Hemophagocytic Lymphohistiocytosis (HLH)Phase 2/3NCT06951971
Dose-modified Emapalumab and Ruxolitinib (E-Ru) Regimens for Hemophagocytic Lymphohistiocytosis
A Phase 2/3 study testing dose-modified E-Ru and Salvage treatment and follow-up in people with Hemophagocytic Lymphohistiocytosis (HLH), run by The First Affiliated Hospital of Soochow University at 1 site in China.
Trial summary
This study is trying to evaluate the efficacy and safety of dose-modified Emapalumab and Ruxolitinib (E-Ru) regimens for the treatment of active hemophagocytic lymphohistiocytosis. Read more about this study
- Who is it for?
- Ages 1 year to 70 years
- Conditions the study is for
- Hemophagocytic Lymphohistiocytosis (HLH)
- What is being tested?
- dose-modified E-Ru and Salvage treatment and follow-up
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteChina
- Study dates
- 1 Jun 2025to 1 Jun 2028
- Phase
- Phase 2/3
- Are there results?
- Not yet: the study is under way
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 1 year to 70 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Fulfillment of at least five of the eight HLH-2004 criteria for HLH;
- Age 1-70 years old;
- No prior chemotherapy for HLH;
- Confirmed not pregnant and willing to use effective contraceptive measures during the study period, with the last dose of medication administered at least 12 months prior;
- Signed informed consent prior to study participation.
Who cannot join
- According to the New York Heart Association (NYHA) score, patients with heart disease of grade II or above (including grade II);
- HIV-infected patients;
- Patients with severe renal dysfunction (glomerular filtration rate <15 mL/min);
- Patients with severe liver cirrhosis (MELD score>20);
- Uncontrollable infections (including lung infections, intestinal infections, etc.);
- Have serious mental illness;
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- Have a history of active tumor;
- Participate in other clinical investigators at the same time;
- People with central nervous system involvement;
- Exclusion criteria related to concomitant medication.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site
The First Affiliated Hospital of Soochow University
Suzhou, Jiangsu, 215006
Yue Song, Dr., +86 18810253070, xueqifeng1992@163.com
About this study
Official title: A Multiple Center, Prospective, Single Arm, Phase III Clinical Study to Evaluate the Efficacy and Safety of Dose-modified Emapalumab and Ruxolitinib in the Treatment of Hemophagocytic Lymphohistiocytosis
This study is trying to evaluate the efficacy and safety of dose-modified Emapalumab and Ruxolitinib (E-Ru) regimens for the treatment of active hemophagocytic lymphohistiocytosis.
Read the detailed description
This clinical trial is designed to evaluate a new treatment strategy for patients with active Hemophagocytic Lymphohistiocytosis (HLH)-a rare but severe immune disorder characterized by excessive inflammation and immune system activation. HLH can be life-threatening if not treated effectively.
The study is prospective and multicenter, meaning it will be conducted at multiple hospitals and medical institutions, and patients will be followed over time to assess treatment outcomes.
We aim to test the combination of two medications:
- Emapalumab, a monoclonal antibody that blocks interferon-gamma, a key driver of the overactive immune response seen in HLH. In this trial, it will be used at a lower-than-standard dose to reduce potential side effects.
- Ruxolitinib, a JAK1/2 inhibitor that can reduce inflammation by interfering with immune signaling pathways. It will be administered at a higher-than-standard dose to enhance its therapeutic effects.
- Conditions
- Hemophagocytic Lymphohistiocytosis (HLH)
- Being tested
- dose-modified E-Ru, Salvage treatment and follow-up
- Medical subject headings
- Lymphohistiocytosis, Hemophagocytic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
dose-modified E-RuDrug
Emapalumab (1-2 mg/kg intravenously once weekly for 8 weeks) + Ruxolitinib (15-30 mg orally twice daily for 8 weeks)
Salvage treatment and follow-upOther
- for patients who did not respond to the E-Ru regimen after 7 days or who relapsed at any point during treatment, a rescue regimen such as HLH-94 or doxorubicin-etoposide-methylprednisolone (DEP) was administered; 2) for patients diagnosed with lymphoma after enrollment, chemotherapy was initiated; 3) for patients who completed 8 weeks of treatment without recurrence and had no detected HLH-related gene mutations, follow-up was initiated; and 4) for patients who met the criteria for allo-HSCT, allo-HSCT was performed.
| Study group | Role | What they receive |
|---|---|---|
| E-Ru for HLH | Experimental | dose-modified Emapalumab+Ruxolitinib for the treatment of HLH patients. Receives: Drug: dose-modified E-Ru; Other: Salvage treatment and follow-up |
What is being measured
The primary outcome is the main question the study is designed to answer.
60-days OS
overall survival (OS) rate at 60 days (2-month OS)
Measured: 60 days
Secondary outcomes (5)
Response rate
The ORR was defined as the percentage of patients with a complete response (CR), a partial response (PR), or an improvement in the measures of HLH.
Measured: 7, 14, 28, and 56 days
Security
Adverse events (AEs) were assessed according to the National Cancer Institute Common Terminology Criteria for Adverse Events, version 5.0 (CTCAE 5.0).
Measured: 7, 14, 28, and 56 days
EFS
event-free survival, defined as the time from the first administration of E-Ru until no response on day 7, disease progression or death from any cause
Measured: 6 months
Overall survival
OS (defined as the time from the first administration of E-Ru until death from any cause)
Measured: 6 months
Trend of related indicators
Changes in biomarkers between baseline and post-treatment.
Measured: 7, 14, 28, and 56 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Apr 2025
- Study start1 Jun 2025 (estimated)
- Primary completion1 Jun 2027 (estimated)
- Study completion1 Jun 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophagocytic Lymphohistiocytosis (HLH)Studies of the same condition, open and under way.
- More trials run by The First Affiliated Hospital of Soochow UniversityThe sponsor's other registered studies.
- 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 2/3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Xuefeng He, The First Affiliated Hospital of Soochow University
- Investigators
- Xuefeng He, Dr., Principal investigator, The First Affiliated Hospital of Soochow University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06951971
- Sponsor's protocol ID
- E-Ru
- Status last verified
- Apr 2025
- First submitted
- 23 Apr 2025
- Last updated
- 30 Apr 2025
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 30 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multiple Center, Prospective, Single Arm, Phase III Clinical Study to Evaluate the Efficacy and Safety of Dose-modified Emapalumab and Ruxolitinib in the Treatment of Hemophagocytic Lymphohistiocytosis
- ClinicalTrials.gov ID
- NCT06951971
- Acronym / sponsor ID
- E-Ru
- Conditions
- Hemophagocytic Lymphohistiocytosis (HLH)
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- The First Affiliated Hospital of Soochow UniversityUniversity, hospital or other
- Study start
- 1 Jun 2025 (estimated)
- Primary completion
- 1 Jun 2027 (estimated)
- Study completion
- 1 Jun 2028 (estimated)
- First posted
- 30 Apr 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study is trying to evaluate the efficacy and safety of dose-modified Emapalumab and Ruxolitinib (E-Ru) regimens for the treatment of active hemophagocytic lymphohistiocytosis.
Detailed description
This clinical trial is designed to evaluate a new treatment strategy for patients with active Hemophagocytic Lymphohistiocytosis (HLH)-a rare but severe immune disorder characterized by excessive inflammation and immune system activation. HLH can be life-threatening if not treated effectively.
The study is prospective and multicenter, meaning it will be conducted at multiple hospitals and medical institutions, and patients will be followed over time to assess treatment outcomes.
We aim to test the combination of two medications:
- Emapalumab, a monoclonal antibody that blocks interferon-gamma, a key driver of the overactive immune response seen in HLH. In this trial, it will be used at a lower-than-standard dose to reduce potential side effects.
- Ruxolitinib, a JAK1/2 inhibitor that can reduce inflammation by interfering with immune signaling pathways. It will be administered at a higher-than-standard dose to enhance its therapeutic effects.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| E-Ru for HLH | Experimental | Drug: dose-modified E-Ru, Other: Salvage treatment and follow-updose-modified Emapalumab+Ruxolitinib for the treatment of HLH patients. |
Who may qualify
- Ages
- 1 year to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Fulfillment of at least five of the eight HLH-2004 criteria for HLH;
- Age 1-70 years old;
- No prior chemotherapy for HLH;
- Confirmed not pregnant and willing to use effective contraceptive measures during the study period, with the last dose of medication administered at least 12 months prior;
- Signed informed consent prior to study participation.
Key exclusion criteria (10 listed)
- According to the New York Heart Association (NYHA) score, patients with heart disease of grade II or above (including grade II);
- HIV-infected patients;
- Patients with severe renal dysfunction (glomerular filtration rate <15 mL/min);
- Patients with severe liver cirrhosis (MELD score>20);
- Uncontrollable infections (including lung infections, intestinal infections, etc.);
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 |
|---|---|---|
| dose-modified E-RuEmapalumab (1-2 mg/kg intravenously once weekly for 8 weeks) + Ruxolitinib (15-30 mg orally twice daily for 8 weeks) | Drug | E-Ru for HLH |
| Salvage treatment and follow-up1) for patients who did not respond to the E-Ru regimen after 7 days or who relapsed at any point during treatment, a rescue regimen such as HLH-94 or doxorubicin-etoposide-methylprednisolone (DEP) was administered; 2) for patients diagnosed with lymphoma after enrollment, chemotherapy was initiated; 3) for patients who completed 8 weeks of treatment without recurrence and had no detected HLH-related gene mutations, follow-up was initiated; and 4) for patients who met the criteria for allo-HSCT, allo-HSCT was performed. | Other | E-Ru for HLH |
Primary outcome
60-days OS
Time frame: 60 days
overall survival (OS) rate at 60 days (2-month OS)
5 secondary outcomes
Response rate
Time frame: 7, 14, 28, and 56 days
Security
Time frame: 7, 14, 28, and 56 days
EFS
Time frame: 6 months
Overall survival
Time frame: 6 months
Trend of related indicators
Time frame: 7, 14, 28, and 56 days
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Apr 2025.
- Individual participant dataSharing planned: Undecided
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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.
Xuefeng He, Dr. principal investigator
The First Affiliated Hospital of Soochow University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing dose-modified E-Ru and Salvage treatment and follow-up in people with Hemophagocytic Lymphohistiocytosis (HLH), run by The First Affiliated Hospital of Soochow University at 1 site 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 5 inclusion and 10 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: opening soon, as of the sponsor's last update on 30 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Yue Song, Dr (+86 18810253070, xueqifeng1992@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
60-days OS (time frame: 60 days). 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 30 Apr 2025.