ClinicalTrials.gov record NCT05936086

A Randomized Controlled Study of High-dose Cyclophosphamide Induction Therapy in Adult Patients With HLH

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Jul 27, 2026

A Randomized Controlled Study of High-dose Cyclophosphamide Induction Therapy in Adult Patients With HLH

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.The registry record was last updated on Aug 30, 2023, about 3 years ago, so the status shown may be out of date. The study team or the registry can confirm.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
Healthy volunteersAcceptedPeople who do not have the condition being studied who are allowed to take part. Some studies include both people with the condition and healthy volunteers; others may enrol only healthy volunteers.
Age18 years old to 65 years old
SexAll
LocationChina
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Randomized trial comparing a short, very high‑dose cyclophosphamide induction plus dexamethasone with the standard HLH2004 regimen (etoposide, dexamethasone, cyclosporine). It enrolls adults diagnosed by HLH-2004 criteria. The study measures early (up to 30‑day) response rates and safety.

What it is studying. Whether a two-day, high‑dose cyclophosphamide course (Cytoxan 40 mg/kg IV once daily for 2 days) with dexamethasone as induction produces different short-term clinical and laboratory responses and safety outcomes compared with the standard HLH2004 induction regimen (etoposide, dexamethasone, cyclosporine) in adults with non-infective HLH.

What the researchers hope to learn. To compare complete, near‑complete, and partial response rates and individual diagnostic indicators between the two induction approaches; to assess short‑term laboratory and clinical improvements and 30‑day mortality and safety.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

Adult secondary HLH involves tumors, autoimmune diseases and other causes in addition to infection,Infectious factors, theoretically need different treatment methods for different etiology. But adult HLH itself disease .The situation progresses ferociously, which can cause organ damage and blood coagulation disorder and endanger life quickly, with early mortality (30days).It can be more than 50%. On the other hand, although diagnostic techniques have improved significantly, identifying the cause is still costly Time, such as 1-2 weeks for the pathological diagnosis of lymphoma, leads to more patients losing further treatment due to early death. The opportunity to heal. Therefore, it is important to explore effective induction therapy for adult HLH. In the majority ,Early (30-day) mortality was as high as 40% after cardiac induction using HLH2004 or CHOP(cyclophosphamide, hydroxydaunomycin, Oncovin, and prednisone) induction. HLH, on the other hand, usually requires prompt treatment before the cause is established. Due to a specific infection HLH can benefit from anti-infective therapy. Therefore, it is necessary to explore more effective induction therapy for adult non-infective HLH.It has very important clinical significance. Adult secondary HLH has the common features of a large number of T cell proliferation and activation and a significant reduction of NK(natural killer) cells, in which the central liNK(natural killer) is a large number of T cells proliferation and secomplete remission etion of cytokines, which can be used as induction therapy.Common target is also the pathological basis for designing unified induction scheme. Cyclophosphamide is a commonly used alkylated chemotherapy drug,It's also an important immunosuppressant. Based on the treatment of regenerative disorders anemia, allogeneic hematopoietic stem cell transplantation prevention.Experience with Plant versus Host disease (GVHD) has shown that the use of cyclophosphamide exceeds a total dose of 25mg/day,Two days can effectively kill CD8(cluster of differentiation 8 )+ or CD4(cluster of differentiation 4 )+T cells, and the maximum tolerated dose of this drug in humans exceeds 50mg/kg/day for two days. Aiming at the central liNK(natural killer) of adult HLH pathogenesis, The investigators designed for the first time to use a large dose of cyclophosphamide (25mg-50mg/kg/day 2days) to inhibit the activation of T cells, inhibit the production of cytokines and block the development mechanism of HLH. This study intends to conduct a randomized controlled study, with HLH2004 scheme as the control, and the observation is large efficacy and safety of dose cyclophosphamide in induction therapy of non-infective adult HLH in order to complete remission eate a new induction Treatment plan.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • Cytoxan
  • Etoposide
  • Dexamethasone
  • Cyclosporine (CSA)

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

  • Age range from 18 to 65 years old (including the critical value), gender is not limited;
  • According to the diagnostic criteria of HLH-2004, HLH can be diagnosed if any of the following two criteria are met:
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  • Molecular diagnosis is consistent with HLH: Currently known HLH related pathogenic genes exist, such as PRF1, UNC13D, STX11, STXBP2, Rab27a, LYST, SH2D1A, BIRC4, ITK, AP3β1, MAGT1, CD27 (cluster of differentiation antigen 27 )and other pathological mutations.
  • Meet 5 or more of the following 8 indicators:
  • Fever: body temperature \&gt; 38.5 ℃, continuous \&gt; 7 d; ② Splenomegaly;
  • Hemocytopenia (involving two or three peripheral blood lines) : hemoglobin \&lt; 90 g/L (\&lt; 4 weeks infant, hemoglobin \&lt; 100 g/L), platelet \&lt; 100×109/L, neutrophils \&lt; 1.0×109/L and not caused by reduced hematopoietic function of bone marrow; ④ High triglyceride (TG) sepsis and/or low fibrinogenemia: triglyceride \&gt; 3 mmol/L or 3 standard deviations above the same age, fibrinogen \&lt; 1.5g /L or less than 3 standard deviations for the same age; (5) Hematophagy was found in bone marrow, spleen, liver or lymph nodes;
  • The activity of NK cells is decreased or absent;
  • ⑦ Serum ferritin increase: ferritin ≥500 μg/L; Elevated sCD25 (soluble interleukin-2 receptor). (3) Those who can understand the research content, agree to comply with the research plan, and voluntarily sign the informed consent.

Exclusion criteria

  • HLH caused by treatable infectious causes (such as bacteria, fungi, viruses (except Epstein-Barr virus), protozoa, etc.);
  • Have a history of allergy or contraindications to the drugs involved in the program;
  • Organ damage caused by long-term chronic diseases;
  • Extreme physical weakness, unstable vital signs and inability to tolerate large doses of cyclophosphamide;
  • Severe and/or uncontrolled co-morbidivities (e.g., uncontrolled diabetes, pulmonary hypertension, etc.) that the investigator believes may pose an unacceptable safety risk or interfere with protocol compliance;
  • Mental instability or history of severe mental illness
  • Other factors determined by the researcher that subjects are not suitable to participate in this study.

Study description and what participation involves

The registry record has no detailed description beyond the summary above.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

After giving informed consent, participants are randomly assigned to one of two induction treatments: high‑dose IV cyclophosphamide (Cytoxan 40 mg/kg daily for 2 days) with dexamethasone, or a standard regimen of etoposide plus dexamethasone and cyclosporine. Study participation involves regular blood tests and clinical monitoring (including ferritin, sCD25, blood counts, triglycerides and consciousness level) and may include diagnostic tissue or bone‑marrow examination if clinically needed. Responses and safety are assessed over about 30 days. Participants will be monitored closely by the study team throughout this period.

How the study is structured. Interventional, randomized, open‑label, parallel-group drug trial. Participants are assigned to one of two treatment arms (high‑dose cyclophosphamide + dexamethasone versus HLH2004 regimen) and assessed for response and safety over the short term (primary outcomes measured up to 30 days).

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • The listed sites are in China. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

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Important dates

Study start
Apr 20, 2023
Primary completion
Apr 20, 2026 (estimated)
Study completion
May 20, 2027 (estimated)
Target enrollment
160 participants (estimated)
Last updated on registry
Aug 30, 2023
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
shifeng Lou

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (1)

  • The Second affiliated Hosptial of Chongqing medical UniversityRecruiting

    Chongqing · China

    • Shifeng Lou, master
    • Jianchuan Deng

Terms used on this page · explanations by MedicalUpdates.ai

prednisone
Steroid medicines, such as prednisone, that reduce inflammation. Studies often require a stable dose at entry or measure whether the study treatment allows the dose to be reduced.
immunosuppressant
A medicine that reduces the activity of the immune system. Studies of autoimmune conditions often require a stable dose, a prior failure, or a washout of such medicines.
maximum tolerated dose
The highest dose found in a dose-escalation study that can be given without too many dose-limiting side effects.
randomized
Participants are assigned to the study groups by chance, like a coin toss, rather than chosen by the doctor or the participant. This is done so the groups are comparable.
complete remission
No sign of the condition can be found with the tests used in the study. It does not always mean the condition is gone permanently, and the definition depends on the condition and the study.
informed consent
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
contraindications
A reason a particular test or treatment should not be used for a person, for example a metal implant that rules out an MRI scan.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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