Low-dose liposomal amphotericin B to prevent invasive fungal infections in neutropenic children

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a single-center, observational study enrolling 30 pediatric participants who are at high risk for invasive fungal disease due to prolonged neutropenia. All participants receive a fixed low dose of liposomal amphotericin B (1 mg/kg/day IV) as prophylaxis based on clinician judgment after intolerance to other antifungals. The primary outcome is the incidence of proven or probable invasive fungal disease, with secondary outcomes including pneumonia, prolonged fever, use of additional systemic antifungals, and adverse events. Data will be collected at baseline, during treatment, and follow-up to assess safety and efficacy. The study aims to clarify whether this prophylactic approach can reduce fungal infections in this vulnerable population.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Age 3 to 17 years (inclusive), both sexes.
* Meets NCCN 2025 V1 guideline criteria for high-risk invasive fungal disease, including allogeneic hematopoietic stem cell transplantation, autologous hematopoietic cell transplantation with mucosal damage, acute leukemia, grade 3/4 graft-versus-host disease, myelodysplastic syndrome, lymphoma(a), multiple myeloma(a), chronic lymphocytic leukemia(a), treatment with purine analogues (fludarabine, clofarabine, nelarabine), chimeric antigen receptor (CAR) T-cell therapy, alemtuzumab therapy, with expected neutropenia \>7 days(b) and accompanied by agranulocytosis.

Note (a): For these heterogeneous diseases, myeloablative therapy must be met; if neutropenia \>7 days is not met, the patient should be excluded.

Note (b): Agranulocytosis is defined as absolute neutrophil count ≤0.5×10\^9/L, or absolute neutrophil count ≤1×10\^9/L with expected decline to ≤0.5×10\^9/L within 48 hours.

* Assessed by the study physician as having high-risk for invasive fungal infection, intolerant or unable to use other antifungal agents due to toxicity or other reasons, and the treating physician has independently decided in routine clinical practice to initiate liposomal amphotericin B for antifungal prophylaxis for 3-5 days, with the selected dosage regimen of 1 mg/kg/day, intravenous, once daily.
* Patient or legally authorized representative has voluntarily signed the informed consent form.

Exclusion Criteria:

* Allergy to any component of liposomal amphotericin B, or development of serious adverse events during the initial 3-5 days of prophylactic use.
* Prior history of proven or probable invasive fungal disease (IFD).
* Presence of pneumonia, unexplained fever, or clinical/imaging evidence suggestive of or diagnosed as fungal infection during screening.
* Clinically significant hypokalemia (defined as serum potassium \<3.2 mmol/L, or below the lower limit of normal while receiving digitalis therapy) that cannot be corrected before starting trial treatment.
* Hepatic dysfunction with aspartate aminotransferase (AST) or alanine aminotransferase (ALT) ≥5× upper limit of normal (ULN), or total bilirubin ≥3× ULN.
* Renal impairment requiring or currently undergoing hemodialysis or peritoneal dialysis.
* New York Heart Association (NYHA) Class III/IV heart failure.
* Positive for human immunodeficiency virus (HIV) antibody or Treponema pallidum hemagglutination assay (TPHA).
* Expected survival \<3 months.
* Pregnant or breastfeeding women, or women of childbearing potential who are not using contraception and planning pregnancy.
* Any other condition that the investigator considers inappropriate for participation in the clinical trial.

Where it's running

• Haikou Affiliated Hospital of Central South University Xiangya School of Medicine
  Haikou, Hainan, China Recruiting

Study contacts

• Principal investigator: Xiaoyang Yang, MD — Department of Hematology, Haikou People's Hospital
• Study coordinator: Xiaoyang Yang, MD
• Email: y108108@126.com
• Phone: +86-13337647693

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.