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.
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)
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
33
Emapalumab iv infusion
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
Proportion of subjects with complete response (CR) at Week 8 after first administration of emapalumab
Resolution of clinical signs and symptoms present at baseline: The MAS clinical activity will be measured on a visual analog scale (VAS) 10 cm. Resolution of clinical signs and symptoms present at baseline: The MAS clinical activity will be measured on a visual analog scale (VAS) 10 cm. Clinical signs will be considered as resolved if VAS is below or equal to 1/10. And Normalization of laboratory parameters relevant to MAS, as follows: WBC above LLN platelet count above LLN LDH below 1.5 ULN ALT below 1.5 ULN AST below 1.5 ULN fibrinogen higher than 100 mg/dL ferritin levels decreased by at least 80 % from values at screening or baseline (whichever is higher) or below 2000 ng/ml, whichever is low
Time frame: 8 weeks
GCs tapering to a dose below 50% of prednisolone (PDN) equivalent at the time of emapalumab start or to the same (or lower) dose being administered before the occurrence of MAS whichever occurs first
GC tapering as per investigator discretion
Time frame: At any time in the study, up to 1 year
GCs tapering to ≤1mg/kg/day of PDN equivalent at any time during the study.
GC tapering as per investigator discretion
Time frame: At any time in the study, up to 1 year
Time to achieve GCs tapering as defined above.
GC tapering as per investigator discretion
Time frame: At any time in the study, up to 1 year
Time to first Complete Remission
Time to CR
Time frame: At any time in the study, up to 1 year
Proportion of subjects with overall response as defined by CR or PR
Resolution of clinical signs and symptoms present at baseline: The MAS clinical activity will be measured on a visual analog scale (VAS) 10 cm.
Time frame: At any time in the study, up to 1 year
Time to first overall response as defined by CR or PR
CR defined as below: Resolution of clinical signs and symptoms present at baseline: The MAS clinical activity will be measured on a visual analog scale (VAS) 10 cm.
Time frame: At any time in the study, up to 1 year
MAS recurrence at anytime after achievement of CR
Time to MAS recurrence after CR
Time frame: At any time after CR, up to 1 year
Withdrawal from the study due to lack of response as per Investigator decision
Time to withdrawal
Time frame: At any time in the study, up to 1 year
Survival time
Time to Survival
Time frame: At any time in the study, up to 1 year
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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
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