Primary Hemophagocytic LymphohistiocytosisPhase 4NCT05744063
A Post-authorization Study to Describe the Safety and Efficacy of Emapalumab for the Treatment of pHLH in Treatment Experienced Chinese Patients
A Phase 4 study testing Emapalumab-Lzsg 5 MG/ML [Gamifant] in people with Primary Hemophagocytic Lymphohistiocytosis, run by Swedish Orphan Biovitrum at 7 sites in China.
Trial summary
The goal of this post-authorization study is to describe safety and efficacy of emapalumab in treatment experienced Chinese patients with pHLH. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Primary Hemophagocytic Lymphohistiocytosis
- What is being tested?
- Emapalumab-Lzsg 5 MG/ML [Gamifant]
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 13 participants
- Where
- 7 sitesChina
- Study dates
- 3 Feb 2023to 8 Aug 2025
- Phase
- Phase 4
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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)
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- 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.
Who cannot join
- 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).
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- 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.
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
China7 sites
Swedish Orphan Biovitrum Research site
Beijing, Xicheng
Swedish Orphan Biovitrum Research site
Beijing
Swedish Orphan Biovitrum Research site
Chongqing
Swedish Orphan Biovitrum Research site
Guangzhou
Swedish Orphan Biovitrum Research site
Nanjing
Swedish Orphan Biovitrum Research site
Shanghai, Fudan
Swedish Orphan Biovitrum Research site
Zhengzhou
About this study
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
The goal of this post-authorization study is to describe safety and efficacy of emapalumab in treatment experienced Chinese patients with pHLH.
Read the 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
- Being tested
- Emapalumab-Lzsg 5 MG/ML [Gamifant]
- Keywords
- pHLH
- Medical subject headings
- Lymphohistiocytosis, Hemophagocytic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Emapalumab-Lzsg 5 MG/ML [Gamifant]Drug
Also called Gamifant
iv
| Study group | Role | What they receive |
|---|---|---|
| emapalumab | Experimental | emapalumab solution for infusion twice weekly at a starting dose of 1 mg/kg Receives: Drug: Emapalumab-Lzsg 5 MG/ML [Gamifant] |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Until conditioning for hematopoietic stem cell transplant (HSCT), likely within 6 months from first dose
Secondary outcomes (6)
Overall Response
Number of participants with an overall response i.e., achievement of either Complete Response or Partial Response or HLH Improvement, at end of treatment or week 8 (whichever occurs earlier).
Measured: End of treatment or week 8 (whichever occurs earlier)
Time to First Overall Response
Time to first overall response from first dose of study drug to the first achievement of response (Complete Response, Partial Response, or HLH Improvement)
Measured: End of treatment, likely within 6 months from first dose
Cumulative Duration of Response
Cumulative duration of response is defined as total time in response from the first achievement of an Overall Response until EOT.
For patients who achieve a response, lost that response, and then achieve it subsequently, the total time in response is calculated by adding together these separate periods in response.
Measured: End of treatment, likely within 6 months from first dose
Ability to Reduce Glucocorticoids by 50% or More
Number of participants able to reduce glucocorticoids by 50% or more of the baseline dose at any time point of the treatment period
Measured: End of treatment, likely within 6 months from first dose
Investigator Assessed Response
Investigator's assessment of how patient responds to treatment and rated as complete response, partial response, or no response
Measured: End of treatment
Survival
Number of participants surviving to start of HSCT conditioning and Number of participants that underwent HSCT surviving after HSCT to end of study
Measured: End of study (1 year)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Feb 2023
- Study start3 Feb 2023
- Primary completion21 Feb 2025
- Study completion8 Aug 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Primary Hemophagocytic LymphohistiocytosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- 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)
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- Swedish Orphan Biovitrum
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Rui Zhang, MD, Prof, Principal investigator, Beijing Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05744063
- Sponsor's protocol ID
- Sobi.emapalumab-104
- Status last verified
- Sep 2025
- First submitted
- 13 Feb 2023
- Last updated
- 26 Aug 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT05744063
- Acronym / sponsor ID
- Sobi.emapalumab-104
- Keywords
- pHLH
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 13 participants (actual)
- Lead sponsor
- Swedish Orphan BiovitrumIndustry (pharma, biotech, devices)
- Study start
- 3 Feb 2023 (actual)
- Primary completion
- 21 Feb 2025 (actual)
- Study completion
- 8 Aug 2025 (actual)
- First posted
- 24 Feb 2023 (actual)
- Results
- Posted 13 Mar 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- 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.
| Arm | Type | Receives |
|---|---|---|
| emapalumab | Experimental | Drug: Emapalumab-Lzsg 5 MG/ML [Gamifant]emapalumab solution for infusion twice weekly at a starting dose of 1 mg/kg |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (20 listed)
- 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:
- Presence of active HLH disease as assessed by the investigator.
- Patients must fulfil one of the following criteria as assessed by the investigator:
- Expectation of survival beyond 1 week as judged by the investigator.
Key exclusion criteria (11 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Emapalumab-Lzsg 5 MG/ML [Gamifant]Also: Gamifantiv | Drug | emapalumab |
Primary outcome
Permanent Discontinuation of Study Drug Due to Emapalumab-related Adverse Event
Time frame: Until conditioning for hematopoietic stem cell transplant (HSCT), likely within 6 months from first dose
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
6 secondary outcomes
Overall Response
Time frame: End of treatment or week 8 (whichever occurs earlier)
Time to First Overall Response
Time frame: End of treatment, likely within 6 months from first dose
Cumulative Duration of Response
Time frame: End of treatment, likely within 6 months from first dose
Ability to Reduce Glucocorticoids by 50% or More
Time frame: End of treatment, likely within 6 months from first dose
Investigator Assessed Response
Time frame: End of treatment
Survival
Time frame: End of study (1 year)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Aug 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Rui Zhang, MD, Prof principal investigator
Beijing Children's Hospital
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Emapalumab-Lzsg 5 MG/ML [Gamifant] in people with Primary Hemophagocytic Lymphohistiocytosis, run by Swedish Orphan Biovitrum at 7 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 20 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 26 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 13 Mar 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Permanent Discontinuation of Study Drug Due to Emapalumab-related Adverse Event (time frame: Until conditioning for hematopoietic stem cell transplant (HSCT), likely within 6 months from first dose). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Aug 2026.