Study of emapalumab in children and adults with Macrophage Activation Syndrome (MAS) occurring in Still’s Disease or Systemic Lupus Erythematosus

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What is this study about?

This study focuses on treating Macrophage Activation Syndrome (MAS), a severe inflammatory condition that can occur in patients with Still’s Disease (including both children and adults) and Systemic Lupus Erythematosus. The condition being studied causes the immune system to become overactive, leading to inflammation throughout the body. The study will test a medication called emapalumab, which is given through an intravenous infusion.

The purpose of this research is to determine if emapalumab can effectively treat MAS in patients who have not responded well enough to standard treatments with high-dose steroids. The medication works by targeting specific proteins in the body that are involved in inflammation. The study will include both children and adults between 6 months and 80 years of age.

During the study, participants will receive emapalumab through an intravenous infusion for up to 28 days. The maximum daily dose will be 6 milligrams per kilogram of body weight. Patients will be monitored for 8 weeks after starting the treatment to evaluate how well the medication works. The study will look at how the treatment affects inflammation levels in the body and whether it allows patients to reduce their steroid medication use.

1 Initial eligibility verification

Your age should be between 6 months and 80 years

You must have a confirmed diagnosis of active MAS (Macrophage Activation Syndrome) with fever and specific blood test results

You must have already received high-dose intravenous steroids for at least 3 days without adequate improvement

2 Treatment initiation

You will receive emapalumab through an intravenous infusion

The treatment will continue while monitoring your response

Your current steroid dose may be gradually reduced based on your response to treatment

3 Monitoring period

Your response to treatment will be evaluated over 8 weeks after starting emapalumab

Regular blood tests will be performed to check your condition

Your doctor will monitor for any signs of MAS recurrence

4 Safety requirements

If you are a woman who can become pregnant, you must use effective birth control during treatment and for 6 months after the last dose

Acceptable birth control methods include hormone-containing contraceptives, intrauterine devices, or other approved methods

Regular safety assessments will be conducted throughout the treatment period

5 Treatment completion

The study will evaluate if your condition has improved completely or partially

Your steroid dose will be reviewed to determine if it can be reduced to less than half of the starting dose

Long-term monitoring will continue to check for any return of MAS symptoms

Who Can Join the Study?

Age requirement: Patients must be between 6 months and 80 years old at the time of active MAS diagnosis
Must provide informed consent (for adults) or have consent from legal representatives with patient assent when applicable
Must have active MAS (Macrophage Activation Syndrome) with:
- Fever and high ferritin levels (above 684 ng/mL)
- At least 2 of the following:
  - Low blood platelet count (≤181 x109/L)
  - Elevated liver enzymes (AST >48 U/L)
  - High blood fat levels (triglycerides >156 mg/dL)
  - Low fibrinogen levels (≤360 mg/dL)
Must have inadequate response to high-dose intravenous steroids given for at least 3 days
For Cohort 1 patients:
- Must have confirmed systemic juvenile idiopathic arthritis (sJIA) or Adult onset Still’s disease (AOSD)
For Cohort 2 patients:
- Must have confirmed systemic lupus erythematosus (SLE)
For women who can become pregnant:
- Must use effective birth control methods during treatment and for 6 months after the last dose
- Acceptable methods include hormonal contraception, intrauterine devices, tubal ligation, or having a partner with vasectomy

Who Cannot Join the Study?

Active or chronic infections, including but not limited to tuberculosis (TB), HIV, or hepatitis B or C
Previous treatment with any cell therapy or gene therapy within 6 months before starting the study
History of cancer within the past 5 years (except successfully treated non-melanoma skin cancer)
Severe heart conditions, including heart failure or uncontrolled high blood pressure
Severe liver problems or abnormal liver function tests
Severe kidney problems or significantly reduced kidney function
Current pregnancy or breastfeeding
Known allergic reactions to similar medications
Participation in another clinical trial within the past 30 days
Mental conditions that could interfere with following study procedures
Use of live vaccines within 6 weeks before starting the study
Any condition that, in the opinion of the study doctor, would make participation unsafe

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

No sites found in this category

Other Sites

Site Name	City	Country
IRCCS Istituto Giannina Gaslini	Genoa	Italy
Istituto Di Ricovero E Cura A Carattere Scientifico Materno Infantile Burlo Garofolo	Trieste	Italy
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico	Milan	Italy
Vseobecna Fakultni Nemocnice V Praze	Prague	Czechia
University Hospital Olomouc	Olomouc	Czechia
Ospedale Pediatrico Bambino Gesu’	Rome	Italy
Wrwsddlzgs Ccotumxoo Hzzxhuef	Utrecht	The Netherlands

Trial status

Country	Status	Recruitment Start
Czechia	Not recruiting	12.11.2021
Italy	Not recruiting	12.11.2021
The Netherlands	Not recruiting	12.11.2021

Trial locations

Investigated drugs:

Emapalumab

Emapalumab is a medication that works by targeting and blocking a protein called interferon-gamma, which plays a role in inflammation. It is used to treat Macrophage Activation Syndrome (MAS), a severe inflammatory condition that can occur in patients with Still’s disease and systemic lupus erythematosus. The medication helps control the overactive immune system response and reduce inflammation in the body.

Investigated diseases:

Haemophagocytic lymphohistiocytosis

Macrophage Activation Syndrome (MAS) – A severe inflammatory condition characterized by the abnormal activation and growth of immune system cells called macrophages. It can develop as a complication of other inflammatory diseases, causing fever, enlarged liver and spleen, and changes in blood cell counts.

Systemic Juvenile Idiopathic Arthritis (sJIA) – A form of arthritis that affects children, causing inflammation in joints and other body parts. It typically begins with high fevers, rash, and joint pain, and can affect internal organs.

Adult Onset Still’s Disease (AOSD) – A rare inflammatory disorder that affects adults, similar to systemic juvenile idiopathic arthritis. It causes high fevers, salmon-colored rash, joint pain, and may affect various organs.

Systemic Lupus Erythematosus (SLE) – A chronic autoimmune disease where the immune system attacks healthy tissues throughout the body. It can affect multiple organs, causing inflammation and various symptoms including joint pain, skin rashes, and fatigue.

Trial ID:

2024-516153-52-00

Protocol code:

NI-0501-14

Trial Phase:

Therapeutic use (Phase IV)

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Study of emapalumab in children and adults with Macrophage Activation Syndrome (MAS) occurring in Still’s Disease or Systemic Lupus Erythematosus

What is this study about?

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