Emapalumab for MAS in Still's Disease or Lupus

(EMERALD Trial)

Recruiting at 46 trial locations

Overseen By Anna Westerdahl

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: Swedish Orphan Biovitrum

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 68

Duration: 8 weeks

41 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing emapalumab, a medication that helps control an overactive immune system, in children and adults with certain immune conditions who haven't responded to high-dose steroids. The goal is to see if it is safe and effective in reducing excessive inflammation. Emapalumab is a monoclonal antibody that targets interferon-γ and was first approved for treating primary hemophagocytic lymphohistiocytosis (HLH) in patients with refractory, recurrent, or progressive disease.

Research Team

Brian Jamieson, MD

Principal Investigator

Swedish Orphan Biovitrum

Eligibility Criteria

This trial is for children and adults aged 6 months to 80 years with macrophage activation syndrome (MAS) in Still's disease or systemic lupus erythematosus (SLE), who haven't responded well to high-dose steroids. Participants must meet specific criteria including fever, low platelets, high liver enzymes, and others. Pregnant women, those with certain infections or allergies to the study drug, or recent recipients of live vaccines cannot join.

Inclusion Criteria

Confirmed diagnosis of AOSD as per Yamaguchi criteria.

I am willing to use effective birth control from the start of the study until 6 months after the last dose.

Cohort 1:

Exclusion Criteria

My cancer diagnosis has been confirmed.

I am not taking canakinumab, JAK inhibitors, TNF inhibitors, or tocilizumab when starting emapalumab.

Pregnancy or lactating female subjects.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive emapalumab for 8 weeks to assess efficacy, safety, and tolerability

8 weeks

Every treatment visit until week 8

Follow-up

Participants are monitored for safety, efficacy, and other outcomes up to 1 year after the last dose

1 year

Day 60, Day 100, 6 months, 1 year

Treatment Details

Interventions

• Emapalumab

Trial Overview The trial tests Emapalumab's safety and effectiveness in treating MAS associated with Still's disease or SLE when standard steroid treatments fail. It involves patients receiving Emapalumab under controlled conditions to see if it improves their symptoms compared to previous treatments.

Participant Groups

Group I: Cohort 1 (sJIA and AOSD) and Cohort 2 (SLE) Experimental Treatment 1 Intervention

MAS in the context of systemic juvenile idiopathic arthritis and adult onset Still's disease (sJIA and AOSD) or SLE

Emapalumab is already approved in United States for the following indications:

Approved in United States as Gamifant for:

• Primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy

Who Is Running the Clinical Trial?

Swedish Orphan Biovitrum

Lead Sponsor

Trials

103

Recruited

13,300+

Dr. Guido Oelkers

Swedish Orphan Biovitrum

Chief Executive Officer since 2017

PhD in Economics

Dr. Lydia Abad-Franch

Swedish Orphan Biovitrum

Chief Medical Officer since 2023

MD, MBA