A Real-world Prospective Observational Study on the Efficacy and Safety of L-AmB(Liposomal Amphotericin B) for br- IFD(Breakthrough Invasive Fungal Disease) in Children and Adolescent Patients With Hematological Malignancies Receiving Triazoles or Echinocandins Prophylaxis

NCT07357038 | Recruiting

• 1 other identifier: QTJC2024077
• Study Type: observational
• Target: 38
• Locations: 1 country
• Sites: 1

Brief Summary

The goal of this observational study is to evaluate the efficacy the favorable response rate and safety of L-AmB(liposomal amphotericin B) for the treatment of br-IFD(breakthrough invasive fungal disease) in Chinese children and adolescent patients with hematological malignancies receiving triazoles/echinocandins antifungal prophylaxis. The main question it aims to answer is:

1. 1.Whether the L-AmB have the same efficacy in the treatment of br-IFD in Chinese children and adolescent with hematological malignancies who are receiving triazoles or echinocandins antifungal prophylaxis as in adults(compared with historical data)
2. 2.Whether L-AmB may have better renal safety in Chinese children and adolescent than in adults (compared with historical data).

Conditions

Fungal Disease

Keywords

malignant hematological diseases; breakthrough; invasive fungal disease; liposomal amphotericin B

Outcome Measures

Primary Outcomes (1)

• The favorable response rate .

  The favorable response rate at the end of L-AmB therapy( an average of 2 weeks). The favorable response rate: proportion of patients achieving complete or partial remission.

Secondary Outcomes (1)

• Safety data

  Through study completion, an average of 14 days.

Study Arms (1)

children and adolescent patients with hematological malignancies who have breakthrough IFD

children and adolescent patients with hematological malignancies who have breakthrough IFD after receiving triazoles or echinocandins prophylaxis

Drug: Patients received the Drug: liposomal amphotericin B（AmBisome） for treatment

Interventions

Patients received the Drug: liposomal amphotericin B（AmBisome） for treatment DRUG

1. This is a single center, prospective ,single-arm and observational study. 2. Patients who meet the inclusion and exclusion criteria receive liposomal amphotericin B (L-AmB)(AmBisome) for antifungal therapy. Regarding the combination therapy: During the research period, it is allowed to combine with other antifungal drugs in combination and accurately record them. Researchers determine it based on the severity of the underlying disease, the recovery of immune suppression, and clinical response. Regarding the sequential therapy: During the research period, it is allowed to accept sequential therapy with other antifungal drugs and accurately record them. Researchers determine it based on the severity of the underlying disease, the recovery of immune suppression, and clinical response. Regarding the surgical treatment: If the patient's condition permits, surgical treatment can be accepted and explained. Dosage: All medication doses are to be administered in accordance with the drug

children and adolescent patients with hematological malignancies who have breakthrough IFD

Eligibility Criteria

• Age: 1 Month - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Probability Sample

Study Population

breakthrough IFD in children and adolescent patients with hematological malignancies receiving triazoles or echinocandins prophylaxis

You may qualify if:

• The children and adolescent patients with hematological malignancy received echinocandin/triazole for antifungal prophylaxis and diagnosed with br-IFD.
• Age: from 1 month to 18 years old.
• Diagnosed patients with hematological malignancy.
• Received echinocandin/triazole for antifungal prophylaxis at least 7 days.
• 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.
• ECOG-PS(Eastern Cooperative Oncology Group Performance Status): 0-2 points.
• There are no organ dysfunction restrictions during the screening period that limit the use of this protocol.
• The guardian understands the research and signs written informed consent form.

You may not qualify if:

• (a) Received AmB formulation for prophylaxis or treatment within the past 30 days.

Sponsors & Collaborators

• Institute of Hematology & Blood Diseases Hospital, China: lead
• Gilead Sciences: collaborator

Study Sites (1)

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

MeSH Terms

Conditions

Mycoses; Invasive Fungal Infections

Interventions

Therapeutics

Condition Hierarchy (Ancestors)

Bacterial Infections and Mycoses; Infections

Central Study Contacts

Wenyu Yang, Doctor

CONTACT

+86 13821596186; yangwenyu@ihcams.ac.cn

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 12 Weeks
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 23, 2025
• First Posted: January 21, 2026
• Study Start: September 30, 2025
• Primary Completion (Estimated): February 28, 2027
• Study Completion (Estimated): May 31, 2027
• Last Updated: January 21, 2026

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)