Clinical Study of CLL-1 CAR-T in the Treatment of Children With R/R AML

August 24, 2026 updated by: Yunyan He, First Affiliated Hospital of Guangxi Medical University

A Single-Center Clinical Study Evaluating the Safety and Preliminary Efficacy of CLL-1 CAR-T in the Treatment of Children With Relapsed/Refractory Acute Myeloid Leukemia

A study to evaluate the safety and preliminary efficacy of CLL-1-targeted CAR-T cell therapy in children aged 3 to 18 years with relapsed or refractory acute myeloid leukemia (r/r AML).

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Withdrawn

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

It's a single-center, single-arm, investigator-initiated clinical study. After thorough consideration and signing of the informed consent form by the subject and/or their legal guardian(s), subjects meeting the eligibility criteria during the screening period will provide blood samples for CAR-T manufacturing. Following lymphodepleting chemotherapy, CAR-T cell infusion will be administered on D0.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Early Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Voluntarily sign the ICF and are expected to complete the study's follow-up examinations and procedures.
  2. Aged 3 to 18 years (inclusive), and body weight ≥10 kg.
  3. Diagnosis of AML according to the 2016 WHO classification, meeting the diagnostic criteria for relapse and refractoriness as per the "Chinese Guidelines for Diagnosis and Treatment of Relapsed/Refractory Acute Myeloid Leukemia (2017 Edition)", and currently having no clinically relevant treatment options or suitable registered clinical trials available.
  4. Confirmation of CLL-1 expression ≥50% on AML blasts by flow cytometry.
  5. Recovery from toxicities of prior therapies.
  6. Karnofsky score (for age ≥16 years) ≥70 or Lansky score (for age <16 years) ≥50 at screening, and an expected survival >3 months.
  7. Suitable function of the liver, kidneys, hematological system, lungs and heart is required.
  8. Females subjects of childbearing potential must have a negative blood pregnancy test at screening and agree to use effective contraception during the study and within 1 year after the last dose of study drug.
  9. Male subjects with reproductive potential must agree to use barrier contraception or practice complete abstinence until 1 year after the last study treatment.
  10. Eligible blood cell specimen can be provided.

Exclusion Criteria:

  1. Diagnosis of APL.
  2. History of other malignancies within 3 years prior to screening, except for adequately treated carcinoma in situ of the cervix, papillary thyroid carcinoma, basal cell or squamous cell skin cancer, localized prostate cancer treated with radical surgery, and ductal carcinoma in situ treated with radical surgery.
  3. Evidence of CNS involvement or cranial nerve pathology.
  4. Subjects with active infections such as hepatitis B, hepatitis C, etc., are to be excluded.
  5. Subjects with a history of severe allergies or known allergy to any drug component included in this study are to be excluded.
  6. Subjects with severe cardiac diseases, refractory hypertension, active neurological autoimmune or inflammatory diseases, clinically significant active cerebrovascular disease, oncologic emergencies requiring urgent intervention, acute or chronic GVHD, or any uncontrolled infections requiring antibiotic therapy, etc., are unsuitable for enrollment.
  7. Previous organ transplant or planned organ transplant (except for hematopoietic stem cell transplantation).
  8. Received allo-HSCT within 6 weeks prior to screening.
  9. Subjects who have recently undergone major surgery or plan to undergo major surgery, excluding diagnostic procedures and biopsies.
  10. Subjects with severe mental disorders, alcoholism, or drug abuse.
  11. Subjects who, in the judgment of the investigator, have other conditions that make them unsuitable for enrollment.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: CAR-T
CAR-T therapy
Biological: CAR-T
A infusion of CLL-1 CAR-T cells following a lymphodepleting chemotherapy regimen.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Incidence of Treatment-Related Adverse Events
Time Frame: Up to 2 years.
Proportion of subjects experiencing all kinds of treatment-related AEs.
Up to 2 years.

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Overall Response Rate
Time Frame: Up to 2 years.
Proportion of subjects achieving CR, CRi, morphologic leukemia-free state (MLFS), or PR.
Up to 2 years.
Duration of Response
Time Frame: Up to 2 years.
Time from first assessment of CR, CRi, MLFS, or PR to first assessment of disease recurrence/progression or death from any cause.
Up to 2 years.
Relapse-Free Survival
Time Frame: Up to 2 years.
For subjects achieving CR, CRi, or CR MRD-only. Time from achievement of response to relapse or death (any cause).
Up to 2 years.
Event-Free Survival
Time Frame: Up to 2 years.
For all subjects. Time from cell infusion to treatment failure, relapse, or death (any cause).
Up to 2 years.
Overall Survival
Time Frame: Up to 2 years.
For all subjects. Time from cell infusion to death from any cause.
Up to 2 years.
Proportion of CLL-1-Positive Tumor Cells
Time Frame: Up to 2 years.
Changes in the proportion of CLL-1-positive tumor cells after CLL-1 CAR-T infusion.
Up to 2 years.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
First Affiliated Hospital of Guangxi Medical University

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
Guangzhou Bio-gene Technology Co., Ltd

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Principal Investigator: Yunyan He, PhD, First Affiliated Hospital of Guangxi Medical University

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
January 5, 2026

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
March 30, 2026

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
March 30, 2026

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
December 22, 2025

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 5, 2026

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
January 13, 2026

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
August 26, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 24, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
August 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • BG-CT-25-012

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

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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