AzTrials
AzTrials

Leukemia, Myeloid, Acute(AML)Early Phase 1UHKT-CAR123-01NCT06765876

CART123 T Cells in Relapsed or Refractory CD123+ Hematologic Malignancies: A Dose Escalation Phase I Trial

A Early Phase 1 study testing Autologous CAR123 T lymphocytes in people with Leukemia, Myeloid, Acute(AML) and Precursor Cell Lymphoblastic Leukemia-Lymphoma, run by Institute of Hematology and Blood Transfusion, Czech Republic at 1 site in Czechia.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
Search all trials

Trial summary

Adult patients with refractory or relapsed CD123+ hematologic malignancies, including acute myeloid leukemia, myelodysplastic syndrome, acute lymphoblastic leukemia, or blastic plasmocytoid dentritic cell neoplasm will be recruited in the trial. CART123 cells will be manufatured from blood of each patient. Read more about this study

Who is it for?
Ages 18 to 70 years
What is being tested?
Autologous CAR123 T lymphocytes
Could I receive a placebo?
No, everyone receives an active treatment
How many people
18 participants
Where
1 siteCzechia
Study dates
23 Oct 2024to 31 Dec 2028
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

  • Patients with AML, MDS-IB2, BPDCN or ALL positive for CD123 antigen, who meet one of disease specific criteria below:
  • Patients with AML will be eligible if they meet one of the following criteria:
  • Patient with refractory AML defined as failure to achieve CR or CRi after at least 2 cycles of induction chemotherapy or 1 cycle of high dose salvage regimen or 4 cycles of venetoclax with azacytidine OR
  • Second or subsequent relapse of AML OR
  • Relapse after allogeneic HSCT.
  • Patients with ALL will be eligible if they meet one of following criteria:
Show all 20Show fewer
  • disease refractory to or relapsed after CAR-19 cell therapy OR
  • CD19 negative relapse ineligible for treatment with TKI inhibitors and inotuzumab ozogamicin.
  • Patients with BPDCN will be eligible if they meet following criteria:
  • Refractory or relapsing after chemotherapy with or without allogeneic stem cell transplantation.
  • Patients with MDS-IB2 will be eligible if they meet one of following criteria:
  • Disease refractory to at least four cycles of azacytidine or progression on azacytidine-based therapy OR
  • Disease refractory to induction chemotherapy OR
  • Relapse after haematopoietic stem cell transplantation.
  • CD123 expression on malignant cells confirmed by flow cytometry or by immunohistochemistry.
  • Age between 18 and 70 years.
  • Patient has a suitable donor for allogeneic hematopoietic stem cell transplantation. Workup and clearance of the donor must be completed before IMP administration.
  • Patient able to understand and sign informed consent.
  • Women of child-bearing potential: negative pregnancy test at enrolment (PSV) and at Visit 1.
  • Patient for whom there are no standard-of-care treatments available or such treatment options have been exhausted.

Who cannot join

  • Known hypersensitivity to any component of the IMP.
  • Allogeneic HSCT within 3 months prior to IMP administration.
  • Severe, uncontrolled active infection.
  • Life expectancy < 8 weeks.
  • Respiratory insufficiency (need for oxygen therapy).
  • Significant liver impairment: bilirubin > 50 µmol/L, AST or ALT > 4 times normal upper limit.
Show all 15Show fewer
  • Acute kidney injury with serum creatinine > 180 µmol/L, oliguria or need for acute dialysis.
  • Heart failure with LVEF < 50% by echocardiography.
  • Presence of active grade 3 - 4 acute GvHD or severe chronic GvHD.
  • Serious uncontrolled neurological comorbidity.
  • Vaccination with live virus vaccines in the 4 weeks before IMP administration and within 90 days after the IMP dose.
  • Women: pregnancy or breast-feeding.
  • Subjects of fertile age, unless permanent sexual abstinence is their lifestyle choice:
  • female patients of childbearing potential not willing to use a highly effective method of contraception during the study,
  • male patients whose sexual partner(s) are women of childbearing potential who are not willing to use a highly effective method of contraception during the study.

Trial locations and contacts

1 site in 1 country.

Czechia1 site

  • Ustav hematologie a krevni transfuze / Institute of Hematology and Blood Transfusion

    Prague, 12800

Other trials in Czechia

About this study

Official title: Safety and Efficacy of Anti-CD123 Chimeric Antigen Receptor-Modified Autologous T Cells (CART123) in Patients With Relapsed/Refractory CD123+ Hematologic Malignancies: A Dose Escalation, Open-Label, Phase I Study

Adult patients with refractory or relapsed CD123+ hematologic malignancies, including acute myeloid leukemia, myelodysplastic syndrome, acute lymphoblastic leukemia, or blastic plasmocytoid dentritic cell neoplasm will be recruited in the trial. CART123 cells will be manufatured from blood of each patient. During the production of CAR123 cells, patients may receive appropriate bridging therapy. After cells are produced, participants will undergo a single course of lymphodepleting chemotherapy and receive a single dose of CAR123 T cells. The trial will establish the recommended dose for further studies, either the Maximum Tolerated Dose (MTD) or Maximum Feasible Dose (MFD). Patients must be eligible for hematopoietic stem cell transplantation in order to participate in the trial.

Read the detailed description

This is an open-label, single arm study on up to 18 adult subjects with refractory or relapsed CD123+ AML, MDS, ALL or BPDCN. Following lymphodepleting conditioning regimen, the subjects will receive a single dose of autologous CAR123 T lymphocytes supplied by the sponsor´s manufacturing facility.

CART123 dose will be increased in three predefined steps using the accelerated Bayesian optimal interval (BOIN) design in order to establish recommended CART123 dose for further study, which will be either Maximum Tolerated Dose (MTD) or Maximum Feasible Dose (MFD), whichever is reached first.

Alternative dosing schedule will be adopted in case of dose limitation due to insufficient CART123 expansion during IMP manufacture.

Due to concern for potentially prolonged or irreversible hematologic toxicity of CART123, all patients recruited in the study must be eligible for hematopoietic stem cell transplantation (HSCT) and have a donor of allogeneic hematopoietic stem cells identified and cleared by the transplant center. Decision to perform HSCT will be made on a case-by-case basis.

Being tested
Autologous CAR123 T lymphocytes
Keywords
CAR123 T lymphocytes, CART123, CD123+ Hematologic Malignancies, Anti-CD123, Chimeric Antigen Receptor (CAR) T Cells, Autologous T Cells, Hematopoietic Malignancies, Immunotherapy, Personalized Medicine, Biological Therapy, Phase I Clinical Trial, Acute Lymphoblastic Leukemia, in Relapse, Acute Lymphoblastic Leukemia, Refractory, Relapsed Myelodysplastic syndrome, Relapsed Blastic Plasmacytoid Dendritic Cell Neoplasm, Acute Myeloid Leukaemia Recurrent
Medical subject headings
Leukemia, Myeloid, Acute, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Myelodysplastic Syndromes, Blastic Plasmacytoid Dendritic Cell Neoplasm, Hematologic Neoplasms

What participants receive

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

  • Autologous CAR123 T lymphocytesBiological

    Also called CART123, CAR123 T Cells, Autologous T-cell Therapy

    Anti-CD123 Chimeric Antigen Receptor (CAR) T-Cells (CART123)

Study groups in NCT06765876
Study groupRoleWhat they receive
Treatment ArmExperimentalExperimental: Autologous CAR123 T lymphocytes
Receives: Biological: Autologous CAR123 T lymphocytes

What is being measured

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

  1. Safety and tolerability of autologous CART123 cells

    Cumulative incidence of IMP-related adverse events (AEs) graded by ASTCT consensus grading criteria for Cytokine Release Syndrome (CRS) and Immune effector cell-associated neurotoxicity syndrome (ICANS) and by Common Terminology Criteria for Adverse Events (CTCAE) v 5.0 for other AEs

    Measured: 28 Days, Months 24

  2. Rate of hematological recovery

    Rate of hematological recovery, defined as absolute neutrophil count (ANC) > 1x 10^9/L and platelet count > 20x10^9/L without transfusion support. Hematological recovery before and after HSCT will be evaluated separately.

    Measured: 14 Days, 28 Days, 1Year, Months 12, Months 24

  3. A dose of CART123 cells for further study

    Incidence of dose-limiting toxicities (DLTs) and other safety data after IMP administration.

    Measured: 14 Days, 28 Days, 1year, Months 12, Months 24

Secondary outcomes (4)

  1. Morphologic Leukemia Free State (MLFS).

    Efficacy of IMP administration in patients with refractory or relapsed AML, MDS, BPDCN and ALL evaluated by rate of morphologic leukemia-free state (MLFS).

    Measured: at day 14 and 28 after IMP administration.

  2. Complete Remission (CR) rate

    Efficacy of IMP administration in patients with refractory or relapsed AML, MDS, BPDCN and ALL evaluated by Complete Remission (CR) rate.

    Measured: at 28 days and at any later point after IMP administration, before and after HSCT.

  3. Median Overall Survival and Overall Survival

    Efficacy of IMP administration in patients with refractory or relapsed AML, MDS, BPDCN and ALL evaluated by overall survival (OS)

    Measured: at one year after IMP administration.

  4. Feasibility and need for allogeneic hematopoietic cell transplantation after IMP administration

    Proportion of patients who did not recover blood counts within 28 days of IMP administration, proportion of patients who received allogeneic haematopoietic stem cell transplantation, and who engrafted after transplant.

    Measured: within 28 days of IMP administration

Timeline

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

  1. First posted9 Jan 2025
  2. Study start23 Oct 2024
  3. Primary completion31 Dec 2028 (estimated)
  4. Study completion31 Dec 2028 (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
Model description
Experimental: Autologous CAR123 T lymphocytes
Masking
None (open label)
Enrolment
18 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06765876
Sponsor's protocol ID
UHKT-CAR123-01
Other IDs
2022-503165-30-00
Acronym
UHKT-CAR123-01
Status last verified
Sep 2026
First submitted
18 Dec 2024
Last updated
14 Sep 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Early Phase 1 study testing Autologous CAR123 T lymphocytes in people with Leukemia, Myeloid, Acute(AML) and Precursor Cell Lymphoblastic Leukemia-Lymphoma, run by Institute of Hematology and Blood Transfusion, Czech Republic at 1 site in Czechia.

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 20 inclusion and 15 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: under way, not looking for participants, as of the sponsor's last update on 14 Sep 2026. 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 Czechia. 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 and tolerability of autologous CART123 cells (time frame: 28 Days, Months 24). 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 NCT06765876.

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 14 Sep 2026.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Keep searching with a free account

You’ve used your 3 free searches for this session. Log in or create a free AzTrials account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in