Ongoing, recruitingPhase II and Phase III (Integrated)Interventional

A two-part randomized, double-blind, placebo-controlled multicenter dose ranging and confirmatory study to assess the safety and efficacy of VAY736 in autoimmune hepatitis patients with incomplete response to or intolerance of standard therapy (AMBER)

This study aims to find out if a new medicine, VAY736, can help adults who have autoimmune hepatitis. This is a condition where the body's immune system mistakenly attacks the liver. The study is for people whose current treatments aren't fully working or are causing problems. Researchers want to see if VAY736 is safe and if it can help bring their liver enzyme levels, specifically ALT, back to normal. Participants will either receive VAY736 or a placebo (a dummy treatment) to compare their effects. This will help understand if VAY736 could be a better option for people living with this liver condition.

At a glance

Status

Ongoing, recruiting

Phase

Phase II and Phase III (Integrated)

Sponsor

Novartis Pharma AG

Enrolment target

Start

31 Jan 2024

Autoimmune hepatitis

What is this study about?

This research study is about a new medicine called VAY736, for adults who have a liver condition known as autoimmune hepatitis. In this condition, your body's immune system mistakenly attacks and damages your liver. Currently, there are treatments available, but they don't always work perfectly for everyone, or they might cause side effects that are hard to manage.

The main goal of this study is to see if VAY736 is safe and effective in helping your liver get healthier. Specifically, the researchers are looking to see if it can help bring a particular liver enzyme, called ALT, back to a normal level. High ALT levels can be a sign that your liver is inflamed or damaged. By taking part, you'd be helping doctors understand if VAY736 could be a new and better way to manage autoimmune hepatitis for people who haven't had good results with standard treatments.

Participants in the study will be randomly assigned to either receive VAY736 or a 'placebo.' A placebo looks just like the real medicine but contains no active drug. This comparison helps researchers truly understand if any improvements are due to VAY736 itself, rather than other factors. This type of study design, where neither you nor your doctor knows which treatment you're getting, is common and helps ensure the results are accurate and unbiased.

Key takeaways

This study is testing a new drug, VAY736, for autoimmune hepatitis.
It's for adults whose current treatments aren't working well or cause side effects.
The main goal is to see if VAY736 is safe and improves liver health (ALT levels).
Participants will receive either VAY736 or a dummy treatment (placebo).
Close medical monitoring and regular clinic visits are part of taking part.
You can stop participating at any time.

Who may be eligible?

To join this study, you need to be an adult, aged 18 or older. Both men and women can take part.

Crucially, you must have been diagnosed with autoimmune hepatitis. The study is specifically for those whose existing treatments for autoimmune hepatitis are either not fully controlled or are causing significant side effects that you can't tolerate.

The medical team will carefully review your health information and medical history to make sure that the study is a safe and suitable option for you. There will be specific health requirements that need to be met to ensure your safety and the accuracy of the study results.

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.

Are you 18 years old or older?
Have you been diagnosed with autoimmune hepatitis?
Are your current treatments for autoimmune hepatitis not fully effective or causing problems?
Are you able to attend regular clinic visits and follow study instructions?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you'll be involved in this study over a period of time, which will include regular visits to a clinic. During these visits, the study team will carry out health checks, take blood samples to measure your liver health (like ALT levels), and ask about how you're feeling and any side effects you might be experiencing. You'll be given either the new medicine (VAY736) or a dummy treatment (placebo) as an infusion or injection, according to a set schedule.

The study involves both a dose-finding part, where different amounts of VAY736 might be tested, followed by a larger part to confirm the best dose. You'll be closely monitored throughout this time to ensure your safety. There will also be follow-up appointments after your treatment period finishes to check on your long-term health. The total duration of your participation in the study will be explained in detail by the study team.

Potential risks and benefits

Participating in this study might offer some potential benefits. You would receive close medical attention and monitoring from a team of specialists, and you might get access to a new treatment, VAY736, before it's widely available. This new treatment could potentially help improve your autoimmune hepatitis if your current treatments aren't working as well as hoped. However, there are also potential risks involved. VAY736 is an investigational drug, meaning its full side effects and risks are not yet completely known. You might experience side effects from the medication or from the procedures involved, like blood tests or infusions. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (2)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Czechia
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Unverified
Germany

Common questions

What is autoimmune hepatitis?

It's a liver condition where your body's immune system mistakenly attacks your own liver, causing inflammation and damage.

What does 'placebo' mean?

A placebo is a dummy treatment. It looks exactly like the real medicine but doesn't contain any active drug. It helps researchers compare the true effects of the new medicine.

Why would I get a placebo?

By comparing the new medicine to a placebo, doctors can be sure that any improvements seen are truly due to the medicine and not just other factors, making the study results more reliable.

What does 'ALT normalization' mean?

ALT is a liver enzyme. 'Normalization' means that the level of this enzyme in your blood has returned to a healthy, normal range, which is a sign of improved liver health.

Will I know if I'm getting the new drug or the placebo?

No, in this type of study, neither you nor your doctor will know whether you are receiving VAY736 or the placebo until the study is complete. This helps to keep the results unbiased.