NCT05001737

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

A Two-cohort, Open-label, Single Arm, Multicenter Study to Evaluate Efficacy, Safety and Tolerability, PK and PD, of Emapalumab in Children and Adults With MAS in Still's Disease or With MAS in Systemic Lupus Erythematous

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 33 (actual)
• Sponsor: Swedish Orphan Biovitrum · Industry
• Sex: All
• Age: 6 Months – 80 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

Study NI-0501-14 is a two-cohort trial that enrolls subjects who are diagnosed with sHLH/MAS (MAS being a form of secondary HLH) and who are presenting an inadequate response to high doses of GCs. These subjects will be enrolled in 2 cohorts as per their background disease. The cohorts are defined as follows: * Cohort 1: MAS in the context of sJIA and AOSD. * Cohort 2: MAS in the context of pediatric and adult SLE. The study has the objectives to investigate the efficacy, safety and tolerability, for 8 weeks, and PK and PD, QoL and immunogenicity in these 2 cohorts for up to 1 year after last dose of of emapalumab. Macrophage Activation Syndrome (MAS) Secondary Hemophagocytic Lymphohistiocytosis (sHLH) systemic Juvenile Idiopathic Arthritis (sJIA) Adult-onset Still's Disease (AOSD) Systemic Lupus Erythematosus (SLE)

Conditions

• Macrophage Activation Syndrome
• Secondary Hemophagocytic Lymphohistiocytosis
• Still Disease
• Systemic Lupus Erythematosus
• SJIA
• AOSD
• MAS

Interventions

Type	Name	Description
DRUG	Emapalumab	Emapalumab iv infusion

Timeline

• Start date: 2021-12-15
• Primary completion: 2024-06-30
• Completion: 2025-06-04
• First posted: 2021-08-12
• Last updated: 2026-02-19
• Results posted: 2026-02-19

Locations

44 sites across 13 countries: United States, Belgium, Canada, China, Czechia, France, Germany, Italy, Japan, Netherlands, Poland, Spain, United Kingdom

Regulatory

• FDA-regulated drug study