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New drug aims to calm dangerous immune storms in Still's and lupus patients

NCT ID NCT05001737

Disease control Sponsor: Swedish Orphan Biovitrum Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Aug 27, 2026 · Updated 2 times

Summary

This study tested a drug called emapalumab in 33 children and adults with macrophage activation syndrome (MAS), a life-threatening immune overreaction, linked to Still's disease or lupus. The goal was to see if the drug could safely control the condition and reduce the need for high-dose steroids. Participants received emapalumab by IV, and researchers measured how many achieved a complete response by week 8.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
emapalumab (a drug given by IV infusion that blocks a protein called interferon gamma)
What this could lead to
If it works, this could offer a new treatment option for people with severe immune overreaction (MAS) linked to Still's disease or lupus, potentially reducing the need for high-dose steroids.
What could go wrong
This is a small, single-arm study (no placebo group), so results are preliminary. The drug may not work for everyone, and side effects like infections are possible since it suppresses the immune system.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

33 people

The number who actually took part.

Started

Dec 2021

Finished

Jun 2025

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

6 months to 80 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion criteria Run-in phase in all cohorts 1. Informed consent provided by the subject or by the subject s' legally authorized representative(s) with the assent of subjects who are legally capable of providing it, as required by local law. 2. Male and female subjects aged between 6 months and 80 years of age at the time of diagnosis of MAS. 3. MAS defined as per the criteria defined below for each cohort and requiring treatment with GCs. Interventional phase in all cohorts 1. Informed consent provided by the subject or by the subject's legally authorized representative(s) with the assent of subjects who are legally capable of providing it, as as required by local law. 2. Male and female subjects aged between 6 months and 80 years of age at the time of diagnosis of active MAS. 3. Subjects who have shown an inadequate response to high dose intravenous (i.v.) GCs administered for at least 3 days according to local standard clinical practice, including but not limited to pulses of 30 mg/kg PDN on 3 consecutive days. High i.v. GCs dose is recommended not to be lower than 2 mg/kg/ day PDN equivalent (or at least 60 mg/day in pediatric subjects of 30 kg or more, and at least 1g/day in adult MAS subjects). In case of rapid worsening of the subject's condition and/or laboratory parameters, as per Investigator judgment, inclusion may occur within less than 3 days from starting high dose GCs. 4. Diagnosis of active MAS confirmed by the treating rheumatologist, having ascertained the followings: a. Febrile subjects presenting with ferritin \> 684 ng/mL. b. and any 2 of: i. Platelet count ≤ 181 x109/L ii. AST-level \> 48 U/L iii. Triglycerides \> 156 mg/dL iv. Fibrinogen level ≤ 360 mg/dL 5. Female subjects of child-bearing potential willing to use highly effective methods of contraception from study drug initiation to 6 months after the last dose of study drug. Specific inclusion criteria to Cohort 1 and Cohort 2 6. Cohort 1: 1. Confirmed sJIA diagnosis. For subjects presenting with MAS in the context of the onset of sJIA, high presumption of sJIA will suffice for eligibility. 2. Confirmed diagnosis of AOSD as per Yamaguchi criteria. 7. Cohort 2: 1. Confirmed diagnosis of SLE as per SLICC'12 criteria. Exclusion criteria 1. Primary HLH documented by either the presence of a known causative genetic mutation or abnormal perforin expression and CD107a degranulation assay as described with primary hemophagocytic lymphohistiocytosis or by the presence of family history. 2. Confirmed malignancy. Note: subjects with a suspected malignancy should have mononuclear cells typed by flow cytometry and/or tissue biopsy, as applicable, to rule out malignancy. 3. Treatment with canakinumab, JAK inhibitors, TNF inhibitors and tocilizumab at the time of emapalumab initiation. 4. Ongoing treatment with anakinra at a dose above 4 mg/kg at time of emapalumab initiation. 5. Subjects treated with etoposide for MAS in the last 1 month. 6. Clinically active mycobacteria (typical and atypical), Histoplasma Capsulatum, or Salmonella infections. 7. Evidence of leishmania infections. 8. Evidence of latent tuberculosis. 9. History of hypersensitivity or allergy to any component of the study drug. 10. Receipt of a Bacillus Calmette-Guerin (BCG) vaccine within 12 weeks prior to screening. 11. Receipt of a live or attenuated live (other than BCG) vaccine within 4 weeks prior to screening. 12. Pregnancy or lactating female subjects.

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Conditions

The condition(s) this trial relates to.

adult-onset Still disease juvenile idiopathic arthritis macrophage activation syndrome secondary hemophagocytic lymphohistiocytosis systemic lupus erythematosus systemic-onset juvenile idiopathic arthritis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

AOSD Macrophage activation syndrome MAS Secondary hemophagocytic lymphohistiocytosis SJIA Still disease Systemic lupus erythematosus

Contacts and locations

Locations

  • Akron Children's Hospital

    Akron, Ohio, 44308, United States

  • Alberta Children's Hospital

    Calgary, Canada

  • Beijing Children's Hospital

    Beijing, China

  • Beijing Friendship Hospital

    Beijing, China

  • Centre Hospitalier Universitaire Sainte-Justine

    Montreal, Canada

  • Centre Hospitalier de l'Université de Montréal

    Montreal, Canada

  • Charité Universitätsmedizin

    Berlin, Germany

  • Children's Healthcare of Atlanta

    Atlanta, Georgia, 30329, United States

  • Children's Hospital of Fudan University

    Shanghai, China

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229, United States

  • Fakultní nemocnice Olomouc

    Olomouc, Czechia

  • Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

    Milan, Italy

  • Great Ormond Street Hospital

    London, United Kingdom

  • Hospital Sant Joan de Déu

    Barcelona, Spain

  • Hospital Universitari i Politècnic La Fe

    Valencia, Spain

  • Hospital Universitario

    La Paz, Spain

  • Hospital Universitario La Paz

    La Paz, Spain

  • Hospital for sick children

    Toronto, Canada

  • Hôpital Claude Huriez

    Lille, France

  • Hôpital De La Conception

    Marseille, France

  • Hôpital Necker-Enfants Malades

    Paris, France

  • Hôpital Universitaire Pitié Salpêtrière

    Paris, France

  • IRCCS - Materno-Infantile Burlo Garofolo

    Trieste, Italy

  • IRCCS G. Gaslini

    Genova, Italy

  • Imperial College Healthcare NHS Trust

    London, United Kingdom

  • Nationwide Children's Hospital, Abigail Wexner Research Institute

    Columbus, Ohio, 43205, United States

  • Ortopedyczno - Rehabilitacyjny Szpital Kliniczny im. Wiktora Degi Uniwersytetu Medycznego im. Karola Marcinkowskiego w Poznaniu

    Poznan, Poland

  • Osaka Medical and Pharmaceutical University Hospital

    Takatsuki, Japan

  • Ospedale Pediatrico Bambino Gesù

    Roma, Italy

  • St. Marianna University School of Medicine Hospital

    Kawasaki, Japan

  • Szpital Specjalistyczny im. J. Dietla w Krakowie

    Krakow, Poland

  • Tokyo Medical and Dental University Hospital

    Tokyo, Japan

  • UAB Hospital

    Birmingham, Alabama, 35233, United States

  • UCLA Health

    Los Angeles, California, 90095, United States

  • UMC Utrecht

    Utrecht, Netherlands

  • UPMC Children's Hospital of Pittsburgh

    Pittsburgh, Pennsylvania, 15213, United States

  • Universitair Ziekenhuis Leuven

    Leuven, Belgium

  • University of Calgary

    Calgary, Canada

  • University of Florida

    Gainesville, Florida, 32610, United States

  • University of Minnesota Masonic Children's Hospital

    Minneapolis, Minnesota, 55455, United States

  • Universitätsklinikum Heidelberg

    Heidelberg, Germany

  • Vseobecna Fakultni Nemocnice v Praze

    Prague, Czechia

  • Wojewódzki Specjalistyczny Szpital Dziecięcy im. św. Ludwika w Krakowie

    Krakow, Poland

  • Yokohama City University Hospital

    Yokohama, Japan

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