In short
CARCIK-CD123/33 is an investigational cell therapy being studied in adults and children with relapsed or refractory acute myeloid leukemia (AML) and blastic plasmacytoid dendritic cell neoplasm (BPDCN). One authorised Phase 1/2 trial with a planned enrollment of 18 participants is evaluating the feasibility and safety of this therapy. The study is currently authorised but not yet recruiting.
Key points
- CARCIK-CD123/33 is an investigational cell therapy for blood cancers.
- It is being studied in relapsed or refractory acute myeloid leukemia and blastic plasmacytoid dendritic cell neoplasm.
- The current trial is Phase 1/2 with a planned enrollment of 18 participants.
- The trial status is authorised, meaning it has received approval but is not yet recruiting.
- The primary goal is to evaluate feasibility and safety of CARCIK-CD123/33 infusions.
What is CARCIK-CD123/33?
CARCIK-CD123/33 is an investigational cell therapy being developed for the treatment of certain blood cancers. It is a type of CAR-T cell therapy, a treatment approach in which a patient's own immune cells are modified in a laboratory to recognise and attack cancer cells. The name CARCIK-CD123/33 suggests that this therapy targets two proteins found on cancer cells: CD123 and CD33. It is not yet an authorised medicine and is currently being evaluated in clinical trials.
How does CARCIK-CD123/33 work?
The specific mechanism of action of CARCIK-CD123/33 has not been fully described in the available trial registry data. Based on its name, CARCIK-CD123/33 is designed to target CD123 and CD33, which are proteins found on the surface of certain leukemia and lymphoma cells. CAR-T cell therapies work by modifying a patient's own T cells to recognise specific proteins on cancer cells, allowing the immune system to attack the cancer. The trial is designed to evaluate the feasibility and safety of this approach.
Which conditions is CARCIK-CD123/33 being studied for?
CARCIK-CD123/33 is being studied for two conditions:
- Acute myeloid leukemia (AML) — a cancer of the blood and bone marrow in which abnormal white blood cells grow rapidly and crowd out normal blood cells.
- Blastic plasmacytoid dendritic cell neoplasm (BPDCN) — a rare and aggressive cancer of the blood and bone marrow that affects cells called plasmacytoid dendritic cells.
Both conditions are being studied in the relapsed or refractory setting, meaning the cancer has returned after previous treatment or has not responded to prior therapy.
How is CARCIK-CD123/33 given?
CARCIK-CD123/33 is given as an intravenous infusion, which means it is administered directly into a vein. The trial registry describes it as an intravenous infusion of CARCIK-CD123/33 cells.
What do the clinical trials show?
There is currently one clinical trial investigating CARCIK-CD123/33. This trial is a Phase 1/2 study, which is an early-stage trial that first evaluates safety and dosing in a small number of participants, and then begins to look at whether the treatment has an effect on the disease. The trial is currently authorised, meaning it has received regulatory approval but has not yet started recruiting participants. The planned enrollment is 18 participants.
The study is designed to evaluate the feasibility and safety of CARCIK-CD123/33 cell infusions in both adults and children with these blood cancers.
Who can take part in these trials?
The trial is open to adults and children with relapsed or refractory acute myeloid leukemia or blastic plasmacytoid dendritic cell neoplasm. Relapsed means the cancer has returned after previous treatment, and refractory means it has not responded to prior therapy. The trial has a planned enrollment of 18 participants.
What are the trials measuring?
The primary outcomes of the trial include:
- The rate of enrolled patients who eventually receive the CARCIK-CD123/33 infusion.
- The rate of dose-limiting toxicity, which refers to side effects that are severe enough to prevent giving a higher dose, assessed during the first 28 days after the infusion.
- The incidence of adverse events and laboratory abnormalities, which are monitored continuously throughout the study and reported at each scheduled visit.
These measures focus on establishing the safety and feasibility of the therapy in this patient population.