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

• Status: Active, not recruiting
• Trial end date: 2022-10-01
• Target enrollment: 0
• Participant gender: All

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, with special focus on long-term outcome 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, 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).

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: NovImmune SA; Swedish Orphan Biovitrum
• Treatments: Interferon-gamma; Interferons

Criteria

Inclusion Criteria:

• Primary HLH patients with active disease.

• Treatment naïve patients or patients having already received HLH conventional therapy, but having failed or unable to tolerate current standard of care.

• Informed consent signed by the patient or by the patient's legally authorized representative.

• Guidance on contraception

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.