Liposomal amphotericin B (AmBisome) for breakthrough invasive fungal infections in children and teens with blood cancers
A Real-world Prospective Observational Study on the Efficacy and Safety of L-AmB for br- IFD in Children and Adolescent Patients With Hematological Malignancies Receiving Triazoles or Echinocandins Prophylaxis
This project will see if liposomal amphotericin B (AmBisome) works and is safe for treating breakthrough invasive fungal infections in children and adolescents with hematological malignancies who were taking triazole or echinocandin prevention.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 38 (estimated) |
| Ages | 1 Month to 18 Years |
| Sex | All |
| Sponsor | Institute of Hematology & Blood Diseases Hospital, China Academic / other |
| Locations | 1 site (Tianjin, Tianjin Municipality) |
| Trial ID | NCT07357038 on ClinicalTrials.gov |
What this trial studies
This is a single-center, prospective, single-arm observational study enrolling children and adolescents (1 month to 18 years) with hematological malignancies who develop breakthrough invasive fungal disease while on triazole or echinocandin prophylaxis. Enrolled patients receive liposomal amphotericin B (AmBisome) as part of routine care and investigators will record clinical response and safety outcomes, including renal function, using EORTC/MSG 2020 definitions for proven, probable, and possible IFD. The planned sample size is 43 (allowing for 10% dropout) to estimate a presumed 67% favorable response rate with a 95% confidence interval. Data will be compared to historical adult benchmarks and baseline characteristics that predict response will be explored.
Who should consider this trial
Good fit: Children and adolescents aged 1 month to 18 years with a hematological malignancy who developed a proven, probable, or possible breakthrough invasive fungal disease at least 7 days after starting triazole or echinocandin prophylaxis are the intended participants.
Not a fit: Patients who already received any amphotericin formulation for prophylaxis or treatment before enrollment, or those without breakthrough IFD or outside the age range, are unlikely to be eligible or to benefit from these specific observations.
Why it matters
Potential benefit: If results are favorable, this could support using L‑AmB as an effective option with an acceptable renal safety profile for pediatric breakthrough IFD and guide clinical care in similar patients.
How similar studies have performed: Liposomal amphotericin B has documented efficacy in adults with invasive fungal disease, but pediatric-specific prospective data on breakthrough IFD and comparative renal safety are limited, so this study addresses a relatively undertested pediatric question.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * The children and adolescent patients with hematological malignancy received echinocandin/triazole for antifungal prophylaxis and diagnosed with br-IFD. 1. Age: from 1 month to 18 years old. 2. Diagnosed patients with hematological malignancy. 3. Received echinocandin/triazole for antifungal prophylaxis at least 7 days. 4. Br-IFD is defined as a proven, probable, and possible IFD diagnosed at least 7 days after the start of primary antifungal prophylaxis and by 7 days from the end of primary antifungal prophylaxis. Definitions of proven, probable and possible IFD based on the EORTC/MSG 2020 criteria. 5. ECOG-PS(Eastern Cooperative Oncology Group Performance Status): 0-2 points. 6. There are no organ dysfunction restrictions during the screening period that limit the use of this protocol. 7. The guardian understands the research and signs written informed consent form. Exclusion Criteria: * (a) Received AmB formulation for prophylaxis or treatment within the past 30 days. (b) Confirmed allergy/rapid onset severe allergic reaction/intolerance to L-AmB (c) Has a history of other tumors and has received any treatment for this tumor within the past 3 years (d) HIV, active hepatitis B and active hepatitis C virus or syphilis infection (e) Suffering from mental illness or other conditions that prevent cooperation with research, treatment, and monitoring requirements (f) Serum creatinine level 2 times the upper limit of normal; Liver transaminase or alkaline phosphatase levels 5 times the upper limit of normal, bilirubin levels 3 times the upper limit of normal (g) Researchers believe that patients who are not suitable for inclusion
Where this trial is running
Tianjin, Tianjin Municipality
- National Clinical Research Center for Blood Diseases, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College Hospital — Tianjin, Tianjin Municipality, China (Recruiting)
Study contacts
- Study coordinator: Wenyu Yang, Doctor
- Email: yangwenyu@ihcams.ac.cn
- Phone: +86 13821596186
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.