CAR-T cell therapy targeting CD33 and CD123 for treating acute myeloid leukemia

Sequential CAR-T Cell Infusion Targeting CD33 and CD123 for Refractory/Relapsed Acute Myeloid Leukaemia

This study is testing a new CAR-T cell therapy that targets specific markers in the blood to see if it can help people with acute myeloid leukemia who haven't responded to other treatments.

Quick facts

• Phase: PHASE1; PHASE2
• Study type: Interventional
• Enrollment: 85 (estimated)
• Ages: 6 Years to 90 Years
• Sex: All
• Sponsor: Essen Biotech (other)
• Drugs / interventions: CAR-T, chimeric antigen receptor, immunotherapy, CAR T
• Locations: 1 site (Beijing, Beijing Municipality)
• Trial ID: NCT06420063 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the safety and efficacy of chimeric antigen receptor T cell (CAR-T) immunotherapy targeting CD33 and CD123 in patients with acute myelocytic leukemia (AML). It is an open, single-arm study that aims to assess the feasibility of using CAR-T cells sequentially against these targets in patients who have relapsed or refractory AML. The primary focus is on monitoring adverse effects, including cytokine storm responses, and evaluating disease status post-treatment. The study also seeks to understand the persistence and function of CAR-T cells in the AML patient population.

Who should consider this trial

Good fit: Ideal candidates include patients aged over 6 months with confirmed expression of CD33 or CD123 in their AML cells and a Karnofsky performance status score higher than 80.

Not a fit: Patients with severe uncontrolled medical conditions or those who do not meet the eligibility criteria may not benefit from this study.

Why it matters

Potential benefit: If successful, this therapy could provide a new treatment option for patients with relapsed or refractory acute myelocytic leukemia.

How similar studies have performed: Other studies have shown promising results with CAR-T therapies targeting different antigens, suggesting potential for success with this novel approach.

Eligibility criteria

Inclusion Criteria:

* Subjects with acute myeloid leukemia who voluntarily signed informed consent and met the following criteria:
* 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.
* Hgb≥80g/L.
* No cell separation contraindications.
* Abilities to understand and the willingness to provide written informed consent.

Exclusion Criteria:

* 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.
* Use of glucocorticoid for systemic therapy within one week prior to entering the trial.
* Patients, in the opinion of investigators, may not be able to comply with the study.

Where this trial is running

Beijing, Beijing Municipality

• District One Hospital — Beijing, Beijing Municipality, China (RECRUITING)

Study contacts

• Study coordinator: Rhoda M Smith, Phd

Conditions: AML, Acute Myeloid Leukemia, AML, Adult Recurrent, AML, Adult, aml, cd33, cd123, CAR-T