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

NCT03312751 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 35

Last updated 2024-03-12

Study results available
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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) 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).

Conditions

  • Primary Hemophagocytic Lymphohistiocytosis

Interventions

DRUG

Emapalumab

Emapalumab will be administered by intravenous infusion, twice weekly.

Sponsors & Collaborators

  • Swedish Orphan Biovitrum

    lead INDUSTRY

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-02-06
Primary Completion
2021-08-18
Completion
2022-09-14
FDA Drug
Yes

Countries

  • United States
  • Canada
  • Germany
  • Italy
  • Spain
  • Sweden
  • Switzerland
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03312751 on ClinicalTrials.gov