatment
5. Negative HIV test
6. Patients with chronic hepatitis B must be under antiviral treatment and hepatitis B DNA must be <500 IU/mL
7. Variceal status must be known and if present, adequate medical or endoscopic treatment is required
8. ECOG Performance Status 0-1
9. Child-Pugh class A-B8
10. Adequate hematological and end-organ function, defined as follows: -AST and ALT < 10 x ULN -Serum bilirubin <3.5 mg/dL - Albumin ≥ 28 g/L - Serum creatinine ≤ 1.5 mg/dL - Hemoglobin ≥ 8 mg/dL - Platelet count ≥ 50 G/L - Leukocytes ≥ 2.5 G/L -Patients not receiving therapeutic anticoagulation: INR ≤ 2.3 or thromboplastin time ≥ 40%
11. Women of childbearing potential must agree to remain abstinent (refrain from heterosexual intercourse) or use contraceptive methods
12. Men must agree to remain abstinent (refrain from heterosexual intercourse) or use a condom

Exclusion criteria 15

1. Known fibrolamellar carcinoma or mixed cholangiocellular carcinoma
2. Massive tumor progression (>100% increase in target lesions or progression associated with significant clinical deterioration)
3. Uncontrolled ascites
4. Overt hepatic encephalopathy or concomitant treatment with rifaximin
5. Prior allogeneic stem cell or solid organ transplantation
6. Active or history of severe autoimmune disease
7. History of idiopathic pulmonary fibrosis, organizing pneumonia (e.g., bronchiolitis obliterans), drug-induced pneumonitis, or idiopathic pneumonitis, or evidence of active pneumonitis
8. Significant cardiovascular disease (such as New York Heart Association Class II or greater cardiac disease, myocardial infarction, or cerebrovascular accident) within 3 months prior to study inclusion or unstable angina
9. Severe infection within 4 weeks prior to study inclusion
10. Pregnant or breastfeeding women
11. Treatment with systemic immunosuppressive medication with the following exceptions: Acute, low-dose systemic immunosuppressant medication or a one-time pulse dose of systemic immunosuppressant medication (e.g., 48 hours of corticosteroids for contrast allergy) -Mineralocorticoids (e.g., fludrocortisone), corticosteroids for chronic obstructive pulmonary disease or asthma, or low-dose corticosteroids for adrenal insufficiency
12. Significant vascular disease (e.g., peripheral arterial thrombosis) within 6 months prior to study inclusion
13. Major surgery within 4 weeks prior to study inclusion or minor surgery (excluding placement of a vascular access device) within 3 days prior to study inclusion
14. History of gastrointestinal fistula or perforation, or intraabdominal abscess within 6 months prior to study inclusion
15. Serious, non-healing wound or active ulcer

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. The primary endpoint will be analyzed using descriptive statistics. In detail, the incidence and severity of treatment-related adverse events determined according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 5.0 will be described.

Secondary endpoints 2

1. Efficacy will be evaluated by the number (percentage) of study participants achieving complete response (CR), partial response (PR), stable disease (SD) or progressive disease (PD) as best radiological response evaluated according to mRECIST and RECIST v1.1 criteria. Objective response is defined as either complete or partial response, while disease control rate comprises complete/partial response as well as stable disease. Kaplan-Meier method will be used to calculate progression-free survival
2. Distribution of quality of life parameters will be assessed by plotting histograms. Baseline and follow-up values will be demonstrated as mean (+/- standard deviation) or median (IQR), as applicable. Changes of quality of life during the study period as assessed by EQ-5D-5L questionnaire will be evaluated using paired T-test or Wilcoxon signed rank test.

Investigational products

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

Test 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Medical University Of Vienna

Sponsor organisation

Medical University Of Vienna

Address

Spitalgasse 23, Alsergrund Alsergrund

City

Vienna

Postcode

1090

Country

Austria

Scientific contact point

Organisation

Medical University Of Vienna

Contact name

Division of Gastroenterology and Hepatology

Public contact point

Organisation

Medical University Of Vienna

Contact name

Division of Gastroenterology and Hepatology

Locations

1 EU/EEA country · 1 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

Summary of results Art. 37(4) CTR

Layperson summary Annex V

Documents 12 files

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

Application history

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