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

NI-0501-09

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).

2019-02-06

2021-08-18

2022-09-14

Completed

Early phase I

35

Main Inclusion Criteria: * Primary HLH patients with active disease. * Treatment naïve patients or patients having already received HLH conventional therapy, but having not responded, not achieved a satisfactory response or worsened, or reactivated, or are unable to tolerate current standard of care. * Informed consent signed by the patient or by the patient's legally authorized representative. * Received guidance on contraception. Main Exclusion Criteria: * Diagnosis of secondary HLH consequent to a proven rheumatic, metabolic or neoplastic disease. * Active mycobacteria, Histoplasma capsulatum, Shigella, Salmonella, Campylobacter or Leishmania infections. * Evidence of latent tuberculosis. * Presence of malignancy. * Concomitant disease or malformation severely affecting cardiovascular, pulmonary, central nervous system (CNS), liver, or renal function, that in the opinion of the Investigator may significantly affect the likelihood to respond to treatment and/or the assessment of emapalumab safety and/or efficacy. * History of hypersensitivity or allergy to any component of the study regimen. * Receipt of a BCG vaccine within 12 weeks prior to Screening. * Receipt of a live or attenuated-live (other than BCG) vaccine within 6 weeks prior to Screening. * Pregnant or lactating female patients.

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2024-03-12