Actively Recruiting
Safety and Efficacy of Anti-CD123 CAR-Modified Autologous T Cells in Relapsed or Refractory CD123+ Blood Cancers A Dose Escalation, Open-Label Phase I Study
Led by Institute of Hematology and Blood Transfusion, Czech Republic · Updated on 2026-01-12
18
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating a new treatment for adult patients with relapsed or refractory CD123-positive blood cancers, including acute myeloid leukemia, myelodysplastic syndrome, acute lymphoblastic leukemia, and blastic plasmacytoid dendritic cell neoplasm. This early-phase trial aims to find the recommended dose of CAR123 T cells, a type of genetically modified immune cell therapy, and assess its safety and tolerability. Participants must be eligible for hematopoietic stem cell transplantation and have a suitable donor identified before receiving the therapy. Patients will have their own blood used to create CAR123 T cells, which will be given back after a single course of chemotherapy to reduce their existing immune cells. The dose of CAR123 cells will be increased in steps to determine the highest safe or feasible dose. During cell manufacturing, patients may receive bridging treatments if needed. The study is open-label and involves one treatment group receiving autologous CAR123 T lymphocytes. The decision to perform stem cell transplantation after treatment will be individualized. Throughout the trial, participants will be monitored for safety and recovery of blood cells at multiple time points up to 24 months. Researchers will assess the recommended dose, response to treatment, survival, and the need for stem cell transplantation. Blood tests, disease assessments, and follow-up visits will be conducted regularly to evaluate outcomes and detect any side effects. The total duration of participant involvement can extend to two years for safety and efficacy follow-up.
CONDITIONS
Brief Title
CART123 T Cells in Relapsed or Refractory CD123+ Hematologic Malignancies: A Dose Escalation Phase I Trial
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Adults aged 18 to 70 years
- Diagnosis of AML, MDS-IB2, BPDCN, or ALL positive for CD123 antigen meeting disease-specific refractory or relapse criteria
- CD123 expression confirmed by laboratory testing
- Suitable donor for allogeneic hematopoietic stem cell transplantation cleared before treatment
- Able to understand and sign informed consent
- Negative pregnancy test for women of child-bearing potential at enrollment
- No standard treatments available or exhausted
You will not qualify if you...
- Known allergy to any component of the investigational product
- Allogeneic stem cell transplant within 3 months before treatment
- Severe uncontrolled active infection
- Life expectancy less than 8 weeks
- Respiratory failure requiring oxygen therapy
- Significant liver impairment beyond specified limits
- Acute kidney injury requiring dialysis or with high creatinine
- Heart failure with ejection fraction below 50%
- Active severe graft-versus-host disease
- Serious uncontrolled neurological conditions
- Recent live virus vaccination within specified timeframes
- Pregnancy or breastfeeding
- Fertile patients not using highly effective contraception or abstinence as lifestyle choice for the study duration
AI-Screening
AI-Powered Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 28 days
Participants receive a lymphodepleting conditioning regimen followed by a single dose of autologous CAR123 T lymphocytes to treat relapsed or refractory CD123+ blood cancers.
Multiple visits during the first 28 days for assessments and treatment administration
Duration - Up to 24 months
Participants are monitored for safety, hematological recovery, and treatment outcomes including possible allogeneic hematopoietic cell transplantation.
Scheduled visits at 14 days, 28 days, 1 year, 12 months, and 24 months after treatment
Trial Site Locations
Total: 1 location
1
Ustav hematologie a krevni transfuze / Institute of Hematology and Blood Transfusion
Prague, Czechia, 12800
Actively Recruiting
Research Team
P
Petr Lesny, MD, PhD
J
Jan Vydra, MD, PhD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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