NCT03312751

Study to Assess the Efficacy and Safety of Emapalumab in Primary Haemophagocytic Lymphohistiocytosis

An Open-label, Single Arm, Multicenter Study to Broaden Access to Emapalumab, an Anti-Interferon Gamma (Anti-IFNγ) Monoclonal Antibody, and to Assess Its Efficacy, Safety, Impact on Quality of Life, and Long-term Outcome in Pediatric Patients With Primary Hemophagocytic Lymphohistiocytosis

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 35 (actual)
• Sponsor: Swedish Orphan Biovitrum · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to expand the knowledge on the efficacy and safety of emapalumab (previously known as NI-0501) as a treatment for primary haemophagocytic lymphohistiocytosis (HLH) patients, including on long-term outcomes and quality of life assessments. Emapalumab can be administered as the first-line therapy to patients not previously treated with the current standard of care, or can be given to patients who have either failed or were unable to tolerate the available standard of care. Emapalumab is to be administered until the start of conditioning for hematopoietic stem cell transplantation (HSCT), with an anticipated duration ranging from a minimum of 4 weeks to approximately 12 weeks and not exceeding 6 months. After treatment completion, patients will continue in the study for long-term follow-up until 1 year after either HSCT or last emapalumab infusion (if HSCT is not performed).

Conditions

• Primary Hemophagocytic Lymphohistiocytosis

Interventions

Type	Name	Description
DRUG	Emapalumab	Emapalumab will be administered by intravenous infusion, twice weekly.

Timeline

• Start date: 2019-02-06
• Primary completion: 2021-08-18
• Completion: 2022-09-14
• First posted: 2017-10-18
• Last updated: 2024-03-12
• Results posted: 2024-03-12

Locations

27 sites across 8 countries: United States, Canada, Germany, Italy, Spain, Sweden, Switzerland, United Kingdom

Regulatory

• FDA-regulated drug study