NI-0501-14 : Study of emapalumab in children and adults with macrophage activation syndrome (MAS)
Research type
Research Study
Full title
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 arthritis and Adult onset Still’s disease) or with MAS in Systemic lupus erythematous
IRAS ID
1003938
Contact name
Jeanette Bachir
Contact email
Sponsor organisation
Swedish Orphan Biovitrum AG (Sobi AG)
Eudract number
2021-001577-24
Research summary
Study NI-0501-14 is an open-label two-cohort, single arm, multicenter, interventional, phase 2/3 study. This study will be conducted internationally.
The study will enrol male and female paediatric and adult participants who will be assigned to different cohorts as per the underlying disease:
# Cohort 1: Macrophage activation syndrome (MAS) in the context of Systemic juvenile inflammatory arthritis (sJIA) and Adult onset Still’s disease (AOSD).
# Cohort 2: MAS in the context of paediatric and adult Systemic lupus erythematous (SLE).Each cohort in this study is designed as a single arm study and will be composed of 2 phases: one run-in phase and one interventional phase.
A total of 41 participants are planned to be enrolled in the interventional phase: 25 participants (of these at least 16 paediatric subjects) in cohort 1 and 16 participants (of these at least 6 paediatric subjects) in cohort 2.
Participants enrolled in the interventional phase will receive emapalumab. Emapalumab will be administered by intravenous infusion every 3 days until study day 16, and then twice-a-week for additional 2 weeks. Participants will have different procedures done in both phases of the study, including, but not limited to physical examinations, vital sign measurements, blood sampling.
REC name
London - City & East Research Ethics Committee
REC reference
22/LO/0011
Date of REC Opinion
14 Feb 2022
REC opinion
Further Information Unfavourable Opinion