Targeted treatment for relapsed or refractory acute myeloid leukemia using neoantigen cytotoxic T cells

A Clinical Study to Evaluate the Safety and Initial Efficacy of Targeted AML1-ETO Neoantigen Cytotoxic T Cells (CTL) in the Treatment of Relapsed or Refractory t(8; 21) AML

This study is testing a new treatment using specially targeted immune cells for people with relapsed or hard-to-treat acute myeloid leukemia to see if it helps them get better.

Quick facts

• Phase: Early Phase 1
• Study type: Interventional
• Enrollment: 16 (estimated)
• Ages: 18 Years to 75 Years
• Sex: All
• Sponsor: BGI, China (Academic / other)
• Locations: 1 site (Shenzhen, Guangdong)
• Trial ID: NCT06499025 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the safety and effectiveness of targeted AML1-ETO neoantigen cytotoxic T cells (CTL) in patients with relapsed or refractory acute myeloid leukemia (AML). It consists of two phases: a dose exploration phase to determine the maximum tolerated dose and a dose extension phase to further assess safety and efficacy. Participants will receive CTL therapy along with other treatments such as Cyclophosphamide, Decitabine, and Liposome mitoxantrone. The study aims to measure complete response rates and overall survival among participants.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18-75 with relapsed or refractory t(8;21) acute myeloid leukemia who have specific HLA types.

Not a fit: Patients who do not have the required HLA types or those with contraindications for mononuclear cell collection may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new targeted therapy option for patients with difficult-to-treat AML.

How similar studies have performed: While this approach is innovative, similar studies using targeted neoantigen therapies have shown promise in other cancers, suggesting potential for success.

Eligibility criteria

Inclusion Criteria:

1. Age 18-75 years old (including 18, 75 years old), gender is not limited;
2. According to WHO (2020) criteria, the subjects are diagnosed for recurrent or refractory t(8:21) acute myeloid leukemia;
3. The subjects voluntarily participate in the study and sign the Informed Consent Form by themselves or their legal guardians;
4. The HLA types of subjects are HLA-A\* 11:01 or HLA-A\*02:01 ;
5. Verified to have AE expression in tumor samples by histopathological examination ;
6. Disease progression after adequate first-line systemic treatment for remission, or disease progression after first-line or above systemic systemic treatment for ≥2 cycles , or without remission (CR or PR) after≥4 cycles of treatment ;
7. No contraindications for collection of mononuclear cells from peripheral blood ;
8. ECOG score ≤1;
9. The survival time is exspected to be≥ 3 months;
10. Have the ability to understand and be willing to sign the informed consent for this test.

Exclusion Criteria:

1. Tumor cells do not express AML1-ETO neoantigen;
2. Active infection;
3. Abnormal liver function \[TBil（total bilirubin)\>1.5×ULN, ALT\>2.5×ULN\], abnormal kidney function \[Scr（serum creatinine)\>1.5×ULN\];
4. Unstable angina or 3/4 class of congestive heart failure according to New York Heart Association, or multiple organ dysfunction;
5. HIV/AIDS patients;
6. Participants who need treatment of long-term anticoagulation (warfarin or heparin) or antiplatelet(aspirin\>300mg/d; Clopidogrel\>75mg/d) ;
7. Participants who received radiotherapy within 4 weeks ,prior to study initiation (blood collection);
8. Known or suspected drug abuse or alcohol dependence;
9. Patients with mental disorders or other medical conditions are unable to obtain informed consent and cooperate to complete the requirements of experimental treatment and examination procedures;
10. Participants in other clinical trials within 30 days;
11. Pregnant or lactating women and male subjects (or their partners) or female subjects who plan to become pregnant during the study period and within 6 months after the end of the study ,and do not wish to use a medically approved effective contraceptive method (such as an IUD or condom) during the study period;
12. The investigator evaluates that the subject is unable or unwilling to comply with the requirements of the study protocol;

Where this trial is running

Shenzhen, Guangdong

• Shenzhen University General Hospital — Shenzhen, Guangdong, China (Recruiting)

Study contacts

• Principal investigator: LiXin Wang, Doctor — Shenzhen University General Hospital
• Study coordinator: LiXin Wang

Conditions AML; t(8; 21); Neoantigen