High-dose Cyclophosphamide treatment for adult patients with Hemophagocytic Syndrome

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

This study is testing if high doses of Cyclophosphamide can help adults with Hemophagocytic Syndrome get better and live longer.

Quick facts

• Phase: Not applicable
• Study type: Interventional
• Enrollment: 160 (estimated)
• Ages: 18 Years to 65 Years
• Sex: All
• Sponsor: The Second Affiliated Hospital of Chongqing Medical University (Academic / other)
• Drugs / interventions: cyclophosphamide, chemotherapy, prednisone
• Locations: 1 site (Chongqing)
• Trial ID: NCT05936086 on ClinicalTrials.gov

What this trial studies

This clinical trial investigates the effectiveness of high-dose Cyclophosphamide induction therapy in adult patients diagnosed with Hemophagocytic Lymphohistiocytosis (HLH). The study aims to address the urgent need for effective treatment options due to the high early mortality rates associated with this condition. Participants will receive a combination of Cytoxan, Etoposide, Dexamethasone, and Cyclosporine as part of the intervention. The trial seeks to improve patient outcomes by potentially reducing the time to effective treatment and improving survival rates.

Who should consider this trial

Good fit: Ideal candidates for this study are adults aged 18 to 65 who meet the diagnostic criteria for HLH.

Not a fit: Patients with HLH caused by factors not addressed by this treatment or those outside the specified age range may not benefit.

Why it matters

Potential benefit: If successful, this treatment could significantly reduce early mortality rates in patients with HLH.

How similar studies have performed: While there have been studies on HLH treatments, this specific high-dose Cyclophosphamide approach is novel and has not been extensively tested.

Eligibility criteria

Inclusion Criteria:

1. Age range from 18 to 65 years old (including the critical value), gender is not limited;
2. 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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1. 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.
2. 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:

1. HLH caused by treatable infectious causes (such as bacteria, fungi, viruses (except Epstein-Barr virus), protozoa, etc.);
2. Have a history of allergy or contraindications to the drugs involved in the program;
3. Organ damage caused by long-term chronic diseases;
4. Extreme physical weakness, unstable vital signs and inability to tolerate large doses of cyclophosphamide;
5. 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;
6. Mental instability or history of severe mental illness
7. Other factors determined by the researcher that subjects are not suitable to participate in this study.

Where this trial is running

Chongqing

• The Second affiliated Hosptial of Chongqing medical University — Chongqing, China (Recruiting)

Study contacts

• Study coordinator: shifeng Lou, master

Conditions Hemophagocytic Syndrome; Hemophagocytic syndrome（HLH）