This is a phase I, interventional, single arm, open label, treatment study to evaluate the safety and tolerability of anti-CD33 CAR-T cells in patients with relapsed and/or refractory, high risk hematologic malignancies.
AML is a rapidly progressing blood cancer and treated by high-dose multi-agent chemotherapy potentially followed by hematopoietic stem cell transplantation. Despite such intensive therapies, which are often associated with considerable toxicities and even death, about 60-70% of AML patients still relapse. Furthermore, the five-year survival rate from AML remains at a dismal 27%. AML is composed mostly of CD33+ leukemic blast cells. Therefore, CD33 is a potential good target by CAR T cells.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
10
Anti-CD33 CAR T cells are used to treat patients. Patient will be administered either fresh or thawed CAR T cells by IV injection after receiving lymphodepleting chemotherapy.
Hebei Yanda Lu Daopei Hospital
Langfang, Hebei, China
The number and incidence of adverse events after anti-CD33 CAR infusion.
Determine the toxicity profile of anti-CD33 CAR T cell therapy including the number, incidence, and severity of symptoms such as cytokine release syndromes and neurotoxicity
Time frame: 1 year, particularly the first 3 months after CAR infusion
The disease response to anti-CD33 CAR T cells
The disease response to anti-CD33 CAR T cells is evaluated by bone marrow biopsy and aspirate at 1, 2, 3, and 4 weeks. The proportion of subjects receiving anti-CD33 CAR T infusion to 1) morphological remission (blasts \<5%): 2) flow cytometry analysis was blast negative, and 3) molecular biological remission (if applicable).
Time frame: 4 weeks
Allogeneic hematopoietic stem cell transplantation (HCT)
Allogeneic hematopoietic stem cell transplantation (HCT) is performed after anti-CD33 CAR T treatment. The time after HCT engraftment \[time range: 42 days after HCT ingraftemnt\] is calculated from the day of HCT until the absolute neutrophil count (ANC) is greater than 500 / ul for three consecutive days.
Time frame: 42 days after HCT ingraftment
HCT 100% chymerism time
HCT 100% chymerism time
Time frame: 2 weeks after HCT
Overall survival
The time from the start of anti-CD33 CAR T injection to death is determined as the overall survival
Time frame: 1 year after HCT
Progress-free survival
Progress-free survival is measured from the injection of anti-CD33 CAR T cells until the record of disease progression or death due to any reason, whichever comes first.
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Time frame: one year after HCT
Treatment-related mortality
Treatment-related mortality calculated from one year after HCT.
Time frame: one year after HCT