Universal CAR-T Cells Targeting AML

Last updated: October 10, 2023
Sponsor: Shenzhen Geno-Immune Medical Institute
Overall Status: Active - Recruiting

Phase

1

Condition

Acute Myeloid Leukemia

Leukemia

Platelet Disorders

Treatment

CLL-1, CD33, CD38 and/or CD123-specific universal CAR- T cells

Clinical Study ID

NCT05995041
GIMI-IRB-23004
  • Ages 6-75
  • All Genders

Study Summary

The purpose of this clinical trial is to assess the feasibility, safety and efficacy of universal CAR T-cell products targeting CLL-1, CD33, CD38 and CD123 in patients with relapsed and refractory AML. The study also aims to learn more about the function of the universal CAR T cells and their persistency in AML patients.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. Age older than 6 months.
  2. Confirmed expression of CLL-1, CD123, CD38 and/or CD33 in AML blasts byimmuno-histochemical staining or flow cytometry.
  3. Karnofsky performance status (KPS) score is higher than 80 and life expectancy > 3months.
  4. Adequate bone marrow, liver and renal function as assessed by the following laboratoryrequirements: cardiac ejection fraction ≥ 50%, oxygen saturation ≥ 90%, creatinine ≤ 2.5 × upper limit of normal, aspartate aminotransferase (AST) and alanineaminotransferase (ALT) ≤ 3 × upper limit of normal, total bilirubin ≤ 2.0mg/dL.
  5. Hgb≥80g/L.
  6. No cell separation contraindications.
  7. Abilities to understand and the willingness to provide written informed consent.

Exclusion

Exclusion Criteria:

  1. Sever illness or medical condition, which would not permit the patient to be managedaccording to the protocol, including active uncontrolled infection.
  2. Active bacterial, fungal or viral infection not controlled by adequate treatment.
  3. Known HIV or active hepatitis C virus (HCV) infection.
  4. Pregnant or nursing women may not participate.
  5. Use of glucocorticoid for systemic therapy within one week prior to entering thetrial.
  6. Previous treatment with any gene therapy products.
  7. The bone marrow AML burden (MRD) is above 50%.
  8. Patients, in the opinion of investigators, may not be able to comply with the study.

Study Design

Total Participants: 30
Treatment Group(s): 1
Primary Treatment: CLL-1, CD33, CD38 and/or CD123-specific universal CAR- T cells
Phase: 1
Study Start date:
October 31, 2023
Estimated Completion Date:
December 31, 2026

Study Description

Acute myeloid leukemia (AML) is a malignant disease characterized by the rapid growth of myeloblasts that grow in the bone marrow and interferes with the generation of normal blood cells.Over the past years, several groups have demonstrated that CLL1, CD33, CD38 and CD123 are potential AML targets. CLL-1 (C-type lectin-like molecule-1) is a transmembrane glycoprotein, which is overexpressed in leukemic stem cells but absent in normal hematopoietic stem cells, suggesting that CLL-1 can be a promising target for targeted AML therapy. Although CAR-T cells have shown impressive anti-leukemic effect in B cell disease, CAR-T treatment for AML has proven to be more difficult. One of the reasons is because AML patients often has highly suppressed bone marrow function, and it is often difficult to obtain good quality of T cells for CAR-T preparation. In addition, AML progression can be acute and rapid, which can outpace the CAR-T expansion, and the time-consuming CAR-T manufacture process makes it more difficult to treat AML with autologous source of T cells By using universal type of CAR-T cells, the product can be supplied off-the-shelf without being customized from individual patients. In addition, the immediate availability means that patients with short disease remission time under severe bone marrow suppression may get a chance to be treated with CAR-T cells to achieve disease remission. In addition, those patients who suffer from long-term immunosuppression due to tumor microenvironment or myelosuppressive chemotherapy would have the option of treatment with the universal CAR-T cells.

The purpose of this study is to assess the feasibility, safety and efficacy of several AML-specific universal CAR-T products. Another goal is to learn more about the function of the universal CAR T cells and their persistency in the patients.

Connect with a study center

  • Beijing Jingdu Children's Hospital

    Beijing, Beijing
    China

    Site Not Available

  • Shenzhen Geno-Immune Medical Institute

    Shenzhen, Guangdong 518000
    China

    Active - Recruiting

  • The Second Affiliated Hospital of Hainan Medical University

    Haikou, Hainan 570100
    China

    Site Not Available

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