Study to Assess the Efficacy and Safety of Emapalumab in Primary Haemophagocytic Lymphohistiocytosis
NCT ID: NCT03312751
Last Updated: 2024-03-12
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
35 participants
INTERVENTIONAL
2019-02-06
2022-09-14
Brief Summary
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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).
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Emapalumab
Emapalumab
Emapalumab will be administered by intravenous infusion, twice weekly.
Interventions
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Emapalumab
Emapalumab will be administered by intravenous infusion, twice weekly.
Eligibility Criteria
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Inclusion Criteria
* 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.
Exclusion Criteria
* 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.
18 Years
ALL
No
Sponsors
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Swedish Orphan Biovitrum
INDUSTRY
Responsible Party
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Locations
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Phoenix Children Hospital
Phoenix, Arizona, United States
Children's Hospital Los Angeles
Los Angeles, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
Alfred I. duPont Hospital for Children - Nemours Center for Cancer and Blood Disorders - Division of Pediatric Hematology Oncology
Wilmington, Delaware, United States
Children's Healthcare of Atlanta
Atlanta, Georgia, United States
Dana-Farber Cancer Institute (DFCI)
Boston, Massachusetts, United States
Spectrum Health Helen DeVos Children's Hospital
Grand Rapids, Michigan, United States
Cincinnati Children's Hospital
Cincinnati, Ohio, United States
Texas Children's Hospital - Feigin Center
Houston, Texas, United States
Seattle Children's Hospital
Seattle, Washington, United States
Hopital Ste-Justine Research Center
Montreal, , Canada
Hospital for Sick Children
Toronto, , Canada
Children's and Women's Health Centre of British Columbia
Vancouver, , Canada
Universitätsklinikum Essen
Essen, , Germany
Medical Center- University of Freiburg
Freiburg im Breisgau, , Germany
Universitätsklinikum Eppendorf
Hamburg, , Germany
Istituto Giannina Gaslini
Genova, , Italy
Fondazione MBBM, Ospedale San Gerardo
Monza, , Italy
Ospedale Pediatrico Bambino Gesù
Rome, , Italy
Ospedale della Donna e del Bambino
Verona, , Italy
Hospital Universitario Vall d'Hebron
Barcelona, , Spain
Hospital Universitario Niño Jesús
Madrid, , Spain
Karolinska University Hospital Huddinge
Stockholm, , Sweden
University Children's Hospital Zurich
Zurich, , Switzerland
Leeds Children's Hospital
Leeds, , United Kingdom
Great Ormond Street Hospital
London, , United Kingdom
Royal Manchester Children's Hospital
Manchester, , United Kingdom
Countries
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Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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NI-0501-09
Identifier Type: -
Identifier Source: org_study_id
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