Safety profile of autologous macrophage therapy for liver cirrhosis.
Francesca Moroni, Benjamin J Dwyer, Catriona Graham...
https://pubmed.ncbi.nlm.nih.gov/31591593Actively Recruiting
Phase 1
Phase 2
Age: 18Years - 75Years
All Genders
ID06823713
An Open-label Phase 1/2 Study to Evaluate the Safety, Tolerability, and Efficacy of RTX001 Autologous Macrophages in Liver Cirrhosis with Hepatic Decompensation
Led by Resolution Therapeutics Limited · Updated on 2026-01-28
30
Participants Needed
14
Research Sites
13 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and effectiveness of RTX001, an autologous engineered macrophage cell therapy, in patients with end-stage liver disease who have recently experienced hepatic decompensation. This first-in-human Phase 1/2 open-label study aims to understand how RTX001, made from a person's own white blood cells, affects liver function and disease progression compared to data from a similar patient population in an external natural history study. Participants will undergo leukapheresis to collect white blood cells, which are then used to manufacture RTX001. After manufacturing, participants are assigned to one of two groups based on their current liver stability: those who are stable and those who have had another decompensation event but have since stabilized. Both groups will receive up to four intravenous infusions of RTX001. Treatment timing depends on the participant's clinical status. During the study, participants will be monitored closely for safety and tolerability at each infusion and up to two weeks afterwards, with follow-up continuing for 2.5 years. Researchers will assess clinical events, mortality, and changes in liver disease severity using the Model for End Stage Liver Disease score. Participants will have regular evaluations including medical assessments and laboratory tests throughout the study to track the therapy's impact and ensure safety.
CONDITIONS
Brief Title
RTX001 Autologous Engineered Macrophages for Liver Cirrhosis
Who Can Participate
Age: 18Years - 75Years
All Genders
Eligibility Criteria
You may qualify if you...
- Male or female aged 18 to 75 years
- Willing and able to comply with all study procedures
- Diagnosis of liver cirrhosis by clinical and radiological features, Fibroscan >15 kPa, or prior biopsy
- Liver disease caused by steatotic liver disease including MASLD, Met-ALD, or ALD with controlled alcohol intake
- Hospitalized recently for a major hepatic decompensation event within 6 months or medically refractory ascites recurring within 6 months
- Confirmed alcohol test below 200 ng/ml
- MELD score between 12 and 20 within two weeks of qualifying event
- No known contraindications to filgrastim or leukapheresis
- Use of contraception consistent with local regulations
- Able and willing to give signed informed consent or assent
You will not qualify if you...
- Liver cirrhosis caused by viral hepatitis, autoimmune or cholestatic diseases
- Acute liver disease without cirrhosis
- Organ failure needing more than outpatient care unrelated to qualifying event
- Known splenomegaly ≥16 cm
- Platelet count below 50 x10⁹/L
- History of organ transplant, ACLF, sepsis unless stable after antibiotics, HIV, syphilis, HTLV-1, pulmonary embolism, hepatocellular carcinoma or active cancer in last 5 years
- Portal vein thrombosis or significant co-hepatic conditions
- Clinically significant hydrothorax needing intervention
- Chronic kidney failure on dialysis or unresolved acute kidney injury
- Severe heart failure (NYHA Grade III/IV), porto-pulmonary hypertension
- Severe chronic lung disease with low lung function
- Hepatopulmonary syndrome
- Previous multiple albumin infusions for therapy
- Unstable or untreated psychiatric disease
- Transjugular intrahepatic portosystemic shunt (TIPSS)
- Intercurrent life-threatening or significant illness
- Current or planned immunomodulator or immunosuppressive use (low dose corticosteroids or inhaled steroids allowed)
- Previous gene or cell therapy
- Recent or planned live attenuated vaccine use
- Recent investigational product use or participation in another trial
- Known allergy to dimethyl sulfoxide (DMSO)
- Unlikely to comply with study procedures
- Pregnant, breastfeeding, or planning pregnancy within a year
- Alcohol misuse or binge drinking before screening
- Use of non-medically supervised drugs posing high risk or non-compliance
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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2
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Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Treatment
Duration - Up to 2.5 years with infusions and monitoring
Participants undergo leukapheresis to collect white blood cells which are then used to manufacture RTX001, an autologous engineered macrophage cell therapy. Following manufacturing, participants receive up to four intravenous infusions of RTX001. Participants are assigned to groups based on their liver cirrhosis compensation status, but treatment is the same for all.
1 leukapheresis visit and up to 4 infusion visits with safety monitoring at each infusion and for two weeks post-infusion
Follow-up
Duration - Up to 2.5 years after the last infusion
Participants are monitored for safety, tolerability, and efficacy outcomes including clinical events, mortality, and liver function changes after treatment completion.
Regular follow-up visits over 2.5 years
Trial Site Locations
Total: 14 locations
1
Hospital Universitari Vall d'Hebron
Barcelona, Spain, 08035
Actively Recruiting
2
Hospital Universitario Reina Sofía
Córdoba, Spain, 14004
Actively Recruiting
3
Hospital General Universitario Gregorio Marañon
Madrid, Spain, 28007
Actively Recruiting
4
Hospital Universitario Ramón y Cajal
Madrid, Spain, 28034
Actively Recruiting
5
Hospital Universitario La Paz
Madrid, Spain, 28046
Actively Recruiting
6
Hospital Universitario Virgen del Rocío
Seville, Spain, 41013
Actively Recruiting
7
Bristol Royal Infirmary
Bristol, United Kingdom, BS2 8HW
Actively Recruiting
8
Royal Infirmary of Edinburgh
Edinburgh, United Kingdom, EH16 4SA
Actively Recruiting
9
Glasgow Royal Infirmary
Glasgow, United Kingdom, G4 0SF
Actively Recruiting
10
Royal Liverpool University Hospital
Liverpool, United Kingdom, L7 8YE
Actively Recruiting
11
King's College Hospital
London, United Kingdom, SE5 9RS
Actively Recruiting
12
St George's Hospital
London, United Kingdom, SW17 0QT
Actively Recruiting
13
St Mary's Hospital
London, United Kingdom, W2 1NY
Actively Recruiting
14
Nottingham University Hospital
Nottingham, United Kingdom, NG5 1PB
Actively Recruiting
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Research Team
R
Resolution Therapeutics Clinical Enquiries
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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