Low-Dose Liposomal Amphotericin B for Invasive Fungal Infection Prophylaxis in Neutropenic Children

NCT07725263 | Recruiting DMC

• 1 other identifier: SC20260053
• Study Type: observational
• Target: 30
• Locations: 1 country
• Sites: 1

Brief Summary

This is a single-center, single-arm, observational clinical study evaluating the efficacy and safety of low-dose liposomal amphotericin B (1 mg/kg/day, intravenous, once daily) for the prevention of invasive fungal infections in children aged 3-17 years with hematological malignancies who develop prolonged neutropenia (absolute neutrophil count ≤ 0.5×10\^9/L, expected to last \> 7 days) and are at high risk for invasive fungal disease. Participants are those who, per the treating physician's routine clinical decision, have been initiated on liposomal amphotericin B prophylaxis at 1 mg/kg/day due to intolerance or toxicity to other antifungal agents. The primary outcome is the incidence of proven or probable invasive fungal disease. Secondary outcomes include incidence of pneumonia, persistent unexplained fever \>4 days, use of additional systemic antifungal therapy, and adverse events. A total of 30 participants will be enrolled. Data will be collected at baseline, during treatment, and within 7 days after treatment completion.

Conditions

Thalassemia; Neutropenia; Invasive Fungal Infections; Hematopoietic Stem Cell Transplantation; Graft vs Host Disease

Keywords

Thalassemia; Allogeneic Hematopoietic Stem Cell Transplantation; Antifungal Prophylaxis; Liposomal Amphotericin B; Invasive Fungal Disease; Neutropenia

Outcome Measures

Primary Outcomes (1)

• Incidence of Proven or Probable Invasive Fungal Disease

  Proven or probable invasive fungal disease (IFD) diagnosed according to the Sixth Revised Edition of the Diagnostic Criteria and Treatment Principles for Invasive Fungal Disease in Patients with Hematological Malignancies. Proven IFD requires histopathological evidence or positive culture from a sterile site. Probable IFD requires the presence of host factors, clinical features, and mycological evidence.

  From baseline to 7 days after the end of antifungal prophylaxis treatment

Secondary Outcomes (5)

• Incidence of Pneumonia With No Identified Pathogen

  From baseline to 7 days after the end of antifungal prophylaxis treatment

• Proportion of Participants With Persistent Unexplained Fever >4 Days

  From baseline to 7 days after the end of antifungal prophylaxis treatment

• Proportion of Participants Requiring Additional Systemic Antifungal Therapy

  From baseline to 7 days after the end of antifungal prophylaxis treatment

• Discontinuation Rate of Liposomal Amphotericin B Due to Adverse Effects or Intolerance

  Throughout the treatment period (from Day 1 to the end of liposomal amphotericin B therapy)

• Incidence of Adverse Events

  From baseline to 7 days after the end of antifungal prophylaxis treatment

Study Arms (1)

Liposomal Amphotericin B Prophylaxis Group

Children with thalassemia undergoing allogeneic hematopoietic stem cell transplantation who meet high-risk criteria for invasive fungal disease per NCCN 2025 V1 guidelines, with expected neutropenia \>7 days (absolute neutrophil count ≤0.5×10\^9/L). Participants receive liposomal amphotericin B at 1 mg/kg/day, intravenous, once daily, as antifungal prophylaxis initiated by the treating physician in routine clinical practice due to intolerance or toxicity to other antifungal agents.

Drug: Liposomal Amphotericin B (LAmB)

Interventions

Liposomal Amphotericin B (LAmB) DRUG

Liposomal amphotericin B at 1 mg/kg/day, administered intravenously once daily, for antifungal prophylaxis in children with prolonged neutropenia. Liposomal amphotericin B is a broad-spectrum polyene antifungal agent with activity against most pathogenic fungi, including Candida, Aspergillus, and Mucorales species. The liposomal formulation reduces nephrotoxicity compared to conventional amphotericin B deoxycholate while maintaining equivalent antifungal activity.

Also known as: L-AmB, Amphotericin B Liposome

Liposomal Amphotericin B Prophylaxis Group

Eligibility Criteria

• Age: 3 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• Sampling Method: Non-Probability Sample

Study Population

Children aged 3-17 years with thalassemia or other hematological conditions who undergo allogeneic hematopoietic stem cell transplantation or other intensive therapies associated with prolonged neutropenia (\>7 days), meet NCCN 2025 V1 high-risk criteria for invasive fungal disease, and have been initiated on liposomal amphotericin B prophylaxis at 1 mg/kg/day by the treating physician in routine clinical practice due to intolerance or toxicity to other antifungal agents.

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Haikou Affiliated Hospital of Central South University Xiangya School of Medicine: lead

Study Sites (1)

Haikou Affiliated Hospital of Central South University Xiangya School of Medicine

Haikou, Hainan, 570208, China

RECRUITING

MeSH Terms

Conditions

Thalassemia; Neutropenia; Invasive Fungal Infections; Graft vs Host Disease

Interventions

liposomal amphotericin B; Amphotericin B

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Agranulocytosis; Leukopenia; Cytopenia; Leukocyte Disorders; Mycoses; Bacterial Infections and Mycoses; Infections; Immune System Diseases

Intervention Hierarchy (Ancestors)

Macrolides; Polyketides; Lactones; Organic Chemicals

Study Officials

• Xiaoyang Yang, MD

  Department of Hematology, Haikou People's Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Xiaoyang Yang, MD

CONTACT

+86-13337647693; y108108@126.com

Guang Cheng, MMed

CONTACT

+86-18077944163; chengg4163@163.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 7 Days
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 21, 2026
• First Posted: July 24, 2026
• Study Start: February 1, 2026
• Primary Completion (Estimated): February 28, 2027
• Study Completion (Estimated): March 31, 2027
• Last Updated: July 24, 2026

Record last verified: 2026-07

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)