CART123 T Cells in Relapsed or Refractory CD123+ Hematologic Malignancies: A Dose Escalation Phase I Trial
NCT ID: NCT06765876
Last Updated: 2026-01-12
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
EARLY_PHASE1
18 participants
INTERVENTIONAL
2024-10-23
2028-12-31
Brief Summary
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Detailed Description
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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.
Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Treatment Arm
Experimental: Autologous CAR123 T lymphocytes
Autologous CAR123 T lymphocytes
Anti-CD123 Chimeric Antigen Receptor (CAR) T-Cells (CART123)
Interventions
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Autologous CAR123 T lymphocytes
Anti-CD123 Chimeric Antigen Receptor (CAR) T-Cells (CART123)
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
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
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.
18 Years
70 Years
ALL
No
Sponsors
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Institute of Hematology and Blood Transfusion, Czech Republic
OTHER
Responsible Party
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Principal Investigators
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Jan Vydra, MD, PhD
Role: PRINCIPAL_INVESTIGATOR
Institute of Hematology and Blood Transfusion, Prague, Czech Republic
Petr Lesný, MD, PhD
Role: STUDY_DIRECTOR
Institute of Hematology and Blood Transfusion, Prague, Czech Republic
Locations
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Ustav hematologie a krevni transfuze / Institute of Hematology and Blood Transfusion
Prague, , Czechia
Countries
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Central Contacts
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Facility Contacts
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Related Links
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Other Identifiers
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2022-503165-30-00
Identifier Type: CTIS
Identifier Source: secondary_id
UHKT-CAR123-01
Identifier Type: -
Identifier Source: org_study_id
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