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

NCT07338357 | Not Yet Recruiting Early Phase 1

• 1 other identifier: BG-CT-25-012
• Study Type: interventional
• Target: 10
• Locations: 0 countries
• Sites: N/A

Brief 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).

Conditions

AML (Acute Myeloid Leukemia); CAR-T

Keywords

R/R AML; CAR-T

Outcome Measures

Primary Outcomes (1)

• Incidence of Treatment-Related Adverse Events

  Proportion of subjects experiencing all kinds of treatment-related AEs.

  Up to 2 years.

Secondary Outcomes (6)

• Overall Response Rate

  Up to 2 years.

• Duration of Response

  Up to 2 years.

• Relapse-Free Survival

  Up to 2 years.

• Event-Free Survival

  Up to 2 years.

• Overall Survival

  Up to 2 years.

Study Arms (1)

CAR-T

EXPERIMENTAL

CAR-T therapy

Biological: CAR-T

Interventions

CAR-T BIOLOGICAL

A infusion of CLL-1 CAR-T cells following a lymphodepleting chemotherapy regimen.

CAR-T

Eligibility Criteria

• Age: 3 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.
• 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.

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• First Affiliated Hospital of Guangxi Medical University: lead
• Guangzhou Bio-gene Technology Co., Ltd: collaborator

MeSH Terms

Conditions

Leukemia, Myeloid, Acute

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Yunyan He, PhD

  First Affiliated Hospital of Guangxi Medical University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Yunyan He, PhD

CONTACT

+86 13607868275; yunyanhe@aliyun.com

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: PhD

Study Record Dates

• First Submitted: December 22, 2025
• First Posted: January 13, 2026
• Study Start: January 5, 2026
• Primary Completion (Estimated): October 31, 2028
• Study Completion (Estimated): October 31, 2028
• Last Updated: January 13, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share