CART123 T Cells in Relapsed or Refractory CD123+ Hematologic Malignancies: A Dose Escalation Phase I Trial (UHKT-CAR123-01)

January 8, 2026 updated by: Institute of Hematology and Blood Transfusion, Czech Republic

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Estimated)

18

Phase

  • Early Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • Czechia
      • Prague, Czechia, 12800
        • Recruiting
        • Ustav hematologie a krevni transfuze / Institute of Hematology and Blood Transfusion
        • Contact:
        • Contact:
        • Principal Investigator:
          • Jan Vydra, MD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Patients with AML, MDS-IB2, BPDCN or ALL positive for CD123 antigen, who meet one of disease specific criteria below:

    a) Patients with AML will be eligible if they meet one of the following criteria:

    i) 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

    ii) Second or subsequent relapse of AML OR

    iii) Relapse after allogeneic HSCT.

    b) Patients with ALL will be eligible if they meet one of following criteria:

    i) disease refractory to or relapsed after CAR-19 cell therapy OR

    ii) CD19 negative relapse ineligible for treatment with TKI inhibitors and inotuzumab ozogamicin.

    c) Patients with BPDCN will be eligible if they meet following criteria:

    i) Refractory or relapsing after chemotherapy with or without allogeneic stem cell transplantation.

    d) Patients with MDS-IB2 will be eligible if they meet one of following criteria:

    i) Disease refractory to at least four cycles of azacytidine or progression on azacytidine-based therapy OR

    ii) Disease refractory to induction chemotherapy OR

    iii) Relapse after haematopoietic stem cell transplantation.

  2. CD123 expression on malignant cells confirmed by flow cytometry or by immunohistochemistry.
  3. Age between 18 and 70 years.
  4. Patient has a suitable donor for allogeneic hematopoietic stem cell transplantation. Workup and clearance of the donor must be completed before IMP administration.
  5. Patient able to understand and sign informed consent.
  6. Women of child-bearing potential: negative pregnancy test at enrolment (PSV) and at Visit 1.
  7. Patient for whom there are no standard-of-care treatments available or such treatment options have been exhausted.

Exclusion Criteria:

  1. Known hypersensitivity to any component of the IMP.
  2. Allogeneic HSCT within 3 months prior to IMP administration.
  3. Severe, uncontrolled active infection.
  4. Life expectancy < 8 weeks.
  5. Respiratory insufficiency (need for oxygen therapy).
  6. Significant liver impairment: bilirubin > 50 µmol/L, AST or ALT > 4 times normal upper limit.
  7. Acute kidney injury with serum creatinine > 180 µmol/L, oliguria or need for acute dialysis.
  8. Heart failure with LVEF < 50% by echocardiography.
  9. Presence of active grade 3 - 4 acute GvHD or severe chronic GvHD.
  10. Serious uncontrolled neurological comorbidity.
  11. Vaccination with live virus vaccines in the 4 weeks before IMP administration and within 90 days after the IMP dose.
  12. Women: pregnancy or breast-feeding.

  13. Subjects of fertile age, unless permanent sexual abstinence is their lifestyle choice:

    1. female patients of childbearing potential not willing to use a highly effective method of contraception during the study,
    2. 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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Treatment Arm
Experimental: Autologous CAR123 T lymphocytes
Biological: Autologous CAR123 T lymphocytes
Anti-CD123 Chimeric Antigen Receptor (CAR) T-Cells (CART123)
Other Names:
  • CART123
  • CAR123 T Cells
  • Autologous T-cell Therapy

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Safety and tolerability of autologous CART123 cells
Time Frame: 28 Days, Months 24
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
28 Days, Months 24
Rate of hematological recovery
Time Frame: 14 Days, 28 Days, 1Year, Months 12, Months 24
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.
14 Days, 28 Days, 1Year, Months 12, Months 24
A dose of CART123 cells for further study
Time Frame: 14 Days, 28 Days, 1year, Months 12, Months 24
Incidence of dose-limiting toxicities (DLTs) and other safety data after IMP administration.
14 Days, 28 Days, 1year, Months 12, Months 24

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Morphologic Leukemia Free State (MLFS).
Time Frame: at day 14 and 28 after IMP administration.
Efficacy of IMP administration in patients with refractory or relapsed AML, MDS, BPDCN and ALL evaluated by rate of morphologic leukemia-free state (MLFS).
at day 14 and 28 after IMP administration.
Complete Remission (CR) rate
Time Frame: at 28 days and at any later point after IMP administration, before and after HSCT.
Efficacy of IMP administration in patients with refractory or relapsed AML, MDS, BPDCN and ALL evaluated by Complete Remission (CR) rate.
at 28 days and at any later point after IMP administration, before and after HSCT.
Median Overall Survival and Overall Survival
Time Frame: at one year after IMP administration.
Efficacy of IMP administration in patients with refractory or relapsed AML, MDS, BPDCN and ALL evaluated by overall survival (OS)
at one year after IMP administration.
Feasibility and need for allogeneic hematopoietic cell transplantation after IMP administration
Time Frame: within 28 days of 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.
within 28 days of IMP administration

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
Determination of persistence and phenotype of CART123 cells in peripheral blood and bone marrow.
Time Frame: Days 1, 3, 5, 7, 14, 28, 49, 70, 100. Months 6, 12, 24.
Number (absolute and percentage of T lymphocytes) of CART123 cells in peripheral blood and bone marrow.
Days 1, 3, 5, 7, 14, 28, 49, 70, 100. Months 6, 12, 24.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Institute of Hematology and Blood Transfusion, Czech Republic

Investigators

  • Principal Investigator: Jan Vydra, MD, PhD, Institute of Hematology and Blood Transfusion, Prague, Czech Republic
  • Study Director: Petr Lesný, MD, PhD, Institute of Hematology and Blood Transfusion, Prague, Czech Republic

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

October 23, 2024

Primary Completion (Estimated)

December 31, 2028

Study Completion (Estimated)

December 31, 2028

Study Registration Dates

First Submitted

December 18, 2024

First Submitted That Met QC Criteria

January 3, 2025

First Posted (Actual)

January 9, 2025

Study Record Updates

Last Update Posted (Estimated)

January 12, 2026

Last Update Submitted That Met QC Criteria

January 8, 2026

Last Verified

January 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • UHKT-CAR123-01
  • 2022-503165-30-00 (Ctis)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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