Acute Myeloid Leukemia (AML)Early Phase 1NCT06642025
EX02 CAR-T Cells for Relapsed and Refractory Acute Myeloid Leukemia
A Early Phase 1 study testing CAR-T Cell Injection in people with Acute Myeloid Leukemia (AML) and CAR-T, run by Zhimin Zhai at 1 site in China.
Trial summary
This is a early Phase 1 open-label, single-arm clinical study of EX02 CAR-T therapy for relapsed and refractory acute myeloid leukemia. Each participant will undergo leukapheresis after enrolment, receive treatment of the conditioning chemotherapy of cyclophosphamide and fludarabine, and an an intravenous infusion of… Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Acute Myeloid Leukemia (AML), CAR-T
- What is being tested?
- CAR-T Cell Injection
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteChina
- Study dates
- 1 Sep 2024to 1 Sep 2029
- 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 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
- Peripheral blood, bone marrow, or extramedullary lesion specimens showing leukemia cells with positive surface expression of EX02;
- ECOG performance status score of 0-2;
- Age ≥18 and ≤70 years, any gender;
- 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;
- 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;
- 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.
Who cannot join
- Active hepatitis A, B, C, HIV infection, or other severe active infections that are not yet controlled;
- History of acquired immune deficiency syndrome (AIDS) or long-term use of immunosuppressants due to other diseases (including steroids, equivalent to prednisone >15mg/day);
- 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;
- Pregnant or lactating women; participants (both male and female) unwilling to use contraception;
- 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);
- History of severe allergic reactions to any drugs planned for use in this study;
- 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;
- Other conditions deemed unsuitable for participation in this trial by the investigator.
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, 1 recruiting
- Recruiting
The Second Affliated Hospital of Anhui Medical University
Hefei, Anhui, 230031
Zhimin Zhai, +86-0551-63869571, zzzm889@163.com
About this study
Official title: An Open-label, Single-arm Clinical Study of EX02 CAR-T Therapy for Relapsed and Refractory Acute Myeloid Leukemia
This is a early Phase 1 open-label, single-arm clinical study of EX02 CAR-T therapy for relapsed and refractory acute myeloid leukemia.
Each participant will undergo leukapheresis after enrolment, receive treatment of the conditioning chemotherapy of cyclophosphamide and fludarabine, and an an intravenous infusion of CAR-T cells.
Each participant will proceed through the following study procedures:
- Screening
- Enrollment/Leukapheresis
- Conditioning chemotherapy
- CAR T treatment
- Post-treatment assessment
- Long-term follow-up
- Conditions
- Acute Myeloid Leukemia (AML), CAR-T
- Being tested
- CAR-T Cell Injection
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CAR-T Cell InjectionBiological
Conditioning chemotherapy: Lymphodepletion regimen consisting of fludarabine 25 mg/m2/day and cyclophosphamide 250 mg/m2/day for 3 consecutive days, administered 48 hours before intraperitoneal infusion.
On the day of administration, premedication with 500mg of acetaminophen orally and 20mg of promethazine intramuscularly or intravenously (or other non-steroidal anti-inflammatory drugs and antihistamines) should be given 30 minutes before intraperitoneal infusion.
The therapeutic dose of CAR-T cells is defined as intravenous injection of 1.0E6 CAR-T cells/kg as the initial standard dosage.
| Study group | Role | What they receive |
|---|---|---|
| CAR-T Cell Injection | Experimental | Not described Receives: Biological: CAR-T Cell Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Treatment-Emergent Adverse Events (TEAE)
TEAE is defined as any adverse event that occurs within 90 days after the first injection of EX02 CAR-T, or that existed before treatment and worsened after treatment. All adverse events are graded according to CTCAE v4.0, classified, and then the incidence rate is calculated. In addition, according to the definition of this study, the occurrence of CAR-T cell therapy-specific CRS and ICANS, as well as their grading, management, and prognosis are evaluated, and the incidence rates are calculated separately, with specific patient details recorded.
Measured: From the infusion (Day 0) to 3 months
Overall Response Rate (ORR)
Overall response rate=number of complete remission(CR) (including incomplete hematologic recovery (CRi)) + number of partial remission (PR) / total number of treated cases, determined by the investigator according to the 2023 Chinese Diagnosis and Treatment Guidelines for Adult Acute Myeloid Leukemia (non-acute promyelocytic leukemia).
Measured: From the infusion (Day 0) to Day 60
Secondary outcomes (3)
PFS
The time from the onset of leukocyte apheresis to the appearance of tumor progression or death
Measured: 12 months
OS
OS, The time between leukocyte apheresis and death from any cause
Measured: 12 months
ECOG scoring
Zubrod-ECOG-WHO method for assessing and comparing the physical status of patients before and after treatment
Measured: 2 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Oct 2024
- Study start1 Sep 2024
- Primary completion1 Aug 2026 (estimated)
- Study completion1 Sep 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid Leukemia (AML)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- 9 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Zhimin Zhai
- Sponsor type
- University, hospital or other
- Collaborators
- Zeno Therapeutics Pte. Ltd
- Responsible party
- Sponsor-investigator, Zhimin Zhai, The Second Hospital of Anhui Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06642025
- Sponsor's protocol ID
- TXB2024-001(F1)
- Status last verified
- Oct 2024
- First submitted
- 12 Oct 2024
- Last updated
- 15 Oct 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Single-arm Clinical Study of EX02 CAR-T Therapy for Relapsed and Refractory Acute Myeloid Leukemia
- ClinicalTrials.gov ID
- NCT06642025
- Acronym / sponsor ID
- TXB2024-001(F1)
- Conditions
- Acute Myeloid Leukemia (AML), CAR-T
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 9 participants (estimated)
- Lead sponsor
- Zhimin ZhaiUniversity, hospital or other
- Collaborators
- Zeno Therapeutics Pte. Ltd
- Study start
- 1 Sep 2024 (actual)
- Primary completion
- 1 Aug 2026 (estimated)
- Study completion
- 1 Sep 2029 (estimated)
- First posted
- 15 Oct 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a early Phase 1 open-label, single-arm clinical study of EX02 CAR-T therapy for relapsed and refractory acute myeloid leukemia.
Each participant will undergo leukapheresis after enrolment, receive treatment of the conditioning chemotherapy of cyclophosphamide and fludarabine, and an an intravenous infusion of CAR-T cells.
Each participant will proceed through the following study procedures:
- Screening
- Enrollment/Leukapheresis
- Conditioning chemotherapy
- CAR T treatment
- Post-treatment assessment
- Long-term follow-up
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CAR-T Cell Injection | Experimental | Biological: CAR-T Cell Injection |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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.
- Peripheral blood, bone marrow, or extramedullary lesion specimens showing leukemia cells with positive surface expression of EX02;
- ECOG performance status score of 0-2;
- Age ≥18 and ≤70 years, any gender;
- 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;
Key exclusion criteria (8 listed)
- Active hepatitis A, B, C, HIV infection, or other severe active infections that are not yet controlled;
- History of acquired immune deficiency syndrome (AIDS) or long-term use of immunosuppressants due to other diseases (including steroids, equivalent to prednisone >15mg/day);
- 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;
- Pregnant or lactating women; participants (both male and female) unwilling to use contraception;
- 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);
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CAR-T Cell InjectionConditioning chemotherapy: Lymphodepletion regimen consisting of fludarabine 25 mg/m2/day and cyclophosphamide 250 mg/m2/day for 3 consecutive days, administered 48 hours before intraperitoneal infusion. On the day of administration, premedication with 500mg of acetaminophen orally and 20mg of promethazine intramuscularly or intravenously (or other non-steroidal anti-inflammatory drugs and antihistamines) should be given 30 minutes before intraperitoneal infusion. The therapeutic dose of CAR-T cells is defined as intravenous injection of 1.0E6 CAR-T cells/kg as the initial standard dosage. | Biological | CAR-T Cell Injection |
Primary outcome
Treatment-Emergent Adverse Events (TEAE)
Time frame: From the infusion (Day 0) to 3 months
TEAE is defined as any adverse event that occurs within 90 days after the first injection of EX02 CAR-T, or that existed before treatment and worsened after treatment. All adverse events are graded according to CTCAE v4.0, classified, and then the incidence rate is calculated. In addition, according to the definition of this study, the occurrence of CAR-T cell therapy-specific CRS and ICANS, as well as their grading, management, and prognosis are evaluated, and the incidence rates are calculated separately, with specific patient details recorded.
Overall Response Rate (ORR)
Time frame: From the infusion (Day 0) to Day 60
Overall response rate=number of complete remission(CR) (including incomplete hematologic recovery (CRi)) + number of partial remission (PR) / total number of treated cases, determined by the investigator according to the 2023 Chinese Diagnosis and Treatment Guidelines for Adult Acute Myeloid Leukemia (non-acute promyelocytic leukemia).
3 secondary outcomes
PFS
Time frame: 12 months
OS
Time frame: 12 months
ECOG scoring
Time frame: 2 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Oct 2024.
- Individual participant dataSharing planned: No
Related trials for Acute Myeloid Leukemia (AML)
RecruitingPhase 1
A Study of CHM-029 in Participants With NPM1 Mutated, KMT2A or NUP98 Rearranged AML
AML (Acute Myeloid Leukemia)
RecruitingPhase 2
Testing New Therapies for Patients With Acute Myeloid Leukemia Who Are Newly Diagnosed and Have Not Yet Started Treatment (A MyeloMATCH Treatment Trial)
Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic/Myeloproliferative Neoplasm
Active, not recruitingPhase 2
Clofarabine and Melphalan Before Donor Stem Cell Transplant in Treating Patients With Myelodysplasia, Acute Leukemia in Remission, or Chronic Myelomonocytic Leukemia
Adult Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome
RecruitingPhase 1/2
Venetoclax and CLAG-M for the Treatment of Acute Myeloid Leukemia and High-Grade Myeloid Neoplasms
Acute Biphenotypic Leukemia, Acute Myeloid Leukemia
Not yet recruitingPhase 1
ATRN-119 in Combination With Decitabine in Patients With TP53-Mutated AML or Higher-Risk MDS
Acute Myeloid Leukemia, High-risk Myelodysplastic Syndrome
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing CAR-T Cell Injection in people with Acute Myeloid Leukemia (AML) and CAR-T, run by Zhimin Zhai 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 7 inclusion and 8 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: looking for participants now, as of the sponsor's last update on 15 Oct 2024. 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?
Contact Zhimin Zhai, MD (+86-0551-63869571, zzzm889@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Treatment-Emergent Adverse Events (TEAE) (time frame: From the infusion (Day 0) to 3 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 15 Oct 2024.