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

Status:	Recruiting
Healthy:	No
Age Range:	Any - 18
Updated:	4/6/2019
Start Date:	September 24, 2018
End Date:	October 2022
Contact:	Maria Ballabio, MD
Email:	mballabio@novimmune.com
Phone:	+41 (0)61 2011 324

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

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).

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 contrqaception

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.

We found this trial at

sites

Cincinnati Children's Hospital

Cincinnati, Ohio 45229

Principal Investigator: Michael Jordan, MD

Phone: 513-636-9461

from

Cincinnati, OH