Ongoing, recruitingPhase II and Phase III (Integrated)Interventional

A Two-cohort, Open-label, Single-arm, Multicenter Study to Evaluate Efficacy, Safety and Tolerability, Pharmacokinetics and Pharmacodynamics of Emapalumab in Children and Adults with Macrophage Activation Syndrome (MAS) in Still's Disease (Including Systemic Juvenile Idiopathic Arthitis and Adult Onset Still's Disease) or with MAS in Systemic Lupus Erythematosus

This research study is investigating a new medication called emapalumab. It's designed for people who have a serious inflammatory condition called Macrophage Activation Syndrome (MAS). MAS can happen when you have certain other illnesses, such as Still's disease (including Systemic Juvenile Idiopathic Arthritis in younger people or Adult Onset Still's Disease) or Systemic Lupus Erythematosus (lupus). The study aims to find out if emapalumab can safely and effectively help treat MAS. Researchers will be looking at how well the treatment works and if there are any side effects. Ultimately, they hope to find a better way to manage MAS for patients.

At a glance

Status

Ongoing, recruiting

Phase

Phase II and Phase III (Integrated)

Sponsor

Swedish Orphan Biovitrum AG

Enrolment target

Start

19 Sep 2024

What is this study about?

This study is about a health problem called Macrophage Activation Syndrome (MAS). MAS is a severe reaction where your immune system, which normally fights off infections, becomes overactive and harms your own body. It can be a complication of other diseases like Still's disease (which includes Systemic Juvenile Idiopathic Arthritis in children and teenagers, and Adult Onset Still's Disease) or Systemic Lupus Erythematosus (commonly known as lupus). These conditions cause inflammation and can lead to MAS, which needs urgent treatment.

The researchers are testing a new medicine called emapalumab. This medicine is designed to calm down the overactive immune system in people with MAS. The main goals of this study are to see how well emapalumab works in bringing MAS under control and if it is safe for patients. They will also look at how the body handles the medicine and how it affects the immune system.

This is a combined Phase 2 and Phase 3 study, meaning it's moving from early testing into larger trials. By participating, you could help doctors understand if emapalumab is an effective and safe option for people living with MAS caused by Still's disease or lupus. This knowledge could help many others in the future.

Key takeaways

This study is testing a new medicine, emapalumab, for Macrophage Activation Syndrome (MAS).
It's for MAS linked to Still's disease (including types in children and adults) or lupus.
The main goal is to see how well it works and if it's safe.
It's open to adults aged 18 and over.
Participation involves regular clinic visits and close monitoring.

Who may be eligible?

To be part of this study, you need to have Macrophage Activation Syndrome (MAS).

This MAS must be linked to either Still's disease (which includes types seen in children and adults) or Systemic Lupus Erythematosus (lupus). The study is open to adults of any age, starting from 18 years old. Both men and women can take part.

There will be other specific health requirements that your doctor will check to make sure the study is a good fit for you and safe for your health.

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.

Do you have Macrophage Activation Syndrome (MAS)?
Is your MAS linked to Still's disease or lupus?
Are you 18 years old or older?
Are you able to attend study visits regularly?

Answer every question to see your result.

What does participation involve?

If you join this study, you will receive the study medication, emapalumab. The total length of your participation will depend on how you respond to the treatment and the study plan, but it will involve regular visits to the clinic. At these visits, doctors will perform examinations, take blood samples, and ask you about your health and any side effects. They will closely monitor your condition to see how the MAS is responding and how you are feeling generally. You may also need to have other tests done throughout the study to track your progress. The study will also look at how your current medications for your underlying condition might be adjusted.

Potential risks and benefits

Taking part in any research study has potential benefits and risks. A potential benefit of this study is that emapalumab might help to control your MAS. However, it's also possible it may not work for you, or you might experience side effects. Your doctor will discuss all the known risks and benefits with you in detail. You are always free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (3)

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

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Unverified
Italy
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Unverified
Czechia
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Unverified
Netherlands

Common questions

What is Macrophage Activation Syndrome (MAS)?

MAS is a serious condition where your immune system becomes overactive and causes widespread inflammation in your body. It often happens as a complication of other conditions like Still's disease or lupus.

What is emapalumab?

Emapalumab is the name of the new medicine being tested in this study. It aims to calm down the overactive immune response in people with MAS.

What is Still's disease?

Still's disease is an inflammatory condition that can affect children (Systemic Juvenile Idiopathic Arthritis) and adults (Adult Onset Still's Disease), causing fever, rash, and joint pain, and can sometimes lead to MAS.

What is lupus?

Lupus (Systemic Lupus Erythematosus) is a long-term autoimmune disease where your immune system mistakenly attacks healthy tissues in many parts of the body, and it can also cause MAS.

Will I still take my regular medicines if I join?

The study will look at how your current medications for your underlying condition might be adjusted. Your study doctor will discuss this with you.