In short
CD19-CART01 is an investigational CAR T-cell therapy being studied in a Phase 1 basket trial for eight severe autoimmune and alloimmune conditions, including systemic lupus erythematosus and chronic graft-versus-host disease. The trial, authorised in the EU, plans to enrol 24 patients. Its goal is to assess safety and find the recommended dose of this anti-CD19 cell therapy.
Key points
- CD19-CART01 is an investigational CAR T-cell therapy that targets the CD19 protein on B cells.
- The single authorised trial is a Phase 1 basket study covering eight autoimmune and alloimmune conditions.
- The trial plans to enrol 24 patients and is currently authorised in the EU.
- The main goal is to assess safety and determine the recommended dose of CD19-CART01.
- CD19-CART01 is given as a single intravenous infusion.
- The study uses a 3+3 dose-escalation design to find the maximum tolerated dose.
What is CD19-CART01?
CD19-CART01 is an investigational anti-CD19 chimeric antigen receptor (CAR) T-cell therapy. It is a cell-based immunotherapy, meaning it is made from a patient's own immune cells that are modified in a laboratory to recognise and attack cells carrying the CD19 protein. CD19 is found on the surface of B cells, a type of white blood cell involved in many autoimmune diseases. The substance is not an authorised medicine; it is being studied in a clinical trial to determine whether it is safe and what dose should be used. In the trial record, CD19-CART01 is listed as a drug given intravenously.
How does CD19-CART01 work?
CD19-CART01 is designed to target the CD19 protein on B cells. B cells play a central role in autoimmune diseases by producing antibodies and driving inflammation. The therapy works by redirecting a patient's own T cells to recognise and eliminate CD19-positive B cells. This is intended to deplete the B-cell population that contributes to disease activity. The trial does not describe a more detailed mechanism beyond this anti-CD19 targeting.
Which conditions is it studied for?
The authorised Phase 1 basket trial studies CD19-CART01 in eight conditions:
- Systemic lupus erythematosus (SLE) — a chronic autoimmune disease that can affect the skin, joints, kidneys and other organs.
- Systemic sclerosis (SSc) — a rare disease causing hardening and tightening of the skin and connective tissues.
- Dermatomyositis/polymyositis (DM/PM) — inflammatory muscle diseases that cause muscle weakness and skin rashes.
- Immune-mediated necrotizing myopathy (IMNM) — a rare autoimmune muscle disease with severe muscle weakness.
- ANCA-associated vasculitis (AAv) — inflammation of small blood vessels caused by autoantibodies.
- Myasthenia gravis (MG) — an autoimmune condition causing weakness of voluntary muscles.
- Neuromyelitis optica spectrum disorder (NMOSD) — an autoimmune disease affecting the optic nerves and spinal cord.
- Chronic graft-versus-host disease (cGVHD) — a complication after stem cell transplant where donor immune cells attack the recipient's body.
How is CD19-CART01 given?
CD19-CART01 is given as an intravenous infusion. The trial record lists the pharmaceutical form as a solution for infusion and the route of administration as intravenous use. In the basket trial, patients receive a single infusion of CD19-CART01 after their T cells have been collected, modified and expanded in the laboratory.
What do the clinical trials show?
There is one authorised clinical trial of CD19-CART01 in the EU Clinical Trials Information System. It is a Phase 1 basket trial, meaning it tests the therapy in several different diseases at the same time. The trial plans to enrol 24 patients across the eight conditions listed above. It is currently authorised, which means it has received regulatory approval to begin but no results have been published yet.
The primary goal of the trial is to assess safety and determine the recommended dose. The study uses a 3+3 dose-escalation design, in which small groups of three patients receive increasing doses until the maximum tolerated dose is identified. The trial will specifically measure the incidence and severity of cytokine release syndrome (CRS), immune effector cell-associated neurotoxicity syndrome (ICANS), immune effector cell-associated haemophagocytic lymphohistiocytosis-like syndrome (IEC-HS), and grade 2 or higher infections within the first four weeks after infusion. It will also assess whether the manufacturing process produces enough modified CD19-CART01 cells for each patient.
The trial is open to patients with SLE, SSc, DM/PM, IMNM, AAv, MG, NMOSD or cGVHD. Specific eligibility criteria are available in the trial registry record. The study is interventional, meaning participants receive the therapy as part of the trial.
What side effects are known?
The trial record does not list known side effects of CD19-CART01. Because the trial is measuring safety, side effects will be collected during the study. The primary outcome specifically tracks cytokine release syndrome, neurotoxicity, haemophagocytic lymphohistiocytosis-like syndrome and infections, which are known risks of CAR T-cell therapies in general.