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

Primary Hemophagocytic Lymphohistiocytosis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT05744063
• Other study ID #: Sobi.emapalumab-104
• Status: Active, not recruiting
• Phase: Phase 4
• Start date: February 3, 2023
• Est. completion date: July 2025

Study information

• Verified date: February 2024
• Source: Swedish Orphan Biovitrum
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 13
• Est. completion date: July 2025
• Est. primary completion date: July 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• Lymphohistiocytosis, Hemophagocytic
• Primary Hemophagocytic Lymphohistiocytosis

Intervention

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

Locations

Country	Name	City	State
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	Shanghai	Fudan
China	Swedish Orphan Biovitrum Research site	Zhengzhou

Sponsors (1)

Lead Sponsor	Collaborator
Swedish Orphan Biovitrum

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Permanent 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
Secondary	Overall response	measured as complete response, partial response, or HLH improvement	end of treatment or week 8 (whichever occurs earlier)
Secondary	Time to first overall response	from first dose of study drug to first response	end of treatment, likely within 6 months from first dose
Secondary	Cumulative duration of response	total time in response	end of treatment, likely within 6 months from first dose
Secondary	Ability to reduce glucocorticoids by 50% or more	reduction from baseline dose during emapalumab treatment	end of treatment, likely within 6 months from first dose
Secondary	Investigator assessed response	Investigator's assessment of how patient responds to treatment and rated as complete response, partial response, or no response	end of treatment or week 8 (whichever occurs earlier)
Secondary	Survival	To start of HSCT conditioning and after time from HSCT to death	end of study