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).
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
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
10
A infusion of CLL-1 CAR-T cells following a lymphodepleting chemotherapy regimen.
Incidence of Treatment-Related Adverse Events
Proportion of subjects experiencing all kinds of treatment-related AEs.
Time frame: Up to 2 years.
Overall Response Rate
Proportion of subjects achieving CR, CRi, morphologic leukemia-free state (MLFS), or PR.
Time frame: 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.
Time frame: 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).
Time frame: Up to 2 years.
Event-Free Survival
For all subjects. Time from cell infusion to treatment failure, relapse, or death (any cause).
Time frame: Up to 2 years.
Overall Survival
For all subjects. Time from cell infusion to death from any cause.
Time frame: 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.
Time frame: Up to 2 years.
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