Study to Assess the Efficacy and Safety of Emapalumab in Primary Haemophagocytic Lymphohistiocytosis
Completed · Phase 3
Conditions studied: Primary Hemophagocytic Lymphohistiocytosis
In brief
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).
Key facts
- Study ID
- NCT03312751
- Run by
- Swedish Orphan Biovitrum
- People needed
- 35
- Starts
- 2019-02-06
- Expected to finish
- 2022-09-14
- Last updated by the study team
- 2024-03-12
Who can join
Age: any, up to 18. Sex: any. Healthy volunteers: not accepted.
Where it is running
- 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
Full record on ClinicalTrials.gov
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