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Ucart22

UCART22 is an innovative cellular therapy being studied in clinical trials for patients with relapsed or refractory B-cell Acute Lymphoblastic Leukemia (B-ALL). This article explores the ongoing research into UCART22, its potential benefits, and what patients should know about participating in these groundbreaking clinical trials.

Table of Contents

What is UCART22?

UCART22 is an innovative cell therapy being developed to treat patients with relapsed or refractory B-cell Acute Lymphoblastic Leukemia (B-ALL). It is classified as a type of CAR T-cell therapy, which stands for Chimeric Antigen Receptor T-cell therapy. This treatment is also known as “Universal Engineered Chimeric Antigen Receptor T-cells targeting CD22”.[1]

UCART22 is considered an advanced therapy and is classified as a gene therapy product. It is administered as a cell suspension for injection through an intravenous route.[1]

Target Condition: Relapsed or Refractory B-cell Acute Lymphoblastic Leukemia

B-cell Acute Lymphoblastic Leukemia (B-ALL) is a type of blood cancer that affects B lymphocytes, a type of white blood cell. When the disease is described as “relapsed,” it means the cancer has returned after initial treatment. “Refractory” means the cancer has not responded well to treatment. UCART22 is specifically designed to help patients whose B-ALL has either relapsed or become refractory to standard treatments.[1]

How UCART22 Works

UCART22 works by targeting a specific protein called CD22 found on the surface of B-ALL cancer cells. The therapy uses genetically modified T-cells (a type of immune cell) that have been engineered to recognize and attack cells expressing CD22.[1]

These modified T-cells are equipped with a Chimeric Antigen Receptor (CAR) that specifically targets CD22. When infused into the patient, these engineered T-cells can identify and destroy cancer cells that have CD22 on their surface.[1]

Clinical Trial Details

UCART22 is currently being studied in a clinical trial called BALLI-01. This is a Phase 1/2a study, which means it’s an early-stage trial designed to test the safety and initial effectiveness of the treatment.[1]

The trial is divided into two main parts:

  1. Dose Escalation Phase: This phase aims to determine the safest and most effective dose of UCART22.
  2. Dose Expansion Phase: This phase will further evaluate the chosen dose in a larger group of patients.

The main goals of the trial include assessing the safety of UCART22, determining the best dose, and evaluating how well it works in treating B-ALL.[1]

Eligibility Criteria

To participate in the UCART22 trial, patients must meet certain criteria. Some key eligibility factors include:

  • Age: 15-70 years for the dose escalation phase, 12-70 years for the dose expansion phase
  • Diagnosed with relapsed or refractory B-ALL
  • Previous treatment with standard chemotherapy and at least one salvage regimen
  • At least 70% of B-ALL blast cells must express CD22
  • Adequate organ function

There are also several factors that may exclude a patient from participating, such as certain prior treatments, active infections, or other medical conditions.[1]

Potential Benefits

While the effectiveness of UCART22 is still being studied, the therapy offers potential benefits for patients with relapsed or refractory B-ALL who have limited treatment options. The main potential benefits being evaluated include:

  • Overall response rate (complete or partial remission of the disease)
  • Minimal residual disease (MRD) negativity (very low levels of cancer cells remaining)
  • Progression-free survival (time without the disease getting worse)
  • Overall survival
  • Duration of response (how long the treatment effect lasts)

Additionally, for some patients, UCART22 might serve as a bridge to potentially curative treatments like stem cell transplantation.[1]

Safety Considerations

As with any new treatment, safety is a primary concern in the UCART22 trial. The study is carefully monitoring for any side effects or adverse events. Some specific safety considerations include:

  • Cytokine release syndrome (a condition caused by rapid activation of immune cells)
  • Neurological toxicities
  • Infections
  • Graft-versus-host disease (in patients who have had prior stem cell transplants)

The trial includes careful monitoring and management plans for these potential side effects.[1]

Aspect Details
Drug Name UCART22
Drug Type Allogeneic engineered T-cells expressing Anti-CD22 Chimeric Antigen Receptor
Target Condition Relapsed or refractory CD22+ B-cell Acute Lymphoblastic Leukemia (B-ALL)
Trial Phase Phase 1/2a
Trial Design Open-label, dose-escalation and dose-expansion study
Main Objectives Assess safety, tolerability, and determine optimal dose; Evaluate anti-leukemic activity
Key Eligibility Criteria Age 12-70 years; R/R B-ALL; Prior standard chemotherapy and salvage regimen; CD22 expression ≥70% on B-ALL blast cells
Administration Intravenous injection
Primary Endpoints Incidence and severity of adverse events; Proportion of patients with dose-limiting toxicity

FAQ

  • What is UCART22? UCART22 is an allogeneic engineered T-cell therapy that targets CD22, a protein found on B-cell leukemia cells. It's being studied as a potential treatment for patients with relapsed or refractory B-cell Acute Lymphoblastic Leukemia (B-ALL).
  • Who is eligible for the UCART22 clinical trial? Eligibility varies between the dose escalation and dose expansion phases. Generally, patients aged 12-70 years with relapsed or refractory B-ALL, who have received prior standard chemotherapy and salvage therapy, may be eligible. Specific CD22 expression levels and other medical criteria also apply.
  • What are the main objectives of the UCART22 clinical trial? The main objectives are to assess the safety and tolerability of UCART22, determine the appropriate dose, and evaluate its effectiveness in treating relapsed or refractory B-ALL. The trial also aims to study the drug's ability to eliminate minimal residual disease.
  • How is UCART22 administered? UCART22 is administered as a cell suspension for injection, given intravenously. Before receiving UCART22, patients undergo a lymphodepletion regimen to prepare their body for the treatment.
  • What are the potential benefits and risks of participating in this trial? Potential benefits include access to a novel therapy that may be effective against B-ALL when other treatments have failed. Risks may include side effects related to the treatment or lymphodepletion regimen. The trial closely monitors for adverse events and serious adverse events to ensure patient safety.

Ongoing Clinical Trials on Ucart22

  • Study of UCART22 for Patients with Relapsed or Refractory B-cell Acute Lymphoblastic Leukemia

    Not yet recruiting

    2 1 1 1
    Investigated diseases:
    Investigated drugs:
    France Italy Spain

Glossary

  • Acute Lymphoblastic Leukemia (ALL): A type of cancer that affects the blood and bone marrow, causing rapid growth of immature white blood cells called lymphoblasts.
  • Allogeneic: Refers to cells or tissues that are taken from a genetically different donor of the same species.
  • CAR T-cell therapy: A type of treatment in which a patient's T cells (a type of immune system cell) are changed in the laboratory so they will attack cancer cells.
  • CD22: A protein found on the surface of B cells, including B-cell leukemia cells, which is targeted by UCART22.
  • Chimeric Antigen Receptor (CAR): A special receptor created in the laboratory and inserted into immune cells (like T cells) to help them better identify and attack cancer cells.
  • Dose escalation: A method of determining the safest and most effective dose of a new drug by gradually increasing the amount given to patients.
  • Dose expansion: A phase in clinical trials where the determined dose from the escalation phase is given to a larger group of patients to further evaluate safety and efficacy.
  • Lymphodepletion: A process of reducing the number of lymphocytes (a type of white blood cell) in the body, often done before cell therapies to improve their effectiveness.
  • Minimal Residual Disease (MRD): Small numbers of cancer cells that remain in the body during or after treatment, often undetectable by standard tests.
  • Relapsed: The return of a disease or the signs and symptoms of a disease after a period of improvement.
  • Refractory: A condition that does not respond to treatment or stops responding after initial improvement.

References

  1. http://clinicaltrials.eu/trial/study-of-ucart22-for-patients-with-relapsed-or-refractory-b-cell-acute-lymphoblastic-leukemia/