Evaluate Efficacy, Safety and Tolerability, PK and PD of Emapalumab in Children and Adults With MAS in Still's or SLE

Completed · Phase 3

Conditions studied: Macrophage Activation Syndrome, Secondary Hemophagocytic Lymphohistiocytosis, Still Disease, Systemic Lupus Erythematosus, SJIA, AOSD, MAS

In brief

The purpose of this study is to assess the safety, tolerability and efficacy of emapalumab in children and adults with macrophage activation syndrome (sHLH/MAS) in Still's disease (including systemic juvenile idiopathic arthritis and adult onset Still's disease) or with sHLH/MAS in systemic lupus erythematous, resenting an inadequate response to high dose glucocorticoid treatment.

Key facts

Study ID

NCT05001737

Run by

Swedish Orphan Biovitrum

People needed

33

Starts

2021-12-15

Expected to finish

2025-06-04

Last updated by the study team

2026-02-19

Who can join

Age: 1 and older, up to 80. Sex: any. Healthy volunteers: not accepted.

You may not qualify if…

• 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.
• 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.
• Treatment with canakinumab, JAK inhibitors, TNF inhibitors and tocilizumab at the time of emapalumab initiation.
• Ongoing treatment with anakinra at a dose above 4 mg/kg at time of emapalumab initiation.
• Subjects treated with etoposide for MAS in the last 1 month.
• Clinically active mycobacteria (typical and atypical), Histoplasma Capsulatum, or Salmonella infections.
• Evidence of leishmania infections.
• Evidence of latent tuberculosis.
• History of hypersensitivity or allergy to any component of the study drug.
• Receipt of a Bacillus Calmette-Guerin (BCG) vaccine within 12 weeks prior to screening.
• Receipt of a live or attenuated live (other than BCG) vaccine within 4 weeks prior to screening.
• Pregnancy or lactating female subjects.

Where it is running

• UAB Hospital — Birmingham, Alabama, United States
• UCLA Health — Los Angeles, California, United States
• University of Florida — Gainesville, Florida, United States
• Children's Healthcare of Atlanta — Atlanta, Georgia, United States
• University of Minnesota Masonic Children's Hospital — Minneapolis, Minnesota, United States
• Akron Children's Hospital — Akron, Ohio, United States
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
• Nationwide Children's Hospital, Abigail Wexner Research Institute — Columbus, Ohio, United States
• UPMC Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania, United States
• Universitair Ziekenhuis Leuven — Leuven, Belgium
• Alberta Children's Hospital — Calgary, Canada
• University of Calgary — Calgary, Canada
• Centre Hospitalier de l'Université de Montréal — Montreal, Canada
• Centre Hospitalier Universitaire Sainte-Justine — Montreal, Canada
• Hospital for sick children — Toronto, Canada
• Beijing Children's Hospital — Beijing, China
• Beijing Friendship Hospital — Beijing, China
• Children's Hospital of Fudan University — Shanghai, China
• Fakultní nemocnice Olomouc — Olomouc, Czechia
• Vseobecna Fakultni Nemocnice v Praze — Prague, Czechia
• Hôpital Claude Huriez — Lille, France
• Hôpital De La Conception — Marseille, France
• Hôpital Necker-Enfants Malades — Paris, France
• Hôpital Universitaire Pitié Salpêtrière — Paris, France
• Charité Universitätsmedizin — Berlin, Germany

Full record on ClinicalTrials.gov

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