NCT05744063|CompletedPhase 4Results

A Post-authorization Study to Describe the Safety and Efficacy of Emapalumab for the Treatment of pHLH in Treatment Experienced Chinese Patients

An Open Label, Single Arm, Multi-Centre, Post-authorization Study to Describe the Safety and Efficacy of Emapalumab for the Treatment of Primary Hemophagocytic Lymphohistiocytosis in Treatment Experienced Chinese Patients

Swedish Orphan Biovitrum ClinicalTrials.gov

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1 other identifier

Sobi.emapalumab-104

Study Type

interventional

Target

Locations

1 country

Sites

Timeline

RegisteredFeb 2023

StartedFeb 2023

CompletionAug 2025

Brief Summary

The goal of this post-authorization study is to describe safety and efficacy of emapalumab in treatment experienced Chinese patients with pHLH.

Trial Health

On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment

participants targeted

Target at below P25 for phase_4

Timeline

Completed

Started Feb 2023

Typical duration for phase_4

Geographic Reach

1 country

7 active sites

Status

completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

2.5 years study duration

Study Start

First participant enrolled

February 3, 2023

Completed

10 days until next milestone

First Submitted

Initial submission to the registry

February 13, 2023

Completed

11 days until next milestone

First Posted

Study publicly available on registry

February 24, 2023

Completed

2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 21, 2025

Completed

6 months until next milestone

Study Completion

Last participant's last visit for all outcomes

August 8, 2025

Completed

7 months until next milestone

Results Posted

Study results publicly available

March 13, 2026

Completed

Last Updated

March 13, 2026

Status Verified

September 1, 2025

Enrollment Period

2.1 years

First QC Date

February 13, 2023

Results QC Date

February 4, 2026

Last Update Submit

March 12, 2026

Conditions

Primary Hemophagocytic Lymphohistiocytosis

Keywords

pHLH

Outcome Measures

Primary Outcomes (1)

Permanent Discontinuation of Study Drug Due to Emapalumab-related Adverse Event
Number of participants permanently discontinuation of study drug due to emapalumab-related adverse event as judged by Investigator, until conditioning for hematopoietic stem cell transplant (HSCT), likely within 6 months from first dose
Until conditioning for hematopoietic stem cell transplant (HSCT), likely within 6 months from first dose

Secondary Outcomes (6)

Overall Response
End of treatment or week 8 (whichever occurs earlier)
Time to First Overall Response
End of treatment, likely within 6 months from first dose
Cumulative Duration of Response
End of treatment, likely within 6 months from first dose
Ability to Reduce Glucocorticoids by 50% or More
End of treatment, likely within 6 months from first dose
Investigator Assessed Response
End of treatment
+1 more secondary outcomes

Study Arms (1)

emapalumab

EXPERIMENTAL

emapalumab solution for infusion twice weekly at a starting dose of 1 mg/kg

Drug: Emapalumab-Lzsg 5 MG/ML [Gamifant]

Interventions

Emapalumab-Lzsg 5 MG/ML [Gamifant]DRUG

Also known as: Gamifant

emapalumab

Eligibility Criteria

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

Male and female HLH patients of any age.
Patients diagnosed with confirmed or suspected pHLH, based on; a molecular diagnosis or familial history consistent with pHLH or fulfilment of HLH-2004 diagnostic criteria, i.e., five out of eight of the criteria below:
Fever
Splenomegaly
Cytopenias affecting 2 of 3 lineages in the peripheral blood (hemoglobin \<90 g/L; platelets \<100 x 109/L; neutrophils \<1 x 109/L)
Hypertriglyceridemia (fasting triglycerides ≥3 mmol/L or ≥265 mg/dL) and/or hypofibrinogenemia (≤1.5 g/L)
Hemophagocytosis in bone marrow, spleen, or lymph nodes, with no evidence of malignancy.
Low or absent NK-cell activity
Ferritin ≥500 μg/L
Soluble CD25 (sCD25; i.e., soluble IL-2 receptor) ≥2400 U/mL
Presence of active HLH disease as assessed by the investigator.
Patients must fulfil one of the following criteria as assessed by the investigator:
Having not responded to previous conventional treatment of HLH
Having not achieved a satisfactory response to previous conventional treatment of HLH or worsened
Having reactivated HLH
+5 more criteria

You may not qualify if:

Diagnosis of secondary HLH consequent to a proven rheumatic, metabolic or neoplastic disease.
Active mycobacteria, Histoplasma capsulatum, Salmonella, or Leishmania infections.
Evidence of latent tuberculosis.
Presence of malignancy.
Existence of any severe co-morbidity or any other medical condition which, in the opinion of the investigator, makes the patient unsuitable for the treatment
History of hypersensitivity or allergy to any component of the study regimen (e.g., polysorbate).
Receipt of a Bacillus Calmette-Guérin (BCG) vaccine within 12 weeks prior to Screening.
Receipt of a live or attenuated live (other than BCG) vaccine within 4 weeks prior to Screening.
Pregnant or lactating female patients.
Any condition or circumstance that in the opinion of the Investigator may make the patient unlikely to complete the study or comply with study procedures or requirements.

Contact the study team to confirm eligibility.