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

Phase 3

Completed

• Conditions: Macrophage Activation Syndrome; SJIA; MAS; Secondary Hemophagocytic Lymphohistiocytosis; Still Disease; Systemic Lupus Erythematosus; AOSD
• Interventions: Drug: Emapalumab

• Registration Number: NCT05001737
• Lead Sponsor: Swedish Orphan Biovitrum

Brief 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)

Study Timeline

• Study First Submitted: 2021-06-16
• Study First Submitted QC: 2021-08-03
• Study First Posted: 2021-08-12
• Study Start: 2021-12-15
• Primary Completion: 2024-06-30
• Status Verified: 2025-06
• Study Completion: 2025-06-04
• Last Update Submitted: 2025-06-10
• Last Update Posted: 2025-06-11

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 33

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Cohort 1 (sJIA and AOSD) and Cohort 2 (SLE)	Emapalumab	MAS in the context of systemic juvenile idiopathic arthritis and adult onset Still's disease (sJIA and AOSD) or SLE

Primary Outcome Measures

Name	Time	Method
Proportion of subjects with complete response (CR) at Week 8 after first administration of emapalumab	8 weeks	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

Secondary Outcome Measures

Name	Time	Method
Time to achieve GCs tapering as defined above.	At any time in the study, up to 1 year	GC tapering as per investigator discretion
Time to first Complete Remission	At any time in the study, up to 1 year	Time to CR
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	At any time in the study, up to 1 year	GC tapering as per investigator discretion
Proportion of subjects with overall response as defined by CR or PR	At any time in the study, up to 1 year	Resolution of clinical signs and symptoms present at baseline: The MAS clinical activity will be measured on a visual analog scale (VAS) 10 cm.
Survival time	At any time in the study, up to 1 year	Time to Survival
GCs tapering to ≤1mg/kg/day of PDN equivalent at any time during the study.	At any time in the study, up to 1 year	GC tapering as per investigator discretion
MAS recurrence at anytime after achievement of CR	At any time after CR, up to 1 year	Time to MAS recurrence after CR
Time to first overall response as defined by CR or PR	At any time in the study, up to 1 year	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.
Withdrawal from the study due to lack of response as per Investigator decision	At any time in the study, up to 1 year	Time to withdrawal

Trial Locations

Locations (44)

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

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