Amphotericin B for Breakthrough Fungal Infections After Triazole Prophylaxis in Hematological Patients

A Multicenter, Prospective, Observational Study to Evaluate the Efficacy and Safety of Amphotericin B in the Treatment of Breakthrough Invasive Fungal Disease in Hematological Patients Receiving Triazole Prophylaxis

The goal of this clinical trial is to learn if amphotericin B works to treat breakthrough invasive fungal disease (IFD) in patients with hematological diseases who have received azole-based prophylaxis. It will also learn about the safety of amphotericin B in this population.

The main questions it aims to answer are:

1. What is the overall response rate (complete remission + partial remission) of amphotericin B in treating breakthrough IFD after azole prophylaxis?
2. What medical problems do participants have when taking amphotericin B?
3. What is the 6-week (42-day) overall survival rate after starting treatment?

This is a single-arm, prospective study. Participants will:

1. Receive intravenous liposomal amphotericin B (3-5 mg/kg daily) for 4-6 weeks.
2. Undergo weekly clinical and laboratory assessments, including serum G/GM tests, microbiology tests, and imaging (CT) at 2, 4, and 6 weeks.
3. Have safety monitoring including liver and kidney function, electrolytes, and ECG.
4. Be followed for treatment response and survival.

Study Overview

• Status: Not yet recruiting
• Conditions: Invasive Fungal Disease
• Intervention / Treatment: Drug: Amphotericin B

• Study Type: Interventional
• Enrollment (Estimated): 15
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Rui Ma; Phone Number: +86 18834948122; Email: marui_pku_ed@163.com

Study Locations

• China
  • Beijing Municipality
    • Beijing, Beijing Municipality, China
      • Peking University People's Hospital
      • Contact: Rui Ma; Phone Number: +86 18834948122; Email: marui_pku_ed@163.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Age ≥ 18 years old.
2. Diagnosed with hematological diseases and receiving triazole prophylaxis.
3. Confirmed or suspected breakthrough invasive fungal disease (IFD) according to EORTC/MSG 2020 criteria.
4. Able to understand and sign the informed consent form voluntarily.

Exclusion Criteria:

1. Severe liver or renal dysfunction (ALT/AST > 3×ULN, Cr > 2×ULN).
2. Known allergy to amphotericin B or any component of the study drug.
3. Pregnant or lactating women.
4. Severe underlying diseases with expected survival < 3 months.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Amphotericin B Treatment	Drug: Amphotericin B; Amphotericin B is a polyene antifungal drug used to treat breakthrough fungal infections. The dosage and duration will be determined based on pathogen results and clinical guidelines.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall Response Rate of Antifungal Therapy at 6 Weeks (42 Days)	The overall response rate will be assessed at 6 weeks (42 days) after the initiation of amphotericin B treatment, evaluated according to the EORTC/MSG 2020 criteria for invasive fungal disease, including complete response, partial response, stable disease, and progressive disease.	6 weeks (42 days) after treatment initiation

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall Survival Rate at 6 Weeks	Proportion of participants alive at 6 weeks (42 days) after initiation of amphotericin B therapy.	6 weeks (42 days) after treatment initiation
Change in serum G test level	Change from baseline in serum (1,3)-β-D-glucan level. Unit of measure: pg/mL.	From enrollment to the end of 6-week treatment
Change in serum GM test level	Change from baseline in serum galactomannan test value. Unit of measure: ug/L.	From enrollment to the end of 6-week treatment
Microbiological clearance	Proportion of participants with negative conversion of baseline positive microbiological evidence (e.g., blood culture, sputum culture, mNGS, or site smear/culture).	From enrollment to the end of 6-week treatment
Change in chest CT findings	Change from baseline in chest CT lesion characteristics (e.g., reduction in size, new lesions, complete resolution).	Baseline to 6 weeks after treatment initiation
Proportion of participants with treatment-emergent adverse events (AEs)	Proportion of participants experiencing any AE, graded according to WHO toxicity criteria (grade 3 or higher AEs leading to drug discontinuation).	From first dose to 30 days after last dose.
Proportion of participants discontinuing amphotericin B due to adverse events	Proportion of participants who permanently stop amphotericin B because of any AE.	During treatment period (up to 6 weeks).
Change in serum alanine aminotransferase (ALT)	Change from baseline in ALT level. Unit of measure: U/L.	Baseline to end of treatment up to 6 weeks (twice weekly).
Change in serum aspartate aminotransferase (AST)	Change from baseline in AST level. Unit of measure: U/L.	Baseline to end of treatment up to 6 weeks (twice weekly).
Change in serum total bilirubin	Change from baseline in total bilirubin level. Unit of measure: μmol/L.	Baseline to end of treatment up to 6 weeks (twice weekly).
Change in serum creatinine	Description: Change from baseline in serum creatinine level. Unit of measure: μmol/L.	Baseline to end of treatment up to 6 weeks (twice weekly).
Change in serum electrolyte levels (potassium, sodium, calcium, magnesium)	Change from baseline in serum levels of potassium, sodium, calcium, and magnesium. Each electrolyte will be reported separately as absolute change (mmol/L) from baseline.	Baseline to end of treatment up to 6 weeks (twice weekly).
Change in electrocardiogram (ECG) findings	Change from baseline in ECG parameters (e.g., QTc interval).	Baseline to end of treatment up to 6 weeks (weekly).

Collaborators and Investigators

• Sponsor: Peking University People's Hospital

Study record dates

Study Major Dates

• Study Start (Estimated): April 1, 2026
• Primary Completion (Estimated): December 1, 2028
• Study Completion (Estimated): December 31, 2028

Study Registration Dates

• First Submitted: April 12, 2026
• First Submitted That Met QC Criteria: April 26, 2026
• First Posted (Actual): April 30, 2026

Study Record Updates

• Last Update Posted (Actual): April 30, 2026
• Last Update Submitted That Met QC Criteria: April 26, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Keywords: Amphotericin B; Invasive fungal disease; Hematological diseases; Breakthrough fungal infection; Triazole prophylaxis
• Additional Relevant MeSH Terms: Infections; Bacterial Infections and Mycoses; Mycoses; Hemic and Lymphatic Diseases; Invasive Fungal Infections; Hematologic Diseases; Organic Chemicals; Macrolides; Lactones; Polyketides; Amphotericin B
• Other Study ID Numbers: PKUPH20260411

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No