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Relapsed/Refractory Acute Myeloid Leukemia(AML)Phase 1CT0991NCT06709131

A Clinical Study to Explore the Safety and Efficacy of CT0991 in Relapsed/Refractory Acute Myeloid Leukemia

A Phase 1 study testing CAR-T cells（ chimeric antigen receptor T cells） in people with Relapsed/Refractory Acute Myeloid Leukemia(AML), run by He Huang at 1 site in China.

Suspended

Paused. Enrolment is on hold for now and may resume.

Official record
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Trial summary

A Clinical Study to Investigate the Safety and Efficacy of CT0991 in Patients with Relapsed/Refractory Acute Myeloid Leukemia Read more about this study

Who is it for?
Ages 18 to 75 years
What is being tested?
CAR-T cells（ chimeric antigen receptor T cells）
Could I receive a placebo?
No, everyone receives an active treatment
How many people
24 participants
Where
1 siteChina
Study dates
8 Jan 2025to 17 Aug 2026
Phase
Phase 1
Are there results?
None posted in the registry

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

  • Volunteer to participate in the clinical trial; Fully understand and are informed of this study and sign the informed consent form; Willing to follow and able to complete all trial procedures；
  • Age 18-75 years (inclusive), male or female；
  • Estimated survival > 12 weeks;
  • Patients with relapsed or refractory AML as defined in the Chinese Guidelines for the Diagnosis and Treatment of Relapsed and Refractory Acute Myeloid Leukemia (Version 2023);
  • ECOG score 0-2;
  • Participants should meet the following test results (no ongoing ongoing supportive care): a)Left ventricular ejection fraction (LVEF) > 50%; b)ALT ≤ 2.5 × ULN, AST ≤ 2.5 × ULN, total bilirubin ≤ 2 × ULN; c)Endogenous creatinine clearance ≥ 30 mL/min (creatinine clearance calculated using the Cockcroft-Gault formula); d) Activated partial thromboplastin time (APTT) ≤ 1.5 × ULN and prothrombin time (PT) ≤ 1.5 × ULN.
  • Female participants of childbearing potential must have a negative serum pregnancy test at screening and before receiving cleansing, be willing to use a highly effective and reliable method of contraception for 1 year after receiving trial treatment, and be absolutely prohibited from donating eggs for 1 year after receiving trial treatment during the trial;
  • Male participants willing to use a highly effective and reliable method of contraception for 1 year following trial treatment if sexually active with a woman of childbearing potential. All male participants were absolutely prohibited from donating sperm for 1 year after receiving trial treatment during the trial.

Who cannot join

  • The participant has any serious illness, laboratory abnormality, or psychiatric disorder that may impair the ability to receive or tolerate planned trial treatment; or the investigator judges that the participant 's participation in the clinical trial is not in his/her best interest (e.g., compromised health), or may hinder, limit, or confound protocol-specific assessments;
  • Participants were diagnosed with acute promyelocytic leukemia (APL), BCR-ABL positive leukemia (chronic myeloid leukemia in acute phase), secondary AML (other than MDS), central nervous system leukemia;
  • Participants with a history of epilepsy or other central nervous system disease;
  • Participants who have received autologous stem cell transplantation within 12 weeks or allogeneic stem cell transplantation within 6 months prior to screening;
  • Participant has clinically significant active GVHD or is receiving systemic corticosteroids for GVHD;
  • Participant has a second primary malignancy other than AML that has required treatment or has not been in complete remission within the past 2 years. The following cancers were considered curable, including nonmetastatic basal cell or squamous cell skin cancer, nonmetastatic prostate cancer, breast or cervical carcinoma in situ, and nonmuscle-invasive bladder cancer. Participants receiving ongoing hormonal therapy may be included in this trial at the discretion of the medical monitor in consultation with the investigator;
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  • Participants had positive serology for human immunodeficiency virus (HIV) and syphilis, active hepatitis C virus (HCV) infection, or active hepatitis B virus (HBV) infection. Participants with a history of previously treated hepatitis B or C were allowed to be included in this trial if viral load could not be detected by qPCR and/or nucleic acid testing;
  • Major surgery within 14 days prior to washout or planned major surgery within 28 days after receiving CT0991 infusion. If required, the medical monitor and investigator must discuss to confirm whether the surgery is considered a major surgery before the participant is included in this trial;
  • Received anti-tumor therapy 14 days before Preconditioning, including chemotherapy, molecular targeted therapy;
  • Systemic therapeutic doses of glucocorticoids (defined as ≥ 20 mg prednisone or other equivalent per day) within 14 days prior to cleansing; however topical glucocorticoids such as topical dermal, eye drops, nasal sprays, inhalations, and physiologic replacement therapy doses of glucocorticoids are permitted ;
  • Vaccination with live attenuated vaccine or mRNA vaccine within 4 weeks prior to clear shower;
  • Allergic or intolerant to clear shower drugs and tocilizumab, or allergic to DMSO in cell infusion preparations, or previous history of other serious allergies such as anaphylactic shock;
  • Toxicities caused by previous treatment did not recover to Common Terminology Criteria for Adverse Events (CTCAE) ≤ Grade 1, except alopecia, peripheral neuropathy, and other events that were judged by the investigator to be unlikely to be cumulative toxicities due to clear shower or CT0991 infusion;
  • Pregnant or lactating women.

Trial locations and contacts

1 site in 1 country.

China1 site

  • The first affiliated hospital of medical college of zhejiang university

    Hangzhou, Zhejiang, 310003

Other trials in China

About this study

Official title: A Clinical Study to Investigate the Safety and Efficacy of CT0991 in Patients With Relapsed/Refractory Acute Myeloid Leukemia

A Clinical Study to Investigate the Safety and Efficacy of CT0991 in Patients with Relapsed/Refractory Acute Myeloid Leukemia

Read the detailed description

This is a single-arm, open-label, dose-escalation clinical trial to evaluate the safety, efficacy, and cellular pharmacokinetics of CT0991 in patients with relapsed or refractory acute myeloid leukemia. It is planned to enroll 9-24 participants in this trial.

Being tested
CAR-T cells（ chimeric antigen receptor T cells）
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 cells（ chimeric antigen receptor T cells）Drug

    CAR-T cells（ chimeric antigen receptor T cells）

Study groups in NCT06709131
Study groupRoleWhat they receive
CAR-T cells（ chimeric antigen receptor T cells）ExperimentalCAR-T cells（ chimeric antigen receptor T cells）
Receives: Drug: CAR-T cells（ chimeric antigen receptor T cells）

What is being measured

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

  1. Adverse Events (AE) after CT0991infusion

    An assessment of severity grade will be made according to the National Cancer Institute Common Terminology Criteria

    Measured: 12 months after CT0991 infusion

  2. Dose-limiting toxicity （DLT）

    The DLT is evaluated as the proportion of patients who experienced adverse events related to CT0991 that meet the criteria for DLT events after the first infusion.

    Measured: Up to 28 days after CAR-T cells infusion

  3. MTD and/or dose range

    Evaluate Dose limited toxicity and recommended dosage range after CT0991 infusion

    Measured: Up to 28 days after CAR-T cells infusion

Secondary outcomes (7)

  1. Complete response (CR), and complete response with incomplete hematologic recovery (CRi)

    Performed according to the Technical Guidelines for Clinical Development of New Drugs for Acute Myeloid Leukemia and ELN 2022 Criteria for AML defined of Achieve CR and CRi.

    Measured: 12 months after CT0991 infusion

  2. complete response with partial hematologic recovery (CRh), Morphologic leukemia-free status (MLFS) and partial response (PR)

    Performed according to the Technical Guidelines for Clinical Development of New Drugs for Acute Myeloid Leukemia and ELN 2022 Criteria for AML defined of Achieve CRh, MLFS and PR.

    Measured: 12 months after CT0991 infusion

  3. Duration of response (DOR)

    Participants achieving CR/CRi/CRh will be included in the analysis set for DOR. DOR is defined as the time from the date of confirmed response until the date of disease relapse or death from any cause, whichever occurs first.

    Measured: 12 months after CT0991 infusion

  4. Event-free survival (EFS)

    defined as the time from the date of receiving the infusion to the date of treatment failure (failure to achieve CR/CRh/CRi/MLFS/PR after both efficacy assessments), or relapse (hematologic relapse or extramedullary relapse after CR/CRh/CRi), or death from any cause, whichever occurs first. When an EFS event was "Ineffective Therapy", the primary analysis of EFS was performed on a 1-day basis (ie, time to treatment received as the event). For a more comprehensive assessment, sensitivity analyses could be performed using the actual date of treatment failure, end of treatment, or start of next-line anti-leukemia therapy as the end of EFS for treatment failure, respectively.

    Measured: 12 months after CT0991 infusion

  5. Overall survival (OS)

    defined as the time from the date of receiving the infusion to the date of death from any cause.

    Measured: 12 months after CT0991 infusion

  6. Minimal Residual Disease (MRD) Negative Rate

    tested in all participants who achieved CR/CRh/CRi. MRD negativity was defined as abnormal cells detected by the MFC method accounting for < 0.1% of CD45-positive cells (at least 500,000 CD45-positive cells detected).

    Measured: 12 months after CT0991 infusion

  7. Test Copy number of CAR

    Evaluate cellular pharmacokinetics of CT0991

    Measured: 12 months after CT0991 infusion

Timeline

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

  1. First posted29 Nov 2024
  2. Study start8 Jan 2025
  3. Primary completion17 Aug 2025
  4. Study completion17 Aug 2026 (estimated)

Why it stopped: Product manufacturing process improvement

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
Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
24 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06709131
Sponsor's protocol ID
TXB2024024
Acronym
CT0991
Status last verified
Nov 2025
First submitted
22 Nov 2024
Last updated
24 Nov 2025
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing CAR-T cells（ chimeric antigen receptor T cells） in people with Relapsed/Refractory Acute Myeloid Leukemia(AML), run by He Huang 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 8 inclusion and 14 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: paused, as of the sponsor's last update on 24 Nov 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?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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

Are results available for this study?

Results are not available in the source record.

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?

Adverse Events (AE) after CT0991infusion (time frame: 12 months after CT0991 infusion). The study lists 3 primary outcomes in all. 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 NCT06709131.

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 24 Nov 2025.

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