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AzTrials

AMLEarly Phase 1NCT06499025

Treatment of Relapsed or Refractory t(8; 21) AML With Targeted AML1-ETO Neoantigen Cytotoxic T Cells (CTL)

A Early Phase 1 study testing targeted AML1-ETO neoantigen cytotoxic T cells (CTL), Cyclophosphamide injection and Decitabine Injection in people with AML and t(8;21), run by BGI, China at 1 site in China.

Enrolling by invitation

Enrolling by invitation. The study team selects and invites participants, usually from an existing group of patients.

Sites and contacts
Official record
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Trial summary

1. To evaluate the safety and tolerability of targeted AML1-ETO neoantigen cytotoxic T cells (CTL) in the treatment of relapsed or refractory acute myeloid leukemia . 2. To evaluate the effectiveness of targeted AML1-ETO neoantigen cytotoxic T cells (CTL)，by the complete response rate(CRR) and overall survival (OS)… Read more about this study

Who is it for?
Ages 18 to 75 years
Conditions the study is for
AML, t(8;21), Neoantigen
What is being tested?
targeted AML1-ETO neoantigen cytotoxic T cells (CTL), Cyclophosphamide injection and Decitabine Injection
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
16 participants
Where
1 siteChina
Study dates
1 Feb 2024to 30 Dec 2027
Phase
Early Phase 1
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 75 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Age 18-75 years old (including 18, 75 years old), gender is not limited;
  • According to WHO (2020) criteria, the subjects are diagnosed for recurrent or refractory t(8:21) acute myeloid leukemia or demonstrated persistant AML1-ETO positiving or genetic MRD recurrence after ≥3 cycles of intensive chemotherapy, as confirmed by quantitative PCR;
  • The subjects voluntarily participate in the study and sign the Informed Consent Form by themselves or their legal guardians;
  • The HLA types of subjects are HLA-A* 11:01 or HLA-A*02:01;
  • Possessing the AML1-ETO(RUNX1-RUNX1T1) funsion gene;
  • 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 ;
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  • No contraindications for collection of mononuclear cells from peripheral blood ;
  • ECOG score ≤1;
  • The survival time is exspected to be≥ 3 months;
  • Have the ability to understand and be willing to sign the informed consent for this test.

Who cannot join

  • Tumor cells do not express AML1-ETO neoantigen;
  • Active infection;
  • Abnormal liver function [TBil（total bilirubin)>1.5×ULN, ALT>2.5×ULN], abnormal kidney function [Scr（serum creatinine)>1.5×ULN];
  • Unstable angina or 3/4 class of congestive heart failure according to New York Heart Association, or multiple organ dysfunction;
  • HIV/AIDS patients;
  • Participants who need treatment of long-term anticoagulation (warfarin or heparin) or antiplatelet(aspirin>300mg/d; Clopidogrel>75mg/d) ;
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  • Participants who received radiotherapy within 4 weeks ,prior to study initiation (blood collection);
  • Known or suspected drug abuse or alcohol dependence;
  • 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;
  • Participants in other clinical trials within 30 days;
  • 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;
  • The investigator evaluates that the subject is unable or unwilling to comply with the requirements of the study protocol;

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

China1 site

  • Shenzhen University General Hospital

    Shenzhen, Guangdong

Other trials in China

About this study

Official title: 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

  1. To evaluate the safety and tolerability of targeted AML1-ETO neoantigen cytotoxic T cells (CTL) in the treatment of relapsed or refractory acute myeloid leukemia .
  2. To evaluate the effectiveness of targeted AML1-ETO neoantigen cytotoxic T cells (CTL)，by the complete response rate(CRR) and overall survival (OS) followed.
Read the detailed description

This is a single arm、open label and non-randomied clinical trial ，divided into dose exploration phase (Part A) and dose extension phase (Part B).

Part A: Plan to enroll six subjects to evaluate the safety and tolerabilty of targeted neoantigen cytotoxic T cells (CTL),determine dose-limiting toxicity(DLT),explore the maximum tolerated dose (MTD) or the recommended dose for later clilnical studies.The DLT observation period is 28 days after the infusion of targeted neoantigen cytotoxic T cells (CTL) iniection. One dose group(total number of cells is 5×10^7/bag) and the another one (total number of cells is 10×10^7 /bag )is setted by the 3+3 test design.

Part B: Ten subjects are planned to be enrolled in the dose-exploration phase with the recommended dose, to further evaluate the safety、tolerability and its efficacy of targeted neoantigen cytotoxic T cells (CTL) in relapsed or refractory acute myeloid leukemia.

Conditions
AML, t(8;21), Neoantigen
Being tested
targeted AML1-ETO neoantigen cytotoxic T cells (CTL), Cyclophosphamide injection, Decitabine Injection, Liposome mitoxantrone

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • targeted AML1-ETO neoantigen cytotoxic T cells (CTL)Biological

    After subject screening, peripheral blood mononuclear cell #PBMC# donors matching half or more of the subject's HLA matching will undergo blood collection to prepare neoantigen cytotoxic T cells. Neoantigen cytotoxic T cells preparation is expected to be 25-30 days after blood collection. In this study, the bridging therapy will be allowed before Chemotherapy preconditioning. Chemotherapy preconditioning will be performed before neoantigen cytotoxic T cells transfusion.

  • Cyclophosphamide injectionDrug

    Cyclophosphamide injection will be performed in -10 to -8d before neoantigen cytotoxic T cells transfusion

  • Decitabine InjectionDrug

    Decitabine Injection will be performed in -12 to -8d before neoantigen cytotoxic T cells transfusion

  • Liposome mitoxantroneDrug

    Liposome mitoxantrone will be performed in -9 to -8d before neoantigen cytotoxic T cells transfusion

Study groups in NCT06499025
Study groupRoleWhat they receive
targeted AML1-ETO neoantigen cytotoxic T cells (CTL)ExperimentalThe escalating doses of cells (CTL) in this study will be 5*10^7 cells and 1*10^8 cells.
Receives: Biological: targeted AML1-ETO neoantigen cytotoxic T cells (CTL); Drug: Cyclophosphamide injection; Drug: Decitabine Injection; Drug: Liposome mitoxantrone

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Safety assessment (Evaluation of treatment-related adverse events according to CTCAEv5.0)

    To determine the incidence of AE and SAE in clinical trials

    Measured: From preconditioning or cell reinfusion to one year after cell reinfusion or the initiation of other antitumor therapy or the discontinuation of the trial for other reasons, whichever occurred first.

Secondary outcomes (2)

  1. Complete response rate

    The total number of subjects with complete response (CR) confirmed and the proportion of subjects in the corresponding analysis set

    Measured: Up to 48 weeks

  2. Overall survival (OS)

    The time from cell transfusion to death who have completed the prescribed dose. If the sujects is lost to follow-up, the last known date of surival wil be OS deletion time

    Measured: Up to 72 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted12 Jul 2024
  2. Study start1 Feb 2024
  3. Primary completion30 Dec 2026 (estimated)
  4. Study completion30 Dec 2027 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Early Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
16 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06499025
Sponsor's protocol ID
BGI-007
Other IDs
KYLLHS-20230402A
Status last verified
Aug 2025
First submitted
7 Jul 2024
Last updated
28 Aug 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • Godugu K, Rajabi M, Mousa SA. RETRACTED: Godugu et al. Anti-Cancer Activities of Thyrointegrin alphavbeta3 Antagonist Mono- and Bis-Triazole Tetraiodothyroacetic Acid Conjugated via Polyethylene Glycols in Glioblastoma. Cancers 2021, 13, 2780. Cancers (Basel). 2024 May 15;16(10):1880. doi: 10.3390/cancers16101880.

    PubMed 38792024

  • Falini B, Mecucci C, Tiacci E, Alcalay M, Rosati R, Pasqualucci L, La Starza R, Diverio D, Colombo E, Santucci A, Bigerna B, Pacini R, Pucciarini A, Liso A, Vignetti M, Fazi P, Meani N, Pettirossi V, Saglio G, Mandelli F, Lo-Coco F, Pelicci PG, Martelli MF; GIMEMA Acute Leukemia Working Party. Cytoplasmic nucleophosmin in acute myelogenous leukemia with a normal karyotype. N Engl J Med. 2005 Jan 20;352(3):254-66. doi: 10.1056/NEJMoa041974.

    PubMed 15659725

  • Biernacki MA, Foster KA, Woodward KB, Coon ME, Cummings C, Cunningham TM, Dossa RG, Brault M, Stokke J, Olsen TM, Gardner K, Estey E, Meshinchi S, Rongvaux A, Bleakley M. CBFB-MYH11 fusion neoantigen enables T cell recognition and killing of acute myeloid leukemia. J Clin Invest. 2020 Oct 1;130(10):5127-5141. doi: 10.1172/JCI137723.

    PubMed 32831296

  • Blankenstein T, Leisegang M, Uckert W, Schreiber H. Targeting cancer-specific mutations by T cell receptor gene therapy. Curr Opin Immunol. 2015 Apr;33:112-9. doi: 10.1016/j.coi.2015.02.005. Epub 2015 Feb 27.

    PubMed 25728991

  • Sahin U, Tureci O. Personalized vaccines for cancer immunotherapy. Science. 2018 Mar 23;359(6382):1355-1360. doi: 10.1126/science.aar7112.

    PubMed 29567706

  • van der Lee DI, Reijmers RM, Honders MW, Hagedoorn RS, de Jong RC, Kester MG, van der Steen DM, de Ru AH, Kweekel C, Bijen HM, Jedema I, Veelken H, van Veelen PA, Heemskerk MH, Falkenburg JHF, Griffioen M. Mutated nucleophosmin 1 as immunotherapy target in acute myeloid leukemia. J Clin Invest. 2019 Feb 1;129(2):774-785. doi: 10.1172/JCI97482. Epub 2019 Jan 14.

    PubMed 30640174

Questions about this trial

What is this clinical trial studying?

A Early Phase 1 study testing targeted AML1-ETO neoantigen cytotoxic T cells (CTL), Cyclophosphamide injection and Decitabine Injection in people with AML and t(8;21), run by BGI, China at 1 site in China.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 10 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: enrolling by invitation, as of the sponsor's last update on 28 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Safety assessment (Evaluation of treatment-related adverse events according to CTCAEv5.0) (time frame: From preconditioning or cell reinfusion to one year after cell reinfusion or the initiation of other antitumor therapy or the discontinuation of the trial for other reasons, whichever occurred first.). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06499025, which also lists 6 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Aug 2025.

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