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)

PHASE2CompletedINTERVENTIONAL
Enrollment

14

Participants

Timeline

Start Date

February 20, 2018

Primary Completion Date

May 19, 2020

Study Completion Date

May 19, 2020

Conditions
Macrophage Activation SyndromeLymphohistiocytosis, HemophagocyticArthritis, JuvenileAdult 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).

Trial Locations (5)

45229

Cincinnati Children'S Hospital, Cincinnati

75743

Hôpital Necker-Enfants Malades, Unité d'Immunologie-hématologie et Rhumatologie pédiatriques, Paris

00165

IRCCS Ospedale Pediatrico, Bambino Gesù, Rome

08950

Hospital Sant Joan de Deu, Barcelona

WC1N 3JH

Great Ormond Street Hospital for Children, London

Sponsors
All Listed Sponsors
lead

Swedish Orphan Biovitrum

INDUSTRY

NCT03311854 - 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) | Biotech Hunter | Biotech Hunter