CD19 CAR-T therapy for young patients with high-risk B-cell leukemia

Treatment of CD19 Chimeric Antigen Receptor T Cells for Pediatric Patients With CD19-positive B-cell Acute Lymphoblastic Leukemia Who Are Indicated for Hematopoietic Stem Cell Transplantation

This study is testing a new CAR-T therapy for young patients with high-risk B-cell leukemia to see if it can be a good alternative to stem cell transplants.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 50 (estimated)
• Ages: N/A to 25 Years
• Sex: All
• Sponsor: Seoul National University Hospital (Academic / other)
• Drugs / interventions: CAR-T, chemotherapy
• Locations: 1 site (Seoul)
• Trial ID: NCT06247501 on ClinicalTrials.gov

What this trial studies

This phase 2 clinical trial focuses on pediatric and adolescent patients diagnosed with CD19-positive B-cell acute lymphoblastic leukemia (B-ALL) who are considered very high-risk. The study aims to evaluate the effectiveness of CD19 CAR-T therapy as an alternative treatment for patients eligible for hematopoietic stem cell transplantation. Participants must be 25 years old or younger and have achieved hematologic complete remission after initial chemotherapy. The trial will assess the safety and efficacy of the SNUH-CD19-CAR-T intervention in this specific patient population.

Who should consider this trial

Good fit: Ideal candidates for this study are pediatric and adolescent patients under 25 years old with CD19-positive B-ALL who have achieved hematologic complete remission.

Not a fit: Patients who are older than 25 years or those who do not have CD19-positive B-ALL may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for young patients with very high-risk B-ALL, potentially improving survival rates.

How similar studies have performed: Other studies have shown promising results with CAR-T therapies in treating B-cell malignancies, indicating a potential for success in this approach.

Eligibility criteria

Inclusion Criteria:

* Subject eligibility criteria include individuals who are under the age of 25 at the time of SNUH-CD19-CAR-T administration. Confirmation of B-cell acute lymphoblastic leukemia diagnosed at the initial assessment by a treating physician for the first diagnosis is required. Immunophenotypic analysis of CD19 expression on leukemia cells must be confirmed through immune profiling at the time of the initial diagnosis of B-cell acute lymphoblastic leukemia. In patients experiencing hematologic relapse of B-cell acute lymphoblastic leukemia, CD19 expression should also be confirmed at the time of relapse confirmation.

Patients must achieve hematologic complete remission defined as less than 5% blasts in the bone marrow after the first-line or second-line chemotherapy (first salvage therapy). Eligible patients for hematopoietic stem cell transplantation, defined by indications for transplantation, include those meeting at least one of the following criteria:

1. Philadelphia chromosome-positive due to t(9;22)(q34;q11) translocation.
2. Hypodiploidy defined by fewer than 44 chromosomes.
3. E2A-HLF gene fusion due to t(17;19) translocation.
4. Detection of minimal residual disease (MRD) positive at 0.01% or higher by next-generation sequencing confirmed after consolidation therapy and maintenance therapy.
5. Failure of the first-line therapy.
6. Cases not meeting any of the above criteria but deemed eligible for hematopoietic stem cell transplantation based on the investigator's judgment.

Exclusion Criteria:

* Patients who have undergone hematopoietic stem cell transplantation.
* Individuals for whom an adequate or sufficient leukapheresis product suitable for the production of SNUH-CD19-CAR-T cannot be obtained or is unavailable.
* Those known to be infected with the human immunodeficiency virus (HIV).
* Presence of uncontrolled active infections, determined by the investigator's assessment. If appropriate treatment has been administered for the infection, and there are no signs of progression at the time of enrollment, it is considered controlled. Persistent fever without other symptoms is not interpreted as progressive infection.
* Women who are pregnant or breastfeeding.
* Individuals deemed clinically inappropriate for participation in the clinical trial based on the investigator's clinical judgment.

Where this trial is running

Seoul

• Seoul National University Children's Hospital — Seoul, South Korea (Recruiting)

Study contacts

• Study coordinator: Hyoung Jin Kang, Professor

Conditions Very High Risk Acute Lymphoblastic Leukemia; CD19 CAR-T Therapy