Adult Refractory Secondary Hemophagocytic LymphohistiocytosisNCT07531069
Efficacy and Safety of Interferon-Gamma Monoclonal Antibody Combined With Dexamethasone in the Treatment of Refractory Adult Secondary Hemophagocytic Lymphohistiocytosis
An observational study of Adult Refractory Secondary Hemophagocytic Lymphohistiocytosis and Secondary Hemophagocytic Lymphohistiocytosis, run by Fudan University at 1 site in China.
Trial summary
The goal of this observational study is to learn if interferon-gamma monoclonal antibody combined with dexamethasone works to treat adults with refractory secondary hemophagocytic lymphohistiocytosis (HLH). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Adult Refractory Secondary Hemophagocytic Lymphohistiocytosis, Secondary Hemophagocytic Lymphohistiocytosis, HLH
- Type of treatment
- Pill or oral medicine, Injection or infusion
- How many people
- 22 participants
- Where
- 1 siteChina
- Study dates
- 21 May 2024to 31 Aug 2025
- How long will I take part?
- 12 Months
- Phase
- Observational
- Are there results?
- None posted in the registry
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: This study will enroll adult participants aged 18 years and older with a confirmed diagnosis of refractory secondary hemophagocytic lymphohistiocytosis (HLH) based on the HLH-2004 diagnostic criteria. Participants must have demonstrated inadequate response or disease progression following standard first-line or second-line HLH-directed therapy. All participants must be able to provide written informed consent prior to any study-related procedures.
Who may be able to join
- Age ≥ 18 years
- Confirmed diagnosis of secondary hemophagocytic lymphohistiocytosis (HLH) according to HLH-2004 criteria
- Refractory HLH defined as inadequate response or progression after at least 2 weeks of standard first-line or second-line therapy
- Able to provide written informed consent
Who cannot join
- Active infection with mycobacteria, histoplasma, CMV, herpes zoster, or HIV
- Left ventricular ejection fraction < 50%
- Concurrent use of other anti-neoplastic or investigational agents
- Other uncontrolled infections judged by the investigator
- Psychiatric disorders or poor protocol compliance
Trial locations and contacts
1 site in 1 country.
China1 site
Huadong Hospital, Fudan University
Shanghai, Shanghai Municipality, 200040
About this study
The goal of this observational study is to learn if interferon-gamma monoclonal antibody combined with dexamethasone works to treat adults with refractory secondary hemophagocytic lymphohistiocytosis (HLH). The main questions it aims to answer are:
How well does this treatment help patients recover from refractory secondary HLH? How safe is this treatment for these patients? Participants will receive interferon-gamma monoclonal antibody and dexamethasone as part of their clinical care. Researchers will monitor participants with regular blood tests, physical exams, and safety checks for up to 8 weeks of treatment. They will collect information about how participants respond to treatment and any side effects that occur.
This study will include about 22 adult participants at Huadong Hospital Affiliated to Fudan University.
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Observation group | Participants receive IFN-γ monoclonal antibody by weight-based intravenous infusion. Dexamethasone is administered orally or intravenously. Prophylaxis for infections and standard supportive care are provided as clinically needed. Treatment continues for up to 8 weeks, with regular safety and efficacy assessments. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Response Rate (ORR)
The proportion of participants who achieve complete response (CR) or partial response (PR) at the end of 8-week treatment, defined by normalized or improved clinical and laboratory parameters including ferritin, triglycerides, sCD25, blood cell counts, and hemophagocytosis.
Measured: Up to 8 weeks after initiation of treatment
Secondary outcomes (3)
Progression-Free Survival (PFS)
Time from treatment start to disease progression or death from any cause, whichever occurs first.
Measured: Up to 12 months after treatment initiation
Overall Survival (OS)
Time from treatment start to death from any cause.
Measured: Up to 12 months after treatment initiation
Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs)
Number and severity of treatment-emergent adverse events and serious adverse events, graded according to NCI-CTCAE v5.0.
Measured: Up to 28 days after the last dose of study treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Apr 2026
- Study start21 May 2024
- Primary completion31 Aug 2025
- Study completion31 Aug 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Adult Refractory Secondary Hemophagocytic LymphohistiocytosisStudies of the same condition, open and under way.
- More trials run by Fudan 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
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 22 participants (actual)
- Target duration
- 12 Months
Sponsor and investigators
- Lead sponsor
- Fudan University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Zhijun Bao, Fudan University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07531069
- Sponsor's protocol ID
- 2024K017
- Status last verified
- Apr 2026
- First submitted
- 2 Apr 2026
- Last updated
- 15 Apr 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 15 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of Interferon-Gamma Monoclonal Antibody Combined With Dexamethasone in the Treatment of Refractory Adult Secondary Hemophagocytic Lymphohistiocytosis
- ClinicalTrials.gov ID
- NCT07531069
- Acronym / sponsor ID
- 2024K017
- Conditions
- Adult Refractory Secondary Hemophagocytic Lymphohistiocytosis, Secondary Hemophagocytic Lymphohistiocytosis, HLH
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 22 participants (actual)
- Lead sponsor
- Fudan UniversityUniversity, hospital or other
- Study start
- 21 May 2024 (actual)
- Primary completion
- 31 Aug 2025 (actual)
- Study completion
- 31 Aug 2025 (actual)
- First posted
- 15 Apr 2026 (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.
The goal of this observational study is to learn if interferon-gamma monoclonal antibody combined with dexamethasone works to treat adults with refractory secondary hemophagocytic lymphohistiocytosis (HLH). The main questions it aims to answer are:
How well does this treatment help patients recover from refractory secondary HLH? How safe is this treatment for these patients? Participants will receive interferon-gamma monoclonal antibody and dexamethasone as part of their clinical care. Researchers will monitor participants with regular blood tests, physical exams, and safety checks for up to 8 weeks of treatment. They will collect information about how participants respond to treatment and any side effects that occur.
This study will include about 22 adult participants at Huadong Hospital Affiliated to Fudan University.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Target follow-up
- 12 Months
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Observation group | – | –Participants receive IFN-γ monoclonal antibody by weight-based intravenous infusion. Dexamethasone is administered orally or intravenously. Prophylaxis for infections and standard supportive care are provided as clinically needed. Treatment continues for up to 8 weeks, with regular safety and efficacy assessments. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- This study will enroll adult participants aged 18 years and older with a confirmed diagnosis of refractory secondary hemophagocytic lymphohistiocytosis (HLH) based on the HLH-2004 diagnostic criteria. Participants must have demonstrated inadequate response or disease progression following standard first-line or second-line HLH-directed therapy. All participants must be able to provide written informed consent prior to any study-related procedures.
Key inclusion criteria (4 listed)
- Age ≥ 18 years
- Confirmed diagnosis of secondary hemophagocytic lymphohistiocytosis (HLH) according to HLH-2004 criteria
- Refractory HLH defined as inadequate response or progression after at least 2 weeks of standard first-line or second-line therapy
- Able to provide written informed consent
Key exclusion criteria (5 listed)
- Active infection with mycobacteria, histoplasma, CMV, herpes zoster, or HIV
- Left ventricular ejection fraction < 50%
- Concurrent use of other anti-neoplastic or investigational agents
- Other uncontrolled infections judged by the investigator
- Psychiatric disorders or poor protocol compliance
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Overall Response Rate (ORR)
Time frame: Up to 8 weeks after initiation of treatment
The proportion of participants who achieve complete response (CR) or partial response (PR) at the end of 8-week treatment, defined by normalized or improved clinical and laboratory parameters including ferritin, triglycerides, sCD25, blood cell counts, and hemophagocytosis.
3 secondary outcomes
Progression-Free Survival (PFS)
Time frame: Up to 12 months after treatment initiation
Overall Survival (OS)
Time frame: Up to 12 months after treatment initiation
Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: Up to 28 days after the last dose of study treatment
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Apr 2026.
- Individual participant dataSharing planned: Yes
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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Adult Refractory Secondary Hemophagocytic Lymphohistiocytosis and Secondary Hemophagocytic Lymphohistiocytosis, run by Fudan 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 4 inclusion and 5 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 15 Apr 2026. 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.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Overall Response Rate (ORR) (time frame: Up to 8 weeks after initiation of treatment). 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 15 Apr 2026.