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

Primary Purpose

Primary Hemophagocytic Lymphohistiocytosis

Status
Recruiting
Phase
Phase 4
Locations
China
Study Type
Interventional
Intervention
Emapalumab-Lzsg 5 MG/ML [Gamifant]
Sponsored by
Swedish Orphan Biovitrum
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Primary Hemophagocytic Lymphohistiocytosis focused on measuring pHLH

Eligibility Criteria

undefined - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria: 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 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. Expectation of survival beyond 1 week as judged by the investigator. Patient has expectation of proceeding to HSCT 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. 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: 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. Enrollment in another concurrent clinical interventional study, or intake of an IMP, within three months prior to inclusion in this study 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.

Sites / Locations

  • Swedish Orphan Biovitrum Research site
  • Swedish Orphan Biovitrum Research siteRecruiting
  • Swedish Orphan Biovitrum Research site
  • Swedish Orphan Biovitrum Research site
  • Swedish Orphan Biovitrum Research site
  • Swedish Orphan Biovitrum Research site
  • Swedish Orphan Biovitrum Research site

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

emapalumab

Arm Description

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

Outcomes

Primary Outcome Measures

Permanent discontinuation of study drug due to emapalumab-related adverse event
as judged by Investigator

Secondary Outcome Measures

Overall response
measured as complete response, partial response, or HLH improvement
Time to first overall response
from first dose of study drug to first response
Cumulative duration of response
total time in response
Ability to reduce glucocorticoids by 50% or more
reduction from baseline dose during emapalumab treatment
Investigator assessed response
Investigator's assessment of how patient responds to treatment and rated as complete response, partial response, or no response
Survival
To start of HSCT conditioning and after time from HSCT to death

Full Information

First Posted
February 13, 2023
Last Updated
February 23, 2023
Sponsor
Swedish Orphan Biovitrum
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1. Study Identification

Unique Protocol Identification Number
NCT05744063
Brief Title
A Post-authorization Study to Describe the Safety and Efficacy of Emapalumab for the Treatment of pHLH in Treatment Experienced Chinese Patients
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
Study Type
Interventional

2. Study Status

Record Verification Date
February 2023
Overall Recruitment Status
Recruiting
Study Start Date
February 3, 2023 (Actual)
Primary Completion Date
July 2025 (Anticipated)
Study Completion Date
July 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Swedish Orphan Biovitrum

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Primary Hemophagocytic Lymphohistiocytosis
Keywords
pHLH

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Model Description
This is an open-label, single-arm, multi-centre study to collect safety and efficacy data on emapalumab in treatment experienced male and female patients diagnosed with pHLH. The study will be performed in China.
Masking
None (Open Label)
Allocation
N/A
Enrollment
10 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
emapalumab
Arm Type
Experimental
Arm Description
emapalumab solution for infusion twice weekly at a starting dose of 1 mg/kg
Intervention Type
Drug
Intervention Name(s)
Emapalumab-Lzsg 5 MG/ML [Gamifant]
Other Intervention Name(s)
Gamifant
Intervention Description
iv
Primary Outcome Measure Information:
Title
Permanent discontinuation of study drug due to emapalumab-related adverse event
Description
as judged by Investigator
Time Frame
until conditioning for hematopoietic stem cell transplant (HSCT), likely within 6 months from first dose
Secondary Outcome Measure Information:
Title
Overall response
Description
measured as complete response, partial response, or HLH improvement
Time Frame
end of treatment or week 8 (whichever occurs earlier)
Title
Time to first overall response
Description
from first dose of study drug to first response
Time Frame
end of treatment, likely within 6 months from first dose
Title
Cumulative duration of response
Description
total time in response
Time Frame
end of treatment, likely within 6 months from first dose
Title
Ability to reduce glucocorticoids by 50% or more
Description
reduction from baseline dose during emapalumab treatment
Time Frame
end of treatment, likely within 6 months from first dose
Title
Investigator assessed response
Description
Investigator's assessment of how patient responds to treatment and rated as complete response, partial response, or no response
Time Frame
end of treatment or week 8 (whichever occurs earlier)
Title
Survival
Description
To start of HSCT conditioning and after time from HSCT to death
Time Frame
end of study

10. Eligibility

Sex
All
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 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 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. Expectation of survival beyond 1 week as judged by the investigator. Patient has expectation of proceeding to HSCT 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. 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: 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. Enrollment in another concurrent clinical interventional study, or intake of an IMP, within three months prior to inclusion in this study 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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Ines Margalet, MD
Phone
+41 61 508 72 13
Email
ines.margalet@sobi.com
First Name & Middle Initial & Last Name or Official Title & Degree
Per Hedlund
Phone
+46 8 697 20 00
Email
Per.hedlund@sobi.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Rui Zhang, MD, Prof
Organizational Affiliation
Beijing Children's Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
Swedish Orphan Biovitrum Research site
City
Shanghai
State/Province
Fudan
Country
China
Individual Site Status
Not yet recruiting
Facility Name
Swedish Orphan Biovitrum Research site
City
Beijing
State/Province
Xicheng
Country
China
Individual Site Status
Recruiting
Facility Name
Swedish Orphan Biovitrum Research site
City
Beijing
Country
China
Individual Site Status
Not yet recruiting
Facility Name
Swedish Orphan Biovitrum Research site
City
Chongqing
Country
China
Individual Site Status
Not yet recruiting
Facility Name
Swedish Orphan Biovitrum Research site
City
Guangzhou
Country
China
Individual Site Status
Not yet recruiting
Facility Name
Swedish Orphan Biovitrum Research site
City
Nanjing
Country
China
Individual Site Status
Not yet recruiting
Facility Name
Swedish Orphan Biovitrum Research site
City
Zhengzhou
Country
China
Individual Site Status
Not yet recruiting

12. IPD Sharing Statement

Plan to Share IPD
Undecided

Learn more about this trial

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

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