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A Study to Investigate the Safety and Efficacy of Emapalumab, an Anti-IFN-gamma mAb in Patients With Systemic Juvenile Idiopathic Arthritis (sJIA) or Adult-onset Still's Disease (AOSD) Developing Macrophage Activation Syndrome/Secondary HLH (MAS/sHLH)

NCT03311854 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 14

Last updated 2022-05-17

Study results available
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Summary

Macrophage Activation Syndrome (MAS) is a rare, life-threatening condition characterized by uncontrolled hyperinflammation which may develop on the background of systemic Juvenile Idiopathic Arthritis (sJIA) or Adult-onset Still's Disease (AOSD). Emapalumab is a monoclonal antibody neutralizing interferon-gamma (IFN-gamma), a key cytokine which contributes to the inflammation and tissue damage seen in MAS. The purpose of this study is to assess the safety, tolerability and efficacy of emapalumab in sJIA or AOSD participants developing MAS, presenting an inadequate response to high dose glucocorticoid treatment.

Conditions

  • Macrophage Activation Syndrome
  • Lymphohistiocytosis, Hemophagocytic
  • Arthritis, Juvenile
  • Adult Onset Still Disease

Interventions

DRUG

Emapalumab

Emapalumab was administered at an initial dose of 6 mg/kg by intravenous infusion. Emapalumab treatment continued at a dose of 3 mg/kg, every 3 days until study day 15, and then twice-a-week for an additional 2 weeks, i.e., until study day 28. The emapalumab regimen could be adapted (the frequency between infusions shortened, the dose increased, or the treatment prolonged beyond 4 weeks) upon assessment of a favourable benefit-risk profile. There was a 4-week off-drug follow-up period (up to Week 8).

Sponsors & Collaborators

  • Swedish Orphan Biovitrum

    lead INDUSTRY

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
0 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-02-20
Primary Completion
2020-05-19
Completion
2020-05-19
FDA Drug
Yes

Countries

  • United States
  • France
  • Italy
  • Spain
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03311854 on ClinicalTrials.gov