Anti-CD33 CAR-T Cells for the Treatment of Relapsed/Refractory CD33+ Acute Myeloid Leukemia

iCell Gene Therapeutics

Status and phase: Not yet enrolling; Phase 1

Conditions: Relapsed and/or Refractory Acute Myeloid Leukemia; High Risk Hematologic Malignancies

Treatments: Biological: anti-CD33 CAR T cells

Study type: Interventional

Funder types: Industry; Other

Identifiers

NCT05445765

ICG165-001

Details and patient eligibility

About

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.

Full description

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.

Enrollment: 10 estimated patients

Sex: All

Ages: 2 to 60 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Signed written informed consent; Patients volunteer to participate in the clinical trial;
2. Diagnosis is mainly based on the World Health Organization (WHO) 2008;
3. Complete remission cannot be achieved after induction therapy; recurrence occurs after completion remission; the burden of leukemic blasts in the peripheral blood or bone marrow is greater than 5%;
4. Leukemic blast cells express CD33 (CD33 positive by flow cytometry or immunohistochemistry ≥70%);
5. The expected survival period is greater than 12 weeks;
6. ECOG score ≤2;
7. Age 2-60 years old;
8. HGB≥70g/L (can be transfused);
9. Total bilirubin does not exceed 3 times the upper limit of normal value, and AST and ALT do not exceed 5 times the upper limit of normal value.

Exclusion criteria

1. Patients declining to consent for treatment
2. Prior solid organ transplantation
3. One of the following cardiac issues: atrial fibrillation; myocardial infarction within the past 12 months; prolonged QT syndrome or secondary QT prolongation; clinically significant pericardial effusion; cardiac insufficiency NYHA (New York Heart Association) III or IV;
4. History of severe pulmonary dysfunction diseases;
5. Severe infection or persistent infection cannot be effectively controlled;
6. Severe autoimmune disease or congenital immunodeficiency;
7. Active hepatitis;
8. Human immunodeficiency virus (HIV) infection;
9. Clinically significant viral infections, or uncontrollable viral reactivation, including EBV (Epstein-Barr virus).

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Sequential Assignment

Masking: None (Open label)

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10 participants in 1 patient group

anti-CD33 CAR T cells

Experimental group

Description:

Dose escalation phase: anti-CD33 CAR T cells will be transduced with a lentiviral vector to express anti-CD33 CARs

Treatment:

Biological: anti-CD33 CAR T cells