AML (Acute Myeloid Leukemia)Early Phase 1NCT07338357
Clinical Study of CLL-1 CAR-T in the Treatment of Children With R/R AML
A Early Phase 1 study testing CAR-T in people with AML (Acute Myeloid Leukemia) and CAR-T, run by First Affiliated Hospital of Guangxi Medical University.
Trial summary
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). Read more about this study
- Who is it for?
- Ages 3 to 18 years
- Conditions the study is for
- AML (Acute Myeloid Leukemia), CAR-T
- What is being tested?
- CAR-T
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 5 Jan 2026to 30 Mar 2026
- Phase
- Early Phase 1
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 3 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Voluntarily sign the ICF and are expected to complete the study's follow-up examinations and procedures.
- Aged 3 to 18 years (inclusive), and body weight ≥10 kg.
- 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.
- Confirmation of CLL-1 expression ≥50% on AML blasts by flow cytometry.
- Recovery from toxicities of prior therapies.
- Karnofsky score (for age ≥16 years) ≥70 or Lansky score (for age <16 years) ≥50 at screening, and an expected survival >3 months.
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- Suitable function of the liver, kidneys, hematological system, lungs and heart is required.
- 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.
- Male subjects with reproductive potential must agree to use barrier contraception or practice complete abstinence until 1 year after the last study treatment.
- Eligible blood cell specimen can be provided.
Who cannot join
- Diagnosis of APL.
- 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.
- Evidence of CNS involvement or cranial nerve pathology.
- Subjects with active infections such as hepatitis B, hepatitis C, etc., are to be excluded.
- Subjects with a history of severe allergies or known allergy to any drug component included in this study are to be excluded.
- 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.
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- Previous organ transplant or planned organ transplant (except for hematopoietic stem cell transplantation).
- Received allo-HSCT within 6 weeks prior to screening.
- Subjects who have recently undergone major surgery or plan to undergo major surgery, excluding diagnostic procedures and biopsies.
- Subjects with severe mental disorders, alcoholism, or drug abuse.
- Subjects who, in the judgment of the investigator, have other conditions that make them unsuitable for enrollment.
About this study
Official title: 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).
Read the 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.
- Conditions
- AML (Acute Myeloid Leukemia), CAR-T
- Being tested
- CAR-T
- Keywords
- R/R AML, CAR-T
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CAR-TBiological
A infusion of CLL-1 CAR-T cells following a lymphodepleting chemotherapy regimen.
| Study group | Role | What they receive |
|---|---|---|
| CAR-T | Experimental | CAR-T therapy Receives: Biological: CAR-T |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Treatment-Related Adverse Events
Proportion of subjects experiencing all kinds of treatment-related AEs.
Measured: Up to 2 years.
Secondary outcomes (6)
Overall Response Rate
Proportion of subjects achieving CR, CRi, morphologic leukemia-free state (MLFS), or PR.
Measured: Up to 2 years.
Duration of Response
Time from first assessment of CR, CRi, MLFS, or PR to first assessment of disease recurrence/progression or death from any cause.
Measured: Up to 2 years.
Relapse-Free Survival
For subjects achieving CR, CRi, or CR MRD-only. Time from achievement of response to relapse or death (any cause).
Measured: Up to 2 years.
Event-Free Survival
For all subjects. Time from cell infusion to treatment failure, relapse, or death (any cause).
Measured: Up to 2 years.
Overall Survival
For all subjects. Time from cell infusion to death from any cause.
Measured: Up to 2 years.
Proportion of CLL-1-Positive Tumor Cells
Changes in the proportion of CLL-1-positive tumor cells after CLL-1 CAR-T infusion.
Measured: Up to 2 years.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jan 2026
- Study start5 Jan 2026 (estimated)
- Primary completion30 Mar 2026 (estimated)
- Study completion30 Mar 2026 (estimated)
Why it stopped: Strategic adjustment.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AML (Acute Myeloid Leukemia)Studies 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
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- First Affiliated Hospital of Guangxi Medical University
- Sponsor type
- University, hospital or other
- Collaborators
- Guangzhou Bio-gene Technology Co., Ltd
- Responsible party
- Principal investigator, Yunyan He, First Affiliated Hospital of Guangxi Medical University
- Investigators
- Yunyan He, PhD, Principal investigator, First Affiliated Hospital of Guangxi Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07338357
- Sponsor's protocol ID
- BG-CT-25-012
- Status last verified
- Aug 2026
- First submitted
- 22 Dec 2025
- Last updated
- 26 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No