Study to Assess the Efficacy and Safety of Emapalumab in Primary Haemophagocytic Lymphohistiocytosis
Public ClinicalTrials.gov record NCT03312751. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
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
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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).
Study identification
- NCT ID
- NCT03312751
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 35 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- Up to 18 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Feb 5, 2019
- Primary completion
- Aug 17, 2021
- Completion
- Sep 13, 2022
- Last update posted
- Mar 11, 2024
2019 – 2022
United States locations
- U.S. sites
- 10
- U.S. states
- 10
- U.S. cities
- 10
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Phoenix Children Hospital | Phoenix | Arizona | 85016 | — |
| Children's Hospital Los Angeles | Los Angeles | California | 90027 | — |
| Children's Hospital Colorado | Aurora | Colorado | 80045-7106 | — |
| Alfred I. duPont Hospital for Children - Nemours Center for Cancer and Blood Disorders - Division of Pediatric Hematology Oncology | Wilmington | Delaware | 19803 | — |
| Children's Healthcare of Atlanta | Atlanta | Georgia | 30329 | — |
| Dana-Farber Cancer Institute (DFCI) | Boston | Massachusetts | 02215 | — |
| Spectrum Health Helen DeVos Children's Hospital | Grand Rapids | Michigan | 49503 | — |
| Cincinnati Children's Hospital | Cincinnati | Ohio | 45229 | — |
| Texas Children's Hospital - Feigin Center | Houston | Texas | 77030 | — |
| Seattle Children's Hospital | Seattle | Washington | 98109 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 17 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT03312751, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Mar 11, 2024 · Synced Sep 9, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT03312751 live on ClinicalTrials.gov.