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

NCT ID: NCT05744063

Last Updated: 2025-09-17

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE4
• Total Enrollment: 13 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-02-03
• Study Completion Date: 2025-08-08

Brief Summary

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

Detailed Description

This is an open-label, multi center, single arm, post-authorization study aiming to describe safety and efficacy of emapalumab in treatment experienced Chinese patients with confirmed or suspected primary hemophagocytic lymphohistiocytosis (pHLH). The main objectives of the study are to collect safety and efficacy data on emapalumab in treatment experienced Chinese pHLH patients

Conditions

Primary Hemophagocytic Lymphohistiocytosis

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

emapalumab

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

Group Type: EXPERIMENTAL

Emapalumab-Lzsg 5 MG/ML [Gamifant]

Intervention Type: DRUG

iv

Interventions

Emapalumab-Lzsg 5 MG/ML [Gamifant]

iv

Intervention Type: DRUG

Other Intervention Names

Gamifant

Eligibility Criteria

Inclusion Criteria

1. Male and female HLH patients of any age.

2. 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

3. Presence of active HLH disease as assessed by the investigator.

4. 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
• Showing intolerance to previous conventional treatment of HLH At the time of enrollment, eligible patients might still be receiving treatment (induction or maintenance) or might have already discontinued it.

5. Expectation of survival beyond 1 week as judged by the investigator.

6. Patient has expectation of proceeding to HSCT

7. Informed consent signed by the patient (as required by local law), or by the patient's legally authorized representative(s) with the assent of patients who are legally capable of providing it, as applicable.

8. Willing to use highly effective methods of contraception from study drug initiation to 6 months after the last dose of study drug, if female and of childbearing potential.

Exclusion Criteria

1. Diagnosis of secondary HLH consequent to a proven rheumatic, metabolic or neoplastic disease.

2. Active mycobacteria, Histoplasma capsulatum, Salmonella, or Leishmania infections.

3. Evidence of latent tuberculosis.

4. Presence of malignancy.

5. 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

6. History of hypersensitivity or allergy to any component of the study regimen (e.g., polysorbate).

7. Receipt of a Bacillus Calmette-Guérin (BCG) vaccine within 12 weeks prior to Screening.

8. Receipt of a live or attenuated live (other than BCG) vaccine within 4 weeks prior to Screening.

9. Pregnant or lactating female patients.

10. Enrollment in another concurrent clinical interventional study, or intake of an IMP, within three months prior to inclusion in this study

11. 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.

• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Swedish Orphan Biovitrum

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Rui Zhang, MD, Prof

Role: PRINCIPAL_INVESTIGATOR

Beijing Children's Hospital

Locations

Swedish Orphan Biovitrum Research site

Shanghai, Fudan, China

Swedish Orphan Biovitrum Research site

Beijing, Xicheng, China

Swedish Orphan Biovitrum Research site

Beijing, , China

Swedish Orphan Biovitrum Research site

Chongqing, , China

Swedish Orphan Biovitrum Research site

Guangzhou, , China

Swedish Orphan Biovitrum Research site

Nanjing, , China

Swedish Orphan Biovitrum Research site

Zhengzhou, , China

Countries

China

Other Identifiers

Sobi.emapalumab-104

Identifier Type: -

Identifier Source: org_study_id