Status:
COMPLETED
Study to Assess the Efficacy and Safety of Emapalumab in Primary Haemophagocytic Lymphohistiocytosis
Lead Sponsor:
Swedish Orphan Biovitrum
Conditions:
Primary Hemophagocytic Lymphohistiocytosis
Eligibility:
All Genders
Up to 18 years
Phase:
PHASE3
Brief 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) patient...
Eligibility Criteria
Inclusion
- Main
- 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
- 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.
Key Trial Info
Start Date :
February 6 2019
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
September 14 2022
Estimated Enrollment :
35 Patients enrolled
Trial Details
Trial ID
NCT03312751
Start Date
February 6 2019
End Date
September 14 2022
Last Update
March 12 2024
Active Locations (27)
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1
Phoenix Children Hospital
Phoenix, Arizona, United States, 85016
2
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027
3
Children's Hospital Colorado
Aurora, Colorado, United States, 80045-7106
4
Alfred I. duPont Hospital for Children - Nemours Center for Cancer and Blood Disorders - Division of Pediatric Hematology Oncology
Wilmington, Delaware, United States, 19803