Study in Patients With Decompensated Liver Cirrhosis

NCT06380335 | Active Not Recruiting

• 1 other identifier: RTX001-01S
• Study Type: observational
• Target: 24
• Locations: 2 countries
• Sites: 12

Brief Summary

OPAL is a multicenter observational study, following the natural disease trajectory of participants who have permanent damage to their liver caused by scarring, sometimes also referred to as liver cirrhosis. These participants will also have recently had an acute worsening of their liver disease, which is also known as a hepatic decompensating event, which has resulted in them being admitted to hospital or required them to seek medical attention as an outpatient.

Conditions

Liver Cirrhosis

Keywords

Liver cirrhosis; Cirrhotic; Hepatic Cirrhosis; Chronic Liver Disease; Liver Fibrosis; Decompensated liver cirrhosis; Child-Pugh Score; MELD score; Liver function tests; Hepatic encephalopathy; End Stage Liver Disease (ESLD); Retractable ascites; Variceal Bleeding

Outcome Measures

Primary Outcomes (6)

• Examine the characteristics of patients admitted to hospital with hepatic decompensation.

  Demographics

  Baseline and up to 96 weeks

• Examine the characteristics of patients admitted to hospital with hepatic decompensation.

  Aetiology of liver disease

  Baseline and up to 96 weeks

• Examine the characteristics of patients admitted to hospital with hepatic decompensation.

  Disease co-morbidities

  Baseline and up to 96 weeks

• Examine the characteristics of patients admitted to hospital with hepatic decompensation.

  Alcohol use

  Baseline, and up to 96 weeks

• Examine the characteristics of patients admitted to hospital with hepatic decompensation.

  Changes in Model for End-Stage Liver Disease (MELD) score

  Baseline, and up to 96 weeks

• Examine the characteristics of patients admitted to hospital with hepatic decompensation.

  Safety laboratory (biochemistry and haematology) parameters

  Baseline, and up to 96 weeks

Secondary Outcomes (11)

• Follow the natural history of patients admitted to hospital with hepatic decompensation.

  Screening up to 96 weeks

• Follow the natural history of patients admitted to hospital with hepatic decompensation.

  Screening up to 96 weeks

• Follow the natural history of patients admitted to hospital with hepatic decompensation.

  Screening up to 96 weeks

• Follow the natural history of patients admitted to hospital with hepatic decompensation.

  Screening up to 96 weeks

• Examine the characteristics and natural history of patients with medically refractory ascites that do not require hospitalisation.

  Screening up to 96 weeks

Other Outcomes (2)

• Explore clinical and laboratory parameters.

  Screening up to 96 weeks

• Evaluate the effect of disease progression on biomarkers of inflammatory activity

  Screening up to 96 weeks

Study Arms (2)

Group 1

Patients with liver cirrhosis who have been hospitalised following a recent hepatic decompensation which is at least 6 months after any hospitalisation for a prior decompensation event.

Group 2

Out-patients with medically refractory ascites that recurs (i.e., second LVP) within a 6-month period.

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

1. Patients with liver cirrhosis who have been hospitalized for hepatic decompensation. 2. Out-patients with refractory ascites that does not require in-patient admission.

You may qualify if:

• Male or female age ≥18-75 years.
• Patient is willing and able to provide informed consent to participate in the study.
• Patient confirms willingness/ability to comply with all study procedures.
• Has a diagnosis of liver cirrhosis determined by a physician based on at least one of the following:
• clinical and radiological features that correlate with a diagnosis of cirrhosis;
• transient elastography (TE) (FibroscanTM) \>15kPa;
• previous liver biopsy confirming histological features of cirrhosis.
• \. For eligibility at Screen Part 2 - Aetiology of liver disease of steatotic liver disease (SLD) including pure metabolic dysfunction associated steatotic liver disease (MASLD) or metabolic and alcohol related/associated liver disease (Met-ALD), or alcohol-related liver diseases (ALD).
• a. Patients with ALD or Met-ALD only if they are confirmed to not be drinking alcohol above Met-ALD limits defined in this protocol AND have phosphatidyl ethanol (PEth) test \<200 ng/ml. (N.B. No more than 34% of the total patients in this protocol will be ALD \[excludes Met-ALD\]).
• Meets one of the following criteria:
• a. Hospitalised as an in-patient for a recent major hepatic decompensation event (qualifying event) including ascites, HE or variceal bleed, HRS-AKI or SBP, this being the only hospitalisation for an hepatic decompensation event within the last 6 months, and where recent is defined as within 6 weeks of hospital discharge OR
• Out-patient: Medically refractory ascites is defined by the repeated (≥ 2) need for LVP (i.e., therapeutic, not diagnostic) at least once per 8 weeks despite best medical attempts to control the ascites by sodium restriction and diuretic treatment, as confirmed by the Investigator, with date on of onset \[defined as the date of the second therapeutic paracentesis\] occurring within the past 6 months.
• MELD 3.0 score of 12-20 taken within 2 weeks of qualifying event.
• Has stabilised post-hepatic decompensation event, as defined by two MELD assessments (within 1 point of each) other taken within 2 weeks, or physician assessed as stable. with the ability to safely cell mobilise with GCSF, apherese and received RTX001 treat in the interventional Phase 1/2 study.

You may not qualify if:

• Liver cirrhosis due to:
• any viral hepatitis , or
• autoimmune and cholestatic aetiologies including, but not limited to, primary biliary cholangitis and primary sclerosing cholangitis.
• Acute liver disease in the absence of underlying liver cirrhosis, including, but not limited to, drug induced liver injury.
• Any current organ failure requiring more than out-patient non-invasive supportive care, and not associated with the patient's qualifying hepatic decompensation event.
• Known splenomegaly ≥16cm.
• Thrombocytopenia \<50,000 mm3.
• Sepsis (with positive microbial cultures) or as defined by the Investigator, unless stable and is at least 4 weeks after having completed a full course of intravenous antibiotics.
• Presence or suspicion of any of the following co-morbidities:
• a. history of liver transplantation or other organ transplant;
• ACLF;
• known human immunodeficiency virus;
• pulmonary embolism;
• hepatocellular carcinoma, or active malignant disease within the last 5 years, (excluding non-melanoma skin cancer, cervical carcinoma in situ, superficial bladder cancer, benign polyps etc.);
• co-hepatic morbidities e.g., portal vein thrombosis;

Sponsors & Collaborators

• Resolution Therapeutics Limited: lead

Study Sites (12)

Hospital Universitario Reina Sofía

Córdoba, 14004, Spain

Location

Hospital General Universitario Gregorio Marañón

Madrid, 28007, Spain

Location

Bristol Royal Infirmary

Bristol, BS2 8HW, United Kingdom

Location

Royal Infirmary Edinburgh

Edinburgh, EH16 4SA, United Kingdom

Location

Leeds Teaching Hospital NHS Trust

Leeds, LS9 7TF, United Kingdom

Location

Royal Liverpool Hospital

Liverpool, L7 8XP, United Kingdom

Location

King's College Hospital

London, SE5 9RS, United Kingdom

Location

St George's Hospital

London, SW17 0QT, United Kingdom

Location

St Mary's Hospital

London, W2 1NY, United Kingdom

Location

Nottingham University Hospital

Nottingham, NG5 1PB, United Kingdom

Location

Southampton General Hospital

Southampton, SO16 6YD, United Kingdom

Location

Sunderland Royal Hospital

Sunderland, SR4 7TP, United Kingdom

Location

Biospecimen

Retention: SAMPLES WITHOUT DNA

Plasma

MeSH Terms

Conditions

Liver Cirrhosis; Hepatic Encephalopathy; End Stage Liver Disease

Condition Hierarchy (Ancestors)

Liver Diseases; Digestive System Diseases; Fibrosis; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Liver Failure; Hepatic Insufficiency; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: PROSPECTIVE
• Sponsor Type: NETWORK
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 9, 2024
• First Posted: April 23, 2024
• Study Start: October 10, 2023
• Primary Completion (Estimated): April 3, 2030
• Study Completion (Estimated): April 3, 2030
• Last Updated: January 29, 2026

Record last verified: 2026-01

Locations

ES (2); GB (10)