Sequential CAR-T Cells Targeting CD33/CD123 in Patients With Acute Myelocytic Leukemia AML

Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age6-90
SponsorEssen Biotech

About this trial

This is an open, single-arm, clinical study to evaluate the efficacy and safety of chimeric antigen receptor T cell immunotherapy (CAR-T) targeting CD33 or CD123 or both sequentially in the treatment of Acute Myelocytic Leukemia.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Age older than 6 months.

Confirmed expression of CLL-1, CD123 and/or CD33 in blast AML by immuno-histochemical staining or flow cytometry.

Karnofsky performance status (KPS) score is higher than 80 and life expectancy > 3 months.

Adequate bone marrow, liver and renal function as assessed by the following laboratory requirements: cardiac ejection fraction ≥ 50%, oxygen saturation ≥ 90%, creatinine ≤ 2.5 × upper limit of normal, aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 3 × upper limit of normal, total bilirubin ≤ 2.0mg/dL.

Disqualifiers

Severe illness or medical condition, which would not permit the patient to be managed according to the protocol, including active uncontrolled infection.

Active bacterial, fungal or viral infection not controlled by adequate treatment.

Known HIV, hepatitis B virus (HBV) or hepatitis C virus (HCV) infection.

Pregnant or nursing women may not participate.

Trial design

Design model

Single group

Treatments tested in this trial

  • CD123/CD33 CART

    Biological/Vaccine

    The intervention in this clinical trial involves a novel approach using CD22/123-Chimeric Antigen Receptor T (CAR T) cells combined with chemotherapy. The goal is to assess safety and efficacy in patients with specific hematologic malignancies. Treatment Regimen: Patients in the trial will undergo the following regimen: Fludarabine Phosphate (Days -4 to -2): IV administration of fludarabine phosphate over 30 minutes on days -4 to -2. It's part of the preparatory regimen to enhance the body's response to CAR T-cell therapy. Cyclophosphamide (Day -2): IV cyclophosphamide over 60 minutes on day -2. CD33/123-Chimeric Antigen Receptor T Cells (Day 0): IV administration of investigational therapy, CD33/123-CAR T cells, over 10-20 minutes on day 0. Additional Doses: Eligible patients responding well to the initial CD33/123 CAR-T cell infusion without unacceptable side effects and sufficient CAR T cell availability may receive 2 or 3 additional doses.

Treatment groups

85 Participants
are divided into 1 treatment group
Group A: Treatment (CD33/CD123 CAR T cells, chemotherapy)Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Incidence and severity of dose-limiting toxicities (DLTs) following chemotherapy preparative regimen and infusion of CD5/CD7 chimeric antigen receptor (CAR) T cells

Will be recorded and graded according to the Common Terminology Criteria for Adverse Events (CTCAE) version 4.03 at three dose levels until the maximum tolerated dose (MTD) is determined.

Time frame
28 days

Secondary outcomes

1

Rate of successful manufacture and expansion of the CD33/123 chimeric antigen receptor (CAR) T cells

satisfy the targeted dose level and meet the required release specifications outlined in the Certificate of Analysis (COA)

Time frame
10-14 days

Other outcomes

Sponsors and contacts

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