Fungal DiseaseNCT07357038
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
An observational study of Fungal Disease, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
Trial 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… Read more about this study
- Who is it for?
- Ages 1 month to 18 years
- Conditions the study is for
- Fungal Disease
- What is being tested?
- Patients received the Drug: liposomal amphotericin B（AmBisome） for treatment
- How many people
- 38 participants
- Where
- 1 siteChina
- Study dates
- 30 Sep 2025to 31 May 2027
- How long will I take part?
- 12 Weeks
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 month to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: breakthrough IFD in children and adolescent patients with hematological malignancies receiving triazoles or echinocandins prophylaxis
Who may be able to join
- 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.
Who cannot join
- (a) Received AmB formulation for prophylaxis or treatment within the past 30 days.
- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
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
wenyu Yang, +8613821596186, yangwenyu@ihcams.ac.cn
Yang
About this study
Official title: 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
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:
Read more
- 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)
- Whether L-AmB may have better renal safety in Chinese children and adolescent than in adults (compared with historical data).
Chinese children and adolescent with hematological malignancies will taking L-AmB as part of their regular medical care for br-IFD. The efficacy and safety data will be recorded.
Read the detailed description
This is a single center, prospective, single-arm and observational study. Patients with breakthrough IFD in children and adolescent patients with hematological malignancies receiving triazoles or echinocandins prophylaxis who meet the inclusion and exclusion criteria receive liposomal amphotericin B (L-AmB)(AmBisome) for antifungal therapy. The efficacy and safety will be observed.
The response rate is assumed to be 67%, a sample size of 38 will produce a two-sided 95% confi dence interval for response rate with a width 0.3 (30%). With a 10% dropout consideration, 43 patients will need to be enrolled.
The research objective of this study is to: (a) Evaluate the efficacy and safety of L-AmB for the treatment of br-IFD in Chinese children and adolescent patients with hematological malignancies receiving triazoles/echinocandins prophylaxis.
(b) Identify baseline characteristic that may be associated with better response rates treated with L-AmB.
These research objectives will contribute to a comprehensive understanding of the roles of L-AmB in the treatment of breakthrough IFD in children and adolescent patients, and provide scientific evidence for clinical practice.
- Conditions
- Fungal Disease
- Being tested
- Patients received the Drug: liposomal amphotericin B（AmBisome） for treatment
- Keywords
- malignant hematological diseases, breakthrough, invasive fungal disease, liposomal amphotericin B
- Medical subject headings
- Mycoses, Invasive Fungal Infections
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Patients received the Drug: liposomal amphotericin B（AmBisome） for treatmentDrug
- This is a single center, prospective ,single-arm and observational study.
- 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
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: Drug: Patients received the Drug: liposomal amphotericin B（AmBisome） for treatment |
What is being measured
The primary outcome is the main question the study is designed to answer.
The favorable response rate .
Measured: 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
Monitor and record any adverse reactions that occur during treatment, with adverse reaction names and grades recorded and evaluated according to NCI-CTCAE V 5.0(National Cancer Institute - Common Terminology Criteria for Adverse Events Version 5.0), including but not limited to the following indicators: clinical symptoms such as infusion reactions; Laboratory tests such as nephrotoxicity related indicators (creatinine, glomerular filtration rate), electrolytes (hypokalemia, hypomagnesemia), hepatotoxicity related indicators (alanine aminotransferase, aspartate aminotransferase, presidential bilirubin, direct bilirubin, indirect bilirubin), electrocardiogram, cardiac enzymes, etc
Measured: Through study completion, an average of 14 days.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Jan 2026
- Study start30 Sep 2025
- Primary completion28 Feb 2027 (estimated)
- Study completion31 May 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fungal DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 38 participants (estimated)
- Target duration
- 12 Weeks
Sponsor and investigators
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, China
- Sponsor type
- University, hospital or other
- Collaborators
- Gilead Sciences
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07357038
- Sponsor's protocol ID
- QTJC2024077
- Status last verified
- Sep 2025
- First submitted
- 23 Sep 2025
- Last updated
- 21 Jan 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT07357038
- Acronym / sponsor ID
- QTJC2024077
- Conditions
- Fungal Disease
- Keywords
- malignant hematological diseases, breakthrough, invasive fungal disease, liposomal amphotericin B
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 38 participants (estimated)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Collaborators
- Gilead Sciences
- Study start
- 30 Sep 2025 (actual)
- Primary completion
- 28 Feb 2027 (estimated)
- Study completion
- 31 May 2027 (estimated)
- First posted
- 21 Jan 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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:
- 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)
- Whether L-AmB may have better renal safety in Chinese children and adolescent than in adults (compared with historical data).
Chinese children and adolescent with hematological malignancies will taking L-AmB as part of their regular medical care for br-IFD. The efficacy and safety data will be recorded.
Detailed description
This is a single center, prospective, single-arm and observational study. Patients with breakthrough IFD in children and adolescent patients with hematological malignancies receiving triazoles or echinocandins prophylaxis who meet the inclusion and exclusion criteria receive liposomal amphotericin B (L-AmB)(AmBisome) for antifungal therapy. The efficacy and safety will be observed.
The response rate is assumed to be 67%, a sample size of 38 will produce a two-sided 95% confi dence interval for response rate with a width 0.3 (30%). With a 10% dropout consideration, 43 patients will need to be enrolled.
The research objective of this study is to: (a) Evaluate the efficacy and safety of L-AmB for the treatment of br-IFD in Chinese children and adolescent patients with hematological malignancies receiving triazoles/echinocandins prophylaxis.
(b) Identify baseline characteristic that may be associated with better response rates treated with L-AmB.
These research objectives will contribute to a comprehensive understanding of the roles of L-AmB in the treatment of breakthrough IFD in children and adolescent patients, and provide scientific evidence for clinical practice.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 12 Weeks
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| children and adolescent patients with hematological malignancies who have breakthrough IFD | – | Drug: Patients received the Drug: liposomal amphotericin B（AmBisome） for treatmentchildren and adolescent patients with hematological malignancies who have breakthrough IFD after receiving triazoles or echinocandins prophylaxis |
Who may qualify
- Ages
- 1 month to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- breakthrough IFD in children and adolescent patients with hematological malignancies receiving triazoles or echinocandins prophylaxis
Key inclusion criteria (8 listed)
- The children and adolescent patients with hematological malignancy received echinocandin/triazole for antifungal prophylaxis and diagnosed with br-IFD.
Key exclusion criteria (2 listed)
- (a) Received AmB formulation for prophylaxis or treatment within the past 30 days.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Patients received the Drug: liposomal amphotericin B（AmBisome） for treatment1. 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 | Drug | children and adolescent patients with hematological malignancies who have breakthrough IFD |
Primary outcome
The favorable response rate .
Time frame: 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.
1 secondary outcome
Safety data
Time frame: Through study completion, an average of 14 days.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Jan 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Fungal Disease, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 21 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Wenyu Yang, Doctor (+86 13821596186, yangwenyu@ihcams.ac.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The favorable response rate . (time frame: 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.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Jan 2026.