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Caba-201

A groundbreaking clinical trial is underway to assess the safety and effectiveness of CABA-201, an innovative CAR T-cell therapy, in patients with active Systemic Lupus Erythematosus (SLE). This Phase 1/2 study aims to explore the potential of CABA-201 in treating this chronic autoimmune disorder, which can affect multiple organs and significantly impact patients’ quality of life. The trial will evaluate various aspects of the treatment, including its effects on disease activity, B cell counts, and overall patient well-being.

Table of Contents

What is CABA-201?

CABA-201 is a new type of treatment being developed for Systemic Lupus Erythematosus (SLE), a chronic autoimmune disorder. It belongs to a class of therapies called CAR T-cell therapy, which stands for Chimeric Antigen Receptor T-cell therapy. This innovative approach uses a patient’s own immune cells, specifically T cells, which are modified in a laboratory to target and fight disease.[1]

How does CABA-201 work?

CABA-201 works by targeting CD19, a protein found on the surface of B cells. In SLE, B cells play a crucial role in producing harmful antibodies that attack the body’s own tissues. The process involves:

  1. Collecting T cells from the patient’s blood
  2. Modifying these T cells in a laboratory to recognize and target CD19
  3. Multiplying these modified T cells
  4. Infusing the CAR T cells back into the patient’s body

Once infused, these modified T cells can recognize and eliminate B cells that contribute to the disease, potentially reducing symptoms and improving the patient’s condition.[1]

What conditions does CABA-201 treat?

CABA-201 is being developed to treat active Systemic Lupus Erythematosus (SLE). SLE is a complex autoimmune disease that can affect various parts of the body, including the skin, joints, kidneys, brain, and other organs. It’s characterized by periods of illness and remission.

The treatment is also being studied for a specific severe manifestation of SLE called Lupus Nephritis (LN). LN is a serious kidney inflammation that can lead to significant health problems if left untreated.[1]

Current Research on CABA-201

CABA-201 is currently being studied in a Phase 1/2 clinical trial. This study aims to evaluate the safety, tolerability, and effectiveness of CABA-201 in adult patients with active SLE. The main goals of this research include:

  • Assessing the safety of CABA-201 over 28 days and 156 weeks after infusion
  • Evaluating how CABA-201 affects white blood cell counts, including B cells
  • Measuring the persistence of CABA-201 in the body after infusion
  • Determining the effects of CABA-201 on SLE disease activity and symptoms
  • Assessing how CABA-201 impacts patients’ quality of life

This research will help determine the appropriate dose for future studies and provide valuable information about the potential benefits and risks of this new treatment approach.[1]

Who can participate in the CABA-201 trial?

The trial is open to adults aged 18 to 65 with active SLE. Participants must meet specific criteria, including:

  • A confirmed diagnosis of SLE based on established medical criteria
  • Positive test results for certain antibodies associated with SLE
  • For patients with Lupus Nephritis, specific levels of protein in the urine
  • Adequate kidney and liver function
  • Up-to-date vaccinations, including for COVID-19

However, some factors may exclude participation, such as:

  • Recent treatment with certain medications that deplete B cells
  • Previous CAR T-cell therapy or organ transplants
  • Pregnancy or plans to become pregnant
  • Certain other medical conditions or active infections

It’s important to note that these are just some of the criteria, and the study doctors will carefully evaluate each potential participant to ensure their safety and the study’s integrity.[1]

Potential Benefits of CABA-201

While the research is still ongoing, CABA-201 shows promise in several areas:

  • Potential reduction in SLE disease activity and symptoms
  • Possible improvement in kidney function for patients with Lupus Nephritis
  • Potential decrease in the need for other SLE medications, including steroids
  • Possible improvement in quality of life for patients with SLE

However, it’s crucial to remember that these potential benefits are still being studied, and more research is needed to confirm the effectiveness of CABA-201.[1]

Safety Considerations

As with any new treatment, safety is a top priority in the CABA-201 trial. The researchers will closely monitor participants for any side effects or adverse reactions. Some potential risks associated with CAR T-cell therapies in general include:

  • Cytokine Release Syndrome (CRS): A condition where the immune system becomes highly activated, potentially causing fever, low blood pressure, and other symptoms
  • Neurological effects: Such as confusion, seizures, or difficulty speaking
  • B cell aplasia: A reduction in B cells, which could increase the risk of certain infections

The study includes specific measures to manage these potential risks, including careful patient selection and close monitoring throughout the treatment process.[1]

Aspect Details
Study Type Phase 1/2, open-label study
Main Objective Evaluate safety and tolerability of CABA-201 in active SLE patients over 28 days
Treatment CABA-201 (CD19-specific CAR T-cells) infusion
Preconditioning Regimen Fludarabine (25 mg/m² x 3 doses) and Cyclophosphamide (1,000 mg/m² x 1 dose)
Cell Dose 1 x 10⁶ cells/kg
Eligibility Adults 18-65 years with active SLE, including those with or without lupus nephritis
Follow-up Duration 156 weeks (3 years)
Key Endpoints Safety, B cell counts, SLE disease activity, quality of life measures

FAQ

  • What is CABA-201? CABA-201 is a type of CAR T-cell therapy, which uses a patient's own modified immune cells to target specific cells in the body. In this case, CABA-201 is designed to target CD19-positive cells, which are involved in the autoimmune response in Systemic Lupus Erythematosus (SLE).
  • Who is eligible for this clinical trial? The trial is open to adults aged 18-65 with active SLE, including those with or without lupus nephritis. Participants must meet specific criteria, such as having a positive antinuclear antibody (ANA) titer or anti-dsDNA antibody at screening, and be willing to comply with the study protocol.
  • What does the treatment process involve? The treatment process includes collecting the patient's own T cells through a process called leukapheresis, modifying these cells to create CABA-201, and then infusing the modified cells back into the patient. Before the infusion, patients receive a preconditioning regimen with fludarabine and cyclophosphamide.
  • What are the main goals of this clinical trial? The primary goal is to evaluate the safety and tolerability of CABA-201 in patients with active SLE over 28 days. Secondary objectives include assessing long-term safety, effects on B cell counts, impact on SLE disease activity, and changes in quality of life measures.
  • How long will participants be monitored in this study? Participants will be monitored for up to 156 weeks (3 years) after receiving the CABA-201 infusion. This extended follow-up period allows researchers to assess both short-term and long-term effects of the treatment.

Ongoing Clinical Trials on Caba-201

  • Study on the Safety and Effectiveness of CABA-201, Fludarabine Phosphate, and Cyclophosphamide in Patients with Active Systemic Lupus Erythematosus

    Recruiting

    1 1 1
    Investigated drugs:
    France Spain

  • A study to evaluate the safety and effectiveness of CABA-201, fludarabine, and cyclophosphamide in patients with active inflammatory myopathy

    Not yet recruiting

    1 1 1
    Investigated drugs:
    Germany Italy Spain

Glossary

  • Systemic Lupus Erythematosus (SLE): A chronic autoimmune disorder where the body's immune system attacks its own tissues, causing inflammation and damage to various organs.
  • CAR T-cell therapy: A type of treatment that uses a patient's own T cells (a type of immune cell) that have been genetically modified to target specific cells in the body.
  • Leukapheresis: A medical procedure that separates and collects white blood cells from a patient's blood, used in this study to obtain T cells for modification.
  • Lupus Nephritis (LN): A serious complication of SLE that causes inflammation in the kidneys, potentially leading to kidney damage or failure.
  • Antinuclear Antibody (ANA): A type of autoantibody that targets substances found in the cell nucleus, often present in people with autoimmune disorders like SLE.
  • Anti-dsDNA antibody: An antibody that targets double-stranded DNA, commonly found in patients with SLE and associated with disease activity.
  • SLEDAI-2K: Systemic Lupus Erythematosus Disease Activity Index 2000, a tool used to measure disease activity in SLE patients.
  • BILAG-2004: British Isles Lupus Assessment Group 2004 index, another measure of disease activity in SLE.
  • SRI-4: SLE Responder Index 4, a composite measure used to assess treatment response in SLE clinical trials.

References

  1. http://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-caba-201-fludarabine-phosphate-and-cyclophosphamide-in-patients-with-active-systemic-lupus-erythematosus/