About this trial
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).
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.
Disqualifiers
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.
Trial design
Single group
Treatments tested in this trial
CAR-T
Biological/VaccineA infusion of CLL-1 CAR-T cells following a lymphodepleting chemotherapy regimen.
Treatment groups
Trial outcomes
Primary outcomes
Incidence of Treatment-Related Adverse Events
Proportion of subjects experiencing all kinds of treatment-related AEs.
Secondary outcomes
Overall Response Rate
Proportion of subjects achieving CR, CRi, morphologic leukemia-free state (MLFS), or PR.
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.
Relapse-Free Survival
For subjects achieving CR, CRi, or CR MRD-only. Time from achievement of response to relapse or death (any cause).
Event-Free Survival
For all subjects. Time from cell infusion to treatment failure, relapse, or death (any cause).
Locations
Sponsors and contacts
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First Affiliated Hospital of Guangxi Medical University
Lead sponsor
Guangzhou Bio-gene Technology Co., Ltd
Collaborator
This trial is not recruiting at the moment. You can still explore other options: