A Phase 3, double-blind, randomized, placebo controlled, 2-arm study to investigate the safety and efficacy of efimosfermin alfa injection in adult participants with compensated cirrhosis due to metabolic dysfunction-associated steatohepatitis (NEBULA-1)
This clinical trial is looking at a new injectable medicine called efimosfermin alfa. It is designed for adults with advanced fatty liver disease, known as metabolic dysfunction-associated steatohepatitis (MASH), who have developed compensated cirrhosis. Cirrhosis means the liver has significant scarring, but 'compensated' means it is still able to carry out its normal day-to-day functions. The main goal is to find out if regular injections of efimosfermin alfa can slow down liver damage, reduce scarring, and protect against serious liver complications. Participants will be placed by chance into one of two groups: one receiving the real medicine and the other receiving a dummy injection (placebo). Neither the participants nor their doctors will know who gets which treatment. Researchers will monitor liver health through specialised scans, blood tests, and health questionnaires over the course of the study.
At a glance
What is this study about?
Metabolic dysfunction-associated steatohepatitis (MASH), formerly known as NASH, is an advanced form of fatty liver disease. Over time, an unhealthy build-up of fat causes the liver to become inflamed and swollen. In some people, this persistent inflammation leads to thick scar tissue, a condition called cirrhosis. In 'compensated' cirrhosis, the liver is heavily scarred but still manages to work relatively well. However, without treatment, it can progress to liver failure or cause other serious health problems.
This study, called NEBULA-1, is investigating an experimental medicine called efimosfermin alfa. The treatment is given as an injection under the skin. Scientists hope that it can help reduce liver inflammation, lower fat levels, and even reverse or stabilise liver stiffness and scarring. This could help prevent the disease from getting worse.
Because this is a Phase 3 trial, it is in the final testing stages before a medicine can be approved for general use. Researchers will compare efimosfermin alfa against a placebo (an injection with no active medicine) to see how well it works and to check for any unwanted side effects. Finding effective treatments for MASH-related cirrhosis is vital, as there are currently very few approved medical therapies available.
Key takeaways
- Tests a new injection called efimosfermin alfa for adults with MASH and compensated cirrhosis.
- Compares the study drug to a dummy injection (placebo) to test its safety and effectiveness.
- Aims to find out if the treatment can reduce liver scarring and prevent liver disease from getting worse.
- Includes non-invasive liver scans, blood tests, and quality-of-life surveys.
- Taking part is completely voluntary, and you can withdraw at any point.
Who may be eligible?
This trial is open to adults aged 18 and older who have been diagnosed with compensated cirrhosis linked to MASH. Compensated cirrhosis means that while your liver has scarring, it has not yet caused life-threatening complications such as severe fluid buildup in your tummy or internal bleeding.
To join, participants must be well enough to attend regular clinic appointments and willing to receive regular injections under the skin. Doctors will also review your medical history and run tests to ensure your liver function is stable.
You may not be eligible if your liver disease has become 'decompensated' (meaning the liver is failing), if you have severe liver damage from other causes such as heavy alcohol use or viral hepatitis, or if you have certain other serious medical conditions that could make taking part unsafe.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- I am aged 18 or older.
- I have been diagnosed with MASH (or NASH) fatty liver disease.
- My doctor has told me I have liver cirrhosis, but it is currently stable (compensated).
- I am comfortable receiving injections under the skin.
- I am able and willing to attend regular clinic check-ups and undergo liver scans.
What does participation involve?
Taking part involves attending regular hospital or clinic visits. During these visits, doctors and nurses will closely monitor your general health and liver condition. You will have regular blood tests to check your liver function, cholesterol, and blood sugar levels. You will also have non-invasive liver scans, such as a FibroScan or a specialised MRI, which measure how stiff or scarred your liver is without needing surgery.
You will be taught how to receive the study medicine, which is given as an injection under the skin, or clinic staff will give it to you. You will also fill out short questionnaires asking about your energy levels, tiredness, and daily quality of life. The study lasts over an extended period so that the study team can track your long-term health and see if the treatment prevents liver complications.
Potential risks and benefits
Locations (10)
- —UnverifiedNetherlands
- —UnverifiedGreece
- —UnverifiedAustria
- —UnverifiedSpain
- —UnverifiedItaly
- —UnverifiedFrance
- —UnverifiedBelgium
- —UnverifiedBulgaria
- —UnverifiedGermany
- —UnverifiedPoland
Common questions
What is MASH?
MASH stands for metabolic dysfunction-associated steatohepatitis. It is a form of fatty liver disease where excess fat causes swelling and inflammation, which can eventually lead to liver scarring.
What does compensated cirrhosis mean?
Cirrhosis means severe scarring of the liver. 'Compensated' means the liver is scarred, but it can still do its job and has not yet caused severe symptoms or liver failure.
What is a placebo?
A placebo is a dummy treatment that looks just like the real medicine but contains no active ingredients. It helps doctors confirm whether the study medicine is truly responsible for any health changes.
Will taking part cost me anything?
No, all study-related visits, tests, and study medications are provided free of charge. Most clinical trials also help cover travel expenses for clinic visits.
Can I leave the trial if I change my mind?
Yes. Joining a clinical trial is entirely your choice. You are free to stop participating at any time, and doing so will never affect your standard NHS healthcare.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
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