CAR-T cell therapy for relapsed and refractory acute myeloid leukemia

An Open-label, Single-arm Clinical Study of EX02 CAR-T Therapy for Relapsed and Refractory Acute Myeloid Leukemia

This study is testing a new CAR-T cell therapy to see if it can help people with relapsed and hard-to-treat acute myeloid leukemia feel better and improve their chances of recovery.

Quick facts

• Phase: Early Phase 1
• Study type: Interventional
• Enrollment: 9 (estimated)
• Ages: 18 Years to 70 Years
• Sex: All
• Sponsor: The Second Hospital of Anhui Medical University (Academic / other)
• Drugs / interventions: chemotherapy, prednisone, CAR-T, CAR T, cyclophosphamide, fludarabine
• Locations: 1 site (Hefei, Anhui)
• Trial ID: NCT06642025 on ClinicalTrials.gov

What this trial studies

This early Phase 1 clinical study evaluates the safety and efficacy of EX02 CAR-T cell therapy in patients with relapsed and refractory acute myeloid leukemia (AML). Participants will undergo leukapheresis to collect their T cells, followed by conditioning chemotherapy with cyclophosphamide and fludarabine, before receiving an intravenous infusion of CAR-T cells. The study includes screening, treatment, and long-term follow-up to assess patient outcomes and response to therapy.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 to 70 with relapsed or refractory acute myeloid leukemia who meet specific diagnostic and health criteria.

Not a fit: Patients with acute myeloid leukemia who do not express the EX02 surface marker or have other significant health issues may not benefit from this study.

Why it matters

Potential benefit: If successful, this therapy could provide a new treatment option for patients with difficult-to-treat forms of acute myeloid leukemia.

How similar studies have performed: Other studies using CAR-T cell therapy for hematological malignancies have shown promising results, indicating potential for success in this approach.

Eligibility criteria

Inclusion Criteria:

1. According to the Chinese Adult Acute Myeloid Leukemia (AML) Diagnosis and Treatment Guidelines (2023 edition/previous edition), patients diagnosed with acute myeloid leukemia (AML) and meeting the diagnostic criteria for refractory AML are eligible, including: initial treatment cases with no response after two courses of standard therapy; relapse within 12 months after achieving complete remission (CR) and failure of consolidation therapy; relapse after 12 months with no response to conventional chemotherapy; two or more relapses; and presence of extramedullary leukemia.
2. Peripheral blood, bone marrow, or extramedullary lesion specimens showing leukemia cells with positive surface expression of EX02;
3. ECOG performance status score of 0-2;
4. Age ≥18 and ≤70 years, any gender;
5. Blood cell examination meeting the following conditions: hemoglobin \> 60g/L, normal T lymphocyte count (CD3+) \> 0.5×10\^9/L, platelet count \> 30×10\^9/L;
6. Negative pregnancy test for women of childbearing potential before the start of the trial, and agreement to use effective contraception measures during the trial until the final follow-up; male participants of reproductive potential agreeing to use effective contraception measures during the trial until the final follow-up;
7. Voluntary participation in this clinical study: after fully understanding the study content, voluntary signing of the informed consent form, and commitment to complete all trial procedures and activities.

Exclusion Criteria:

1. Active hepatitis A, B, C, HIV infection, or other severe active infections that are not yet controlled;
2. History of acquired immune deficiency syndrome (AIDS) or long-term use of immunosuppressants due to other diseases (including steroids, equivalent to prednisone \>15mg/day);
3. Conditions of the heart function: a. New York Heart Association (NYHA) class III or IV heart failure; b. Myocardial infarction or coronary artery bypass surgery within the six months prior to enrollment; c. Clinically significant ventricular arrhythmia or unexplained syncope; d. History of severe non-ischemic cardiomyopathy; e. History of cardiac dysfunction (left ventricular ejection fraction \<45%) within 8 weeks before enrollment;
4. Pregnant or lactating women; participants (both male and female) unwilling to use contraception;
5. Severe liver or kidney dysfunction: aspartate aminotransferase (AST) and alanine aminotransferase (ALT) levels exceeding 3 times the upper limit of normal, bilirubin exceeding 3 times the upper limit of normal; serum creatinine ≥ 178 μmol/L (decompensated phase);
6. History of severe allergic reactions to any drugs planned for use in this study;
7. Recipients of hematopoietic stem cell transplantation must have discontinued immunosuppressants for at least 6 weeks before enrollment and have no signs of graft-versus-host disease;
8. Other conditions deemed unsuitable for participation in this trial by the investigator.

Where this trial is running

Hefei, Anhui

• The Second Affliated Hospital of Anhui Medical University — Hefei, Anhui, China (Recruiting)

Study contacts

• Study coordinator: Zhimin Zhai, MD

Conditions Acute Myeloid Leukemia; CAR-T