A first clinical study in humans using the patient's own T-cells that carry a special receptor against a protein called Siglec-6, in cases of hard-to-treat or recurrent acute myeloid leukemia or chronic lymphocytic leukemia

TCX-001 is a novel, personalized therapy in which the patient's own immune cells (T-cells) are processed in the laboratory and equipped with a special receptor. This receptor recognizes the surface marker Siglec-6 on cancer cells and specifically targets them.
Early laboratory studies show that TCX-001 can specifically recognize and kill cancer cells in AML and CLL. In this study, a protein called Siglec-6 is being clinically tested in humans for the first time as a target molecule. The aim is to investigate whether treatment with TCX-001 is safe and whether early signs of efficacy can be observed in patients with limited treatment options.
The main objective of this study is to evaluate the safety of TCX-001 and to determine the maximum tolerated dose (MTD) or the recommended dose for Phase 2 (RP2D).
The study is divided into two sections:
In the first section, the treatment will initially be tested with two very low doses:
• Level 1: 100,000 CAR-T cells per kilogram of body weight
• Level 2: 300,000 CAR-T cells per kilogram of body weight
Each dosage starts with a so-called "sentinel patient," meaning that initially, a single patient is treated and closely monitored over a specified period before additional patients are included.
In the second phase, the dose will be gradually increased depending on how well TCX-001 is tolerated. The planned dosages are:
• Level 3: 1 million CAR-T cells per kilogram of body weight
• Level 4: 3 million CAR-T cells per kilogram
• Level 5: 6 million CAR-T cells per kilogram
Patients will be treated in groups of three. A standardized procedure will be used for dose finding to identify a suitable dose at which no more than 30% of patients experience serious side effects.
Whether the dose is increased or maintained will be decided by an independent committee of investigators and a statistician.

This study investigates whether the investigational drug TCX-001, when administered once intravenously, is safe, well tolerated, and can be used for the treatment of AML or CLL.
The investigational drug TCX-001 consists of the patient's own, genetically modified white blood cells from the immune system (T-cells) that are equipped with a protein complex known as a chimeric antigen receptor (CAR). These T-cells are also called CAR-T cells. The CAR protein complex recognizes a protein structure on other body cells with the scientific name Siglec-6. Current knowledge indicates that Siglec-6 is present on the surface of AML and CLL cancer cells, as well as on a few healthy cells, such as certain immune system defense cells (B-cells, basophilic granulocytes, mast cells). With the help of the CAR protein complex, CAR-T cells bind to Siglec-6 and can neutralize AML and CLL cancer cells. TCX-001 is manufactured from the participant's own T-cells.

acute myeloid leukemia (AML);
chronic lymphocytic leukemia (CLL)