An observational study in Thalassemia, Hematopoietic Stem Cell Transplantation and Neutropenia, sponsored by Haikou Affiliated Hospital of Central South University Xiangya School of Medicine. Recruiting at 1 site in China. Open to participants aged 3 Years to 17 Years. Per ClinicalTrials.gov, last updated 2026-07-24.
Sponsored by Haikou Affiliated Hospital of Central South University Xiangya School of Medicine · Observational
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.
Invasive fungal disease (IFD) is a serious and life-threatening infection caused by pathogenic fungi such as Candida, Aspergillus, and Mucorales, commonly affecting immunocompromised patients with hematological malignancies and those undergoing hematopoietic stem cell transplantation. The incidence of IFD has increased significantly in recent years due to the use of intensive chemotherapy and immunosuppressive agents, which result in prolonged and profound neutropenia (absolute neutrophil count \< 500/μL for ≥10 days). Effective antifungal prophylaxis is a critical strategy to reduce IFD-related morbidity and mortality in this high-risk population.
Liposomal amphotericin B (L-AmB) is a broad-spectrum antifungal agent with potent activity against most pathogenic fungi, including azole-resistant strains such as certain Aspergillus and Mucorales species. It is recommended by multiple guidelines as a first-line treatment for candidemia, invasive aspergillosis, and mucormycosis. However, its optimal prophylactic dosing regimen remains unclear, with published studies using widely varying regimens ranging from fixed low-dose (e.g., 50 mg every other day) to intermittent high-dose schedules (e.g., 5 mg/kg twice weekly).
This study proposes a novel prophylactic regimen of L-AmB at a fixed low dose of 50 mg/day (approximately 1 mg/kg/day) administered intravenously once daily. This regimen is designed to provide sustained, continuous drug exposure throughout the high-risk neutropenic period, potentially offering superior protection compared to intermittent high-dose schedules while maintaining a favorable safety profile.
This is a single-center, single-arm, observational clinical study. Participants will be children aged 3-17 years with hematological malignancies who meet the NCCN 2025 V1 guideline criteria for high-risk IFD, including allogeneic hematopoietic stem cell transplantation, autologous hematopoietic cell transplantation with mucosal damage, acute leukemia, grade 3/4 graft-versus-host disease, myelodysplastic syndrome, or other conditions with expected neutropenia >7 days. Participants must have been initiated on L-AmB prophylaxis at 1 mg/kg/day by the treating physician in routine clinical practice due to intolerance or toxicity to other antifungal agents. Key exclusion criteria include prior proven/probable IFD, active fungal infection at screening, significant hypokalemia, severe hepatic or renal impairment, and NYHA Class III/IV heart failure.
A total of 30 participants will be enrolled. Data will be collected at three time points: baseline (Day -3 to 0), treatment period (from Day 1 until the end of L-AmB therapy), and follow-up (within 7 days after treatment completion). Assessments include complete blood counts, serum biochemistry, imaging (chest CT), microbiological tests (G/GM tests, blood cultures, bronchoalveolar lavage fluid cultures or NGS), and recording of neutropenia episodes, fever, pneumonia, and adverse events. IFD diagnosis will be classified according to the Sixth Revised Edition of the Diagnostic Criteria and Treatment Principles for Invasive Fungal Disease in Patients with Hematological Malignancies.
The primary outcome is the incidence of proven or probable IFD. Secondary outcomes include: (1) incidence of pneumonia with no identified pathogen; (2) proportion of patients with persistent unexplained fever >4 days; (3) proportion of patients requiring additional systemic antifungal therapy; (4) discontinuation rate due to adverse effects or intolerance; and (5) incidence of adverse events graded according to CTCAE Version 5.0.
The study is expected to enroll patients from February 2026 to February 2027, with final data collection and analysis completed by March 2027. This study is approved by the institutional ethics committee and will be conducted in accordance with the Declaration of Helsinki. Informed consent will be obtained from all participants or their legal representatives.
250 studies on the registry are indexed under Thalassemia; 43 are open to participants now.
This study's planned enrollment of 30 is below the median of 106 across 70 observational studies indexed under Thalassemia.
Browse Thalassemia studies →Haikou Affiliated Hospital of Central South University Xiangya School of Medicine is the lead sponsor of 5 studies on the registry; 2 are open to participants now.
Counted across the registry records on this site, refreshed daily.
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.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
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 ≤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.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
After a transplant from a donor, the donor’s immune cells attack the recipient’s body, often the skin, gut or liver.
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Allogeneic and autologous transplant
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GVHD (graft-versus-host disease)
After a transplant from a donor, the donor’s immune cells attack the recipient’s body, often the skin, gut or liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Absolute neutrophil count (ANC)
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
NYHA class (heart failure)
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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)
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
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.
Time frame: From baseline to 7 days after the end of antifungal prophylaxis treatment
Incidence of Pneumonia With No Identified Pathogen
Incidence of pneumonia occurring during the study period with no pathogen identified by microbiological or molecular methods, including but not limited to blood cultures, bronchoalveolar lavage fluid cultures, and NGS.
Time frame: From baseline to 7 days after the end of antifungal prophylaxis treatment
Proportion of Participants With Persistent Unexplained Fever >4 Days
Proportion of participants with persistent unexplained fever lasting more than 4 days during the study period. Unexplained fever is defined as fever without an identifiable infectious source after initial clinical evaluation.
Time frame: From baseline to 7 days after the end of antifungal prophylaxis treatment
Proportion of Participants Requiring Additional Systemic Antifungal Therapy
Proportion of participants who require systemic antifungal therapy other than the study drug (low-dose liposomal amphotericin B) for suspected or confirmed fungal infection during the study period.
Time frame: From baseline to 7 days after the end of antifungal prophylaxis treatment
Discontinuation Rate of Liposomal Amphotericin B Due to Adverse Effects or Intolerance
Proportion of participants who discontinue liposomal amphotericin B prophylaxis due to drug-related adverse effects or intolerance during the treatment period.
Time frame: Throughout the treatment period (from Day 1 to the end of liposomal amphotericin B therapy)
Incidence of Adverse Events
Incidence of adverse events occurring during the study period, graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0. Adverse events include laboratory abnormalities (serum creatinine elevation, hepatic enzyme elevation, hypokalemia) and clinical symptoms (infusion-related reactions, fever, chills, nausea, vomiting, etc.).
Time frame: From baseline to 7 days after the end of antifungal prophylaxis treatment
Plan to share: No — This is a single-center observational study with a small sample size of 30 pediatric participants. Individual participant data will not be shared because the study involves a vulnerable population (children with thalassemia undergoing allogeneic hematopoietic stem cell transplantation), and the research protocol did not include provisions for data sharing with external researchers. Furthermore, the study materials and records will be stored locally at the study site and will not be made publicly available to protect participant privacy, in accordance with the study's confidentiality and privacy protection requirements.
No publications or documents are linked to this record.
Started Feb 2026 (actual). Primary completion is estimated for Feb 2027, 12 months after the start.
Of 23 observational studies under Thalassemia, half reached primary completion within 53 months of starting (the median), counting those still running; a quarter within 29 and three quarters within 74. This study's planned 12 months is below that.
How these figures are worked out, and which studies are left out of them.
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Haikou Affiliated Hospital of Central South University Xiangya School of Medicine